Category Archives: Genetic Engineering

Aspects of genetics including mutation, hybridisation, cloning, genetic engineering, and eugenics have appeared in fiction since the 19th century.

Genetics is a young science, having started in 1900 with the rediscovery of Gregor Mendel's study on the inheritance of traits in pea plants. During the 20th century it developed to create new sciences and technologies including molecular biology, DNA sequencing, cloning, and genetic engineering. The ethical implications were brought into focus with the eugenics movement.

Since then, many science fiction novels and films have used aspects of genetics as plot devices, often taking one of two routes: a genetic accident with disastrous consequences; or, the feasibility and desirability of a planned genetic alteration. The treatment of science in these stories has been uneven and often unrealistic. The film Gattaca did attempt to portray science accurately but was criticised by scientists.

Modern genetics began with the work of the monk Gregor Mendel in the 19th century, on the inheritance of traits in pea plants. Mendel found that visible traits, such as whether peas were round or wrinkled, were inherited discretely, rather than by blending the attributes of the two parents.[1] In 1900, Hugo de Vries and other scientists rediscovered Mendel's research; William Bateson coined the term "genetics" for the new science, which soon investigated a wide range of phenomena including mutation (inherited changes caused by damage to the genetic material), genetic linkage (when some traits are to some extent inherited together), and hybridisation (crosses of different species).[2]

Eugenics, the production of better human beings by selective breeding, was named and advocated by Charles Darwin's cousin, the scientist Francis Galton, in 1883. It had both a positive aspect, the breeding of more children with high intelligence and good health; and a negative aspect, aiming to suppress "race degeneration" by preventing supposedly "defective" families with attributes such as profligacy, laziness, immoral behaviour and a tendency to criminality from having children.[3][4]

Molecular biology, the interactions and regulation of genetic materials, began with the identification in 1944 of DNA as the main genetic material;[5] the genetic code and the double helix structure of DNA was determined by James Watson and Francis Crick in 1953.[6][7] DNA sequencing, the identification of an exact sequence of genetic information in an organism, was developed in 1977 by Frederick Sanger.[8]

Genetic engineering, the modification of the genetic material of a live organism, became possible in 1972 when Paul Berg created the first recombinant DNA molecules (artificially assembled genetic material) using viruses.[9]

Cloning, the production of genetically identical organisms from some chosen starting point, was shown to be practicable in a mammal with the creation of Dolly the sheep from an ordinary body cell in 1996 at the Roslin Institute.[10]

Mutation and hybridisation are widely used in fiction, starting in the 19th century with science fiction works such as Mary Shelley's 1818 novel Frankenstein and H. G. Wells's 1896 The Island of Dr Moreau.[11]

In her 1977 Biological Themes in Modern Science Fiction, Helen Parker identified two major types of story: "genetic accident", the uncontrolled, unexpected and disastrous alteration of a species;[12][13] and "planned genetic alteration", whether controlled by humans or aliens, and the question of whether that would be either feasible or desirable.[12][13] In science fiction up to the 1970s, the genetic changes were brought about by radiation, breeding programmes, or manipulation with chemicals or surgery (and thus, notes Lars Schmeink, not necessarily by strictly genetic means).[13] Examples include The Island of Dr Moreau with its horrible manipulations; Aldous Huxley's 1932 Brave New World with a breeding programme; and John Taine's 1951 Seeds of Life, using radiation to create supermen.[13] After the discovery of the double helix and then recombinant DNA, genetic engineering became the focus for genetics in fiction, as in books like Brian Stableford's tale of a genetically modified society in his 1998 Inherit the Earth, or Michael Marshall Smith's story of organ farming in his 1997 Spares.[13]

Comic books have imagined mutated superhumans with extraordinary powers. The DC Universe (from 1939) imagines "metahumans"; the Marvel Universe (from 1961) calls them "mutants", while the Wildstorm (from 1992) and Ultimate Marvel (20002015) Universes name them "posthumans".[14] Stan Lee introduced the concept of mutants in the Marvel X-Men books in 1963; the villain Magneto declares his plan to "make Homo sapiens bow to Homo superior!", implying that mutants will be an evolutionary step up from current humanity. Later, the books speak of an X-gene that confers powers from puberty onwards. X-men powers include telepathy, telekinesis, healing, strength, flight, time travel, and the ability to emit blasts of energy. Marvel's god-like Celestials are later (1999) said to have visited Earth long ago and to have modified human DNA to enable mutant powers.[15]

James Blish's 1952 novel Titan's Daughter (in Kendell Foster Crossen's Future Tense collection) featured stimulated polyploidy (giving organisms multiple sets of genetic material, something that can create new species in a single step), based on spontaneous polyploidy in flowering plants, to create humans with more than normal height, strength, and lifespans.[16]

Cloning, too, is a familiar plot device. Aldous Huxley's 1931 dystopian novel Brave New World imagines the in vitro cloning of fertilised human eggs.[17][18] Huxley was influenced by J. B. S. Haldane's 1924 non-fiction book Daedalus; or, Science and the Future, which used the Greek myth of Daedalus to symbolise the coming revolution in genetics; Haldane predicted that humans would control their own evolution through directed mutation and in vitro fertilisation.[19] Cloning was explored further in stories such as Poul Anderson's 1953 UN-Man.[20] In his 1976 novel, The Boys from Brazil, Ira Levin describes the creation of 96 clones of Adolf Hitler, replicating for all of them the rearing of Hitler (including the death of his father at age 13), with the goal of resurrecting Nazism. In his 1990 novel Jurassic Park, Michael Crichton imagined the recovery of the complete genome of a dinosaur from fossil remains, followed by its use to recreate living animals of an extinct species.[11]

Cloning is a recurring theme in science fiction films like Jurassic Park (1993), Alien Resurrection (1997), The 6th Day (2000), Resident Evil (2002), Star Wars: Episode II (2002) and The Island (2005). The process of cloning is represented variously in fiction. Many works depict the artificial creation of humans by a method of growing cells from a tissue or DNA sample; the replication may be instantaneous, or take place through slow growth of human embryos in artificial wombs. In the long-running British television series Doctor Who, the Fourth Doctor and his companion Leela were cloned in a matter of seconds from DNA samples ("The Invisible Enemy", 1977) and thenin an apparent homage to the 1966 film Fantastic Voyageshrunk to microscopic size in order to enter the Doctor's body to combat an alien virus. The clones in this story are short-lived, and can only survive a matter of minutes before they expire.[21] Films such as The Matrix and Star Wars: Episode II Attack of the Clones have featured human foetuses being cultured on an industrial scale in enormous tanks.[22]

Cloning humans from body parts is a common science fiction trope, one of several genetics themes parodied in Woody Allen's 1973 comedy Sleeper, where an attempt is made to clone an assassinated dictator from his disembodied nose.[23]

Genetic engineering features in many science fiction stories.[16] Films such as The Island (2005) and Blade Runner (1982) bring the engineered creature to confront the person who created it or the being it was cloned from, a theme seen in some film versions of Frankenstein. Few films have informed audiences about genetic engineering as such, with the exception of the 1978 The Boys from Brazil and the 1993 Jurassic Park, both of which made use of a lesson, a demonstration, and a clip of scientific film.[11][24] In 1982, Frank Herbert's novel The White Plague described the deliberate use of genetic engineering to create a pathogen which specifically killed women.[16] Another of Herbert's creations, the Dune series of novels, starting with Dune in 1965, emphasises genetics. It combines selective breeding by a powerful sisterhood, the Bene Gesserit, to produce a supernormal male being, the Kwisatz Haderach, with the genetic engineering of the powerful but despised Tleilaxu.[25]

Genetic engineering methods are weakly represented in film; Michael Clark, writing for The Wellcome Trust, calls the portrayal of genetic engineering and biotechnology "seriously distorted"[24] in films such as Roger Spottiswoode's 2000 The 6th Day, which makes use of the trope of a "vast clandestine laboratory ... filled with row upon row of 'blank' human bodies kept floating in tanks of nutrient liquid or in suspended animation". In Clark's view, the biotechnology is typically "given fantastic but visually arresting forms" while the science is either relegated to the background or fictionalised to suit a young audience.[24]

Eugenics plays a central role in films such as Andrew Niccol's 1997 Gattaca, the title alluding to the letters G, A, T, C for guanine, adenine, thymine, and cytosine, the four nucleobases of DNA. Genetic engineering of humans is unrestricted, resulting in genetic discrimination, loss of diversity, and adverse effects on society. The film explores the ethical implications; the production company, Sony Pictures, consulted with a gene therapy researcher, French Anderson, to ensure that the portrayal of science was realistic, and test-screened the film with the Society of Mammalian Cell Biologists and the American National Human Genome Research Institute before its release. This care did not prevent researchers from attacking the film after its release. Philim Yam of Scientific American called it "science bashing"; in Nature Kevin Davies called it a ""surprisingly pedestrian affair"; and the molecular biologist Lee Silver described the film's extreme genetic determinism as "a straw man".[26][27]

The geneticist Dan Koboldt observes that while science and technology play major roles in fiction, from fantasy and science fiction to thrillers, the representation of science in both literature and film is often unrealistic.[28] In Koboldt's view, genetics in fiction is frequently oversimplified, and some myths are common and need to be debunked. For example, the Human Genome Project has not (he states) immediately led to a Gattaca world, as the relationship between genotype and phenotype is not straightforward. People do differ genetically, but only very rarely because they are missing a gene that other people have: people have different alleles of the same genes. Eye and hair colour are controlled not by one gene each, but by multiple genes. Mutations do occur, but they are rare: people are 99.99% identical genetically, the 3 million differences between any two people being dwarfed by the hundreds of millions of DNA bases which are identical; nearly all DNA variants are inherited, not acquired afresh by mutation. And, Koboldt writes, believable scientists in fiction should know their knowledge is limited.[29]

See original here:
Genetics in fiction - Wikipedia

Yale researchers have uncovered new details on how a common weed is able to thrive under hot, dry conditions potentially a roadmap to engineering crops that are resistant to the effects of climate change.

The challenge: Higher temperatures, more severe droughts, and the other effects of climate change are now threatening crop yields, imperiling progress in feeding the world made since the Green Revolution.

While corn yields have nearly tripled worldwide since 1961, according to the Food and Agriculture Organization (FAO), a recent NASA study predicts that they could decline by up to 24% before the end of this century.

Climate changeis threateningcrop yields, imperiling progress in feeding the world made since the Green Revolution.

