Category Archives: Gene therapy

CBER maps modernization plan to handle surge in research and applications.

FDAs Center for Biologics Evaluation and Research (CBER) is updating how it manages a growing volume of cellular and gene therapy development programs, seeking added resources and revisions in its oversight of these cutting-edge therapies. Most visible in the elevation of CBERs Office of Tissues and Advanced Therapies (OTAT) into a new super Office of Therapeutic Products (OTP). The change aims to improve functional alignment, increase review capabilities, and add expertise on new cell and gene therapies by establishing multiple branches and divisions in the expanded regulatory unit, as announced in the Federal Register on Sept. 28, 2002.

Stated goals are to help CBER address the substantial growth in innovative, novel products that present new scientific, medical and regulatory challenges that require changes to its structure, including strategies to advance the Regenerative Medicine Advanced Therapy (RMAT) program. The added resources are needed to oversee more than 2000 development programs involving cellular and gene therapies, many involving innovative testing and manufacturing processes. This soaring workload has over-taxed CBER staffers, resulting in serious difficulties in retaining and hiring capable scientists.

The structural changes at CBER reflect agreed-on plans to hire new staffers with funding from recently reauthorized user fee programs. The PDUFA VII commitment letter calls for an additional 132 new hires for CBER in this coming year and another 48 employees the following year, most to support cell and gene therapy reviews at OTP. The reorganization plan calls for OTP to have seven officesfor therapeutic products, clinical evaluation, review management, pharmacology/toxicology, and two for CMCfor gene therapy and for cellular therapy and human tissues. There will be 14 divisions and 32 branches within those offices, providing attractive supervisory opportunities for both new and experienced staffers.

These changes come in the wake of FDA approval of two new gene therapies that have drawn wide attention for both their therapeutic potential and for million-dollar price tags. Bluebird bios Zynteglo was approved by FDA in August for patients with beta thalassemia, an inherited blood disorder causing serious anemia. That was followed a few weeks later with approval of Bluebirds Skysona to treat a rare neurological disorder afflicting young boys. Zynteglo carries a $2.8 million price tag, Skysonas list price is $4 million, but both therapies are expected to target fewer than 1500 patients, limiting the overall cost impact for the US healthcare system. A greater spending effect would come from FDA approval of a new treatment for sickle cell disease from Vertex Pharmaceuticals and CRISPR Therapeutics, which plan to begin a rolling review by FDA in the coming months. The important potential benefits of these treatments, along with concerns about their impact on healthcare spending and access, speaks to the need for a highly capable and sufficiently resourced FDA oversight program.

These developments also highlight the importance of sound testing and production methods for therapies made from living organisms, which are inherently variable and difficult to control and measure to assure product safety, identify, quality, purity, and strength. The surge in applications from a broad range of firms, moreover, has made it difficult for CBER staffers to schedule formal meetings with each sponsor seeking advice on how best to perform manufacturing and testing processes. And publishing new guidance on these changing and emerging issues also takes time and resources.

In response, FDA looks to engage a broad range of sponsors on topics related to product development through a series of virtual town hall meetings. The first was held Sept. 29, 2022 and addressed how manufacturers should describe and inform FDA about chemistry, manufacturing, and controls (CMC) in applications for gene therapies. Wilson Bryan, OTAT (now OTP) director, opened the session by describing plans for establishing OTP as a super office to increase review capabilities and enhance expertise on gene and cellular therapies and set the stage for OTP branch chiefs to field a broad range of queries, ranging from basic CMC policies for various stages of development, to the scope of potency assays and impact of delivery devices on dose potency and quality [a recording of the town hall meeting is available at the FDA events link].

Main topics were comparability testing, assays for product characterization, and process controls. OTP staffers emphasized the importance of determining process requirements early in development to avoid late changes and analytical method variability that could raise uncertainties likely to delay clinical trials. Products with complex mechanisms of action, they advised, stand to benefit from early product characterization and potency assay development. And developers of gene therapies should use multiple production lots during a clinical study to ensure product consistency and quality, even for treatments for very small patient populations.

Manufacturers raised questions about differing CMC issues between early Phase I and late-stage clinical trials and voiced concerns about product characterization related to autologous cell-based gene therapies. A main theme from FDA was the importance of sponsors establishing a well-controlled manufacturing process and qualified analytical testing well before administering any new gene product. While CBER plans to issue guidance on manufacturing changes and comparability for cellular and gene therapy products, the information provided at this session provides unofficial guidance for implementing changes in product manufacturing and the scope of comparability assessments and development studies expected to support such changes.

Jill Wechsler is Washington editor for Pharmaceutical Technology.

Excerpt from:
FDA Expands Oversight of Cell and Gene Therapies - Pharmaceutical Technology Magazine

Gene therapy has a history of presenting possibilities that stay out of reach for a long time. The tantalizing idea of using exogenous good DNA to replace defective DNA was suggested by Stanfield Rogers in 1970. Then, in 1972, the idea was elaborated upon by Theodore Friedmann and Richard Roblin, who wrote that viruses could be tweaked to contain human genes and allowed to infect patients. Once copied into patients cells, the genes could start to function, compensating for defective and disease-causing genes.1

Despite these conceptual advances, clinical progress was slow. Indeed, there were dead ends and reversals. In 1971, Rogers deployed a naturally occurring virus in an attempt to treat an arginase deficiency. And in 1980, Martin J. Cline tried to treat b-thalassemia by using an ex vivo procedure in which bone marrow cells were transfected with a recombinant human globin gene and then reintroduced to patients. Both these efforts were, at best, inconclusive.

Finally, a partial and temporary gene therapy success was reported in 1990. Scientists led by William French Anderson used an ex vivo procedure to treat a four-year-old girl suffering adenosine deaminasedeficient severe combined immunodeficiency disease. They infected the patients own white blood cells with a virus that had been engineered to carry a gene encoding a functional variant of the adenosine deaminase gene. For two years, transfusions were administered that incorporated transfected white blood cells. The transfusions didnt bring about a cure, but they did help reduce the patients symptoms.

