Category Archives: Regenerative Medicine

May21, 20205 min read

Opinions expressed by Entrepreneur contributors are their own.

Education is the crucial step in opening up consumer marketplaces for hemp-based products.

CEO and co-founder of SilverLeaf Global, Gil Laureiros career is deep-rooted in identifying innovative products. His work also ensureseach is certified safe and effective prior to introducing the product into the market. Laureiro has been directly involved with guiding many products through the FDA clearance process. He is at it again.

A big believer in scientific, research-backed health benefits, Laureiro has immersed himself into hemp-based solutions.

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What many people dont realize is that the endocannabinoid system of all mammals vertebrates and invertebrates work the same, said Laureiro. His company SilverLeaf Global isthe foundational platform for NanoSol Pro and NanoSol Pet hemp-based products.

Our goal for ourselves and our furry friends are safe and verifiable products that promote our cells to work optimally, also called 'cellular optimization,'" says Laureiro. "We accomplish cellular optimization through cellular supplementation and thereby, minimize cellular disruption. Said differently, NanoSol formula is designed to optimize cellular activity by minimizing endocannabinoid deficiencies.

While many CBD producers have product lines for pets, Laureiro and his partners at NanoSol Pet set out at the onset to design and formulate hemp-based products intended to promote better health and lifestyle for our four-legged friends.

Laureiro says:We want to educate the public about the health benefits of CBD for our pets. A large portion of our website and marketing efforts are focused on just this.Research is still ongoing.

Green Entrepreneur spoke with SilverLeaf Global'sLaureiro on why focusing on pets was the company's next move, what humans and our pets have in common, and how to find proper dosing.

Green Entrepreneur:Why did you get into CBD? Why pets?

Gil Laureiro: My co-founders and I have spent years advancing healthcare solutions pertaining to both human and pet medical markets. We expanded our business strategy in adopting hemp-based products due to their ability to non-invasively and significantly contribute to cellular optimization. Our hemp product initiative is directly influenced by our business development experience and expertise in the FDA cleared field of non-invasive cellular regenerative medical device solutions launched into the healthcare market.

Both non-invasive cellular regenerative medical devices and hemp-based products contain similarity in that they provide diagnostic and therapeutic solutions to both the animal and human medicine and health optimization markets. Like humans, animals as scientifically proven contain an endocannabinoid system and suffer similar symptoms when the endocannabinoid system becomes deficient.

How does an animals endocannabinoid system work?

Both humans and animals contain anendocannabinoid system (ECS), comprised of cannabinoid receptors, endocannabinoid molecules, and their metabolic enzymes. Together they comprise a crucial molecular system that the body uses to help maintain homeostasis.

The endocannabinoid system is recognized as an important modulatory system that directly affects the function of the brain, endocrine, and immune tissues. Cannabidiol hemp oil acts through cannabinoid receptors located on cells throughout an animals body, which address deficiencies of the endocannabinoid system.

RELATED:CBD For Dogs Is The Next Big Thing In The Supplement Industry

Cannabinoids affect the pets body, as they contain receptors that are made to bind to specific cannabinoid molecules.

How is the animal endocannabinoid system different than humans? How are they the same?

The human and animal endocannabinoid system very similar regarding its intended function and symptoms experienced from a deficiency. Hemp oil supplementation health benefits similar to animals and very much the same for humans.

Humans often dont know if they are taking full spectrum or broad-spectrum hemp oil. Does this matter for animals?

Full Spectrum hemp oils, by definition, contain small traceamounts of THC and when the hemp is processed butthe THC present is not filtered out. Broad Spectrum processes hemp a step further to ensure THC is removed and therefore when tested, no trace amounts of THC are detectable. Science tells us that any tracible amounts of THC ingested are cumulative and may be harmful and therefore, pets should only take broad-spectrumhemp oil.

To ensure you are buying a non-detect THC product, a Certificate of Analysis (COA) should accompany the product and the COA must be provided by a certifiable independent testing laboratory.

We know dosing for humans can sometimes be a matter of trial and error. What about hemp oil dosing for animals?

Pet dosing is calculated by weight and dosing should be clearly suggested on the product label. No different than humans, observing your pet during the first few weeks of dosing helps you determine if the applied dose is working, or possibly the dosing should be increased or decreased.

RELATED:6 Convincing Reasons Why USDA Organic Certification For CBD Is The Trend To Watch

Do vets recommend hemp oil for pets?

Hemp oil recommendations by veterinarians vary from state to state. This said, there are a large number of veterinary practices that not only recommend hemp oil for pets butcarry the product in their practice and sell directly to their clients. Many studies are being conducted due to the empirical evidence already obtained, that supplementing with hemp oil may contain a number of animal health benefits. The most common being inflammation andpain reduction.

The more these studies are made public or published, the more veterinarians will recommend hemp oil supplementation.

