Category Archives: Gene Medicine

IRVINE, Calif. & BASEL, Switzerland--(BUSINESS WIRE)--Urovant Sciences, a wholly-owned subsidiary of Sumitovant Biopharma Ltd., today announced positive topline results from its Phase 2a, double-blind, placebo-controlled exploratory study of URO-902, an investigational, novel, locally injected gene therapy product (plasmid human cDNA encoding maxi-K channel), in patients with overactive bladder (OAB), who were not well managed by oral therapies.

URO-902 showed a clinically meaningful and statistically significant effect on a number of relevant outcome measures in OAB including number of micturitions, urgency episodes, and quality of life indicators compared to placebo, 12 weeks post-administration, said Cornelia Haag-Molkenteller, MD, PhD, executive vice president and chief medical officer of Urovant Sciences. URO-902 was well tolerated, compared to placebo. The most common adverse event was urinary tract infection, in both treatment groups. We are encouraged by these positive results and pending the completion of the study in Fall 2022 and we look forward to discussing next steps for the URO-902 clinical development plan.

The Phase 2a study included 80 female patients and was designed to evaluate the efficacy, safety, and tolerability of a single, physician administered dose of URO-902 of 24 milligrams (mg) and 48 mg, compared with placebo with a primary timepoint at week 12 post-administration. Patients were followed for up to 48 weeks post-administration. URO-902 has the potential to be the first gene therapy for patients with OAB.

These promising results suggest that URO-902 could potentially offer a new treatment option for patients with overactive bladder who have been inadequately managed by oral pharmacologic therapy, said Kenneth Peters, MD, principal investigator, and chief of the department of urology at Beaumont Hospital, Royal Oak; Medical director of the Beaumont Womens Urology and Pelvic Health Center and professor and chair of urology of the Oakland University William Beaumont School of Medicine in Rochester, Michigan.

The company plans to present the topline results of the study at the American Urological Association annual meeting being held May 13-16, 2022 in New Orleans, LA.

About the Phase 2a Study

The study was a randomized, double blind, placebo-controlled trial to evaluate the efficacy, safety, and tolerability of a single physician administered dose of URO-902, a novel gene therapy being developed for patients with OAB who have not been adequately managed with oral or transdermal pharmacologic therapy for OAB. URO-902 is administered via direct intradetrusor injections into the bladder wall under local anesthesia in patients who are experiencing OAB symptoms and urge urinary incontinence (UUI).

The Phase 2a trial enrolled 80 female patients in two cohorts: the first cohort received either a single administration of 24 mg of URO-902 or matching placebo, and the second cohort received 48 mg of URO-902 or matching placebo into the bladder wall. Multiple outcome measures were explored, including the effect on the number of micturitions, urgency episodes, and quality of life indicators compared to placebo, 12 weeks post-administration, as well as an assessment of the safety and tolerability of this potential new therapy. Patients were followed for up to 48 weeks after initial administration.

About URO-902

URO-902 has the potential to be the first gene therapy for patients with OAB. If approved, this innovative treatment may address an unmet need for patients who have not been adequately managed by oral or transdermal pharmacologic OAB therapies and are concerned with potential urinary retention with other minimally invasive therapies or surgical interventions related to existing third-line OAB treatments.

About Urovant Sciences

Urovant Sciences is a biopharmaceutical company focused on developing and commercializing innovative therapies for areas of unmet need, with a dedicated focus in Urology. The Companys lead product, GEMTSA(vibegron), is an oral, once-daily (75 mg) small molecule beta-3 agonist for the treatment of adult patients with overactive bladder (OAB) with symptoms of urge urinary incontinence, urgency, and urinary frequency. GEMTESA was approved by the U.S. FDA in December 2020 and launched in the U.S. in April 2021. GEMTESA is also being evaluated for the treatment of OAB in men with benign prostatic hyperplasia. The Companys second product candidate, URO-902, is a novel gene therapy being developed for patients with OAB who have failed oral pharmacologic therapy. Urovant Sciences, a wholly owned subsidiary of Sumitovant Biopharma Ltd., intends to bring innovation to patients in need in urology and other areas of unmet need. Learn more about us at http://www.urovant.com or follow us on Twitter or LinkedIn.

About Sumitovant Biopharma Ltd.

Sumitovant is a global biopharmaceutical company leveraging data-driven insights to rapidly accelerate development of new potential therapies for unmet patient conditions. Through our unique portfolio of wholly-owned Vant subsidiariesUrovant, Enzyvant, Spirovant, Altavantand use of embedded computational technology platforms to generate business and scientific insights, Sumitovant has supported the development of FDA-approved products and advanced a promising pipeline of early-through late-stage investigational assets for other serious conditions. Sumitovant, a wholly-owned subsidiary of Sumitomo Dainippon Pharma, is also the majority-shareholder of Myovant (NYSE: MYOV). For more information, please visit our website at http://www.sumitovant.com or follow us on Twitter and LinkedIn.

About Sumitomo Dainippon Pharma Co., Ltd.

Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China, and other Asian countries. Sumitomo Dainippon Pharma is based on the 2005 merger between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 7,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at https://www.ds-pharma.com.

About GEMTESA

GEMTESA is a prescription medicine for adults used to treat the following symptoms due to a condition called overactive bladder:

It is not known if GEMTESA is safe and effective in children.

IMPORTANT SAFETY INFORMATION

Do not take GEMTESA if you are allergic to vibegron or any of the ingredients in GEMTESA.

Before you take GEMTESA, tell your doctor about all your medical conditions, including if you have liver problems; have kidney problems; have trouble emptying your bladder or you have a weak urine stream; take medicines that contain digoxin; are pregnant or plan to become pregnant (it is not known if GEMTESA will harm your unborn baby; talk to your doctor if you are pregnant or plan to become pregnant); are breastfeeding or plan to breastfeed (it is not known if GEMTESA passes into your breast milk; talk to your doctor about the best way to feed your baby if you take GEMTESA).

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine.

What are the possible side effects of GEMTESA?

GEMTESA may cause serious side effects including the inability to empty your bladder (urinary retention). GEMTESA may increase your chances of not being able to empty your bladder, especially if you have bladder outlet obstruction or take other medicines for treatment of overactive bladder. Tell your doctor right away if you are unable to empty your bladder.

The most common side effects of GEMTESA include headache, urinary tract infection, nasal congestion, sore throat or runny nose, diarrhea, nausea and upper respiratory tract infection. These are not all the possible side effects of GEMTESA. For more information, ask your doctor or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please click here for full Product Information for GEMTESA.