Then, in 1999, the field suffered a major setback when an 18-year-old patient with a metabolic disorder died after suffering an immune overreaction to an adenovirus designed to restore a missing liver enzyme. And a few years later, several patients with immunodeficiencies developed leukemias after receiving gene therapy, as the viruses caused insertions into cancer-related genome sites. The U.S. Food and Drug Administration (FDA) reacted swiftly, putting many gene trials on hold.2 The development of gene therapy stalled.

In subsequent years, however, researchers learned from these setbacks. For instance, safer viral vectors were identified, such as adeno-associated viruses (AAVs). The genes they deliver typically remain in the cell cytoplasm and are expressed there, rather than being integrated into human cells genomes, making them less likely than some earlier vectors to trigger cancer.

Since 2017, the FDA has approved several gene therapies for disorders caused by defects in single genes, including Luxturna for retinal dystrophy, Zolgensma for young children with spinal muscular atrophy, and Zynteglo for certain patients with b-thalassemia. The agency has also green-lighted several cell-based gene therapies which alter patients cells and reinfuse them into patients. For example, approvals have been granted to several therapies that use modified T cells, specifically, chimeric antigen receptor (CAR) T cells. They have proven effective in treating certain blood cancers.

Meanwhile, hundreds more gene therapy trials are underway. To get a sense of what these trials tell us about the current status and near-term future of gene therapy, GEN spoke with representatives of companies at various stages of clinical development. They took the opportunity to expand on the results they shared at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), which was held last May in Washington, DC. They emphasized that for many diseaseshereditary monogenic disorders, complex diseases, and even cancersingle-dose gene therapies held disease-modifying potential.

The New York Citybased Lexeo Therapeutics has been developing a treatment for Friedreichs ataxia (FA), a rare condition that is currently incurable. Its caused by a mutation in the frataxin gene FXN which leads to progressive degeneration of the nervous system. Rather than targeting the diseases neurological pathology, which is tricky as the viruses fail to transduce efficiently and specifically in affected brain areas, Lexeo tackles the oft-fatal cardiac disease associated with FA, said Jay Barth, MD, Lexeos executive vice president and chief medical officer.

Lexeos therapeutic, LX2006, employs an AAV thats effective at infecting cardiac cells, introducing the FXN gene, increasing frataxin levels, and thereby restoring mitochondrial function. According to data presented at the ASGCT conference, mouse models of FA that received a single intravenous dose of LX2006 had improved heart function, general mobility, and survival compared with untreated rodents, even after they developed fairly advanced cardiac disease, Barth noted.3

Lexeo is planning a Phase I/II trial to assess safety of the therapy in 10 FA patients with cardiomyopathy. One of the goals is to identify the maximum safest dose for LX2006.4 Investigators are taking a cautious approach, Barth said, as overexpression of frataxin has been associated with safety issues.

The first study cohorts will receive the lowest dose thats shown efficacy in mice, and the dose will be incrementally increased in subsequent groups. Frataxin levels will be monitored through heart tissue biopsies. Patients will be followed for one year, and then for an additional four years as the FDA requires. In Barths view, the study could help find some way to prolong the lives of these patients beyond what the disease would give them.

While many gene therapy companies focus on restoring lost functions to normal cells, the Australia-based immuno-oncology company Imugene is employing the method to help kill cancer cells. In 2019, the company acquired an oncolytic virus called CF33, a chimeric vaccinia that infects and selectively replicates in malignant solid tumor cells.5

Imugene scientists have tinkered with CF33 in various ways that are already being tested in patients with specific cancer types. But according to Leslie Chong, the companys chief executive officer and managing director, the crown jewel of Imugene is a version of CF33 that contains a gene encoding the CD19 protein.

This surface protein is expressed on B cells and is the target of several CAR T-cell therapies. Using CF33 to induce uniform expression of CD19 across tumor cells could make CAR T-cell therapy work against solid tumors, which has proven a challenge as the tumors often express a heterogeneous mix of cell surface antigens. But with CF33, Chong explained, We line all your solid tumor [cells] with the CD19-directed targets, such that when you add a CD19-targeted therapy, you then obliterate the solid tumor where it hasnt had markers before.

In 2020, scientists at the City of Hope National Medical Center published data in support of this approach.6 Specifically, the scientists used mouse models of various cancer types to study the effects of administering the CD19-carrying CF33 virus followed by CD19-directed CAR T-cell therapy. Mice that received the antigen-matched therapy survived significantly longer than mice that received only mock T cells or CD19-CAR T cells.

Imugene looks forward to identifying indications that may benefit the most from this onCARlytics approach. The company is also planning a human trial. In our initial in-clinic study, we will be focused on certain indications, Chong noted. However, I think the application could be huge.

The North Carolinabased gene therapy company Asklepios BioPharmaceutical (AskBio) is also pursuing a target that falls outside the usual paradigm of monogenic disorders: congestive heart failure (CHF), a chronic and progressive condition in which the heart cannot pump blood sufficiently. Theres a high unmet medical need to develop additional medicines to reduce mortality and improve quality of life for the patients, said Canwen Jiang, MD, PhD, AskBios chief development officer and chief medical officer.

AskBios approach to tackling this complex disease has been to deliver a gene encoding the phosphatase-1 inhibitor-1, a key protein in regulating cardiac contractions. Introducing the gene via an AAV thats engineered to target cardiac cells could improve heart function as well as reverse and prevent the detrimental remodeling of cardiac muscle that occurs in CHF, Jiang said. The therapy, NAN-101, is delivered via a one-time injection into the hearts coronary arteries.