The FAO estimates that one in three people worldwide currently experiences food insecurity, and the population is growing. If we want to continue to make progress against hunger, or at least protect the gains that have been made, we need not just to prevent a climate change-fueled decline in crop yields, but to continue growing more food on less land.

If we cant, well have to clear more forests for agriculture, releasing the carbon stored in the trees into the atmosphere and making the problem of global warming worse not to mention damaging ecosystems and pressuring endangered species.

The discovery: Genetic engineering offers a potential solution to the problem using tech such as CRISPR, we can give crops characteristics that help them withstand the effects of climate change.

A new Yale study puts us a step closer to making that future a reality by revealing how a common weed, purslane, is able to grow in hot, dry conditions.

Purslane is both drought-resistant and highly productive even in hot climates a rarity for any plant.

Supercharged photosynthesis: While most plants have naturally evolved a single type of photosynthesis, purslane uses two: C4 photosynthesis and CAM photosynthesis.

C4 photosynthesis allows a plant to remain productive when temperatures are high corn and sugarcane use that type of photosynthesis, too. CAM photosynthesis, meanwhile, helps a plant survive with little water its been adapted by cacti and other succulents.

As a result, purslane is both drought-resistant and highly productive even in hot climates a rarity for any plant.

Team effort: Scientists thought that the two types of photosynthesis worked independently in purslanes leaves, but the Yale team has now discovered that the mechanisms are closely integrated, operating in the same cells and working as a single metabolic system.

This knowledge of how the two types of photosynthesis work together could one day be used for engineering plants that are both drought and heat resistant we could one day take a C4 crop like corn, for example, and integrate CAM photosynthesis into it.

[T]here is still a lot of work to do before that could become a reality, said senior author Erika Edwards, but what weve shown is that the two pathways can be efficiently integrated and share products.

Wed love to hear from you! If you have a comment about this article or if you have a tip for a future Freethink story, please email us at [emailprotected].

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Weeds superpower could help feed the planet - Freethink

The following discussion and analysis of our financial condition and results ofoperations should be read in conjunction with our unaudited condensedconsolidated financial statements and related notes appearing elsewhere in thisQuarterly Report on Form 10-Q and our audited consolidated financial statementsand the related notes thereto included in our Annual Report on Form 10-K for theyear ended December 31, 2021, or 2021 Annual Report, as filed with theSecurities and Exchange Commission, or SEC. Operating results are notnecessarily indicative of results that may occur in future periods. Thisdiscussion, particularly information with respect to our future results ofoperations or financial condition, business strategy and plans and objectives ofmanagement for future operations, includes forward-looking statements thatinvolve risks and uncertainties as described under the heading "Special NoteRegarding Forward-Looking Statements" in this Quarterly Report on Form 10-Q. Youshould review the disclosure under the heading "Risk Factors" in this QuarterlyReport on Form 10-Q for a discussion of important factors that could cause ouractual results to differ materially from those anticipated in theseforward-looking statements.

Overview

We are a clinical-stage biopharmaceutical company dedicated to utilizing ourproprietary genetic engineering platform technologies to create next-generationcell and gene therapeutics with the capacity to cure. We were incorporated inDecember 2014 and subsequently spun out from Transposagen, a company that hasbeen developing genetic engineering technologies since 2003. Since ourinception, our operations have focused on organizing and staffing our company,business planning, raising capital, in-licensing, developing and acquiringintellectual property rights and establishing and protecting our intellectualproperty portfolio, developing our genetic engineering technologies, identifyingpotential product candidates and undertaking research and development andmanufacturing activities, including preclinical studies and clinical trials ofour product candidates, and engaging in strategic transactions. We do not haveany product candidates approved for sale and have not generated any revenue fromproduct sales.

We have discovered and are developing a broad portfolio of product candidates ina variety of indications based on our core proprietary platforms, including ournon-viral piggyBac DNA Delivery System, Cas-CLOVER Site-specific Gene EditingSystem and nanoparticle and AAV-based gene delivery technologies. Our coreplatform technologies have utility, either alone or in combination, across manycell and gene therapeutic modalities and enable us to engineer our portfolio ofproduct candidates that are designed to overcome the primary limitations ofcurrent generation cell and gene therapeutics.

Within cell therapy, we believe our technologies allow us to create productcandidates with engineered cells that engraft in the patient's body and drivelasting durable responses that may have the capacity to result in singletreatment cures. Our CAR-T therapy portfolio consists of both autologous andallogeneic, or off-the-shelf, product candidates. We are advancing a broadpipeline and have multiple CAR-T product candidates in the clinical phase inboth hematological and solid tumor oncology indications. Within gene therapy, webelieve our technologies have the potential to create next-generation therapiesthat can deliver long-term, stable gene expression that does not diminish overtime and that may have the capacity to result in single treatment cures.

Our most advanced investigational clinical programs are:

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We manufacture these product candidates using our non-viral piggyBac DNADelivery System. Our fully allogeneic CAR-T product candidates are developedusing well-characterized cells derived from a healthy donor as starting materialwith the goal of enabling treatment of potentially hundreds of patients from asingle manufacturing run. Doses are cryopreserved and stored at treatmentcenters for future off-the-shelf use. In addition, our allogeneic productcandidates use our proprietary Cas-CLOVER site-specific Gene Editing System toreduce or eliminate reactivity, as well as our booster molecule technology formanufacturing scalability.

Our most advanced preclinical cell therapy program is:

Our gene therapy product candidates have been developed by utilizing ourpiggyBac technology together with AAV to overcome the major limitations oftraditional AAV gene therapy. We believe that our approach can result inintegration and long-term stable expression at potentially much lower doses thanAAV technology alone, thus also conferring cost and tolerability benefits. Oureventual goal is to completely replace AAV with our non-viral nanoparticletechnology, freeing future product development in gene therapy of AAVlimitations.

Our most advanced gene therapy programs are:

We expect our expenses and losses to increase substantially for the foreseeablefuture as we continue our development of, and seek regulatory approvals for, ourproduct candidates, including P-PSMA-101 and P-MUC1C-ALLO1, and begin tocommercialize any approved products. While we anticipate an overall increase indevelopment costs as we continue to expand the number of product candidates inour pipeline and pursue clinical development of those candidates, we expect adecrease in our development costs on a per program basis as we are transitioningto our allogeneic platform. In addition, all or some of the development costsrelated to partnered gene therapy programs and cell therapy programs will bereimbursed by Takeda and Roche, respectively. We also expect our general andadministrative expenses will increase for the foreseeable future to support ourincreased research and development and other corporate activities. Our netlosses may fluctuate significantly from quarter-to-quarter and year-to-year,depending on the timing of our clinical trials and our expenditures on otherresearch and development activities.

We do not expect to generate any revenues from product sales unless and until wesuccessfully complete development and obtain regulatory approval for P-PSMA-101and P-MUC1C-ALLO1, or any other product candidates, which will not be for atleast the next several years, if ever. If we obtain regulatory approval for anyof our product candidates, we expect to incur significant commercializationexpenses related to product sales, marketing, manufacturing and distributionactivities. Accordingly, until such time, if ever, as we can generatesubstantial product revenue, we expect to finance our operations through equityofferings, debt financings or other capital sources, including potential grants,collaborations, licenses or other similar arrangements. However, we may not beable to secure additional financing or enter into such other arrangements in atimely manner or on favorable terms, if at all. There can be no assurances thatwe will be able to secure such additional sources of funds to support ouroperations, or, if such funds are available to us, that such additionalfinancing will be sufficient to meet our needs. Our failure to raise capital orenter into such other arrangements when needed would have a negative impact onour financial condition and could force us to delay, reduce or

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terminate our research and development programs or other operations, or grantrights to develop and market product candidates that we would otherwise preferto develop and market ourselves.

The manufacturing process for our allogeneic product candidates is nearlyidentical to the process for our autologous product candidates, except for thegene editing and related steps. We work with a number of third-party contractmanufacturing organizations for production of our product candidates. We alsowork with a variety of suppliers to provide our manufacturing raw materialsincluding media, DNA and RNA components. We have completed construction of aninternal pilot GMP manufacturing facility in San Diego, California adjacent toour headquarters to develop and manufacture preclinical materials and clinicalsupplies of our product candidates for Phase 1 and Phase 2 clinical trials inthe future. We commenced GMP activity in the third quarter of 2021, however weexpect that we will continue to rely on third parties for various manufacturingneeds. In the future, we may also build one or more commercial manufacturingfacilities for any approved product candidates.

Collaboration Agreements

Roche Collaboration Agreement

In July 2022, we entered the Roche Collaboration Agreement with Roche, pursuantto which we will grant to Roche: (i) an exclusive, worldwide license undercertain of our intellectual property to develop, manufacture and commercializeallogeneic CAR-T cell therapy products from each of our existing P-BCMA-ALLO1and P-CD19CD20-ALLO1 programs, or each, a Tier 1 Program; (ii) an exclusiveoption to acquire an exclusive, worldwide license under certain of ourintellectual property to develop, manufacture and commercialize allogeneic CAR-Tcell therapy products from our existing P-BCMACD19-ALLO1 and P-CD70-ALLO1programs, or each, a Tier 2 Program; (iii) an exclusive license under certain ofour intellectual property to develop, manufacture and commercialize allogeneicCAR-T cell therapy products from the up to six Collaboration Programs, asdefined below, designated by Roche; (iv) an option for a non-exclusive,commercial license under certain limited intellectual property to develop,manufacture and commercialize certain Roche proprietary cell therapy productsfor up to three solid tumor targets to be identified by Roche, or LicensedProducts; and (v) the right of first offer for two of our early-stage existingprograms within hematologic malignancies.

For each Tier 1 Program, we will perform development activities through a Phase1 dose escalation clinical trial, and Roche is obligated to reimburse aspecified percentage of certain costs incurred by us in our performance of suchactivities, up to a specified reimbursement cap for each Tier 1 Program. Foreach Tier 2 Program, we will perform research and development activities eitherthrough selection of a development candidate for IND-enabling studies or,subject to Roche's election and payment of an option maintenance fee, throughcompletion of a Phase 1 dose escalation clinical trial. In addition, for eachTier 2 Program for which Roche exercises its option for an exclusive license,Roche is obligated to pay us an option exercise fee. For each Tier 1 Program andTier 2 Program, we will perform manufacturing activities until the completion ofa technology transfer to Roche.