Original post:
Education Is Key In Marketing Hemp Oil And CBD For Pets. Here's Why. - Green Entrepreneur

Back injuries are no joke. Even minor muscle strains can inhibit activities for some time. More serious injuries create greater challenges and often require more stringent treatments, which is where companies like VIVEX Biologics play a significant role.

At the end of April, VIVEX published clinical data assessing VIA Disc as a potential treatment for symptomatic degenerative disc disease. VIVEXs VIA Disc is a non-surgical,injectable treatment option for patients suffering from chronic lower back pain resulting from DDD. Degenerative disc disease is the most common cause of chronic lower back pain in adults, which is the leading cause of disability across the globe, with a cost of about $87.6 billion. There are few treatment options for DDD, with most patients having to use opioids to manage the pain or surgery for patients who cannot manage their disease with medication.

To address this gap in the continuum of care, VIVEX developed VIA Disc to offer patients a novel, non-surgical intervention for treating one to two levels of symptomatic DDD. In the paper, VAST Clinical Trial: Safely Supplementing Tissue Lost to Degenerative Disc Disease, which was published in The International Journal of Spine Surgery, trial data showed that VIA Disc improved pain and function in patients with DDD by greater than 70%, as measured by visual analog scale (VAS) and Oswestry Disability Index (ODI). Additionally, the data from the study showed that the results were durable one year following the treatment.

Peter Wehrly, chief executive officer of VIVEX, said the results are encouraging and validate the therapeutic potential of VID Disc in patients who are in critical need of treatment. The positive data from the study is exciting, particularly given the high, unmet need in this space, Wehrly told BioSpace in an interview. Not only is degenerative disc disease an area of unmet need, the number of patients is growing, Wehrly said. Currently, there are approximately 800,000 cases of DDD, but that continues to increase by about 20,000 each year, Wehrly said.

The study data will lead to the launch of an incremental trial that will follow patients over the course of three years. Wehrly said the data, which has so far looked promising, could prove to be enormously helpful to patients who are dealing with back pain day in and day out. Patients who have been treated with VIA Disc notice a difference almost immediately, he added.

Hopefully this helps patients for at least two years, he said, adding that he will be ecstatic should the studies support that.

Eventually, VIVEX will likely test potential re-administration of VIA Disc in patients who did benefit from an initial treatment. At this point though, Wehrly said the company has yet to delve into that possibility. He stressed that the company will take things one step at a time with VIA Disc as VIVEX has a goal to become the company in this space.

In order to support that goal, VIVEX has been planning an expansion of its footprint in Florida. The company plans to open a new 90,000 square foot facility this summer. It was a facility that VIVEXs employees helped design in order to make it as efficient as possible, Wehrly told BioSpace.

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VIVEX to Expand Footprint in Florida to Support Regenerative Medicine Technologies - BioSpace

To deal with the challenges in regenerative medicine, in 2018, pioneer bioprinting expert Anthony Atala announced the foundation of an organization dedicated solely to advancing the field through manufacturing. Known as the Regenerative Medicine Manufacturing Society (RMMS), it is the first collaborative effort that aspires to help deliver regenerative medicine products to the marketplace. In particular, the RMMS recently announced that working groups of researchers representing industry, government, academia, and non-profit organizations will identify current gaps and solution spaces in regenerative medicine to improve patient outcomes; and among the four impact areas they will investigate, is 3D bioprinting.

RMMS members will share their knowledge and work together to create solutions to manufacturing challenges. The ultimate goal is to develop standardized manufacturing processes to facilitate the smooth and quick transition of new therapies to market, thereby ensuring patient access.

The four impact areas that the societys leadership team has identified as critical in regenerative medicine are cell manufacturing; standards for regenerative medicine; 3D bioprinting; and artificial intelligence-enabled automation.

According to a paper published in Stem Cells Translational Medicine the official journal of the RMMS the working groups will help evaluate the current landscape of work already done in both regenerative medicine and related fields, including cell therapy manufacturing, as well as identify gaps, and propose solution spaces for the field to focus on. One of the targets of their upcoming work will be the development of safety and quality industry standards specifically for regenerative medicine manufacturing.

More precisely, and as far as 3D bioprinting is concerned, the RMMS working groups will look into the development of bioink standards and assist with understanding some of the regulatory hurdles of creating combination products, such as implants consisting of a scaffold and living cells.

Emerging as a powerful tool for building tissue and organ structures, the use of 3D bioprinting technology and the development of user-friendly software platforms have grown tremendously in the past five years. However, experts at the RMMS suggest that the advancement of out of the box customized bioinks that can be sold in tandem with this technology is lagging behind.