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Urovant Sciences Announces Positive Topline Results of Phase 2a Trial of its Potential Novel Gene Therapy, URO-902 - Business Wire

JAVEN, France, March 10, 2022 /PRNewswire/ --HTL Biotechnology, a pioneer and world leader in the development and production of innovative pharmaceutical grade biopolymers, today announced its incubator's first equity investment in GelMEDIX Inc., an early-stage biotechnology company committed to developing the next generation of ocular and regenerative therapies.

This investment supports continued development of the GelMEDIX platform, which enables the delivery of therapeutics from small molecules to cell and gene therapies. Initial research focuses on ophthalmology with lead programs in vision restoring cornea and retina cell therapies and sustained release small molecule therapies.

GelMEDIX's most advanced program is focused on developing a sustained release corticosteroid subconjunctival implant which aims to improve patient care in ocular surface inflammation (postoperative pain and inflammation, dry eye disease, allergic conjunctivitis). One drug-loaded implant replaces 70 patient administered eye drops over the course of one month.

"Instead of using drops, one easy treatment will be administered in the operating theater or in the clinic without any loss of efficacy. In addition to anti-inflammatories, the platform technology can be used for delivery of pro-regenerative therapies that restore ocular health" said Reza Dana, M.D., M.P.H., M.Sc., Scientific Co-founder of GelMEDIX. "As such, this product represents one of the most promising innovations deriving from our proprietary hydrogel platform."

This initial implant constitutes only one of the several research opportunities deriving from GelMEDIX's proprietary photocrosslinkable hydrogel platform, which uniquely enables tunable bioadhesion, tissue regeneration, and biodegradation. HTL's partnership with GelMEDIX also facilitates the development of new applications in regenerative medicine both in ophthalmology and other therapeutic areas.

HTL's incubator will support GelMEDIX through a direct investment and industrial and scientific support throughout its development thanks to its expertise in biopolymers and ophthalmology sectors. HTL will also produce methacrylate hyaluronic acid, a key component for tailoring application specific parameters across the GelMEDIX pipeline including viscosity, bioadhesion, and therapeutic release profiles. Additionally, HTL will help GelMEDIX in the industrialization of its hydrogel production.

"It is an honor to have the support of such a renowned company as HTL. Beyond the financial aspect, its keen understanding of ophthalmology issues, its industrial know-how and the high quality of its products are all crucial assets to accelerate GelMEDIX's development," said Arthur Driscoll, President and Chief Development Officer of GelMEDIX.

HTL's participation will be joined by another equity investment from the venture fund Safar Partners. "The pioneering advancements GelMEDIX is making in the development of ocular and regenerative therapies will lead to dramatic improvements in how these treatments are administered to patients," said Nader Motamedy, a Safar Managing Partner. "The GelMEDIX hydrogel platform is the kind of transformative healthcare technology that Safar Partners highly values as both a long-term position for our portfolio as well as a development that will improve global health."

HTL's incubator is a financial vehicle allowing HTL to take minority investments in innovative biotechs in the biopolymer sector, either as seed funds or as series A investments. The incubator also aims to support these biotechs thanks to HTL's unique knowledge and expertise in the production of biopolymers.

"Innovation is the core of HTL's DNA, which is why we aim at supporting tomorrow's medicine by investing in biotechs that are pushing away the limits of biopolymer use in the medical sector," said Charles Ruban, Deputy CEO of HTL. "We are really excited about this partnership with GelMEDIX, which is a perfect example of the type of biotechs to which we wish to provide strategic and financial support".

This incubator represents one of the strategic axes of HTL's ambitious R&D strategy which positions the French company as the global driver of innovation in the biopolymer sector, developing new markets and applications for biopolymers to address unmet medical needs. The company also relies on its state-of-the-art research facility and numerous partnerships with entities at the forefront of world research to nurture its biopolymer platform for the healthcare industry.

About biopolymers and hyaluronic acidBiopolymers include several types of substances which are naturally produced by the cells of living organisms. Among them, glycosaminoglycans (GAGs) are known for their lubricating and shock-absorbing characteristics, as well as their natural biodegradability within the human body. This is the case, for example, of hyaluronic acid (hyaluronan or HA), a natural substance present in the human body with many biological functions such as skin hydration or lubrication of joints and eye tissue.

HTL produces GAGs by biofermentation, an alternative to animal extraction that maintains the quality required for pharmaceutical grade, allowing the biopolymers to be injectable into patients and used as ingredients for the development of medical treatments. The chemical properties of biopolymers can also be customized by HTL's R&D teams in order to precisely meet the needs of customers and their patients.

Today, the biopolymers which are developed and produced by HTL are used to produce treatments that improve the lives of millions of patients in many fields, such as ophthalmology (cataract surgery, treatment of glaucoma, treatment of dry eye ...), rheumatology (treatment of osteoarthritis), urology (treatment of vesico-ureteral reflux, a rare pediatric congenital disease), or in aesthetic medicine (dermal fillers). Biopolymers are also at the heart of several research programs focused on disruptive innovations in medicine such as bioprinting and regenerative medicine, tissue engineering as well as drug and stem cell delivery.

About GelMEDIXGelMEDIX Inc. is an early-stage biotechnology company committed to innovating the next generation of ocular and regenerative therapies through its proprietary hydrogel platform. GelMEDIX's programs are based upon its photo crosslinked hydrogels, originally developed by Prof. Nasim Annabi (UCLA) and Prof. Reza Dana (Mass Eye and Ear, Harvard Medical School). These hydrogels uniquely enable bioadhesion, tissue regeneration, tunable mechanics, and therapeutic loading across modalities from small molecules to cell and gene therapies.GelMEDIX is developing drug products for indications across the eye focused on cell-based therapies for vision restoration, intraocular sustained release of small molecules and peptides, and in situ forming bioadhesives.GelMEDIX is backed by Safar Partners and HTL Biotechnology along with leading angel investors and is currently raising a Series-A financing. GelMEDIX is based in Cambridge, MA., USA For additional information please inquire with info@gelmedix.com or visit https://gelmedix.com

About HTLHTL is a leading biotech and industrial player in the development and production of innovative, pharmaceutical-grade biopolymers that are used by leading pharmaceutical and medical device companies to transform the lives of millions of patients in multiple therapeutic areas such as ophthalmology, dermatology, medical aesthetics, rheumatology, and urology.

HTL is at the forefront of innovation in the biopolymer industry to meet tomorrow's medical needs by creating new types of biopolymers and chemical modifications, while exploring the untapped potential of biopolymers in innovative applications such as bioprinting or drug delivery.

HTL has a long history in France and in Javen (Ille-et-Vilaine, Brittany) where its production and R&D activities are located. Nearly 180 employees work at this site.