The parties will conduct an initial two-year research program to explore andpreclinically test a specified number of agreed-upon next generation therapeuticconcepts relating to allogeneic CAR-T cell therapies. Subject to Roche'selection and payment of a fee, the parties would subsequently conduct a secondresearch program of 18 months under which the parties would explore andpreclinically test a specified number of additional agreed-upon next generationtherapeutic concepts relating to allogeneic CAR-T therapies. Roche may designateup to six heme malignancy-directed, allogeneic CAR-T programs from the tworesearch programs, for each of which we will perform research and developmentactivities through selection of a development candidate for IND-enablingactivities, or each, a Collaboration Program. Upon its designation of eachCollaboration Program, Roche is obligated to pay a designation fee. After wecomplete lead optimization activities for a Collaboration Program, Roche mayelect to transition such program to Roche with a payment to us or terminate it.Alternatively, Roche may elect, for a limited number of Collaboration Programs,to have us conduct certain additional development and manufacturing activitiesthrough the completion of a Phase 1 dose escalation clinical trial, in whichcase Roche will pay certain milestones and reimburse a specified percentage ofour costs incurred in connection with such development and manufacturingactivities. For each Collaboration Program, we will perform manufacturingactivities until the completion of a technology transfer to Roche.

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Under the Roche Collaboration Agreement, Roche is obligated to make an upfrontpayment to us of $110.0 million. We could also receive up to $110.0 million innear-term fees and milestone and other payments. Subject to Roche exercising itsTier 2 Program options, designating Collaboration Programs, and exercising itsoption for the Licensed Products commercial license and contingent on, amongother things, the products from the Tier 1 Programs, optioned Tier 2 Programsand Collaboration Programs achieving specified development, regulatory, and netsales milestone events, we are eligible to receive certain reimbursements, feesand milestone payments, including the near-term fees and milestone paymentsdescribed above, in the aggregate up to $6.0 billion, comprised of (i) $1.5billion for the Tier 1 Programs; (ii) $1.1 billion for the Tier 2 Programs,(iii) $2.9 billion for the Collaboration Programs; and (iv) $415 million for theLicensed Products.

We are further entitled to receive, on a product-by-product basis, tieredroyalty payments in the mid-single to low double digits on net sales of productsfrom the Tier 1 Programs, optioned Tier 2 Programs and Collaboration Programsand in the low to mid-single digits for Licensed Products, in each case, subjectto certain customary reductions and offsets. Royalties will be payable, ona product-by-product and country-by-country basis, until the latest of theexpiration of the licensed patents covering such product in such country or tenyears from first commercial sale of such product in such country.

The Roche Collaboration Agreement will become effective upon the expiration ortermination of the applicable waiting period under the Hart-Scott-RodinoAntitrust Improvements Act of 1976, as amended, and continue on aproduct-by-product and country-to-country basis until there is no remainingroyalty or other payment obligations. The Roche Collaboration Agreement includesstandard termination provisions, including for material breach or insolvency andfor Roche's convenience. Certain of these termination rights can be exercisedwith respect to a particular product or license, as well as with respect to theentire Roche Collaboration Agreement.

Takeda Collaboration Agreement

In October 2021, we entered into the Takeda Collaboration Agreement, pursuant towhich we granted to Takeda a worldwide exclusive license under ourpiggyBac, Cas-CLOVER, biodegradable DNA and RNA nanoparticle delivery technologyand other proprietary genetic engineering platforms to research, develop,manufacture and commercialize gene therapy products for certain indications,including Hemophilia A. We collaborate with Takeda to initially develop up tosix in vivo gene therapy programs and Takeda also has an option to add twoadditional programs to the collaboration. We are obligated to lead researchactivities up to candidate selection, after which Takeda is obligated to assumeresponsibility for further development, manufacturing and commercialization ofeach program.

Under the Takeda Collaboration Agreement, Takeda made an upfront payment to usof $45.0 million. Takeda is also obligated to provide funding for allcollaboration program development costs including our P-FVIII-101 program;provided that we are obligated to perform certain platform developmentactivities at our own cost. Timelines for P-FVIII-101 and other programs subjectto the Takeda Collaboration Agreement will be driven by Takeda. Under the TakedaCollaboration Agreement, we are eligible to receive preclinical milestonepayments that could potentially exceed $82.5 million in the aggregate ifpreclinical milestones for all six programs are achieved. We are also eligibleto receive future clinical development, regulatory and commercial milestonepayments of $435.0 million in the aggregate per target, with a total potentialdeal value over the course of the collaboration of up to $2.7 billion, ifmilestones for all six programs are achieved and up to $3.6 billion if themilestones related to the two optional programs are also achieved. We areentitled to receive tiered royalty payments on net sales in the mid-single tolow double digits, subject to certain standard reductions and offsets. Royaltieswill be payable, on a product-by-product and country-by-country basis, until thelatest of the expiration of the licensed patents covering such product in suchcountry, ten years from first commercial sale of such product in such country,or expiration of regulatory exclusivity for such product in such country.

In-License Agreements

Below is a summary of our key license agreements. For a more detaileddescription of these and our other license agreements, see the section titled"Business-In-License Agreements" and Note 11 to our annual consolidatedfinancial statements included in our 2021 Annual Report.

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CIRM Grant Funding

In 2017, we were granted an award in the amount of $19.8 million from CaliforniaInstitute of Regenerative Medicine, or CIRM, to support our clinical trial forP-BCMA-101. To date we have received a total of $19.7 million from this grantand we may receive up to $0.1 million in future grant payments upon closeout ofour clinical trial for this program. In the fourth quarter of 2021 we made thedecision to wind down clinical development of the P-BCMA-101 program andderecognized the liability related to amount of the award previously received.In 2018, we were granted an additional award in the amount of $4.0 million fromCIRM to support our preclinical studies for P-PSMA-101, of which we havereceived all proceeds from this grant. The terms of these awards include anoption to repay the grant or convert it to a royalty obligation uponcommercialization of the program. Based upon the terms of the grant agreements,we initially record proceeds as a liability when received and subsequentlyreassess based on our intention to repay the amounts associated with awards orconvert them to a royalty obligation.

Components of Our Results of Operations

Revenues

Collaboration Revenue

Collaboration revenue consists of revenue recognized from our collaboration andlicense agreement with Takeda and reflects the timing and pattern in which wedeliver the contractual deliverables to Takeda.

Operating Expenses

Research and Development

Research and development expenses consist primarily of external and internalcosts incurred for our research and development activities, includingdevelopment of our platform technologies, our drug discovery efforts and thedevelopment of our product candidates.

External costs include:

Internal costs include:

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We expense research and development costs as incurred. External expenses arerecognized based on an evaluation of the progress to completion of specifictasks using information provided to us by our service providers or our estimateof the volume of service that has been performed at each reporting date. Upfrontpayments and milestone payments made for the licensing of technology are relatedto clinical stage programs and expensed as research and development in theperiod in which they are incurred. Advance payments that we make for goods orservices to be received in the future for use in research and developmentactivities are recorded as prepaid expenses or other long-term assets. Theseamounts are expensed as the related goods are delivered or the services areperformed.

At any one time, we are working on multiple research programs. We track externalcosts by the stage of program, clinical or preclinical. Our internal resources,employees and infrastructure are not directly tied to any one program and aretypically deployed across multiple programs. As such, we do not track internalcosts on a specific program basis.

Product candidates in later stages of clinical development generally have higherdevelopment costs than those in earlier stages of clinical development,primarily due to CRO activity and manufacturing expenses. We expect that ourresearch and development expenses will increase substantially in connection withour planned preclinical and clinical development activities in the near term andin the future, including in connection with our ongoing Phase 1 trial ofP-PSMA-101 for the treatment of patients with mCRPC, Phase 1 trial ofP-BCMA-ALLO1 for the treatment of patients with relapsed/refractory multiplemyeloma and Phase 1 trial of P-MUC1C-ALLO1 for the treatment of patients withsolid tumor cancers and additional clinical programs expected to commence as weexpand our pipeline of drug candidates. We cannot accurately estimate or knowthe nature, timing and costs of the efforts that will be necessary to completethe preclinical and clinical development of any of our product candidates. Ourdevelopment costs may vary significantly based on factors such as:

the number and scope of preclinical and IND-enabling studies;

per patient trial costs;

the number of trials required for approval;

the number of sites included in the trials;

the countries in which the trials are conducted;

the length of time required to enroll eligible patients;

the number of patients that participate in the trials;

the drop-out or discontinuation rates of patients;

potential additional safety monitoring requested by regulatory agencies;

the duration of patient participation in the trials and follow-up;

the cost and timing of manufacturing our product candidates;

the phase of development of our product candidates;

the efficacy and safety profile of our product candidates;

the extent to which we establish additional licensing agreements; and

A change in the outcome of any of these variables with respect to thedevelopment of any of our product candidates could significantly change the coststructure and timing associated with the development of respective productcandidates. We may never succeed in obtaining regulatory approval for any of ourproduct candidates. We may obtain unexpected results from our clinical trialsand preclinical studies.

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General and Administrative

General and administrative expenses consist primarily of salaries and relatedcosts, including stock-based compensation, for personnel in executive, financeand administrative functions. General and administrative expenses also includedirect and allocated facility-related costs as well as professional fees forlegal, patent, consulting, investor and public relations, and accounting andaudit services. We anticipate that our general and administrative expenses willincrease in the future as we increase our headcount to support our continuedresearch activities and development of our product candidates, includingP-PSMA-101, P-BCMA-ALLO1 and P-MUC1C-ALLO1, and begin to commercialize anyapproved products.

Interest expense consists of interest expense on outstanding borrowings underour loan agreement and amortization of debt discount and debt issuance costs.

Other Income (Expense), Net

Other income (expense), net consists of interest income and miscellaneous incomeand expense unrelated to our core operations. Interest income is comprised ofinterest earned on our available-for-sale securities.

Results of Operations

Comparison of the Three Months Ended June 30, 2022 and 2021

The following table summarizes our results of operations (in thousands):

Collaboration revenue of $2.7 million for the three months ended June 30, 2022represents revenue recognized from the Takeda Collaboration Agreement that weentered into in the fourth quarter of 2021 and related to the research serviceswe performed for Takeda.