After collecting robust preclinical data, AskBio launched a Phase I study in 2019, enrolling eight individuals with Class III CHF. According to preliminary results presented at the ASGCT meeting, investigators observed efficient transduction of NAN-101 in heart cells of one trial participant from whom a tissue biopsy could be obtained.7 A cohort consisting of three patients who had completed their 12-month follow-up appeared to tolerate the treatment well and saw consistent improvements in heart function.8

If successful, such studies will not only motivate AskBio to expand into broader CHF indications, but also bolster the idea that gene therapy is useful beyond monogenic disorders. It would be a confidence-building example for the industry, for the academic community, as well as for the regulatory agencies, Jiang said.

One of the companies at the Phase III stage is Sarepta Therapeutics, a Cambridge, MAbased biotech firm specializing in rare diseases such as Duchenne muscular dystrophy (DMD), a monogenic disease that causes progressive muscle deterioration.

The gene therapy SRP9001 is based on an AAV virus subtype with an affinity for reaching muscle cells. It contains a gene encoding a form of the dystrophin protein, which is lacking in DMD patients tissues, coupled with a promoter that causes selective expression in skeletal and cardiac muscle cells, explained Jake Elkins, MD, Sareptas senior vice president of research and development and chief medical officer.

In a pilot study that tracked four DMD patients aged four to seven, data was collected four to five years after SRP9001 was taken. The treatment was well tolerated, and patients showed a 7-point improvement on a 17-step mobility scale (the North Star Ambulatory Assessment), despite being at an age where theyd typically experience rapid deterioration of mobility.9 A randomized Phase II study of 41 pediatric participants has so far bolstered these observations at one year of follow up, Elkins noted.10

Currently, Sarepta is closely tracking 120 boys with DMD aged four to seven in a Phase III study. The first half of patients are receiving gene therapy in the first year, during which the second half will receive a placebo until being rolled onto gene therapy after one year. At one year, were able to document clinically meaningful effects of the therapy, Elkins said. We view it as a confirmatory study of our early findings, but [it] will really expand our knowledge base of how this treatment works across the range of ambulatory patients with DMD.

Ultragenyx Pharmaceutical, a California-based rare disease-focused company, has also reached the Phase III stage with DTX401, which tackles glycogen storage disease type IA (GSDIa). This condition is caused by a genetic deficiency of the enzyme glucose-6-phosphatase, which breaks down glycogen reserves into glucose during fasting periods. GSDIa causes low blood sugar and accumulation of glycogen in the liver and kidneys, and patients need to regularly take cornstarch to maintain normal blood sugar levels, explained Eric Crombez, MD, Ultragenyxs chief medical officer for gene therapy and inborn errors of metabolism.

DTX401 is based on a liver-targeting AAV designed to restore glucose-6-phosphatase expression in patients hepatocytes. According to results presented at the ASGCT meeting, a Phase I/II study of DTX401 reported only mild adverse events in adult GSDIa patients.11 And all 12 participants were able to reduce their daily cornstarch intake by around 70% over three years. When interviewed at 52 weeks, most of the patients reported having more energy and better mental clarity.

If the transgene wasnt working, they would be having a lot of problems, Crombez asserted. [We can see that] they dont, [which] shows that weve established the normal breakdown of glycogen to produce glucose.

Motivated by these results, the company launched a Phase III study in 50 patients to compare the efficacy of DTX401 to that of a saline infusion. Primary endpointsincluding patients ability to taper cornstarch usewill be assessed after 48 weeks, but investigators hope to follow patients for as long as possible.

As the liver has high cell turnover, and the therapy doesnt integrate into the genome, the transgene will eventually be lost, Crombez said. Thats why he doesnt describe gene therapy as a cure in the strictest sense. However, he emphasizes that even if you need [another] dose 20 years down the road, [youve still] treated it for a very long period of time.

References1. Friedmann T, Roblin R. Gene Therapy for Human Genetic Disease? Proposals for genetic manipulation in humans raise difficult scientific and ethical problems. Science 1972; 175(4025): 9499552. Pollack A. FDA halts 27 gene therapy trials after illness. New York Times. Published January 15, 2003.3. Zuluaga CM, Gertz M, Yost-Bido M, et al. Identification of the Therapeutically Beneficial Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friederichss Ataxia. Paper presented at: 25th Annual Meeting of the American Society of Gene and Cell Therapy; May 1619, 2022; Washington, DC.4. Lexeo Therapeutics. LEXEO Therapeutics Announces FDA Clearance of Investigational New Drug Application for LX2006, an AAV-Based Gene Therapy Candidate for Friedreichs Ataxia Cardiomyopathy. Published February 16, 2022.5. Imugene. Today we enhanced our portfolio with a compelling oncolytic virus technology. Published July 15, 2019.6. Park AK, Fong Y, Yang SK, et al. Effective combination immunotherapy using oncolytic viruses to deliver CAR targets to solid tumors. Sci. Transl. Med. 2020; 12(559): eaaz1863.7. Tretiakova AP, Ozkan T, Sethna F, et al. Rationally designed cardiotropic AAV capsid demonstrates 30-fold higher efficiency in human vs. porcine heart. Paper presented at: 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), Washington, D.C., May 16-19, 20228. Henry T, Chung ES, Egnaczyk GF, et al. A first in-human phase 1 clinical gene therapy trial for the treatment of heart failure using a novel re-engineered adeno-associated vector. Presented at: 25th Annual Meeting of the American Society of Gene and Cell Therapy; May 1619, 2022; Washington, DC.9. Sarepta Therapeutics. Sarepta Therapeutics Investigational Gene Therapy SRP-9001 for Duchenne Muscular Dystrophy Demonstrates Significant Functional Improvements Across Multiple Studies. Published July 6, 2022.10. Sarepta Therapeutics. Sarepta Therapeutics SRP-9001 Shows Sustained Functional Improvements in Multiple Studies of Patients with Duchenne. Published October 11, 2021.11. Ultragenyx Pharmaceutical. Ultragenyx Announces Positive Longer-Term Durability Data from Two Phase 1/2 Gene Therapy Studies at American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting. Published May 19, 2022.