To learn more about HTL: https://htlbiotech.com/

About Safar PartnersSafar Partners is a seed- to growth-stage venture fund investing primarily in technology companies out of MIT, Harvard, and the University of Rochester. Safar takes advantage of the principles of private equity to create value as our companies scale beyond initial prototypes. We accelerate the scaling of our portfolio companies through the formation of spinouts or joint ventures to address additional markets, industries, or geographies. For more about Safar Partners, visit https://www.safar.partners

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SOURCE htl biotechnology

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HTL Announces Its Incubator's Equity Investment In GelMEDIX, An Early-Stage Biotech Aiming At Revolutionizing Ocular And Regenerative Therapies -...

Precision medicine involves more than molecularly sequencing a tumor and matching it with a targeted therapy, according to Kelvin P. Lee, MD, who argued that bulk sequencing may become an antiquated approach when single-cell RNA sequencing technology becomes available because it can provide a clearer picture of the complexity and heterogeneity of tumors.

Once we move away from bulk sequencing, which essentially says, This whole tumor is all the same and get down to a much finer specificity that [enables us to identify] tumor cells that have this mutation and look like this vs tumor cells that have that mutation and look like that, we will get more and more sophisticated in how we treat patients. The more heterogeneous the tumorand they become more heterogeneous as they are later in treatmentthe harder they are to treat, and the less helpful our current precision genomics technology is in that setting, Lee said in an interview with OncLive following an Institutional Perspectives in Cancer webinar on Precision Medicine.

In the interview, Lee, director of the Indiana University (IU) Simon Comprehensive Cancer Center, the H.H. Gregg Professor of Oncology, professor, Department of Medicine,

Division of Hematology/Oncology, associate dean for cancer research, IU School of Medicine, discussed the nuances of an effective molecular tumor board, explained the practical application of precision oncology genomics, and highlighted the pathways that have the potential to change the treatment landscape for precision oncology.

Lee: The take-home message is that every tumor should be sequenced. If you dont do it now, you may not be able to do it later. The information can be useful in designing a treatment plan, but it also helps us understand that cancer. The more information we have on lung cancers from patients, the better we can design new therapies and move that field forward. If a patient has a tumor specimen that you can send to precision genomics, send it because maybe we dont have an answer today, but maybe in 2 weeks we will have an answer, and that becomes important for that patient.

At IU there is a precision medicine team, and our patients are referred to them. That precision medicine team sets up the testing and the [molecular] sequencing and then does tumor boards, where they go through the results, they talk about what abnormalities and what mutations were found and then they go through what [mutations] are potentially actionable and how we want to utilize [treatment for] those [alterations]. The nice thing about tumor boards is that all the experts are in the room, and now that we have extended [ours] to be virtual, not only can we do precision medicine downtown at University Hospital, but now we have clinics that are in our suburban and metropolitan regions. Were moving those facilities out farther, so we can do precision medicine, hopefully, out in the community. Hopefully, with the expansion, primary care physicians and community oncologists can also utilize this [opportunity to] get the referrals and then sit in virtual tumor boards and get answers for their patients.

The key piece is not only doing the [molecular] sequencing and understanding what the mutations are, but also what youre doing with that information. For many of the mutations, the therapeutic that might be applicable to that patient or that mutation are in clinical trials. An effective precision medicine program has to not only have the expertise as to what these mutations are, what these established targets or established therapeutics are that we might be able to use, but also has to have an ongoing real-time knowledge of all the clinical trials that are available and in the literature.

Not only are there new compounds that a patient might be eligible for in a clinical trial, but there are also drugs that have been used for other things that are being repurposed for targeting mutations that we had not previously expected those drugs to be able to do. The precision medicine team must be aware of the literature, not only published literature but abstracts and journal or meeting work that says, Maybe you can use this malaria drug to target this mutation, which may be something that had not been previously understood. That is, overall, a key piece of what makes precision medicine so effective and so important, but so difficult to do. Its not just sequencing stuff, and then saying, There it is and then figuring out how to manage that [patient].

Next-generation sequencing is now allowing us to get at whole genomes, instead of just testing for BRCAmutations or [other single-gene mutations]. We are identifying other mutations, and now instead of having to re-sequence somebodys tumor, we have all that data thats there. If something pops up that later becomes this polymorphism or this mutation that is important in this cancer, we have that data; we can go back and look at those aspects. What it allows us to do is really what I would consider the next generation of precision medicine: to understand how mutations work together.

For immunotherapy, [tumor] mutational burden has been a key driver of whether checkpoint inhibitors are important. Now as we understand more of what a persons cancer has, in terms of mutations, our informatics, our artificial intelligence, and our machine learning technology is poised to take that data and say, if you have these 2 mutations: Are they compensating for each other, and do you have to target both? Now, if a patient has 1 mutation, they get this 1 drug, but biology is much more complicated. There are mutations that may work in concert with each other that may develop other additional vulnerabilities that we didnt anticipate because one mutation is causing the cell to do something and another mutation is stressing the cells, so maybe there is a target thats not either one of those 2 mutations but that is in the pathways that those mutations are driving that can be gone after. The exciting thing for me is understanding that. Im an immunologist, so understanding the complexity of cells, because immune systems see lots of things simultaneously, is really what we are looking forward to.

Its a very active process; patients are identified at the beginning of the week, and then the team asks: What are the mutations? What are the [alterations] that are actionable? Then they begin to sort through what treatment options are available and what the adverse effects [AEs] are. PharmDs have to ask: What are the AEs, particularly for experimental agents? What are the interactions with other drugs? How do we get these things and what literature supports the use of this agent? Then, are there odd things that we have to understand? Are there subsets of patients who have particularly bad responses that had been reported in the literature? All that gets pulled together.

Our tumor board is later in the week where all that information is presented: the patient case is presented, the genomic description or the description of the genetic changes are reviewed, and then the treatment options are also reviewed if theyre available, and the strength of each [drug] in terms of the data that says that this drug would be particularly good in this patient, or this drug would not be something that wed be looking for in this patient. A lot of what makes tumor boards effective is that research. Its not just, I have a piece of paper says I can give this drug and then you are done. It really is a lot of thoughtful research that goes into understanding the options that a patient might have.

The case study showed what the right process is to analyze the data you get to reach a meaningful action plan. As the technology goes forward, and as our ability to detect things gets more sophisticated, as we start to move toward single-cell sequencing, for example, RNA sequencing, when we start to look at the epigenome, we will have substantially more data than we have now. Then well start looking at what the patients immune system looks like when we start [molecularly] sequencing that. The amount of data that will be collected and the kinds of data that will be collected will grow exponentially.