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Research and Development Expenses

Preclinical stage programs and other

Total research and development expenses $ 35,008 $ 36,008 $ (1,000 )

(1) Clinical stage programs include costs related to P-BCMA-ALLO1, P-MUC1C-ALLO1,

Research and development expenses were $35.0 million for the three months endedJune 30, 2022, compared to $36.0 million for the three months ended June 30,2021. The decrease in research and development expenses of $1.0 million wasprimarily due to a decrease of $4.1 million in external costs related to ourclinical stage programs, driven mainly by the wind-down of our clinicaldevelopment activities associated with the P-BCMA-101 program, as announced inthe fourth quarter of 2021, and an early termination and accelerated expense ofa contract with one of our autologous contract manufacturers in the firstquarter of 2022, partially offset by increases in the number of ongoing clinicaltrials, including enrollment and manufacturing for the P-PSMA-101, P-BCMA-ALLO1,and the P-MUC1C-ALLO1 Phase 1 clinical trials, and a $1.7 million decrease inexternal costs related to our preclinical stage programs, driven mainly by thetransition of the P-BCMA-ALLO1 and P-MUC1C-ALLO1 programs to clinical stage. Theincrease of $4.0 million in personnel expenses was a result of an increase inheadcount which included a $0.4 million increase in stock-based compensationexpense.

General and Administrative Expenses

General and administrative expenses were $9.2 million for the three months endedJune 30, 2022, compared to $8.9 million for the three months ended June 30,2021. The increase in general and administrative expenses of $0.4 million wasprimarily due to an increase of $0.2 million in personnel expenses as a resultof an increase in headcount which included a $0.1 million increase instock-based compensation expense.

Interest Expense

Interest expense was $1.5 million for the three months ended June 30, 2022,compared to $0.8 million for the three months ended June 30, 2021 and consistedof interest on the principal balance outstanding under our term loans withOxford Finance LLC, or Oxford. The increase in interest expense of $0.7 millionwas primarily due to an increase in principal outstanding related to themodification of the terms of our loan pursuant to the 2022 Loan Agreement, asdefined below, which we entered into in February 2022.

Other Income (Expense), Net

Other income, net was less than $0.1 million for each of the three months endedJune 30, 2022 and 2021.

--------------------------------------------------------------------------------

Comparison of the Six Months Ended June 30, 2022 and 2021

The following table summarizes our results of operations (in thousands):

Collaboration revenue of $4.1 million for the six months ended June 30, 2022represents revenue recognized from the Takeda Collaboration Agreement that weentered into in the fourth quarter of 2021 and related to the research serviceswe performed for Takeda.

Research and Development Expenses

Preclinical stage programs and other

Total research and development expenses $ 83,858 $ 65,103 $ 18,755

(1) Clinical stage programs include costs related to P-BCMA-ALLO1, P-MUC1C-ALLO1,

--------------------------------------------------------------------------------

Research and development expenses were $83.9 million for the six months endedJune 30, 2022, compared to $65.1 million for the six months ended June 30, 2021.The increase in research and development expenses of $18.8 million was primarilydue to an increase of $8.7 million in external costs related to our clinicalstage programs from an increase in the number of ongoing clinical trials,including enrollment and manufacturing for the P-PSMA-101 Phase 1, theP-BCMA-ALLO1 Phase 1, and the P-MUC1C-ALLO1 Phase 1 clinical trials, an increaseof $8.4 million in personnel expenses as a result of an increase in headcountwhich included a $1.2 million increase in stock-based compensation expense, anda $1.3 million increase in internal facilities and other costs. The increase inexternal costs related to our clinical stage programs also includes a loss on acontract termination related to an early termination and accelerated expense ofa contract with one of our autologous contract manufacturers during the sixmonths ended June 30, 2022, consisting of future contractual paymentobligations, a write off of deferred milestone payments previously made to ourautologous contract manufacturer, and an impairment of a related right-of-useasset, partially offset by the wind-down of our clinical development activitiesassociated with the P-BCMA-101 program.

General and Administrative Expenses

General and administrative expenses were $18.8 million for the six months endedJune 30, 2022, compared to $17.2 million for the six months ended June 30, 2021.The increase in general and administrative expenses of $1.5 million wasprimarily due to an increase of $1.3 million in personnel expenses as a resultof an increase in headcount which included a $0.7 million increase instock-based compensation expense.

Interest Expense

Interest expense was $2.6 million for the six months ended June 30, 2022,compared to $1.7 million for the six months ended June 30, 2021 and consisted ofinterest on the principal balance outstanding under our term loans with Oxford.The increase in interest expense of $0.9 million was primarily due to anincrease in principal outstanding related to the modification of the terms ofour loan pursuant to the 2022 Loan Agreement, as defined below, which we enteredinto in February 2022.

Other Income (Expense), Net

Other income, net was less than $0.1 million for each of the six months endedJune 30, 2022 and 2021.

Liquidity and Capital Resources

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POSEIDA THERAPEUTICS, INC. Management's Discussion and Analysis of Financial Condition and Results of Operations. (form 10-Q) - Marketscreener.com

Novartis has acknowledged that two patients have died of acute liver failure following treatment with its Zolgensma (onasemnogene abeparvovec-xioi), a one-time gene therapy indicated for some forms of spinal muscular atrophy (SMA).

As a result, the company said, it will revise Zolgensmas label to specify that fatal acute liver failure has been reported.

While this is important safety information, it is not a new safety signal and we firmly believe in the overall favorable risk/benefit profile of Zolgensma, Novartis said in a statement emailed to GEN and other news organizations.

Mani Foroohar, MD, Senior managing director, Genetic Medicines, and a senior research analyst with SVB Securities, wrote in a research note today that the deaths are expected to touch off renewed public discussion over the safety of adeno-associated virus (AAV) gene therapies such as Zolgensma.

We expect these events to rekindle broader debates on safety and management of systemic AAV therapies in fragile or very young patients, one contributor to the overhang on gene therapy stocks across our coverage, which have underperformed the biotech sector as a whole YTD [year to date], Foroohar wrote.

The labelincluding a Boxed Warning in the U.S. Prescribing Information for the gene therapyhas until now included acute liver failure as a known side effect that has been reported following treatment.

A 2020 paper published in The Journal of Pediatrics detailed two cases of transient, drug-induced subacute liver failure following treatment with Zolgensma. A 2021 study in Nature-published Gene Therapy showed that of nine children treated with the gene therapy in Qatar, none experienced failurebut all patients experienced elevated levels of the liver enzymes aspartate aminotransferase (AST) or alanine transaminase (ALT), two experienced high prothrombin time, and one experienced elevated bilirubin. One patient experienced vomiting after infusion.

The deaths are the first fatal cases of acute liver failure that have been linked to Zolgensma, a gene therapy developed by AveXis. Novartis acquired AveXis for $8.7 billion in a deal completed in 2018.

A year later in May 2019, the FDA approved Zolgensma for the treatment of SMA in pediatric patients less than two years of age with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma was the second gene therapy authorized by the FDA for an inherited disease.

During the first half of this year, Zolgensma generated $742 million in net sales, up 17% from JanuaryJune 2021. Zolgensma finished last year with $1.351 billion in net sales, up 47% from 2020. Novartis considers Zolgensma among its key growth brands.

In trading today, Novartis shares dipped 1.16% on the SIX Swiss Exchange, to CHF 80.10 ($85.06).

To date, Novartis stated, Zolgensma has been used to treat more than 2,300 patients worldwide across clinical trials, managed access programs, and commercially.

We have notified health authorities in all markets where Zolgensma is used, including FDA, and are communicating to relevant healthcare professionals as an additional step in markets where this action is supported by health authorities, Novartis added.

Novartis has long asserted that Zolgensmas benefits in halting SMA and facilitating infant development milestones justify its $2.1 million list price, though the company has also long cited its discounted patient-access programs with insurers.

Novartis did not reveal information about the patientswho were both children according to STAT News, which first reported the deaths. However, the company did disclose that one of the fatal cases of acute liver failure took place in Russia and the other, in Kazakhstan.

Both cases occurred at approximately five to six weeks post Zolgensma infusion, and approximately 110 days following the initiation of corticosteroid taper, Novartis stated.

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Novartis Confirms Deaths of Two Patients Treated with Gene Therapy Zolgensma - Genetic Engineering & Biotechnology News

Emerald Isle, N.C.

Aug. 12, 2020

TO THE EDITOR:

Preface to the 1984 Edition of the novel 1984 by Walter Cronkite:

American reporters, given a glimpse of Ayatollah Khomeini's Iran at the End of 1982, were saying that it was like 1984. It's Orwellian, one added.

Big Brother has become a common term for ubiquitous or overreaching authority, and Newspeak is a word we apply to the dehumanizing babble of bureaucracies and computer programs.

Those coinages have come into the language with lives of their own. They are familiar to millions who have never read 1984, who may not even know it as a novel written thirty-five years ago by English socialist Eric Blair, who became famous under the pen name George Orwell.

Seldom has a book provided a greater wealth of symbols for its age and for the generations to follow, and seldom have literary symbols been invested with such power. How is that? Because they were so useful, and became the features of the world he drew, outlandish as they were, also were familiar.

They are familiar today, they were familiar when the book was first published in 1949. We've met Big Brother in Stalin and Hitler and Khomeini. We hear Newspeak in every use of the language to manipulate, deceive, to cover harsh realities with the soft snow of euphemism.

And every time a political leader expects or demands that we believe the absurd, we experience that mental process Orwell called Doublethink. From the show trials of the pre-war Soviet Union to the dungeon courts of post-revolutionary Iran, 1984's vision of justice as foregone conclusion is familiar to us all. As soon as we were introduced to such things, we realized we had always known them.

What Orwell had done was not to foresee the future but to see the implications of the present -- his present and ours -- and he touched a common chord. He had given words and shapes to common but unarticulated fears running deep through all industrial societies.

George Orwell was no prophet, and those who busy themselves keeping score on his predictions and grading his use of the crystal ball miss the point. While here he is a novelist, he is also a sharp political essayist and a satirist with a bite not felt in the English language since Jonathan Swift.

If not prophecy, what was 1984? It was, as many have noticed, a warning: a warning about the future of human freedom in a world where political organization and technology can manufacture power in dimensions that would stunned the imaginations of earlier ages.

Orwell drew upon the technology (and perhaps some of the science fiction) of the day in drawing his picture of 1984. But it was not a work of science fiction he was writing. It was a novelistic essay on power, how it is acquired and maintained, how those who seek it or seek to keep it tend to sacrifice anything and everything in its name.

1984 is an anguished lament and a warning that vibrates powerfully when we may not be strong enough, nor wise enough, nor moral enough to cope with the kind of power we have learned to amass.