Furthermore, the RMMS paper hones in on some of the known materials that are being developed for use across different bioprinting platforms that have ultimately demonstrated a trade-off between printability and biological relevance, such as easier-to-print plant-based bioinks, or extracellular matrix (ECM) component bioinks that are more biologically relevant but more difficult to print.

To close this gap, the RMMS considers assisting organizations that are beginning to strategically circumvent these challenges, like ASTM International. Their work on bioink standards, including printability of bioinks and biomaterials, could be used in automated biofabrication technology, as well as guidance on the development of bioinks, including considerations such as material properties that assist with survival of cells within bioinks. All of this would ultimately lead to the development of new bioinks.

The second limiting factor that RMMS researchers wish to tackle is the regulatory hurdle of developing combination products. Using a 3D bioprinter to create implants consisting of a scaffold and living cells will need to go through review by both device and biologic regulatory agencies, which results in long and expensive regulatory approval pathways compared with the more traditional implants.

As many experts have suggested, regulation is a big issue in the bioprinting field. There are simply too many complex points to be considered with biomanufacturing and it appears that bioprinting research will always outpace the regulatory agencies ability to keep up; that is, unless the quality, risk and safety standards are clearly determined beforehand.

In that regard, the RMMS working groups will assist with understanding some of the regulatory challenges and technology gaps in this rapidly advancing area and highlight member-developed solutions.

RMMS researchers stated in the article that: We envision assisting with the advancement of commercial products with industry partners in this space based on recommendations from our working groups.

Moreover, some early clinical opportunities may include external regenerative medicine targets such as cell-based wound dressings.

Proposed as a collaborative work, the RMMS hopes to accelerate efforts in regenerative medicine manufacturing through partnerships with government, industry, and academia. The primary author of the paper and RMMS Executive Director, researcher Joshua Hunsberger, leads the group of researchers as they work with partners to identify areas where they can make an impact, as well as develop new education and training programs.

Joshua Hunsberger

Education and training are critical to RMMS goals as they expect to accomplish their mission through outreach and education programs and securing grants for public-private collaborations in regenerative medicine manufacturing. From expanding the current talent pool to promoting careers in regenerative medicine, education and training is key to adapt to the growing demands of the workforce in this emerging sector.

RMMS hopes to collaborate in developing learning networks across all levels of education, and in tune with their mission, identify gaps where they could assist in developing new training programs or expanding current ones. The areas they are considering are hands-on cell culture techniques, the latest manufacturing technology, analytical methods, and regulatory requirements.

As the worlds first professional organization dedicated solely to advancing the field of regenerative medicine through manufacturing, RMMS also serves as an information clearinghouse, supports professional and workforce development, and promotes the infrastructure for the fields success.

Back in 2018, during the RMMS inaugural meeting, founding member and director of the Wake Forest Institute for Regenerative Medicine (WFIRM), Atala, described how regenerative medicine therapies are already benefiting small groups of patients through clinical trials. Also revealing that while there is still much to accomplish scientifically, the field is at a tipping point. If we are going to bring high-quality, cost-effective therapies to patients, now is the time to begin the important work of developing the manufacturing processes. Collaboration between stakeholders is vital to success.

Certainly, RMMS is all about collaboration, focused on strengthening the framework of regenerative medicine by developing solutions to accelerate the commercialization of the latest technological advances and growing the workforce in a rapidly expanding sector. And with strong support from researchers coming together from the different branches that ultimately make up the field, it is difficult not to eagerly anticipate the results of this joint effort.

Continue reading here:
RMMS: The Regenerative Medicine Manufacturing Society Will Target Unmet Needs in 3D Bioprinting - 3DPrint.com

The demand within the global stem cell banking market is growing on account of advancements in the field of regenerative medicine. The medical fraternity has become extremely focused towards the development of artificial tissues that can infuse with the human body. Furthermore, medical analysis and testing has gathered momentum across biological laboratories and research institutes. Henceforth, it is integral to develop stem cell samples and repositories that hold relevance in modern-day research. The need for regenerative medicine emerges from the growing incidence of internal tissue rupture. Certain types of tissues do not recover for several years, and may even be damaged permanently. Therefore, the need for stem cell banking is expected to grow at a significant pace.

In a custom report, TMR Research digs into the factors that have aided the growth of the global stem cell banking market. The global stem cell banking market can be segmented on the basis of bank size, application, and region. The commendable developments that have incepted across the US healthcare industry has given a thrust to the growth of the North America stem cell banking market.

Global Stem Cell Banking Market: Notable Developments

The need for improved regenerative medication and anatomy has played an integral role in driving fresh developments within the stem cell banking market.

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Gallant has emerged as a notable market entity that has remained as the torchbearer of innovation within the global stem cell banking market. The company has recently launched stem cell banking for dogs, and has attracted the attention of the masses. As people become increasingly concerned about their pets, the new move by Gallant shall help the company in earning the trust of the consumers. Moreover, it can move several notches higher on the innovation index.