The key piece of molecular tumor boards and the key piece of precision medicine is: How do you analyze that data? The analytical pipeline is going to be the same, the structure is going to be the same. How do you act? How do you take that data in? How do you analyze it, and then how do you use that analysis to come to specific treatment recommendations for that patient? That framework, that pipeline is going to be the same regardless of what the data coming in is. The key thing that was important in that whole process of going through these case studies is to recognize what the steps were that were taken from the very beginning, from the actual case itself where the patient comes in with their history. How were those data put together and analyzed? How was that used to make decisions for the treatment plan for that patient? Its the structure of the analytical process that was the most important aspect of going through those case studies.

The data suggests that with early genomic testing, when you apply it to precision medicine to identify therapeutics, the anti-cancer effect, or the ability to impact a persons cancer is greater on early diagnosis than late diagnosis. There probably are a variety of biologies that are implicated by that. When a tumor has been exposed to lots of things, it probably not only has its initial mutations, but probably has a lot of adaptations that have happened because of chemotherapy that has been given that we dont necessarily pick up; it may not be a genomic abnormality, but it may be overexpression of a particular gene that confers resistance. As those tumors become more resistant to therapy, the initial driver mutations may become less important as things go forward.

It speaks to biology also, because in the beginning, probably, in tumors that have not been treated or not been heavily pretreated at the time of diagnosis, theyre probably less genetically complicated, so maybe they have just one mutation. Maybe all of them have that one mutation, you treat them with a drug, and they all die. As tumors go along, they become much more heterogeneous, so instead of one population of cancer cells, now you have 75 different populations or tribes, for example, that are living, and some of them are sensitive, some of them are different. Some of them have different mutations, and precision genomics is moving towards being able to understand complex tumors, such that some of the cells have one mutation and some of them have a completely different set of mutations. We dont pick that up right now, simply because we dont have the single-cell RNA sequencing technology yet, although thats coming.

We are beginning to see precision medicine in the context of immunotherapy. In that sense, the change in framework is its not the cancer alone thats important because not only do you have to sequence the cancer, and understand its genetic makeup, you also have to sequence the immune systemthe normal part of the patient that is essentially the effector part. Instead of giving chemotherapy, where youre saying, I have a drug, I know everything about that drug, and all I need to do is figure out whats going on in the cancer, if I can find that this drug will hit this piece of the cancer, then lets put those two together.

For immunotherapy, you have the cancer, which is doing stuff and its dynamic, and its activating the immune system and suppressing the immune system. We have to understand that, and some of the suppression that it does is not because it is suppressing the immune system, its making the normal tissue around it suppress the immune system.

People say that cancer is a non-healing wound, so essentially, for wound healing, you dont want your immune system to fire up and start destroying all the tissue around a healing wound. Otherwise, youll never heal. The normal body has perfectly good mechanisms to shut off your immune response and cancers take advantage of that, but that phenomenon is not in the cancer. Its in the surrounding tissue. You have to look in the surrounding tissue to see whats going on there.

Then, you have to look at the immune system because the immune system is the thing thats going to kill the cancer cell, and people have different immune systems. Its very clear that there are lots of genetic variabilities in that. Maybe that genotype within somebodys immune system is not good at getting activated by this immunotherapy. Maybe we should try something different. Its another level of complexity that precision medicine has a tremendous role in, but it becomes that much more complicated because now youre not just looking at the cancer, were now looking at the cancer, the cancers effect on its surrounding microenvironment, and the immune systems ability to target that cancer and perhaps live in that environment that surrounds the tumor. Its an additional level of analysis and complexity. Thats coming though. With the expansion of immunotherapy that will be a much bigger piece of what we do in terms of therapy. Those kinds of analyses and guidance by precision medicine will be a key component of how we deploy immunotherapy in patients with cancer.

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Precision Genomics Moves Toward Increased Granularity in Molecular Sequencing - OncLive

ReportLinker

Abstract: What`s New for 2022? -Global competitiveness and key competitor percentage market shares. -Market presence across multiple geographies - Strong/Active/Niche/Trivial.

New York, March 11, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Gene Therapy Industry" - https://www.reportlinker.com/p05817594/?utm_source=GNW -Online interactive peer-to-peer collaborative bespoke updates -Access to our digital archives and MarketGlass Research Platform -Complimentary updates for one year

Global Gene Therapy Market to Reach US$3.4 Billion by the Year 2027

Amid the COVID-19 crisis, the global market for Gene Therapy estimated at US$970.5 Million in the year 2020, is projected to reach a revised size of US$3.4 Billion by 2027, growing at a CAGR of 19.5% over the period 2020-2027.Viral, one of the segments analyzed in the report, is projected to grow at a 19.7% CAGR to reach US$3 Billion by the end of the analysis period. After an early analysis of the business implications of the pandemic and its induced economic crisis, growth in the Non-Viral segment is readjusted to a revised 17.6% CAGR for the next 7-year period. This segment currently accounts for a 11.1% share of the global Gene Therapy market.

The U.S. Accounts for Over 53.7% of Global Market Size in 2020, While China is Forecast to Grow at a 25.1% CAGR for the Period of 2020-2027

The Gene Therapy market in the U.S. is estimated at US$521.3 Million in the year 2020. The country currently accounts for a 53.71% share in the global market. China, the world second largest economy, is forecast to reach an estimated market size of US$107.9 Million in the year 2027 trailing a CAGR of 25.1% through 2027. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at 17.3% and 18.9% respectively over the 2020-2027 period. Within Europe, Germany is forecast to grow at approximately 18.6% CAGR while Rest of European market (as defined in the study) will reach US$107.9 Million by the year 2027.Select Competitors (Total 154 Featured) -

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Abeona Therapeutics Inc.

Adaptimmune Therapeutics Plc

Advantagene, Inc.

Adverum Biotechnologies, Inc

Akcea Therapeutics

Alnylam Pharmaceuticals, Inc.

Amgen Inc

Anchiano Therapeutics, Inc.

AnGes, Inc.

Applied Genetic Technologies Corporation

Audentes Therapeutics, Inc.

Biogen

bluebird bio, Inc.

Chiesi Farmaceutici S.p.A

CRISPR Therapeutics AG

Editas Medicine, Inc.

Gilead Sciences, Inc.

Intellia Therapeutics, Inc.

Jazz Pharmaceuticals, plc.

Juno Therapeutics, Inc

Merck KGaA

MolMed S.p.A.

Novartis Gene Therapies

Orchard Therapeutics plc

REGENXBIO Inc.