That warning vibrates powerfully when we allow ourselves to sit still and think carefully about orbiting satellites that can read the license plates in a parking lot and computers that can read into thousands of telephone calls and telex transmissions at once and other computers that can do our banking and purchasing, can watch the house and tell a monitoring station what television program we are watching and how many people there are in a room. We think of

Orwell when we read of scientists who believe they have located in the human brain the seats of behavioral emotions like aggression, or learn more about the vast potential of genetic engineering.

And we hear echoes of that warning chord in the constant demand for greater security and comfort, for less risk in our societies.

We recognize, however dimly, that greater efficiency, ease, and security may come at a substantial price in freedom that "law and order" can be a doublethink version of oppression that individual liberties surrendered for whatever good reason are freedoms lost.

Critics and scholars may argue quite legitimately about the particular literary merits of 1984. But none can deny its power, its hold on the imagination of a whole generations, nor the power of its admonitions . . . a power that seems to grow rather than lessen with the passage of time.

It has been said that 1984 fails as a prophecy because it succeeded as a warning -- Orwell's terrible vision has been averted. Well, that kind of self-congratulation is, to say the least, premature.

1984 may not arrive on time, but there's always 1985.

Still, the warning has been effective; and every time we use one of those catch phrases . . . recognize Big Brother in someone, see a 1984 in our future . . . notice something Orwellian . . . we are listening to that warning again.

This was written by Walter Cronkite in 1983. Both Orwell and Cronkite saw the writing on the wall. In the novel, Big Brother (big government) uses fear, oppression and hate to control and manipulate every aspect of people's lives. This includes what you can say, what you can think, and even who you can love.

I think it is especially pertinent today given that we seem to be edging ever closer to 1984. I sincerely hope that Orwell's 1984 is still required reading in our public schools.

JEFFREY WARD

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LETTER TO THE EDITOR: Lessons from 1984 | Letters To Editor | carolinacoastonline.com - Carolinacoastonline

Arkeon Biotechnologies was founded in response to the worlds current food systems, which all three co-founders CEO Gregor Tegl, CSO Simon Rittman, and CTO Gnther Bochmann deem to be unsustainable.

Bochmann and Rittman joined the start-up with an interest in gas fermentation. The duo believes in the potential of repurposing environmentally harmful gas for good.

Tegl, on the other hand, has long been fascinated by enzymes, he recalled.

Thats where we combined. We found that combining two things, notably gas fermentation and enzymes, can upgrade the functionality of whatever is coming out of the bioreactor. Its a powerful tool to convert waste streams, such as CO2, into value-added products.

This is the Austrian start-ups strategy. Combining science with a life-long passion for food, Arkeon wants to fix the worlds broken food systems by producing protein ingredients the most sustainable way possible. We think weve found a pretty good way of doing that, Tegl told FoodNavigator.

Arkeon is developing a novel way of producing the building blocks of protein: amino acids.

Amino acids are monomers that when linked together form the chains we know as proteins. It is the amino acids that make up the protein that our bodies use for energy, explained Tegl.

Proteins are complex in nature and can be difficult to work with, which is why the start-up is interested in producing only the building blocks themselves. We circumvent all the trade-offs that come with the nature of a protein.

Arkeon does this by leveraging microorganisms. The start-up has identified an archaeon capable of producing all 20 amino acids required for human nutrition in one natural fermentation process.

Archaea are single-cell organisms considered a major part of Earths life. They live in extreme environments such as hot springs and salt lakes and are part of the microbiota of all organisms. In the human microbiome, for example, they are found in the gut, mouth, and on the skin.

Producing all amino acids in one fermentation is unheard of, stressed Tegl. Of course, Arkeon is not the first start-up to product alternative proteins with microbes in a lab. However, Arkeons mode of production is unique, the CEO suggested.

Other microbes producing amino acids tend to keep them for themselves to create biomass, Tegl explained. Our microbe is producing all these amino acids and spitting them outside the cell, so we can retrieve these building blocks and already have a really valuable nutrient source from our culture medium.

This is one of Arkeons key advantages, according to the CEO. While a wealth of scientists is studying the physiology of such organisms, very few are interested in their biotechnological exploitation. This gives us a competitive advantage.

Another advantage of Arkeons technology is its carbon source. It turns out the start-ups archaeon of choice likes to feed on carbon dioxide, which is handy given that CO2 is common waste product.

You can use CO2 from any industry. The easiest, at least in the beginning, would be microbially produced such as from breweries of bioethanol plants. Thats very pure CO2 and already food-grade because its coming out of a food production process, Tegl explained, adding that using CO2 in this way makes its production carbon negative. So thats a very attractive source.

The other gas input is hydrogen, which in produced from electricity and water. A green hydrogen approach would require water and renewable energy. That can be done on-site using an electrolyser, but we are also in touch with green hydrogen producersto ensure a strong and secure supply.

The CEO likened the fermentation to that used in beer brewing, with the main differentiator being that in Arkeons case, the carbon source comes from CO2 and the energy source, hydrogen.

The fermentation itself takes place in an off-the-shelf bioreactor operating at atmospheric pressure, which Tegl explained is uncommon in gas fermentation. The higher the pressure in the vessel, the better the gas dissolves. But when you have a microbe like ours, which is so efficient in taking up those gases, you dont need [higher pressure].

In so doing, Arkeons gas fermentation process is economically viable, we were told.

Using these two gas inputs means that the process is independent of agriculture. No part of Arkeons feedstock grows on arable land, which the start-up stressed is a big advantage particularly given the climate catastrophe we are facing, and temperature fluctuations impacting the agricultural sector.

Once the archeon has produced amino acids in the bioreactor, the start-up binds them to peptides. From there, Arkeon says it can make a variety of functional foods.

A key benefit in working with amino acids, rather than protein, is that Arkeons solution is highly soluble. Other advantages in working with amino acids rather than plant proteins lie in avoiding some of the pain points of the protein industry, Tegl explained.

Pea protein, for example, come with off-notes that food formulators often have to mask with additional ingredients. Another key issue is bioavailability.

Protein quality is typically defined in terms of protein digestibility-corrected amino acid score (PDCAAS), which is a measure of its essential amino acid composition and digestibility.

While some plant proteins, such as soy protein, are considered good quality proteins with a PDCAAS score of 1 (the highest possible score), others score much lower. Tree nuts, for example come in under 0.50, with wheat gluten even lower.

Meat products such as chicken, on the other hand, has a PDCAAS score of 0.95 and beef, 0.92.

Arkeons solution offers the nutritional equivalent to meat, which as shown by the PDCAAS ranking, is higher than a lot of plant proteins available. The start-ups powdered ingredients, therefore, can help improve the nutritional profiles of plant-based analogues, Tegl explained.

The next generation of plant-based products will increasingly focus on alt seafood, the CEO predicts, such as smoked, raw fish. However, these products are not remotely close to the nutritional profile of alt meat products on the market.

If we are truly going to swap from conventional meat to alternative protein products, but those offerings dont provide the nutritional value of meat, we will have a huge issue on our hands and nutritional deficiencies.

This is where Arkeon plans to make a difference. The start-up wants to collaborate with vegan start-ups to ensure their products protein content is up to scratch, without compromising on consistency or taste. That is something our protein ingredients can deliver.

Arkeon is looking to commercialisation in Europe and the US, but revealed Singapore is also on its radar.

Its ingredient is classified as a Novel Food under EU law, meaning that regulatory approval will need to be sought before marketing its ingredient on home soil. While it is classified as a novel food, its not because of the ingredients were producing because they are well known.

Its simply due to the microbe were using, which has not been used in food production before.

It may well be that because Arkeons technology does not rely on genetic engineering, and if it can prove its end product is not contaminated with the cells DNA, the process is straightforward.

That is not to say that Arkeon is against genetic engineering. Thanks to current efficiencies, the start-up wont look at incorporating the technology in the short- to mid-term, but wouldnt be against genetically programming its microbe in the future. For a protein ingredient, its simply an incredibly efficient thing to do, and not harmful.

Scale is another challenge, and one that almost all novel fermentation-based protein start-ups are facing. Arkeon is currently transitioning into a 150L bioreactor and expects that spending time identifying its scaling criterion and understanding its bottlenecks will pay off.

Concerning price parity, the start-up believes it can undercut the cost of equivalent proteins by half. When producing at full scale, the start-up expects its product will retail for around 7 per kg of dry weight.

FoodNavigator also queried Tegl about consumer acceptance. Will consumers be willing to eat amino acids produced in a lab? Fermentation has been used in food production for millennia, it's one of the most natural ways to produce protein,"he explained. By the way, when consuming beer, youre not thinking about the steel tanks its produced in.

What is crucial for fermentation start-ups is that fermentation in general is somewhat in vogue, whether it be in the form of kombucha, sauerkraut, or kefir. The process is increasingly understood by consumers, and if its tasty and healthy, the start-up doesnt foresee any issues.

If we do encounter any problems in consumer acceptance in the future, its because we, as an industry, didnt spend enough time educating customers about the product.

Consumer acceptance will also come from how the ingredient will feature on-pack. While this is something that will be decided in the regulatory approval process, Tegl had a preference for cultured protein source. I think thats a very tangible term, and one wed like to follow.

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How Arkeon Biotechnologies is turning CO2 into food: 'Excuse my language, but this is next-level cool' - FoodNavigator.com

SYNB2081 is the second clinical drug candidate developed through the partnership between Ginkgo and Synlogic

CAMBRIDGE, Mass. and BOSTON, Aug. 11, 2022 /PRNewswire/ -- Synlogic, Inc. (Nasdaq: SYBX), a clinical-stage biotechnology company developing medicines for metabolic and immunological diseases through its proprietary approach to synthetic biology, today announced a new drug candidate for the treatment of gout developed in partnership with Ginkgo Bioworks (NYSE: DNA), the leading horizontal platform for cell programming. The new candidate, SYNB2081, is a Synthetic Biotic and is the second product to advance to clinical development through a research collaboration between Synlogic and Ginkgo, following the investigational new drug candidate SYNB1353 for the potential treatment of homocystinuria (HCU).

Gout is a complex form of inflammatory arthritis that occurs when excess uric acid in the body forms crystals in the joints. Patients experience symptoms such as intense joint pain, inflammation and redness, and limited range of motion in the affected joints. Current treatment options present limitations in both safety and efficacy, highlighting a need for new approaches. In addition, gout is a recognized risk factor in chronic kidney disease. SYNB2081 is a Synthetic Biotic designed to lower uric acid.