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Gene Therapy Hits Its Stride in the Clinic - Genetic Engineering & Biotechnology News

With transformative treatments that leverage CRISPR technology, there are many challenges to overcome throughout the journey of development through to patient access. We interviewed a researcher at a top 20 pharma company, multiple patients, and our own market access experts for their insights into the process, obstacles, and opportunities for biopharmaceutical companies to meet patient needs and achieve success in the market.

Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. In the last decade, there has also been rapid development and interest in CRISPR/Cas9 technology and other gene-editing tools that could offer transformative avenues to deliver gene therapies for patients and families affected by devastating diseases such as sickle cell disease and thalassemia.

Beta-thalassemia is a rare blood disorder caused by a genetic defect in hemoglobin. Several manufacturers are developing novel treatments for the disease, including Vertex, which has partnered with CRISPR Therapeutics to develop a gene-editing treatment for beta-thalassemia and sickle cell. In October 2022, the companiesannounced their plan to file exagamglogene autotemcel, the CRISPR/Cas9- edited therapy, for a rolling review with the FDA. Novartis also recently inked an up-to$1.5 billion deal with Precision Biosciences to support its development of one-time treatments for beta-thalassemia and sickle cell. The move marks a continuation in its ongoing commitment to exploring gene-editing technology.

Despite the surge of interest from both scientific and financial stakeholders, there remain numerous unique challenges at every step of this new frontier. From developing and financing the therapy in a timely manner to market access challenges due to the lack of maturity and extensive evidence, and explaining the complex science to physicians and patients, there are a variety of hurdles to overcome before bringing this type of technology to market. And while regenerative and curative therapies are often greatly lauded, there are also challenges in treating affected patient populations, including cost, accessibility, side effects, and other associated risks.

Earlier this year, the US Food and Drug Administration (FDA)approved the first potentially curative gene therapy to treat beta-thalassemia. While multiple companies have been working to bring transformative technologies such as CRISPR to market, approval will require further consideration around the many concerns and challenges of each stakeholder at every step. From the research and development stage all the way to patient access and treatment, remaining considerate of the populations that will ultimately be the end-users and testament to the technologys success is vital.

Diagnosed with beta-thalassemia at six months old, Amar has had regular blood transfusions every few weeks for the majority of his life. As a child, he found himself falling behind others his age. I always felt I was playing catch-up, through school, work, lifealways making up for lost time, he says.

Now in his 40s, he reflects on both the physical and mental impacts of the disease. He says, As Im getting older, my body is deteriorating, and it raises concerns for my future and what my life expectancy will look like. The disease has triggered other health conditions, including osteoarthritis, hypogonadism, and diabetes. Its a constant challenge, both physically and mentally, he says.

Continued here:
The promised land of gene therapy: Commercialization of novel gene-editing technology in beta-thalassemia - PMLiVE

Two years ago, a mother received a phone call with a devastating diagnosis. That mother shares her son's experience with spinal muscular atrophy (SMA), a rare genetic disease, and how early diagnosis and treatment transformed his life.

BANNOCKBURN, Ill., Oct.12, 2022 /PRNewswire/ --

BACKGROUND:

Hannah Weaver did not think much about her son Payne's newborn screening test until she received a phone call five days after bringing him home from the hospital. The results showed Payne tested positive for spinal muscular atrophy (SMA), a rare, progressive neuromuscular disease and a leading genetic cause of infant death when left untreated that affects one in every 11,000 babies born in the U.S.1,2 SMA causes irreversible loss of motor neurons, which can rob infants of their ability to walk, swallow and even breathe.1,2 If left untreated in its most severe forms, 90% of children require permanent feeding and breathing support or pass away by their second birthday.3,4 SMA can progress quickly, making early diagnosis and treatment crucial.

Experience the full interactive Multichannel News Release here: https://www.multivu.com/players/English/9064751-novartis-spinal-muscular-atrophy-health-alert/

Knowing it was imperative to act fast, the Weaver family worked quickly to schedule appointments with specialists to discuss treatment options. Payne's care team went over the available treatment options for SMA, discussing the route of administration and available efficacy and safety data of each. Together, they decided to treat Payne when he was just a few weeks old with a gene therapy called ZOLGENSMA (onasemnogene abeparvovec-xioi), the only SMA treatment designed to directly address the genetic root cause of the disease by replacing the function of the missing or non-working SMN1 gene with a single, one-time dose.

Zolgensma has a boxed warning for acute serious liver injury and acute liver failure. In clinical trials, the most common side effects were elevated liver enzymes and vomiting. Please keep reading for additional important safety information and please see accompanying Full Prescribing Information.

Payne's parents are now able to plan for his future something that would not be possible without early intervention and treatment. They look forward to what he will accomplish next and celebrate every milestone along the way. On the heels of SMA Awareness Month, we kick off Newborn Screening Awareness Month this September, and Hannah is advocating for parents and physicians to recognize the early signs of SMA to avoid a delayed diagnosis.

Dr. Sandra Reyna (Vice President of Global Medical Affairs at Novartis Gene Therapies) and Hannah Weaver (SMA parent advocate) shared information on the signs of SMA, the importance of an early diagnosis, and how Zolgensma has the potential to transform the lives of babies born with this disease.

Results and outcomes vary among children based on several factors, including how far their SMA symptoms progressed prior to receiving treatment.

Please continue reading for Indication and Important Safety Information, and please see accompanying Full Prescribing Information including Boxed Warning.

For more information, please visit: http://www.Zolgensma.com

Interview opportunities are courtesy of Novartis Gene Therapies.

About Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular disease and a leading genetic cause of infant death.3,5Caused by the lack of a functional SMN1 gene, the most severe forms of SMA result in the rapid and irreversible loss of motor neurons, affecting muscle functions including breathing, swallowing and basic movement.1Severity varies across a spectrum of types corresponding to the number of copies of the back-up SMN2 gene.6The majority of patients with two copies of SMN2 develop Type 1, the most common form accounting for 60 percent of cases.4,7,8Type 1 is severe and, left untreated, leads to death or the need for permanent ventilation by the age of two in more than 90 percent of cases.3,5Most patients with three copies of SMN2 develop Type 2, accounting for about 30 percent of cases.4,8 Loss of motor neurons cannot be reversed, so it is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression.9,10

IndicationandImportant Safety InformationforZOLGENSMA(onasemnogeneabeparvovec-xioi)

Whatis ZOLGENSMA?

ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into a vein. ZOLGENSMA was not evaluated in patients with advanced SMA.

WhatisthemostimportantinformationIshouldknowaboutZOLGENSMA?

Whatshould Iwatchforbeforeandafterinfusion withZOLGENSMA?

WhatdoIneedtoknowaboutvaccinationsandZOLGENSMA?

DoIneedtotakeprecautionswiththepatient'sbodilywaste?

Temporarily, small amounts of ZOLGENSMA may be found in the patient's stool. Use good hand hygiene when coming into direct contact with bodily waste for 1 month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.

Whatarethepossible orlikelysideeffectsof ZOLGENSMA?

The most common side effects that occurred in patients treated with ZOLGENSMA were elevated liverenzymesandvomiting.

The safety information provided here is not comprehensive. Talk to the patient's doctor about anysideeffects thatbotherthepatientorthatdon'tgoaway.

You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 orwww.fda.gov/medwatch,orNovartis GeneTherapiesat833-828-3947.

Please see the Full Prescribing Information.

2022 Novartis Gene Therapies, Inc.

US-ZOL-22-0136

References

MEDIA CONTACT: Katie Lesch, [emailprotected]

SOURCE Novartis Gene Therapies

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Health Alert for Parents: How one boy is thriving following treatment with a gene therapy after receiving an early diagnosis - PR Newswire

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces data presentations at the 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Edinburgh, United Kingdom, taking place October 11-14, 2022. Presentations will include clinical data from Rockets lentiviral vector (LV)-based gene therapy programs for Leukocyte Adhesion Deficiency-I (LAD-I), Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD). Donald B. Kohn, MD, Distinguished Professor of Microbiology, Immunology & Molecular Genetics, Pediatrics, and Molecular & Medical Pharmacology at University of California, Los Angeles (UCLA) and Director of the UCLA Human Gene and Cell Therapy Program, will also give an Invited Talk incorporating previously disclosed data from the RP-L201 trial for LAD-I.

Positive Updated Safety and Efficacy Data from Phase 2 Pivotal Trial for Fanconi Anemia (FA)

The poster and presentation include updated safety and efficacy data from the Phase 2 pivotal trial of RP-L102, Rockets ex-vivo lentiviral gene therapy candidate for the treatment of FA.

Positive Top-line Clinical Data from Phase 2 Pivotal Trial for Severe Leukocyte Adhesion Deficiency-I (LAD-I)

The oral presentation includes previously disclosed efficacy and safety data at three to 24 months of follow-up after RP-L201 infusion for all patients and overall survival data for seven patients at 12 months or longer after infusion. RP-L201 is Rockets ex-vivo lentiviral gene therapy candidate for the treatment of severe LAD-I.

Interim Data from Ongoing Phase 1 Trial for Pyruvate Kinase Deficiency (PKD)

The poster and presentation include previously disclosed safety and efficacy data from the Phase 1 trial of RP-L301, Rockets ex-vivo lentiviral gene therapy candidate for the treatment of PKD.

Details for Rockets Invited Talk and poster presentations are as follows:

Title: Interim Results from an ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)Session: Clinical Trials (Plenary 2)Presenter: Donald B. Kohn, MD - University of California, Los Angeles, Distinguished Professor of Microbiology, Immunology & Molecular Genetics (MIMG), Pediatrics, and Molecular & Medical Pharmacology; Director of the UCLA Human Gene and Cell Therapy ProgramSession date and time: Wednesday, 12 October at 11:10-13:15 BSTLocation: Edinburgh International Conference Centre (EICC)Presentation Number: INV20

Title: Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Results from Global RP-L102 Clinical TrialsSession: Poster Session 1Presenter: Julin Sevilla MD, PhD - Fundacin para la Investigacin Biomdica, Hospital Infantil Universitario Nio JessSession date and time: Wednesday, 12 October at 19:30-21:00 BSTLocation: Edinburgh International Conference Centre (EICC)Poster Number: P139

Title: Preliminary Conclusions of the Phase I/II Gene therapy Trial in Patients with Fanconi Anemia-ASession: Blood Diseases: Haematopoietic Cell DisordersPresenter: Juan Bueren, PhD - Unidad de Innovacin Biomdica, Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT)Session date and time: Thursday, 13 October at 15:30-17:30 BSTLocation: Edinburgh International Conference Centre (EICC)Presentation Number: INV41

Title: Interim Results from an Ongoing Global Phase 1 Study of Lentiviral-Mediated Gene Therapy for Pyruvate Kinase DeficiencySession: Poster Session 2Presenter: Jos Luis Lpez Lorenzo, MD, Hospital Universitario Fundacin Jimnez DazSession date and time: Thursday, 13 October at 17:30-19:15 BSTLocation: Edinburgh International Conference Centre (EICC)Poster Number: P128

Abstracts for the presentations can be found online at: https://www.esgct.eu/.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Pyruvate Kinase Deficiency

Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 4,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was in-licensed from the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT), Centro de Investigacin Biomdica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacin Sanitaria de la Fundacin Jimnez Daz (IIS-FJD).