Cells4Life has also remained at the forefront of developments within the global stem cell banking market. After suffering backlash for its error in cord blood stem cell promotion, the company is expected to use effective public relation strategies to regain its value in the market.

Global Stem Cell Banking Market: Growth Drivers

Development of improved facilities for storage of stem cells has played an integral role in driving market demand. Furthermore, the unprecedented demand for improved analysis of regenerative medications has also created new opportunities within the global stem cell banking market. Medical research has attracted investments from global investors and stakeholders. The tremendous level of resilience shown by biological researchers to develop stem cell samples has aided market growth. Henceforth, the total volume of revenues within the global stem cell banking market is slated to multiply.

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Commercialization of stem cell banks has emerged as matter of concern for the healthcare industry. However, this trend has also helped in easy storage and procurement of cells stored during the yester years of children. Presence of sound procedures to register at stem cell banks, and the safety offered by these entities, has generated fresh demand within the global market. New regional territories are opening to the idea of stem cell banking. Several factors are responsible for the growth of this trend. Primarily, improvements in stem cell banking can have favourable impact on the growth of the healthcare industry. Moreover, the opportunities for revenue generation associated with the development of functional stem cell banks has aided regional market growth.

The global stem cell banking market is segmented on the basis of:

Source

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Stem Cell Banking Market - Need for Improved Regenerative Medication and Anatomy - BioSpace

The Indiana University School of Medicine established itself as a leader in regenerative medicine when it recruited Chandan Sen away from Ohio State University in 2018. (Photo courtesy of the IU School ofMedicine)

A dime-size nanochip developed by a world-renowned researcher who recently relocated to Indianapolis could help transform the practice of medicine. It could also turn Indianapolis into a manufacturing and research hub for radically new disease and trauma treatment techniques.

It all began in August 2018, when Chandan Sen, one of the worlds leading experts in the nascent field of regenerative medicine, moved his lab from Ohio State University to the Indiana University School of Medicine. He brought along a team of about 30 researchers and $10 million in research grants, and now serves, among a myriad of other positions, as director of the newly formed Indiana Center for Regenerative Medicine and Engineering, to which IU pledged $20 million over its first five years.

IU recruited Sen away from Ohio State in part because of its desire not just to promote academic research in his field but also to help develop practical, commercial products and uses for hisbreakthroughs.

A scientist prefers to be in the lab and keep on making more discoveries, said Sen, 53.

But I thought that, unless we participate in the workforce development process and the commercialization process, I dont think that the businesspeople would be ready to do it all by themselves. Because its such a nascentfield.

Its definitely newand its potential sounds like the stuff of science fiction.

Regenerative medicine, as its name hints, seeks to develop methods for replacing or reinvigorating damaged human organs, cells and tissues.

For instance, instead of giving a diabetic a lifetimes worth of insulin injections, some of his skin cells could be altered to produce insulin, curing him. Such techniques might also be used for everything from creating lab-grown replacement organs to, someday, regenerating severed limbs.

Regenerative medicine offers a form of medicine that is neither a pill nor a device, Sen said.

It is a completely new platform, where you dont necessarily depend on any given drug, but are instead modifying bodily functions.

A big, tiny breakthrough

Lambert

Sen and his teams signal contribution to the field is a technique theyve dubbed tissue nanotransfection, or TNT. Put simply, it uses a nanotechnology-based chip infused with a special biological cargo that, when applied to the skin and given a brief electrical charge, can convert run-of-the-mill skin cells into other cell types. Potentially, the technique could be used for everything from regrowing blood vessels in burn-damaged tissue to creating insulin-secreting cells that could cure diabetics.

Obviously, such applications are still down the road a ways. But the technology is far enough along that some products are already making it to marketand investors, entrepreneurs and established companies are sniffing around for opportunities. According to the Alliance for Regenerative Medicine, more than 1,000 clinical trials worldwide are using regenerative medicine technologies.

Thousands of patients are already benefiting from early commercial products, and we expect that number will grow exponentially over the next few years, said Janet Lambert, the alliances CEO.

Lambert predicts that the number of approved gene therapies will double in the next one to two years. Last year, the U.S. Food and Drug Administration predicted it would be approving 10 to 20 cell and gene therapies each year by2025.

Shekhar

These new techniques could do more than just revolutionize medicine. They could also upend the medical industry as we know it. And the IU School of Medicineand Indianapoliscould lead the way.

There are really only two or three places in the country that did the kind of comprehensive work that Dr. Sens group was doing, said Anantha Shekhar, executive associate dean for research at IU School of Medicine. And they were doing it from the lab all the way to the clinic, where they were already applying those technologies in patients.

So it was very attractive to think of starting with a bangbringing a comprehensive group here and creating a new center.