"With our second drug candidate into clinical development, this not only demonstrates the value of combining Ginkgo's platform with our Synthetic Biotic platform, but also highlights the potential to develop Synthetic Biotics across a range of diseases, giving us the potential to provide meaningful new treatment options to patients in need," said Dr. David Hava, Chief Scientific Officer, Synlogic.

SYNB2081 is named after one of the largest and best-preserved Tyrannosaurus rex specimens in the world. Nicknamed "Sue," the specimen is housed at the Field Museum in Chicago and is officially named FMNH PR 2081. Data from "Sue" suggests that dinosaurs like the Tyrannosaurus rex suffered from gout much in the same way as other reptiles and birds do.

"The advancement of SYNB2081 and SYNB1353 are clear indicators of the transformative platform Synlogic has created to develop new Synthetic Biotics through synthetic biology," said Patrick Boyle, Head of Codebase for Ginkgo. "We're honored to work with the Synlogic team in this pioneering next step to potentially help patients living with gout. As we've seen the Synlogic pipeline develop over the past year, we're eager to continue supporting Synlogic in generating additional therapeutic candidates."

About Synlogic

Synlogicis a clinical-stage biotechnology company developing medicines through its proprietary approach to synthetic biology. Synlogic's pipeline includes its lead program in phenylketonuria (PKU), which has demonstrated proof of concept with plans to start a pivotal, Phase 3 study in the first half of 2023, and additional novel drug candidates designed to treat homocystinuria (HCU) and enteric hyperoxaluria. The rapid advancement of these potential biotherapeutics, called Synthetic Biotics, has been enabled by Synlogic's reproducible, target-specific drug design.Synlogicuses programmable, precision genetic engineering of well-characterized probiotics to exert localized activity for therapeutic benefit, with a focus on metabolic and immunologic diseases. In addition to its clinical programs,Synlogichas a research collaboration with Roche on the discovery of a novel Synthetic Biotic for the treatment of inflammatory bowel disease. Synlogic has also developed two drug candidates through a research collaboration with Ginkgo Bioworks: SYNB1353, designed to consume methionine for the potential treatment of HCU, and SYNB2081, designed to lower uric acid for the potential treatment of gout. For additional information visitwww.synlogictx.com.

About Ginkgo Bioworks

Ginkgo is building a platform to enable customers to program cells as easily as we can program computers. The company's platform is enabling biotechnology applications across diverse markets, from food and agriculture to industrial chemicals to pharmaceuticals. Ginkgo has also actively supported a number of COVID-19 response efforts, including K-12 pooled testing, vaccine manufacturing optimization and therapeutics discovery. For more information, visit http://www.ginkgobioworks.com.

Forward-Looking Statements of Synlogic

This press release contains "forward-looking statements" that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, clinical development plans, future financial position, future revenue, projected expenses, prospects, plans and objectives of management are forward-looking statements. In addition, when or if used in this press release, the words "may," "could," "should," "anticipate," "believe," "look forward," "estimate," "expect," "intend," on track," "plan," "predict" and similar expressions and their variants, as they relate to Synlogic, may identify forward-looking statements. Examples of forward-looking statements, include, but are not limited to, statements regarding the potential of Synlogic's approach to Synthetic Biotics to develop therapeutics to address a wide range of diseases including: inborn errors of metabolism and inflammatory and immune disorders; our expectations about sufficiency of our existing cash balance; the future clinical development of Synthetic Biotics, including SYNB2081; the approach Synlogic is taking to discover and develop novel therapeutics using synthetic biology; and the expected timing of Synlogic's clinical trials of SYNB1618, SYNB1934, SYNB1353 and SYNB8802 and availability of clinical trial data. Actual results could differ materially from those contained in any forward-looking statements as a result of various factors, including: the uncertainties inherent in the clinical and preclinical development process; the ability of Synlogic to protect its intellectual property rights; and legislative, regulatory, political and economic developments, as well as those risks identified under the heading "Risk Factors" in Synlogic's filings with the U.S Securities and Exchange Commission. The forward-looking statements contained in this press release reflect Synlogic's current views with respect to future events. Synlogic anticipates that subsequent events and developments will cause its views to change. However, while Synlogic may elect to update these forward-looking statements in the future, Synlogic specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Synlogic's view as of any date subsequent to the date hereof.

Forward-Looking Statements of Ginkgo Bioworks

This press release contains certain forward-looking statements within the meaning of the federal securities laws, including statements regarding the potential success of the partnership and Ginkgo's cell programming platform. These forward-looking statements generally are identified by the words "believe," "can," "project," "potential," "expect," "anticipate," "estimate," "intend," "strategy," "future," "opportunity," "plan," "may," "should," "will," "would," "will be," "will continue," "will likely result," and similar expressions. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. Many factors could cause actual future events to differ materially from the forward-looking statements in this press release, including but not limited to: (i) the effect of Ginkgo's business combination with Soaring Eagle Acquisition Corp. ("Soaring Eagle") on Ginkgo's business relationships, performance, and business generally, (ii) risks that the business combination disrupts current plans of Ginkgo and potential difficulties in Ginkgo's employee retention, (iii) the outcome of any legal proceedings that may be instituted against Ginkgo related to its business combination with Soaring Eagle, (iv) volatility in the price of Ginkgo's securities now that it is a public company due to a variety of factors, including changes in the competitive and highly regulated industries in which Ginkgo operates and plans to operate, variations in performance across competitors, changes in laws and regulations affecting Ginkgo's business and changes in the combined capital structure, (v) the ability to implement business plans, forecasts, and other expectations after the completion of the business combination, and identify and realize additional opportunities, (vi) the risk of downturns in demand for products using synthetic biology, (vii) the unpredictability of the duration of the COVID-19 pandemic and the demand for COVID-19 testing and the commercial viability of our COVID-19 testing business, (viii) changes to the biosecurity industry, including due to advancements in technology, emerging competition and evolution in industry demands, standards and regulations, and (ix) our ability to close and realize the expected benefits of pending merger and acquisition transactions. The foregoing list of factors is not exhaustive. You should carefully consider the foregoing factors and the other risks and uncertainties described in the "Risk Factors" section of Ginkgo's quarterly report on Form 10-Q filed with the U.S. Securities and Exchange Commission (the "SEC") on May 16, 2022 and other documents filed by Ginkgo from time to time with the SEC. These filings identify and address other important risks and uncertainties that could cause actual events and results to differ materially from those contained in the forward-looking statements. Forward-looking statements speak only as of the date they are made. Readers are cautioned not to put undue reliance on forward-looking statements, and Ginkgo assumes no obligation and does not intend to update or revise these forward-looking statements, whether as a result of new information, future events, or otherwise. Ginkgo does not give any assurance that it will achieve its expectations.

SYNLOGIC MEDIA CONTACT:Bill Berry Berry & Company Public Relations 212-253-8881; [emailprotected]

SYNLOGIC INVESTOR CONTACT:Andrew Funderburk Kendall Investor Relations 617-914-0008; [emailprotected]

GINKGO BIOWORKS INVESTOR CONTACT:[emailprotected]

GINKGO BIOWORKS MEDIA CONTACT:[emailprotected]

SOURCE Ginkgo Bioworks

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Synlogic Announces Synthetic Biotic for Gout Developed in Partnership with Ginkgo Bioworks - PR Newswire

President Ayub Khan looking at the glow of the nuclear reactor at PINSTECH through a special viewer in a water pool in the mid-1960s. (Courtesy: Ayub Khan Archives/ Tahir Ayub)

SCIENCE matters. Many yearn for science-free times when wars were fought with swords by valiant Ertugrul-like horsemen. Quite a few want still earlier riyasats. But I have yet to meet a fellow Pakistani willing to have a bad tooth pulled out without anaesthesia or who sends emissaries instead of using a cellphone.

These days, electricity and gas loadshedding have triggered a collective nervous breakdown, while the price of petrol is all that people talk about. All of this would be utterly incomprehensible to those who lived a mere hundred years ago. Ancient civilisations had nothing even remotely similar to the science that exists today.

Like it or not, all modern science that which is rapidly changing our world on a day-to-day basis is the 400-year-old child of European modernity. Although many civilisations Egyptian, Babylonian, Chinese, Indian, Greek and Arabian (chronologically ordered) helped create that science, not enough was known earlier to create an overarching picture of a universe run by physical law. Nor did earlier civilisations use science to create functional technologies like we do today. Instead, significant advances in ancient science came from men of genius following scholarly interests rather than economic ends.

But now that civilisation on earth has become science-based, the pursuit of science is systematic and relentless. Every country is rushing to acquire mastery over it and, even more, to use it to create technologies to fulfil social desires. Although science and technology (S&T) are two different worlds, the boundary between them has blurred with time. For example, learning how cells divide was considered pure science in the 1800s. Today, it is crucial to discovering cures for cancer.

There is little appreciation in Pakistan for the centrality of science in every modern economic pursuit. Pervez Hoodbhoy deplores the degradation of our scientific capabilities and wonders whether we can change our worldview.

An attempt to situate Pakistans S&T may be made using two different lenses; to compare todays situation with what existed in 1947 (and even earlier); and to draw parallels between Pakistan and other countries in the region. As a starting point, I will take the advent of modern education in India (as opposed to traditional education) because that is where the bifurcation between modern and conventional ways of life began.

Pre-partition situation

India during the Mughal rule saw spectacular achievements in architecture, art and administrative matters. But there was little curiosity in matters of the intellect, particularly science and philosophy. As a result, no university was built in those three centuries of otherwise brilliant rule. Although internal feuds and succession issues were doubtless a significant cause of decline, this lack of interest in intellectual pursuits eventually led to 40-50,000 Englishmen, armed with technology and the scientific method, overpowering and crushing what had been a magnificent empire. Few understood the secret source of English power better than Mirza Ghalib. Differing from those who craved a return to past glories or who suggested picking up arms against the firangis, his thinking was quintessentially modern:

Go, look at the sahibs of England; Go learn from them their skills and ways; From their hands have sprung wonders and wonders; Go try and see if you can excel them.

Science education in British India was spread by three principal agents: British government, Christian missionaries, and education reformers from both Hindu and Muslim communities. Whereas the Hindus had many well-known reformers, among the Muslims the only well-known one was Sir Syed Ahmad Khan. His vigorous advocacy of science and modernity as a means of uplifting Indian Muslims differed sharply from those who feared learning English and science would diminish their religious faith. He had disagreed with Ghalib earlier, but, upon reflection, he became convinced that Indias Muslims must abandon conservatism and travel new paths.