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rockets future expectations, plans and prospects, including without limitation, Rockets expectations regarding its guidance for 2022 in light of COVID-19, the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Danon Disease, the expected timing and data readouts of Rockets ongoing and planned clinical trials, the expected timing and outcome of Rockets regulatory interactions and planned submissions, Rockets plans for the advancement of its Danon Disease program and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rockets ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rockets dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rockets Annual Report on Form 10-K for the year ended December 31, 2021, filed February 28, 2022 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 29th Annual Congress of the European Society of Gene...

ST. LOUIS--(BUSINESS WIRE)--M6P Therapeutics (M6PT or the Company), a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage disorders (LSDs), today announced a poster presentation titled, A novel AAV9 gene therapy for producing -glucocerebrosidase enzyme with high mannose 6-phosphate content to treat Gaucher disease, by Lin Liu, Ph.D., Director, R&D of M6P Therapeutics, at the 29th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). The Congress takes place in-person and virtually in Edinburgh, Scotland from Oct. 11-14, 2022 at the Edinburgh International Conference Centre.

The data from this preclinical study illustrate the potential of our AAV-GBA-S1S3 gene therapy candidate for the treatment of Gaucher disease, including neuronopathic phenotypes, said Pawel Krysiak, President and CEO of M6P Therapeutics. M012 is part of our pipeline of candidates leveraging a first-in-class co-expression S1S3 platform technology for the treatment of lysosomal storage disorders. Using our technology platform, we enhance mannose 6-phosphate content on lysosomal enzymes for both enzyme replacement and gene therapies, leading to improved enzyme uptake across target tissues.

Gaucher disease is a rare inherited metabolic disorder of defective lipid catabolism caused by deficient -glucocerebrosidase (GCase) encoding by the GBA1 gene resulting in accumulation of glycosphingolipids in the periphery and central nervous system (CNS). Multiple recombinant human GCase enzyme replacement therapies have been approved to treat Gaucher disease type 1, but there are no effective treatments to address the neuronopathic manifestations for Gaucher disease types 2 and 3. Gene therapy could be a potential therapeutic approach to treat all three types of Gaucher disease.

"In this study, gene copy and transcriptome analysis showed that the dosed novel M012 AAV vector enabled transduction of peripheral tissues and various regions of brain in wild-type mice post-intravenous injection, said Dr. Liu. We observed increased GCase enzyme activity with enhanced CI-MPR affinity in peripheral tissues of treated animals and noted strong GCase protein signal with broad distribution detected in M012 AAV vector-treated brain samples. These results strongly suggest that M012 AAV enables effective GCase cross-correction in the CNS. We look forward to sharing additional updates and data as the program progresses.

Presentation highlights from the poster include:

About M6P TherapeuticsM6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted enzyme replacement and gene therapies for lysosomal storage disorders (LSDs). M6P Therapeutics proprietary co-expression S1S3 platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both enzyme replacement and gene therapies, leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product. This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases. M6P Therapeutics team, proven in rare diseases drug development and commercialization, is dedicated to fulfilling the promise of enzyme replacement and gene therapies by harnessing the power of protein phosphorylation using its co-expression S1S3 platform. M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community. For more information, please visit: http://www.m6ptherapeutics.com.

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M6P Therapeutics Presents Novel AAV Gene Therapy Approach for the Treatment of Gaucher Disease at the ESGCT 29th Annual Congress - Business Wire

Breaking News

Approvals of Zynteglo and Skysona mean that Lonzas Houston site now supports three commercial CGT products.

10.11.22

Lonza Houston is dedicated to contract cell and gene therapy development and manufacturing. The facility successfully passed Food and Drug Administration (FDA) pre-Licensing inspections for the viral vector manufacturing or for the cell therapy manufacturing of three commercial cell and gene therapy products that were subsequently approved by the FDA for commercial use in 2021 and 2022.

Lonzas Cell & Gene Technologies (CGT) business, the contract development and manufacturing business unit of its Cell & Gene division, has developed an NPI process to support customers on their journey from early-stage development to commercialization. It provides a roadmap and a systematic approach to development and manufacturing, ensuring necessary quality standards are met for tech transfers, cGMP manufacturing and pre-approval inspection readiness. This NPI process has been designed to support more CGT customers in reaching commercialization with their new and innovative products.

"The two new approvals represent an important milestone for bluebird bio and the patients who can now benefit from these therapies, said Alberto Santagostino, senior, head of cell and gene technologies. The successful approvals reflect our colleagues dedication to supporting our customers in bringing these cell and gene therapies to market. Opened less than five years ago, our Houston site is now manufacturing three commercial cell and gene therapies. We look forward to supporting more customers on their path to commercialization as we continue on our journey towards wider cell and gene therapy adoption."

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Two Cell and Gene Therapies Manufactured at Lonza Houston Reach FDA Approval - Contract Pharma

Exopharms technology has put the company in a leadership-position in both nave exosomes and engineered exosomes as it progresses to a product-first company.

Its been a challenging yet productive two years for the Exopharm (ASX:EX1) team.

The company has made significant progress over this period and now owns a tool chest of 7 important exosome manufacturing technologies, as well as well identifying two new in-house GM (genetic medicine) product programs.

At the centre of Exopharms is its proprietary manufacturing technologies, which enable the company to develop its own exosome-medicine products and harness their unique drug-delivery capabilities.

Preliminary results from a recently completed study detected no immunogenicity or toxicity of exosomes manufactured by Exopharm, which means they are suitable for clinical pursuit.

The study also helped to validate Exopharms exosomes as a safe drug-delivery chassis meaning they could be used as safe delivery vehicles inside a persons body to deliver drug doses at high frequencies.

Separate earlier clinical trials with the company Plexaris product also demonstrated safety and efficacy in wound healing using Exopharm-produced exosomes.

These results have now put Exopharm in a leadership-position in both nave exosomes and engineered exosomes.