Ambitious goals

Instead of merely treating chronic conditions, regenerative medicine could end them, once and for all.

For instance, consider a car with an oil leak. The traditional medical approach might be to live with the chronic condition by pouring in a fresh quart of oil every few days. The regenerative medicine approach would fix the leak. Its good for the car, good for the cars owner but not necessarily good for the guy who was selling all those quarts of oil.

Which is why these new techniques, if they catch on, could cause turmoil in the medical industry.

Because regenerative medicine has the potential to durably treat the underlying cause of disease, rather than merely ameliorating the symptoms, this technology has the potential of being extremely disruptive to the current practice of medicine, Lambert said.

This has the potential to be hugely disruptive, Sen added, because so much of medicine today relies on huge industrial infrastructures to manage, not cure, chronic diseases and disabilities.

Coy

If such disruption comes to pass, the leaders of 16 Tech, a 50-acre innovation district northwest of downtown that aspires to house dozens of medical-related startups and established firms, would love to be its epicenter.

The Center for Regenerative Medicine will be one of the tenants of 16 Techs first building, a $30million, 120,000-square-foot research and office building scheduled to open in June.

Regenerative medicine is probably one of the next major waves of medical innovation in the world, 16 Tech CEO Bob Coy said. To have him here doing this work gives Indianapolis and Indiana an opportunity to develop an industrial cluster in regenerative medicine.

Coy believes the most momentous early step on that road was the recent establishment by Sen of masters and doctoral programs in regenerative medicine at the IU School of Medicine. Its the first degree of its type in the country, earning IU and Indianapolis the enviable status of first mover.

I think, for example, of [Pittsburghs] Carnegie Mellon University, which, back in the late 1960s, created the first college of computer science in the country, Coy said. And now you know Carnegie Mellons reputation in computerscience.

What isnt in place yet is a state or city program to promote development of a regenerative medicine hub.

We need to start doing that, Coy said. That means putting a lot of the infrastructure in place to support startups that are based on this technology, as well as recruiting companies that want to collaborate with Dr. Sen.

In spite of the lack of a coherent recruitment program, Coys phone has started to ring, thanks largely to Sens presence.

There have been a few meetings Ive had with people who already have relationships with him, who, when they come to town, have reached out to meet and talk about what were doing at 16 Tech, he said.

Fueling entrepreneurship

One of the first 16 Tech startups with designs on the regenerative medicine niche is Sexton Biotechnologies.

The company was groomed by Cook Regentec, a division of Bloomington-based Cook Group charged with incubating and accelerating technologies for regenerative medicine and the related field of cell genetherapy.

Any products that show promise are either folded into the company, turned into their own divisions or, as in Sextons case, spun off as an independent entity with Cook retaining a financial stake.

Werner

Its a measure of the newness of this field that Sextons 17 employees arent working on new medicines, but rather marketing basic tools needed to conduct research. The companys offerings include a vial for storing cell and gene products in liquid nitrogen, and a cell culture growth medium.

Theres a ready market for such tailor-made gear, because, for years, researchers in the regenerative medicine field had to make do with jury-rigged equipment.

What most of those companies did was repurpose things like tools from the blood banking industry, or tools from bio pharma, said Sean Werner, Sextons president.

So thats why a lot of newer companies are starting to build tools explicitly for the industry, as opposed to everybody just having to cobble together stuff that was already out there.

Werner said investors recognize the momentous opportunity in regenerative medicine and are flocking to the field.

Its not something you have to explain, he said. Companies and VC groups are trying to get a piece of it.

What has investors and medical researchers charged up is the almost unlimited range of potential applications, from healing burns to, perhaps someday, regenerating limbs.

I think it would be a huge revolution if were able to, for example, regenerate insulin-secreting cells in children who have become juvenile diabetics or have for whatever reason lost their pancreas, Shekhar said. Those are the kinds of things that will start to change the way we see certain diseases.

Lambert predicted that, as the science advances, so will the business case.

While early programs focused primarily on rare genetic diseases and blood cancers, were already seeing the field expand into more common age-related neurological disorders, such as Parkinsons and Alzheimers, shesaid.

I expect this trend to continue in the coming years, greatly increasing the number of patients poised to benefit from these therapies.

Werner said regenerative medicine also is seeking advancements in manufacturing technologies that will lower the cost of product development.

It all adds up to a huge opportunity the state is well-positioned to seize, Wernerbelieves.

Indiana is a perfect place for this kind of thing to really ramp up, he said. Theres no reason we cant lead thefield.

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IU team pursuing breathtaking advancements in regenerative medicine - Indianapolis Business Journal

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, May 11, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX001, an investigational, autologous, gene-edited hematopoietic stem cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

RMAT designation is another important regulatory milestone for CTX001 and underscores the transformative potential of a CRISPR-based therapy for patients with severe hemoglobinopathies, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. We expect to share additional clinical data on CTX001 in medical and scientific forums this year as we continue to work closely with global regulatory agencies to expedite the clinical development of CTX001.