Sir Syeds heroic efforts notwithstanding, Muslim enrolment in schools remained low. The University of Calcutta was the first secular Western-style university in India, and set standards as far away as Punjab. The requirements being rigorous by the standards of the time, only a few Muslims applied or qualified for admission. Although the populations in Bengal were proportional in size, hundreds of Hindus but just two Muslims passed the first BA examination in 1858.

Early years

Let us fast-forward to 1947. Of the 16 universities in British India, Pakistan inherited only one teaching university, i.e. the Punjab University in Lahore. Additionally, there were some 25-30 colleges in the areas that are now Pakistan. Most were in Punjab; Balochistan had none. Because Muslims had entered academia late and in fewer numbers, the senior faculty in almost all institutions of higher learning was predominantly Hindu at the time of partition. Once rioting began, they fled to India and Muslims from lower ranks filled their positions. Academic quality plummeted.

With time, education numbers slowly increased. By 1969 there were a total of eight universities in united Pakistan. The breakup and subsequent emergence of Bangladesh in 1971 temporarily froze further development. However, the quick post-partition promotions of junior faculty had profoundly debilitating consequences in terms of teaching quality. Mediocres rose to become department heads, deans, and vice-chancellors. They blocked bright young entrants lest their authority was challenged. As a result, rote learning became almost as common in universities and colleges as in schools and seminaries.

Nevertheless, in Pakistans early years, there were pockets of excellence in some S&T fields. I will mention only four.

Pakistans space programme began in 1961 with the launch of meteorological rockets provided by the United States. Initiated and headed by Professor Abdus Salam, the Space & Upper Atmosphere Research Commission (Suparco) grew rapidly in the 1960s and was more advanced than the Indian programme at the time.

Pakistans nuclear programme was set in place with the assistance of the US and, until 1972, had been directed towards nuclear power production and basic research. Personnel in the Pakistan Atomic Energy Commission (PAEC) were sent abroad in the 1960s for training. Canada provided Pakistans first nuclear reactor, the Karachi nuclear power plant (Kanupp). The returnees successfully maintained and operated the reactor even after the withdrawal of Canadian fuel and technical support. Indias 1974 nuclear test led to Pakistans open desire to match the Indian bomb, causing the reversal of the Wests nuclear assistance.

In industrial engineering, there was one outstanding institution, the Batala Engineering Company. Founded by entrepreneur C.M. Latif, Beco had relocated itself to Lahore from Batala (in what is now Indian Punjab) after partition. Beco produced a diverse range of heavy and light engineering products, such as diesel engines, machine tools and lathes. Like Indias Tata Industries, it was well set on the path of high growth, but was killed by the wave of nationalisation in 1972.

The creation of Islamabad University in 1967, and in particular the Institute of Physics associated with it, was the high point of academic research in Pakistan. Founded by Riazuddin, a student of Professor Salam, the institute maintained high-quality research in the frontier area of particle physics until its decline in the mid-1970s. At its peak, it compared favourably against a mid-quality physics department in the US.

Assessing the present

Globalisation means no country produces more than a fraction of what it needs and consumes. The more vibrant ones produce relatively more, have higher living standards for more citizens, are better organised, and have cleaner environments. Pakistan also has these aspirations, but is far more reliant on technologies developed elsewhere, such as automobiles, locomotives, aircraft, pharmaceuticals, computers, medical instrumentation, etc.

In principle, a small ecosystem could have developed around imported technologies, but there has been insufficient improvisation and innovation. For example, the once flourishing domestic electric fan industry has been pushed out by cleverer Chinese products. The small domestic output of finished products has led to a staggering trade imbalance that has compounded over time, leading to the current economic crisis.

I have attempted to compare Pakistans S&T in 2022 with other countries in the region based on performance in various domains of science, but the attempt admittedly is qualitative and subjective because a proper methodical study does not exist (Table 1).

Agri-sciences: These aim at raising yields of sugar, cotton, wheat, rice, and other crops by adapting and promoting standard techniques of pesticide use, plantation patterns, sowing methods, etc. As highly practical and relatively simple sciences, they are offshoots of the 1960s Green Revolution and are crucial for feeding Pakistans rapidly expanding population.

Nearly a dozen Pakistani institutions, such as National Institute for Biotechnology and Genetic Engineering (NIBGE), seem to have significantly improved local production and have reportedly developed better varieties of cotton, wheat, rice, tea and various fruits. Drip irrigation, food processing, and scientific livestock management are low-cost, but high-return investments.

Defence technology: Pakistan manufactures fission nuclear weapons and intermediate-range missiles. For both, the basic templates were provided by China, but local manufacturing capabilities had to be developed. The JF-17 fighter and Al-Khalid tank, produced with Chinese collaboration, are now force mainstays. In the 1980s, France provided three Agosta-90B submarines that were serviced locally. Over time a burgeoning Pakistani arms industry developed that now turns out a range of weapons from grenades to tanks, night vision devices to laser-guided weapons. However, the website of the Defence Export Promotion Organisation reveals little of what is being offered for sale. Pakistani arms exports have reportedly stalled in recent years. Poor quality control and lack of innovation are said to be responsible.

Space programme: Suparco has had six decades to mature, but as far as space exploration goes, it has practically folded up. The official website shows no future plans. Instead, it seems to have settled for routine testing of variants of missile series acquired from China. India, on the other hand, has clocked several major achievements, such as two successful orbiter missions to the Moon (2008) and one mission to Mars (2013). In 2017, India launched a record 100 satellites into orbit from the Indian Polar Space Launch Vehicle.

Civilian technology: Pakistans top 10 exports in 2021 were textiles, cotton, cereals, copper, fruits, minerals, sports goods, leather goods, software, and medical instruments. Only the last two items rely on S&T. As of 2020, the last year for which data is available, Pakistans hi-tech exports were 70 times lower than Indias and 2,523 times lower than Chinas (Table 2; the last entry is from the Mundi Index, which defines hi-tech exports as products with high research and development [R&D] intensity, such as in aerospace, computers, pharmaceuticals, scientific instruments and electrical machinery).

The above, however, understates the use of S&T in Pakistans domestic industrial production, which hinges critically upon imported machinery. This is used to produce textiles, Pakistans most important export, as well as cement, vegetable oil, fertiliser, sugar, steel, machinery, tobacco, paper, chemicals and food processing. Imported machinery has created an industrial ecosystem, but finished goods imported from China have adversely impacted many small industries.

Academic research: In developed countries, universities are the engines of scientific progress. Working in tandem with the industry, they help create new products and processes. On the other hand, in developing countries with small industrial bases, universities and colleges are primarily useful in creating a large pool of skilled people who can be gainfully employed in various sectors of the economy.

Irrespective of what area of science a student chooses, the key point that can make a graduate valuable is adaptability. A broad range of interests and knowledge and a good understanding of subject basics enables the students to be useful in different kinds of jobs.

Very few Pakistan institutions have done well at this. Hence, employers in the Middle East generally hire Pakistanis at lower levels relative to Indians, Iranians and Bangladeshis. Leaving aside the imported Cambridge system, rote-centred learning has discouraged students from logical thinking and stunted their cognitive capacities. The mathematical abilities of students and their teachers are generally poor. The only exceptions in the indigenous education system are exceptionally bright students at the right end of the Bell curve.

The poor quality of graduates emerging from Pakistani universities has caused employers to lose trust in grades and degrees. Many with PhDs are all but illiterate in their fields and unable to answer simple questions. At the same time, the number of publications produced by students has skyrocketed. Towards the end of studentship, many are credited with more papers than professors in the 1970s would have published over their lifetimes.

Professors and their students, encouraged by a disastrous policy by the Higher Education Commission (HEC) to reward publication numbers, have created a system where at least 90 per cent of so-called research papers are faulty, trivial or plagiarised. Whereas Chinese, Indian and Iranian speakers are invited to deliver lectures at top US campuses, Pakistans hyper-productive professors are nowhere to be seen there. Still, international university ranking organisations pick up numerical data and use their computers to create misleading rankings.

What not to do

The degradation in Pakistans scientific capabilities is alarming. Just how far Pakistan has fallen into the pit of ignorance and self-delusion was illustrated by a self-styled engineer trained in Khairpurs polytechnic institute who claimed to have invented a water kit that would extract energy from water. Never mind that this violated the rules of thermodynamics, and the rest of the world couldnt do it. He promised a new Pakistan with limitless energy, no need for petrol or gas, and no more loadshedding.

Politicians and media stars can perhaps be excused for being jubilant. But even our famed scientists fell for it and praised the water car publically. No practical joker could have demonstrated more dramatically the true state of science in Pakistan.

In this situation, one needs to carefully think about what to do, and, even more importantly, what not to do.

First, Pakistan does not need any more bricks and mortar for science; there is plenty of that around. A drive along Islamabads Constitution Avenue is lined with Pakistans most important buildings: Presidency, Prime Ministers House, Supreme Court, National Library, etc. On the other side of the road stand science buildings bearing names such as Pakistan Academy of Sciences, Pakistan Science Foundation, Islamic Academy of Sciences, Pakistan Council for Science and Technology, Committee on S&T of Organisation of Islamic Countries (Comstech), Commission on S&T for Sustainable Development in the South (Comsats) and others. A short distance from the Presidency is the head office of the PAEC, the largest single science-based institution in the country. About two miles away, on the campus of Quaid-i-Azam University is the National Centre for Physics (NCP).

Were any or all of these grand buildings to vanish suddenly into thin air, the world of science would simply shrug its shoulders. Shiny new cars parked in their driveways radiate opulence a tragic waste of resources. So-called science incubators in various cities have also proved ineffective. These were supposed to create new products for industry and business as well as new ideas for the world of academia. Nothing is visible. Do we need to spend more money doing this? Can we not understand that chickens may need incubators, but ideas hatch inside the head?

Second, we need to see through the numbers game that was started by the HEC in 2002, and immediately dispense with it. This game had deceived Pakistanis into believing that scientific research had increased when, in fact, the opposite happened.

More research papers and PhDs, and more universities and institutes do not at all translate into actual progress unless certain requirements are met. The most important of these are academic integrity and accurate assessment of scientific worth. As a result of incentivising corruption through cash rewards for papers and grants of PhD degrees, integrity has precipitously declined.