Exosomes are naturally occurring nano-vesicles that are secreted in huge numbers by cells throughout the human body, and are a natural and powerful way that cells communicate and coordinate.

Within the last 7 years, researchers have envisaged using exosomes as delivery vehicles inside a persons body like programmable, targeted and safe nano-sized drones.

Drugs that had delivery problems, like mRNA, DNA and some small molecules, could be packaged into exosomes at a bioprocessing facility to produce modern medicines which were safer, more tolerable, and more specific to the target tissue/destination than their cargo alone.

Over the last few years, Exopharm had worked on developing the technology to manufacture these exosomes, and that led the market to value it as a platform technology company.

But now, the technology has developed significantly and Exopharm believes that market perception is outdated.

Exopharm emphasised that it is now a product-first company after announcing two GM programs under development.

The Exopharm team has assessed hundreds of potential exosome products over the past 15 months using strict assessment criteria and detailed commercial scoping.

From that work now comes two new programs with several potential product variants in each program.

The first lead program is treating Cystic Fibrosis (CF) using exosome-based additive CFTR mRNA gene therapy and nebuliser delivery to lungs.

CF is the most common autosomal recessive disease with >100,000 CF patients worldwide (around 40,000 in US) and the median age of death is 32 years. Lung disorder is the main cause of morbidity and mortality.

There is no cure for cystic fibrosis, but treatment can ease symptoms, reduce complications and improve quality of life.

Current treatment options include antibiotics, anti-inflammatory medications, mucus-thinners, bronchodilators, oral pancreatic enzymes and CFTR modulators.

Exopharm however believes that additive gene therapy is ideal for CF compared to some other genetic diseases.

The Exo-RNA approach, which is gene therapy delivered via exosomes, has several advantages which includes avoiding the use of viral vectors.

The Exo-RNA gene therapy approach also poses no risk of potential insertion into the host genome as the exosomes loaded with CFTR mRNA are formulated to be administered via a nebuliser (inhaler).

This approach is expected to be more efficacious and cost-effective, with no direct competitors identified.

The CF market is predicted to reach US$31 billion by 2027 with 24% compound annual growth rate (CAGR), up from US$5 billion in 2019.

Elastin is a natural molecule in our bodies produced from the ELN gene. Elastin imparts elasticity in various tissues such as skin, lungs, blood vessels and fascia.

With age and exposure to smoke, levels of elastin decrease and medical and aesthetic issues arise from that elastic deficiency.

An additive gene therapy using ELN mRNA would produce additional Elastin in the body and could bring the elastic tissue structure back to normal.

Exopharm has identified several main product opportunities in this program including Chronic obstructive pulmonary disease (COPD) such as pulmonary emphysema and chronic bronchitis.

Destruction of elastin or abnormalities in elastic fibre assembly are major factors in emphysema and COPD, and an estimated 3.1 million Americans have been diagnosed with emphysema and around 11.2 million people in the country have COPD.

Tobacco use is the number one factor in the progression of COPD, along with pollution, infections and genetic factors.

Other potential treatments in this program also include skin conditions and aesthetic dermatology.

Exopharm will also explore hypertension and arterial stiffness, scar prevention, and photoaging in its Elastin program.

Exopharm says these two programs have been selected after considering their potential future commercial value, and as a way to showcase exosomes as a non-viral nanoparticle chassis to overcome drug-delivery problems that GMs face.

With these two lead programs, the company believes its strategy and future-direction is clear and promising.

Exopharm believes there is an emerging opportunity to treat many medical problems with Genetic Medicines to use exosomes as the drug-delivery chassis for potentially hundreds of new products.

Exopharm can also profit from this opportunity by making its exosome technologies available to others on commercial terms.

The company can also generate revenue by developing a small number of its own products in order to build shareholder-value along the way.

This article was developed in collaboration with Exopharm, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.

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Exopharm identifies two new lead programs as exosomes gain prominence in gene therapy - Stockhead

DUBLIN--(BUSINESS WIRE)--The "Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market by Scale of Operation, Type of Vector, Application Area, Therapeutic Area, and Geographical Regions: Industry Trends and Global Forecasts, 2022-2035" report has been added to ResearchAndMarkets.com's offering.

With the increasing number of cell and gene therapies being developed and launched for a wide range of therapeutic areas, these modalities are on their way to become one of the highest valued markets in the biopharmaceutical domain. In fact, in 2021, cell and gene therapy developers raised capital worth more than USD 20 billion, registering an increase of 19% from the amount raised in 2020 (~USD 17 billion).

It is worth highlighting that, in February 2022, the USFDA approved second CAR-T therapy, CARVYKTIT, developed by Johnson and Johnson, which can be used for the treatment of relapsed or refractory multiple myeloma. Additionally, close to 1,500 clinical trials are being conducted, globally, for the evaluation of cell and gene therapies. Over time, it has been observed that the clinical success of these therapies relies on the design and type of gene delivery vector used (in therapy development and / or administration). At present, several innovator companies are actively engaged in the development / production of viral vectors and / or non-viral vectors for cell and gene therapies.

In this context, it is worth mentioning that, over the past few years, multiple viral vector and non-viral vector based vaccine candidates have been developed against COVID-19 (caused by novel coronavirus, SARS-CoV-2) and oncological disorders; this is indicative of lucrative opportunities for companies that have the required capabilities for viral vector manufacturing and gene therapy manufacturing.

The viral vector manufacturing and non-viral vector manufacturing landscape features a mix of industry players (well-established companies, mid-sized firms and start-ups / small companies), as well as several academic institutes. It is worth highlighting that several companies that have the required capabilities and facilities to manufacture vectors for both in-house requirements and offer contract services (primarily to ensure the optimum use of their resources and open up additional revenue generation opportunities) have emerged in this domain.