Sangamo Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Sibiono GeneTech Co. Ltd.

Spark Therapeutics, Inc.

uniQure N.V.

Voyager Therapeutics

Read the full report: https://www.reportlinker.com/p05817594/?utm_source=GNW

I. METHODOLOGY

II. EXECUTIVE SUMMARY

1. MARKET OVERVIEW Influencer Market Insights World Market Trajectories Impact of Covid-19 and a Looming Global Recession A Prelude to Gene Therapy Classification of Gene Therapies COVID-19 Causes Gene Therapy Market to Buckle & Collapse COVID-19 Impact on Different Aspects of Gene Therapy Manufacturing & Delivery Research & Clinical Development Commercial Operations & Access Managing Derailed Operations Focus on Clinical Development Programs Targeting Manufacturing & Delivery Strategies Securing Supplies Remote Working Gene Therapy Set to Witness Rapid Growth Post COVID-19 Gene Therapy - Global Key Competitors Percentage Market Share in 2022 (E) Competitive Market Presence - Strong/Active/Niche/Trivial for Players Worldwide in 2022 (E) By Vector Type VIRAL VECTORS ACCOUNT FOR A MAJOR SHARE OF THE MARKET Adeno-Associated Virus Vectors Lentivirus NON-VIRAL VECTORS TO WITNESS FASTER GROWTH US and Europe Dominate the Gene Therapy Market Oncology Represents the Largest Indication for Gene Therapy Market Outlook WORLD BRANDS

2. FOCUS ON SELECT PLAYERS Recent Market Activity Select Innovations

3. MARKET TRENDS & DRIVERS Availability of Novel Therapies Drive Market Growth Select Approved Gene Therapy Products Adeno-associated Virus Vectors - A Leading Platform for Gene Therapy Lentiviral Vectors Witness Increasing Interest Rising Cancer Incidence Worldwide Spurs Demand for Gene Therapy Global Cancer Incidence: Number of New Cancer Cases in Million for the Years 2018, 2020, 2025, 2030, 2035 and 2040 Global Number of New Cancer Cases and Cancer-related Deaths by Cancer Site for 2018 Number of New Cancer Cases and Deaths (in Million) by Region for 2018 Compelling Level of Technology & Innovation to Ignite Gene Therapy Promising Gene Therapy Innovations for Treatment of Inherited Retinal Diseases Gene Therapy Pivots M&A Activity in Dynamic Domain of Genomic Medicine M&As Rampant in Gene Therapy Space Gene Therapy Deals: 2018 and 2019 Emphasis on Formulating Robust Regulatory Framework Strong Gene Therapy Pipeline Gene Therapy: Phase III Clinical Trials OHSU Implements First-Ever LCA10 Gene Therapy Clinical Trial with CRISPR Growing Funding for Gene Therapy Research Market Issues & Challenges

4. GLOBAL MARKET PERSPECTIVE Table 1: World Recent Past, Current & Future Analysis for Gene Therapy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 2: World Historic Review for Gene Therapy by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 3: World 10-Year Perspective for Gene Therapy by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets for Years 2017, 2021 & 2027

Table 4: World Recent Past, Current & Future Analysis for Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 5: World Historic Review for Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 6: World 10-Year Perspective for Viral by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2021 & 2027

Table 7: World Recent Past, Current & Future Analysis for Non-Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 8: World Historic Review for Non-Viral by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 9: World 10-Year Perspective for Non-Viral by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2021 & 2027

Table 10: World Recent Past, Current & Future Analysis for Oncological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 11: World Historic Review for Oncological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 12: World 10-Year Perspective for Oncological Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2021 & 2027

Table 13: World Recent Past, Current & Future Analysis for Rare Diseases by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 14: World Historic Review for Rare Diseases by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 15: World 10-Year Perspective for Rare Diseases by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2021 & 2027

Table 16: World Recent Past, Current & Future Analysis for Neurological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 17: World Historic Review for Neurological Disorders by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 18: World 10-Year Perspective for Neurological Disorders by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2021 & 2027

Table 19: World Recent Past, Current & Future Analysis for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 20: World Historic Review for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 21: World 10-Year Perspective for Other Applications by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2017, 2021 & 2027

III. MARKET ANALYSIS

UNITED STATES Gene Therapy Market Presence - Strong/Active/Niche/Trivial - Key Competitors in the United States for 2022 (E) Table 22: USA Recent Past, Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 23: USA Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 24: USA 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2021 & 2027

Table 25: USA Recent Past, Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 26: USA Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 27: USA 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2021 & 2027

CANADA Table 28: Canada Recent Past, Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 29: Canada Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 30: Canada 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2021 & 2027

Table 31: Canada Recent Past, Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 32: Canada Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 33: Canada 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2021 & 2027

JAPAN Gene Therapy Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Japan for 2022 (E) Table 34: Japan Recent Past, Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 35: Japan Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 36: Japan 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2021 & 2027

Table 37: Japan Recent Past, Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 38: Japan Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 39: Japan 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2021 & 2027

CHINA Gene Therapy Market Presence - Strong/Active/Niche/Trivial - Key Competitors in China for 2022 (E) Table 40: China Recent Past, Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 41: China Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 42: China 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2021 & 2027

Table 43: China Recent Past, Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 44: China Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 45: China 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2021 & 2027

EUROPE Gene Therapy Market Presence - Strong/Active/Niche/Trivial - Key Competitors in Europe for 2022 (E) Table 46: Europe Recent Past, Current & Future Analysis for Gene Therapy by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 and % CAGR

Table 47: Europe Historic Review for Gene Therapy by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 48: Europe 10-Year Perspective for Gene Therapy by Geographic Region - Percentage Breakdown of Value Sales for France, Germany, Italy, UK and Rest of Europe Markets for Years 2017, 2021 & 2027

Table 49: Europe Recent Past, Current & Future Analysis for Gene Therapy by Vector Type - Viral and Non-Viral - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 50: Europe Historic Review for Gene Therapy by Vector Type - Viral and Non-Viral Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 51: Europe 10-Year Perspective for Gene Therapy by Vector Type - Percentage Breakdown of Value Sales for Viral and Non-Viral for the Years 2017, 2021 & 2027

Table 52: Europe Recent Past, Current & Future Analysis for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 and % CAGR

Table 53: Europe Historic Review for Gene Therapy by Application - Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2017 through 2019 and % CAGR

Table 54: Europe 10-Year Perspective for Gene Therapy by Application - Percentage Breakdown of Value Sales for Oncological Disorders, Rare Diseases, Neurological Disorders and Other Applications for the Years 2017, 2021 & 2027

See the article here:
Global Gene Therapy Market to Reach US$3.4 Billion by the Year 2027 - Yahoo Finance

by Leigh MacMillan

Gene variants increased the risk of acute kidney injury and death in veterans of African ancestry who were hospitalized with COVID-19, according to a new study published in JAMA Internal Medicine.