The first clinical data announced for CTX001 late last year represented a key advancement in our efforts to bring CRISPR-based therapies to people with beta thalassemia and sickle cell disease and demonstrate the curative potential of this therapy, said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. We are encouraged by these recent regulatory designations from the FDA and EMA, which speak to the potential impact this therapy could have for patients.

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review.

In addition to RMAT designation, CTX001 has received Orphan Drug Designation from the U.S. FDA for TDT and from the European Commission for TDT and SCD. CTX001 also has Fast Track Designation from the U.S. FDA for both TDT and SCD.

About CTX001CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients. CTX001 is the most advanced gene-editing approach in development for beta thalassemia and SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

About the CRISPR-Vertex CollaborationCRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the benefits of RMAT designation; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the information provided regarding the status of, and expectations with respect to, the CTX001 clinical development program and related global regulatory approvals, and expectations regarding the RMAT designation. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the development of CTX001 may not proceed or support registration due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167 reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.com orU.S.: +1 617-341-6992orHeather Nichols: +1 617-961-0534orInternational: +44 20 3204 5275

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CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX001 for the...

Except for hard metals like stainless steel, gold and titanium, most synthetic materials in the body create a very significant scarring response. Credit: INSERM

The power of needlework should never be underestimated. Where would we be without the textile industry providing natural and synthetic fibres to thousands of sectors across the globe? In addition to clothing our bodies, textiles are used to help heal them, from wound dressings to sutures and meshes. Some scientists are now going further, using weaving and knitting techniques to create medical fabrics from biological materials such as human cells.

Nicolas LHeureux, director of research at INSERM, the French National Institute of Health and Medical Research is exploring exactly this. He works on repairing damaged blood vessels with biological textiles grown in the laboratory.

Synthetic materials are recognised as not being a normal part of the body, LHeureux explains. Except for hard metals like stainless steel, gold and titanium, most create a very significant scarring response.

He likens it to a splinter. The body recognises the material as foreign and reacts to it by trying to push it out. If its not able to eject it, scar tissue will form and inflammation will be triggered. This can lead to redness, pain, swelling and scarring.

Some parts of the body are better at dealing with scarring than others. Sometimes a scar can actually be helpful, providing mechanical support to the structure. But in a blood vessel, a scarring response could recreate the same blockage surgeons were aiming to fix in the first place. Grafting and stenting can result in restenosis (where the treated vessel closes off again) over time due to this scarring behaviour.

A scarring response will create a lot of tissue that will clog the inner part of your tube. Then your blood will not flow well anymore, LHeureux says.

Synthetic grafts work best for large vessels such as the aorta, which is roughly two centimetres in diameter. If there is a little bit of scarring from the graft, the blood will likely still be able to flow normally. But for smaller blood vessels, rejection of synthetic materials can create a real problem.

LHeureux and his team are developing grafts that wont produce that rejection response. What is more likely to be accepted is a material the body is already familiar with. Researchers cultivate human cells in the laboratory (originally extracted from a skin biopsy), where various chemicals are used to influence them to form sheets of collagen. These sheets are then cut into thin threads of yarn-like material.

The material we collect in sheets is the extracellular matrix outside the cell. Thats what we get the cells to overproduce in the lab, LHeureux reveals. This is the material that they lay down in the right conditions at the bottom of the plastic containers where we grow the cells.

Because the makeup of collagen doesnt vary from person to person, it is hoped that each patient wouldnt need vessels produced from their own cells.

Once the yarn is ready, its time to get sewing. By weaving, braiding or knitting, the team can form tubes of collagen to replace the synthetic structures that are traditionally used in cardiovascular surgery.

We tend to use weaving because it makes a really nice tight wall which is really important if youre making a blood vessel, says LHeureux.

This is weaving as youve never seen it before, but it still requires a loom. The custom-made device has to be tiny so vessels of five millimetres in diameter can be produced. Its made out of stainless steel and plastic so it can be cleaned easily. And it is circular in shape to produce tubes from the collagen yarn.

How well these grafts will be tolerated by the body still needs to be tested though. The next step of the research is to see how the vessels perform in animals. Using genetically modified rodent models that dont reject human tissue, the team will be able to see if the biological textiles adapt well to the body environment.

But one problem with rats and mice is the small size of their blood vessels. So, the researchers are also working on producing similar medical textiles using sheep cells. Once animal model results prove promising, itll be time to think about transplanting the grafts into humans.

The sheep is about the same size as a human in terms of the blood vessels, so well be able to try surgeries that we would do in humans with vessels that would be the same size and using all the same instruments. So, its much more representative, LHeureux explains.