Further, in order to produce more effective and affordable vectors, several stakeholders are integrating various novel technologies; these technologies are likely to improve the scalability and quality of the resultant therapy. In addition, this industry has also witnessed a significant increase in the partnership and expansion activities over the past few years, with several companies having been acquired by the larger firms. Given the growing demand for interventions that require genetic modification, the vector and gene therapy manufacturing market is poised to witness substantial growth in the foreseen future.

Scope of the Report

The "Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market (5th Edition) by Scale of Operation (Preclinical, Clinical and Commercial), Type of Vector (AAV Vector, Adenoviral Vector, Lentiviral Vector, Retroviral Vector, Plasmid DNA and Others), Application Area (Gene Therapy, Cell Therapy and Vaccine), Therapeutic Area (Oncological Disorders, Rare Disorders, Neurological Disorders, Sensory Disorders, Metabolic Disorders, Musco-skeletal Disorders, Blood Disorders, Immunological Diseases, and Others), and Geographical Regions (North America, Europe, Asia Pacific, MENA, Latin America and Rest of the World): Industry Trends and Global Forecasts, 2022-2035" report features an extensive study of the rapidly growing market of vector and gene therapy manufacturing, focusing on contract manufacturers, as well as companies having in-house manufacturing facilities.

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

4. VIRAL VECTOR AND GENE THERAPY MANUFACTURERS (INDUSTRY PLAYERS): MARKET LANDSCAPE

5. PLASMID DNA AND GENE THERAPY MANUFACTURERS (INDUSTRY PLAYERS): MARKET LANDSCAPE

6. VECTOR AND GENE THERAPY MANUFACTURERS (NON-INDUSTRY PLAYERS): MARKET LANDSCAPE

7. VECTOR AND GENE THERAPY MANUFACTURING TECHNOLOGIES: MARKET LANDSCAPE

8. COMPANY COMPETITIVENESS ANALYSIS

9. VECTOR AND GENE THERAPY MANUFACTURERS IN NORTH AMERICA

10. VECTOR AND GENE THERAPY MANUFACTURERS IN EUROPE

11. VECTOR AND GENE THERAPY MANUFACTURERS IN ASIA-PACIFIC

12. OTHER KEY PLAYERS

13. RECENT PARTNERSHIPS

14. RECENT EXPANSIONS

15. STRATEGIC PARTNER ANALYSIS

16. EMERGING VECTORS

17. KEY INSIGHTS

18. COST PRICE ANALYSIS

19. CAPACITY ANALYSIS

20. DEMAND ANALYSIS

21. MARKET SIZING AND OPPORTUNITY ANALYSIS

22. PORTER'S FIVE FORCES ANALYSIS

23. KEY DRIVERS AND CHALLENGES

24. SURVEY ANALYSIS

25. CONCLUDING REMARKS

26. EXECUTIVE INSIGHTS

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/wg8tme

Excerpt from:
Viral Vector Manufacturing, Non-Viral Vector Manufacturing and Gene Therapy Manufacturing Market Report 2022 - ResearchAndMarkets.com - Business Wire

A biotechnology company launched Wednesday by life sciences venture firm ATP is the latest startup to debut with a new twist on genetic editing.

With $50 million in funding, Boston-based Ascidian Therapeutics claims its RNA exon editing approach could match the durability of gene therapy while avoiding some of the risks that come with editing DNA.

Its platform is designed to correct for mutations in exons the regions of DNA that contain information needed to make proteins. Ascidian aims to do this by replacing mutated exons with functional RNA copies as DNA is being converted into its chemical cousin.

The company will first target a genetic eye condition called Stargardt disease, which is the most common form of inherited macular degeneration and results in vision loss.

According to Ascidian, its technology can fix genetic errors that other editing approaches cant, and can be applied to widely varied genes. Its lead program can replace more than 20 exons at a time, said Romesh Subramanian, Ascidians CEO.

We are changing chapters in a book rather than whiting-out one letter at a time, Subramanian, said in an interview with BioPharma Dive. Subramanian came to Ascidian from Dyne Therapeutics, a biotech he founded and led as CEO. He previously founded RNA specialist Translate Bio, which was bought by Sanofi last year.

Subramanian claims that Ascidians approach, by focusing on RNA, maintains genome integrity and thereby sidesteps concerns around off-target edits. His company also doesnt rely on foreign enzymes to work, potentially easing immunogenicity risks, he added.

Along with Stargardt disease, Ascidian is looking at other eye conditions, neurological disorders and rare diseases. Subramanian declined to disclose how many drug research programs Ascidian plans to roll out.

Ascidians name is derived from a class of ocean-dwelling invertebrate creatures, which are sometimes known as sea squirts. These creatures use RNA trans-splicing to alter the RNA messengers used by their cells, a process that Ascidian plans to leverage to rewrite RNA for treating disease.

Ascidian is not ATPs first foray into genetic medicine. Last year, Ascidian co-founder and ATP venture partner Michael Ehlers, a former Biogen executive,launched a startup called Intergalactic Therapeuticsthat focuses on non-viral gene therapy. ATP has also built a company called Replicate, which is developing another kind of RNA medicine.

We think the RNA space is a big way of manipulating biology and treating disease across the board, and this approach we've taken to Ascidian defines a new class of RNA therapeutics, Ehlers said.

The company expects to spend the rest of 2022 and 2023 on pre-clinical studies for its lead program, along with developing proof of concept for other candidates targeting neurological and neuromuscular diseases.

Gene editing research was catalyzed by the discovery of CRISPR, which has now been extended and adapted to support several different gene editing technologies. But biotech companies are also exploring RNA editing, which in part appeals to scientists because it doesnt change the underlying DNA.

It has drawn in larger drugmakers, too: Roche and Eli Lilly have recently formed partnerships with Shape Therapeutics and ProQR Therapeutics, respectively, to develop treatments for a wide variety of diseases.

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Ascidian starts up with $50M and a twist on RNA editing - BioPharma Dive