A team of Vanderbilt investigators led the study as part of the VA Million Veteran Program COVID-19 Science Initiative.

The findings may explain some health disparities associated with COVID-19 and could guide efforts to identify individuals who are at increased risk of acute kidney injury and death and offer personalized treatments, said Adriana Hung, MD, MPH, associate professor of Medicine in the Division of Nephrology and Hypertension and lead author of the paper.

We think it will be very informative to understand if people have these gene variants that put them at increased risk to make decisions about tailoring therapy for them, Hung said.

Acute kidney injury (AKI) sudden kidney failure has been a common complication in patients hospitalized for COVID-19, with higher rates of AKI and death in patients of African ancestry, Hung said.

We wanted to understand what was behind this increased risk, besides being critically ill, she added.

The researchers knew that variants in the gene APOL1 (apolipoprotein L1), found in people of African ancestry, are associated with chronic kidney disease. More than 1 in 10 individuals of African ancestry have two APOL1 variants, which appear to have evolved to protect against infection by the parasites that cause African sleeping sickness. APOL1 variants contribute to health disparities in chronic kidney disease among people with African ancestry.

Hung and her colleagues wondered if APOL1 risk variants are associated with AKI in Black patients hospitalized with COVID-19.

They probed this association using data from the Million Veteran Program (MVP), a national program to study how genes, lifestyle and military exposures affect health and illness. The MVP has enrolled more than 850,000 diverse veterans over the last 10 years, making it the largest DNA biobank in the world.

The teams retrospective study included 990 MVP participants with African ancestry who were hospitalized with COVID-19 between March 2020 and January 2021. The researchers used clinical laboratory data to assess acute kidney injury in the patients, and they adjusted the analysis to account for preexisting diseases, medications and other risk factors for AKI.

Of the 990 MVP participants from 63 different hospitals, 12.6% had two APOL1 variants (high-risk group). Patients in this group were twice as likely to suffer severe AKI and death, compared to participants with only one or no APOL1 risk variants. This increased risk persisted even for high-risk patients who had normal kidney function before hospitalization.

Although case studies have reported an association of APOL1 mutations and FSGS (a rare disease that can cause kidney damage or failure), our study provides for the first time information about the association of APOL1 with acute kidney injury in a large cohort, Hung said.

Hung noted that medications targeting APOL1 are currently being tested and might offer personalized treatment options for patients with high-risk variants.

We also wonder if these findings may be extrapolated to individuals with APOL1 high-risk variants who are critically ill for other reasons, she said.

Using genetic information to inform clinical care is a goal of VUMCs precision medicine initiatives, said Alexander Bick, MD, PhD, assistant professor of Medicine in the Division of Genetic Medicine and a co-author of the current report.

Our goal is to bring more genetic data into the electronic health record, so that its available at clinicians fingertips, Bick said. This study is another example of how genetic information is going to be useful; its just the beginning for bringing this gene mutation into the hospital setting, Bick said.

The study population was 91.4% male, representing a limitation of the MVP, which is striving to increase its female participants, Hung said.

Edward Siew, MD, MSCI, associate professor of Medicine in the Division of Nephrology and Hypertension and a senior author of the paper, noted that having a better understanding of the molecular mechanisms that may explain these findings and predispose to human AKI in general is an important future direction.

There are novel biobanking efforts that are working to obtain tissue and biosamples from patients with AKI, which is an important early step toward this goal, Siew said.

Key members of the Vanderbilt research team included Zhihong Yu, PhD, Ran Tao, PhD, Hua-Chang Chen, PhD, Otis Wilson, Robert Greevy, PhD, Cecilia Chung, MD, MPH, Elvis Akwo, MD, PhD, Michael Matheny, MD, MS, MPH, and Cassianne Robinson-Cohen, PhD.

This research was supported by the Veterans Health Administration MVP COVID-19 Science Program and a VA Clinical Science Research and Development investigator grant to Hung to study the Genetics of Kidney Disease and Hypertension. Siew and Matheny were supported by a VA Health Services Research and Development grant.

Here is the original post:
Gene variants increase risk of kidney failure in Black veterans with COVID-19: study - VUMC Reporter

Later this year, the now-Nobel prize-winning paper authored by Jennifer Doudna and Emmanuelle Charpentier in which they described how a primordial immune system in bacteria could be harnessed to edit the genomes of other organisms will turn 10 years old. The discovery that CRISPR could be turned into an easily programmable tool for rewriting DNA launched biomedical research into warp drive.

In the 10 years leading up to 2012, 200 papers mentioned CRISPR. In 2020 alone, there were more than 6,000. The last decade has seen scientists use CRISPR to cure mice of progeria, fix muscular dystrophy in dogs, and eliminate symptoms for people with genetic blood disorders. Currently, there are more than two dozen human trials of the technology underway around the world.

STAT has created a new tracker of milestone CRISPR studies, and found that the explosion in interest created a positive feedback loop, accelerating the movement of new and better gene editing approaches toward the clinic. For CRISPR 1.0 therapies those using the original Cas9 cutting enzyme described in the Doudna paper four-and-a-half years passed, on average, between the first studies in cells and the first public data in non-human primates. Base editing, or CRISPR 2.0, got it down to three years, according to the CRISPR TRACKR.

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This time-shaving trend is evident in other ways, too. Last November, Beam Therapeutics announced it had gotten the green light to test its base editing technology in humans for treating sickle cell disease. If it begins dosing patients this year, that will put Beam just a few years behind the CRISPR 1.0 companies Intellia, Editas Medicine, and Crispr Therapeutics which began clinical studies of therapies for various genetic disorders in 2021, 2020, and 2019, respectively, effectively shortening the development time from an average of eight years to six.

Were now seeing a real acceleration in progress, said Kiran Musunuru, a gene editing researcher at the University of Pennsylvania and the co-founder of Verve Therapeutics. As the challenges are worked out for version 1.0, it just makes it much much easier to substitute in version 2.0 and then 3.0 and then whatever is next.

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The first five years after Doudna and Charpentiers (and Feng Zhangs and George Churchs) seminal papers were published, the field was consumed with fine-tuning how CRISPR-Cas9 worked in different kinds of cells, setting records for how many cuts it could make, and finding medically relevant applications for its targeted gene-breaking abilities.