The way forward

The state of science in Pakistan, 75 years down the line, is visibly poor. There is little public understanding of science, our exports are largely low-tech textiles and raw materials, all significant weapons systems are imported, the space programme has almost ceased to exist, and scientific research carried out in universities and institutes carries little credibility or usefulness.

It is futile to blame a particular government; between one government and another, there has been little difference. The collective worldview, or weltanschauung, is at the core of the failure. This grim situation should energise us to drastically change our course. This must begin with changing the content and quality of education, beginning at the school level and then upward.

Instead of stuffing minds with propaganda, the goal must be to enhance cognitive capacity and creativity. How this can be done is well known: we can simply copy one of many successful countries. Attitudes acquired in school carry over to all higher levels colleges, universities, research institutes, and every other organisation. Good education encourages questioning and seeking answers. Traditional education, on the other hand, lulls the mind into passivity by endless memorisation and repetition.

As they say, to make an omelette, you must first break an egg. That egg, in Pakistans context, is the traditional value system that clashes with the value system of modernity and science. Pakistan hungers for the fruits of science, but a massive upsurge of zealotry has rendered it attitudinally unfit for nurturing science. Unlike its products, science cannot be acquired without accepting the fundamental premise of strict objectivity and, above all, the scientific method. Yes, it is as plain as that take it or leave it.

The author is an Islamabad-based physicist and writer.

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THE SAD STORY OF THE REJECTION OF SCIENCE - Sp Supplements - DAWN.COM - DAWN.com

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Story continues

What are the Current Market Drivers?

Rising investments in Genome Editing Technologies Governments of numerous nations throughout the world have made large investments in genomics in recent years, which have aided in the development of novel genome editing technologies. Furthermore, the availability of government financing has allowed academic and government institutes to conduct extensive genome editing/engineering research. For instance, in March 2020, Genome Canada received US$ 15 million from the Ministry of Innovation, Science, and Industry (Science) to support 11 genomic research initiatives in the health, agricultural, and environment sectors. Provincial governments, industries, and research partners will contribute a total of US$ 29.7 million to these research projects. The projects involve ovarian and cervical cancer research. The number of genomics research initiatives has increased significantly as a result of major government investments in this sector boosting the genome editing technologies market's growth over the forecast period.

The rise in the incidence of cancer and infectious diseases

Cancer incidence rates are predicted to rise from 20 million new cases per year in 2020 to more than 30 million new cases per year by 2040. Genome editing technologies provide new opportunities in fundamental cancer research and diagnostics, with advantages such as simple design, rapid operation, low cost, and robust scaling, introducing CRISPR/Cas as a rapidly evolving editing technique that is applicable to almost all genomic targets. Several genome editing techniques, including zinc finger endonuclease (ZFN), transcription activator-like effector nuclease (TALEN), and the clustered regularly interspaced short palindromic repeats/CRISPR associated nuclease (CRISPR/Cas) system, have been developed to provide efficient gene editing for the treatment of cancers, infectious diseases, and genetic disorders

Where are the Market Opportunities?

CRISPR Cas9 Technology to widen its applicationCRISPR-Cas9 is one of the most significant discoveries of the twenty-first century. Since its inception in 2012, this gene-editing technology has transformed biology research, making illness research easier and medication discovery faster. The technique is also having a substantial influence on crop development, food production, and industrial fermentation operations. CRISPR-Cas9 technology has huge potential in the pharmaceutical business. Scientists are tackling CRISPR-Cas technology, testing its possibilities and limits as a medical tool. It is being tested for treating diseases in humans such as cancer, blood disorders, blindness, AIDS, and genetic disorder such as Cystic fibrosis, hemophilia, -thalassemia, Alzheimer's, Huntington's, Parkinson's, tyrosinemia, Duchenne muscular dystrophy, Tay-Sachs, and fragile X syndrome disorders.

Competitive LandscapeThe major players operating in the Genome Editing Technologies market are Thermo Fisher Scientific Inc., Merck KGaA, GenScript, Sangamo Therapeutics Inc., Lonza, Editas Medicine, CRISPR Therapeutics AG, Agilent Technologies Inc., Precision Biosciences, and Tecan Life Sciences. These major players operating in this market have adopted various strategies comprising M&A, investment in R&D, collaborations, partnerships, regional business expansion, and new product launch.

Recent Developments

In April 2022, Thermo Fisher Scientific introduced the new GMP-manufactured Gibco CTS TrueCut Cas9 Protein. TrueCut Cas9 proteins are manufactured with United States Pharmacopeia standards in mind, including traceability documentation, aseptic manufacturing, and safety testing.

In February 2022, CRISPR Therapeutics, a biopharmaceutical company focused on developing transformative gene-based medicines for serious diseases, and ViaCyte, Inc., a clinical-stage regenerative medicine company developing novel cell replacement therapies have collaborated to address diseases with significant unmet needs, announced the first patient has been dosed in the Phase 1 clinical trial of VCTX210 for the treatment of type 1 diabetes (T1D)..

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Global Genome Editing Technologies market is projected to grow at a CAGR of 15.96% by 2032: Visiongain Reports Ltd - Yahoo Finance

Photo credit: Julielangford, CC BY-SA 3.0 , via Wikimedia Commons.

Earlier this year, a popular evolution YouTuber, Gutsick Gibbon, or Erika, createda video responseto my post here atEvolution News, Do Statistics Prove Common Ancestry? I had reviewed a paper by Baum et al. (2016), Statistical evidence for common ancestry: Application to primates, and how it presents a flawed and weak argument for separate ancestry that ignores the possibility of common design.

Erika is currently pursuing her Masters of Research in Primate Biology, Behavior and Conservation and is the creator of hundreds of punchy, entertaining YouTube videos. Her channels primary focus seems to be debunking Darwin-skeptics. Unfortunately, she does not seem to apply an equally critical eye to evolutionary theory.

While Erika confidently affirms the conclusions of Baum et al. (2016) in multiple videos here,here, andhere her responses do not negate the arguments raised in my initial post.

Before going further I want to remind you that intelligent design (ID) is compatible with both common ancestry and non-common ancestry views. Some of my colleagues here at Discovery Institute support common ancestry while others (like myself) are more skeptical. Thats OK! We all agree that there is evidence for design in nature. Some of us skeptics are interested in exploring potential models where ID and non-common ancestry histories of life intersect. Design does not rise or fall with these models, but they are interesting questions to explore.

Erikasfirst critiquecan be summarized as a complete misunderstanding of ID proponents objection to the paper. We will deal with that in a post tomorrow.

Hersecond critiqueis that ID proponents shouldnt expect others to test their models, but should test the models themselves. Anyone is welcome to test ID concepts if they like, but I dont think that ID proponents were expecting Baum et al. (2016) to test the hypothesis of separate ancestry. Rather, the paper carried out the normal scientific process where one group of scientists tests another groups scientific hypothesis independently. Only, in this case they tested a hypothesis no one supports more on that later. Perhaps most important, ID proponents are involved in testing models of separate ancestry and the exampleherewas provided in the original post.

Herthird critiqueresponded to my key point there are two known mechanisms (design and ancestry) that can produce genetic similarity. Therefore, genetic similarity should not always be used to provide exclusive support for ancestral relatedness when other explanations are possible.

To elaborate on her third critique, Erika argues that there is no genetic demarcation or separation that would mark a stopping place for comparison between species and higher orders of phyla. She is clearly ignoring reproductive barriers here. While I dont think this argument addresses my bolded point above, I am quite curious what she imagines this stopping point would look like if in fact separate ancestry were correct? I speculate that in such cases people expect the only evidence for a discontinuity in biological relatedness would be a vastly different genetic code for each organism or species. This seems to me a false expectation, because human technology shows that even separately designed structures can have deep similarities that go down to their very blueprints or encoding information. Given that, a design hypothesis would lead us to expect functional similarities. I would also say that there are reasons that a good design would make use of a highly similar genetic code for all organisms.

In this part of her argument, Erika also discusses Last Thursdayism. She says because a seemingly hierarchical ancestral pattern exists, if separate ancestry were correct, the designer must be deceptive to leave us such a pattern. In case you arent familiar with Last Thursdayism, it is a concept that a creator or God could make things look a certain way (billions of years old for example) even if he had created everything last Thursday. While I agree there are problems with Last Thursdayism, Last Thursdayism isnt relevant in this case. There are straightforward reasons to expect some degree of tree-like patterns even in a non-common-ancestry-related dataset.

If the seemingly deceptive pattern exists for a functional reason and has a good design explanation, then there really isnt a deceptive pattern. The deceptive pattern is imposed only by materialist lenses and a poor understanding of functional reasons for the similarities.

To summarize the problems with her third critique,as emphasized in myoriginal post, we know and observe two mechanisms that can result in genetic similarity.Design is one (think genetic engineering) and ancestry (think reproduction) the other.Becausetwoknown mechanisms exist to produce genetic similarity, that means,in and of itself, that genetic similarity does not provide evidence for ancestral relatedness. Certain patterns of genetic similarity may do this, but a design pattern, which isnt randomness, was not considered in the Baum et al. (2016) paper and isnt being considered by many in the academic community. Thats what ID proponents are trying to change.

Erikasfourth critiqueis that Winston Ewerts dependency graph(Ewert 2018)is not an actual model of separate ancestry. Winstons central thesis is that the nested hierarchical pattern observed in subsets of genes is better accounted for by a dependency graph. Erika acknowledges this is outside of her field, but she quotes Joshua Swamidass to dismiss it as a model. Ill talk more about her specific points in a later post.

Finally, Erikaslast pointis to address my argument that Baum et al. (2016) cherry picked which genes they would use when constructing their phylogeny: they only used genes they claimed were phylogenetically informative, which could imply a stacked deck. She really did not address my argument and instead made a comment about orphan genes.

I did not feel that Erika provided evidence for how (experimental) or why (conceptual) common design could not result in genetic similarities between species. Instead, there is evidence of design-dependent genetic similarity exploding all around me. I see it in the artificial selection for dogs (breeding for specific traits). I performed it myself in the lab using recombinant DNA technology. And I see it being dreamed about for the future as bringing about incredible advances in human health using CRISPR-Cas9. These are all proof-of-principle examples that design can and does produce genetic similarity in different organisms. Because this mechanism is well established, when we observe genetic similarity, we cant refuse to include design in the conversation.

In my next post I will explain why I think Gutsick was confused about the objection I raised previously to the separate ancestry model in the Baum et al. (2016) paper and attempt to explain the ID position more clearly.

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I Got Critiqued by YouTuber Gutsick Gibbon - Discovery Institute