There are other research groups working on similar projects.The University of Minnesota Medical School recently grew human-derived blood vessels in a pig. While US company Humacyte is also trying to produce extracellular matrices for vascular and non-vascular applications. In the future, LHeureux hopes to collaborate with groups like these to find the best way of producing biological textiles at scale.

When we bring this material that is completely logical, completely human and integrates well inside the body to patients, well have a solution that weve never had before in medicine.

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Spin me a yarn: the future of medical textiles and regenerative medicine - Medical Device Network

CTX001is an investigational therapy that Vertex Pharmaceuticals and CRISPR Therapeutics are developing to treat inherited disorders of hemoglobin such assickle cell diseaseandbeta-thalassemia.

Sickle cell disease is caused by a mutation in the HBB gene. This gene provides instructions to make the protein hemoglobin. Hemoglobin is a molecule inside red blood cells that is responsible for carrying oxygen. In sickle cell disease, the mutations result in missing or deficient hemoglobin.

CTX001 uses gene-editing technology to make a genetic change to increase the production of fetal hemoglobin in patients red blood cells. Fetal hemoglobin is a form of hemoglobin that exists naturally in newborn babies. The body later replaces it with the adult form of hemoglobin. However, sometimes fetal hemoglobin persists in adults, providing protection for people with sickle cell disease and beta-thalassemia.

For the treatment, researchers first collect a patients hematopoietic stem cells. These are cells from the bone marrow that give rise to all the red and white blood cells. They then genetically modify these cells in the laboratory so they are able to produce high levels of fetal hemoglobin. Finally, they reintroduce them into the patients body, where they will produce large amounts of red blood cells containing fetal hemoglobin.

Researchers presented the results of preclinical experiments with CTX001 at the American Society of Hematology (ASH) Annual Meeting in December 2017. CTX001 was able to efficiently edit the target gene in more than 90% of hematopoietic stem cells to achieve about 40% of fetal hemoglobin production. Investigators believe this is sufficient to improve a patients symptoms. Study results also showed that CTX001 affects only cells at the target site, thereby appearing to be a safe potential treatment.

These positive results prompted CRISPR topartner with Vertex to further develop CTX001. The goal is to market CTX001 as a gene-editing treatment for inherited hemoglobin disorders, including sickle cell disease and beta-thalassemia.

A Phase 1/2 clinical trial (NCT03745287) called CLIMB-SCD-121 was started in November 2018 to investigate the use of CTX001 in sickle cell disease. The open-label, multi-site, single-dose trial is recruiting 45 patients, ages 18 to 35, with severe sickle cell disease in the U.S., Canada, Belgium, Germany, and Italy. Researchers will give participants a single intravenous (into the bloodstream) infusion of CTX001. They will monitor the safety and effectiveness of the treatment for six months to two years.

Researchers reported preliminary results for the first patient in November 2019. Before treatment, the patient averaged seven vaso-occlusive crises (VOCs) a year. At four months after treatment, they were free of VOCs and had hemoglobin levels of 11.3 g/dL. The estimated completion date of the trial is May 2022.

The U.S. Food and Drug Administration (FDA) granted CTX001 fast track designationin January 2019. This designationallows for faster development and review of drugs that treat a serious medical issue and fill an unmet need.

In May 2020, the FDA also granted the therapy the designation of regenerative medicine advanced therapy (RMAT) for treating severe sickle cell disease and transfusion-dependent beta-thalassemia. The purpose of RMAT is to expedite the development and review of new therapies that treat serious or life-threatening medical conditions, or when they show significant clinical benefits over existing therapies.

Last updated: May 13, 2020

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Sickle Cell Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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zge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.

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CTX001 for Treatment of Sickle Cell Disease and Other Blood Disorders - Sickle Cell Anemia News

DUBLIN, May 13, 2020 /PRNewswire/ -- The "Asia-Pacific CAR-T Pipeline" report has been added to ResearchAndMarkets.com's offering.

The Asia-Pacific CAR-T pipeline features over 50 regenerative medicine companies focusing on CAR-T in Asia. The report derives insights from in-depth primary and secondary research studies and a comprehensive pipeline analysis of companies from China, Taiwan, Japan, Korea, India, Australia & SEA. It includes a disease-wise product analysis of the latest technologies and CAR-T developments available, along with insights on stage of development, scale of operation, co-development and technology partners involved and future prospects.

The report will give an assessment of the clinical trial and regulatory approval timelines, financial performance of the companies involved and provide recommendations on possible co-development and technology partnerships in the near future. In addition, it pinpoints the pain points of CAR-T therapy companies and identifies gaps in addressing certain diseases, manufacturing and supply chain capabilities and technologies available. It also provides a company-wise swot analysis. The report seeks to inform the biopharmaceutical industry on the current status of CAR-T therapies in APAC, uncover the potential for CAR-T therapy success in the region and discover future opportunities for partnership.