The next five, driven by a gold rush in finding, engineering, or evolving new CRISPR proteins, saw the gene editing toolbox expand rapidly outward. These newer, shinier, crisper versions of CRISPR pushed forward faster toward the clinic, propelled by all the groundwork that had been laid by its older, clunkier cousin.

The thing thats frankly exhilarating to me, as a gray-haired veteran of editing, is how rich the overall ecosystem has become, said Fyodor Urnov, scientific director of the Innovative Genomics Institute at the University of California, Berkeley, which is headed by Doudna.

Urnov compared the 2000s, when he and others were working on pre-CRISPR versions of genome editing, to medieval times, with a few labs toiling away in their fiefdoms, separated by large tracts of no-mans land. The tools were few, and difficult to come by. Today, Urnov said, dialing up a gene editing experiment is more like clicking open the app store on your smartphone. Not only will you find options for different kinds of editors and modes for delivering them, but each comes with ratings and reviews too.

Ten years ago, the ability to just walk into this enormous smorgasbord of offerings simply didnt exist, he said.

For many of these tools, its still too early to say which ones will take off into components of blockbuster therapies and which ones will burn out upon takeoff. But with the field moving at warp speed, it wont be long before we know.

Read the rest here:
Drugs based on next-generation gene editing are moving toward the clinic faster than CRISPR 1.0 - STAT

BRAZIL - 2021/02/18: In this photo illustration an Editas Medicine logo seen displayed on a ... [+] smartphone with the stock market graphic in the background. (Photo Illustration by Rafael Henrique/SOPA Images/LightRocket via Getty Images)

Our theme of Gene Editing stocks continues to underperform, declining by about 16% year-to-date in 2022, compared to the S&P 500 which is down by about 6% over the same period. The theme also declined by about 11% in 2021. With interest rates rising and monetary policy set to get tighter, the markets are souring on high-growth and futuristic stocks. Gene editing stocks have been hit particularly badly as most players dont generate meaningful revenue as yet and remain deeply unprofitable. Some of the companies in the theme have also witnessed clinical setbacks or seen mixed data from their clinical trials over the last year (see updates below).

So whats the outlook like for the theme? The sector clearly remains out of favor with the market and could see more volatility through 2022 if investors continue to move out of riskier assets amid rising interest rates. Liquidity could also be an issue for smaller players such as Editas Medicine which have been burning through cash, meaning that potentially dilutive fundraises are a possibility.

However, with market capitalizations of gene editing stocks remaining depressed following the sell-off over the last year, some companies could be attractive acquisition targets for larger pharma companies looking for expertise and pipelines in the gene-editing space. The long-term outlook for gene editing as a larger theme also appears promising, given the potentially revolutionary drugs under development, that could cure conditions from cancer to rare genetic disorders that currently lack treatments, to more chronic conditions such as diabetes. Considering this, the theme could see upside in the long term and the recent correction could be a buying opportunity.

Within our theme, Editas Medicine has been the worst performer, declining by about 30% year-to-date in 2022. On the other side, Vertex Pharmaceuticals has been the best performer, with its stock up around 10% year-to-date.

Below youll find our previous coverage of the Gene Editing theme where you can track our view over time.

[8/13/2021] Will Modernas Interest Boost Gene Editing Stocks?

Our indicative theme of Gene Editing stocks has returned about 11% year-to-date, compared to the S&P 500 which is up by about 19% over the same period. However, the gains have overwhelmingly come from a single stock, Intellia Therapeutics, which is up by about 3x year-to-date, after the company announced positive results from early-stage clinical trials for its experimental treatment for transthyretin amyloidosis, marking the first time genome editing was carried out inside the human body to treat disease. The five other stocks in our theme remain down year-to-date. For instance, Editas Medicine remains down by about 6.8%, while bluebird bio remains down by about 56%.

That being said, we think the outlook for gene-editing stocks is looking better. Intellias progress bodes well for the broader gene-editing space, as it validates that gene-editing technology works in humans and also that it remains safe. As more of these companies move candidates into clinical stages and provide readouts, we could see movements in stock prices across the theme. Moreover, gene-editing companies could be ripe for buyouts. For instance, Covid-19 vaccine behemoth Modernas management indicated that it was interested in expanding into other areas, including gene editing. Considering that a majority of gene-editing stocks are small to mid-cap companies, they could easily be acquired by larger players such as Moderna.

[7/1/2021] Gene Editing Stocks Are Worth A Look After Intellias Big Breakthrough

Intellia Therapeutics - a gene-editing company co-founded by CRISPR pioneer and Nobel prize winner Jennifer Doudna - indicated that NTLA-2001, its experimental treatment for transthyretin amyloidosis provided very promising results in an early state trial. Although the study was small, including just six patients, the company noted that there were significant reductions in levels of a harmful liver protein that is associated with the disease after a single infusion. Intellia stock has rallied by almost 80% over the last three trading days following the news.

Now, we think that this could be a big deal for the broader gene editing sector, as well. This was the first report from a clinical trial of genome editing carried out inside the human body to treat disease, and the results should broadly validate that gene-editing technology works in humans and also that it remains safe. Our indicative theme of Gene Editing stocks has rallied considerably over the last week, and remains up by roughly 20% year-to-date, compared to the S&P 500 which is up by about 15% over the same period. That said, the gains are primarily driven by Intellia stock, which is up by almost 3x year-to-date, and the five other stocks in our theme have actually underperformed the market, or declined this year. For example, CRISPR Therapeutics is up by just about 6%, while Vertex Pharmaceuticals and Editas Medicine are down by 15% and 19%, respectively. Sangamo Therapeutics is down 23% (chart, 10-k), while bluebird bio is down by 26%. As more of these companies move candidates into clinical stages and provide readouts, we could see gains in stock prices across the theme.

[6/14/2021] Should You Add Gene Editing Stocks To Your Portfolio?

Our indicative theme of Gene Editing stocks is down by about 12% year-to-date, compared to the S&P 500 which is up by over 13% over the same period. The decline comes as investors move money from high-growth and futuristic sectors to more cyclical and value stocks to ride the post-Covid surge in economic activity over the next few quarters. Gene Editing players have been particularly badly hit by this shift, given that they are mostly clinical or pre-clinical stage biotechs with little or no revenues. Now, although most of the companies in our theme are currently losing money, and are presently out of favor with the market, the longer-term upside could be sizable, given that they are working on potentially revolutionary drugs that could cure conditions from cancer to rare genetic disorders that currently lack treatments, to chronic conditions such as diabetes.