Furthermore, the report covers region specific growth drivers and insights into ongoing CAR-T therapy clinical trials. In addition, the directory presents a regulatory landscape assessment and a review of co-development opportunities. Moreover, in-depth studies on the CAR-T landscape have supplemented the report with a swot analysis and future forecasts. It provides readers a closer look into the pipelines of Nanjing Legend Biotech, CARsgen Therapeutics, JW Therapeutics, AbClon, BiocurePharm, Eutilex, Daiichi Sankyo, Celgene, Otsuka and many more.

Key Topics Covered

Section 1

Section 2: Company-Wise

Section 3: Region-Wise

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/6ywu77

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

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CAR-T Pipeline Assessment in Asia-Pacific, 2020 - Take a Closer Look Into the Pipelines of Nanjing Legend Biotech, CARsgen Therapeutics, JW...

NEWPORT BEACH, Calif.--(BUSINESS WIRE)--jCyte Inc., a biotech company dedicated to improving the lives of patients with rare degenerative retinal diseases, today announced it has entered into a licensing agreement with Santen Pharmaceutical to develop and commercialize its first-in-class, investigational therapy, jCell, outside the U.S., in regions including Europe, Asia and Japan.

Under the terms of the licensing agreement, jCyte will receive $50 million in upfront cash, $12 million in a convertible note offering, and $190 million in clinical and sales milestones based on regulatory approval and initial sales in Europe, Asia and Japan. The total deal is valued at up to $252 million. jCyte is also entitled to receive tiered, double-digit royalty payments on net sales of jCell therapy once commercialized outside the U.S.

We are thrilled to partner with Santen, a global market-leader in ophthalmology. By leveraging Santens large, existing global commercial and medical infrastructure in ophthalmology, as well as its commitment to commercializing cell and gene therapies, we help ensure that more patients with retinitis pigmentosa who live outside the U.S. will have access to this technology, said Paul Bresge, Chief Executive Officer, jCyte, Inc. We intend to use the proceeds from this transaction to continue development of our lead investigational therapy jCell, to improve the lives of patients with retinitis pigmentosa, as well as other degenerative retinal diseases.

jCell is a first-in-class investigational treatment for retinitis pigmentosa, an inherited retinal disease. The treatment is a minimally-invasive intravitreal injection, which can be performed in an ophthalmologists office with topical anesthetic. The entire procedure takes less than 30 minutes. The principal mechanism of action is the release of neurotrophic factors that may rescue diseased retinal cells. jCell therapy aims to preserve vision by intervening in the disease at a time when host photoreceptors can be protected and potentially reactivated. jCell has been developed with support from the California Institute for Regenerative Medicine (CIRM), which has funded previous preclinical development and ongoing clinical studies.

In the United States, the evaluable portion of a Phase 2b clinical trial of jCell for the treatment of retinitis pigmentosa has been completed, and the crossover portion continues. jCell therapy has been administered in over 100 patients. The U.S. Food and Drug Administration (FDA) has granted jCyte Regenerative Medicine Advanced Therapy (RMAT) designation based on early clinical data, making jCell potentially eligible for BLA priority review. In addition to RMAT, jCell has received Orphan Drug designation from the FDA and the European Medicines Agency (EMA).

About Retinitis Pigmentosa (RP)

Retinitis pigmentosa is a rare, genetic condition that progressively destroys the rod and cone photoreceptors in the retina. It often strikes people in their teens, with many patients rendered legally blind by middle age. Worldwide, an estimated 1.9 million people suffer from the disease, including approximately 100,000 people in the U.S., making it the leading cause of inheritable blindness.

About jCyte, Inc.

jCyte, Inc. is a clinical-stage biotech company focused on developing jCell therapy for retinitis pigmentosa (RP) and other degenerative retinal disorders. jCell is a first-in-class investigational treatment for retinitis pigmentosa, an inherited retinal disease. The treatment is minimally-invasive and given as an intravitreal injection. There are currently no FDA approved therapies for RP. The company is pioneering a new era of regenerative therapies to treat patients with unmet medical need. For more information, visit http://www.jcyte.com.

About Santen

As a specialized company dedicated to ophthalmology, Santen carries out research, development, marketing, and sales of pharmaceuticals, over-the-counter products, and medical devices. Santen is the market leader for prescription ophthalmic pharmaceuticals in Japan and its products now reach patients in over 60 countries. With scientific knowledge and organizational capabilities nurtured over a nearly 130-year history, Santen provides products and services to contribute to the well-being of patients, their loved ones and consequently to society. For more information, please visit Santens website (www.santen.com).

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jCyte Inc. Enters into Ex-US Licensing and Commercialization Agreement for jCell Therapy with Global Ophthalmology Leader Santen Pharmaceutical -...