Within our theme, Intellia Therapeutics was the strongest performer, rising by about 57% year-to-date, due to favorable views from brokerages and anticipation surrounding the companys NTLA-2001 drug, which is a single-course, potentially curative therapy for transthyretin amyloidosis. A data readout from the phase 1 study on the drug is due later this month. On the other side, Editas Medicine has been the worst performer in our theme, declining by about -47% year to date, partly due to its big rally late last year, multiple analyst downgrades, and some changes at the top management level.

[3/29/2021] Gene Editing Stocks Have Corrected. What Next?

Our indicative theme of Gene Editing stocks is down by about 19% year-to-date, compared to the S&P 500 which is up by about 6% over the same period. With the economic recovery expected to gather pace, on the back of declining Covid-19 cases and higher vaccination rates, bond yields have been trending higher, causing investors to move funds from highly valued growth names to more cyclical and value bets. Gene Editing players have been particularly badly hit by this shift, given that they are mostly clinical or pre-clinical stage biotechs with little or no revenues. That said, we think that this could be a good time to take a look at the sector, considering that these companies are working on potentially revolutionary developments that could cure conditions from cancer to rare genetic disorders.

Within our theme, Intellia Therapeutics was the strongest performer, rising by about 19% year-to-date. Last November, the company began dosing under its phase 1 study is to evaluate its drug NTLA-2001 which is a single-course, potentially curative therapy for transthyretin amyloidosis. A data readout is due sometime in the next several months. On the other side, Editas Medicine has been the worst performer, declining by about 42% year to date, partly due to its big rally late last year, multiple analyst downgrades, and some changes at the top management level. See our earlier updates below for a detailed look at the components of our Gene Editing stocks theme.

[2/10/2021] Gene Editing Stocks To Watch

Our indicative theme of Gene Editing Stocks is up by about 187% since the end of 2018 and by about 5% year-to-date. Gene editing has received more attention this year, as scientists used the technology to cure progeria syndrome in mice, raising hopes for therapy in humans as well. Progeria is a very rare genetic condition that causes premature aging in children, shortening their lifespan to approximately 14 years. Investors also remain interested in the sector, given that it could revolutionize medicine and also due to the fact that absolute valuations arent too high, with most of the companies remaining in the mid-cap space.

Within our theme, Intellia Therapeutics (NASDAQ: NTLA) has been the strongest performer year-to-date, rising by around 35% since early January. The company recently outlined strategic priorities for 2021, which include the continued advancement of a phase 1 study for a single-course therapy for protein misfolding disorder and the planned submission of regulatory applications for the treatment of acute myeloid leukemia and hereditary angioedema this year. On the other side, Vertex Pharmaceuticals, has declined by about 10% year to date, driven partly by weaker than expected Q4 2020 results. See our updates below for a detailed look at the components in our theme.

[1/27/2021] How Are Gene Editing Stocks Faring?

Gene-editing technology is used to insert, edit, or delete a gene from an organisms genome, and shows promise in treating medical conditions ranging from cancer to rare genetic conditions. Our indicative theme on Gene Editing Stocks has returned over 170% since the end of 2018, compared to the broader S&P 500 which is up by about 54% over the same period. The theme has returned about 2.4% year-to-date. Investor interest in gene-editing remains high, given the upside potential of the sector and considering that absolute valuations arent too high, with most of the stocks remaining in the mid-cap space. Intellia Therapeutics (NASDAQ: NTLA) has been the strongest performer in our theme this year so far, rising 18% since early January. The gains come as the company has outlined strategic priorities for 2021, which include the continued advancement of a phase 1 study for a single-course therapy for protein misfolding disorder and the planned submission of a regulatory application for the treatment of acute myeloid leukemia. [1] On the other side, Editas Medicine has declined by about 13% year to date, after the company indicated that it plans to raise additional capital, issuing about 3.5 million shares at $66 per share. See our update below for a detailed look at the components in our theme.

[1/8/2021] Gene Editing Stocks

Gene editing has emerged as a promising biotech theme. The technology is used to insert, edit, or delete a gene from an organisms genome, helping to replace the defective genes responsible for a medical condition with healthy versions. This technology is being used to develop treatments for a range of diseases from cancer to rare genetic conditions, that are otherwise hard to treat, and is also being considered for diagnostic purposes. While there are broadly three gene-editing technologies, clustered regularly interspaced short palindromic repeats or CRISPR, as it is popularly known, has emerged as the method of choice with most companies, considering that it is relatively inexpensive, simpler, and more flexible compared to other tools such as ZFN and TALEN.

While most gene-editing players remain in the clinical stage with a limited financial track record, funding has risen meaningfully and larger pharma companies are also partnering with these companies, considering that the treatments could be lucrative and the broader technologies may be highly scalable. While the upside remains large, investing in these companies is risky. Being a new technology that has never been used in humans before, there are risks of significant side effects or of the therapies not being effective. The economics of producing and selling these drugs also remains uncertain. These stocks are also volatile, seeing big swings as any new research or data on their potential or risk is outlined. Our indicative theme on Gene Editing Stocks - which includes names such as CRISPR Therapeutics, Editas Medicine, and others - has returned about 230% over the past 2 years, compared to the broader S&P 500 which is up by about 52% over the same period. Below is a bit more about these companies.

CRISPR Therapeutics AG is one of the best-known names in the gene-editing space. The company is working with Vertex Pharmaceuticals to co-develop CTX001, an experimental gene therapy that has provided promising results for people with sickle cell disease, and transfusion-dependent beta-thalassemia - disorders that affect the oxygen-carrying cells in human blood. The company is also developing cancer therapy candidates independently. The company was profitable last year, due to collaboration revenues from Vertex.

CRSP

Editas Medicine, another leading CRISPR-focused biotech company, with a flagship program, EDIT-101 is targeting the treatment of hereditary blindness. The company recently finished dosing for its first group of patients in earlier-stage human trials. The company also recently filed a request with the U.S. FDA to commence phase 1/2 study of EDIT-301 in treating sickle cell disease. The company also has multiple other pre-clinical drugs focused on genetic diseases.

Intellia Therapeutics is developing a drug for a rare and fatal disease known as transthyretin amyloidosis in collaboration with Regeneron. The drug is in phase 1 trials currently. The company is also working on ex-vivo Sickle Cell Anemia treatment with Novartis that involves editing cells outside the body before infusing them into the patient. The candidate is entering Phase 1/2 trails. While the company has 8 other candidates, they are still in the research or pre-clinical stages. [2]

Sangamo BioSciences focuses on multiple areas in the genomic medicine space, including gene therapy, cell therapy, in vivo genome editing, and in vivo genome regulation. The company pioneered the zinc finger nuclease gene-editing method. The companys most advanced development is a treatment for Hemophilia A, which is being developed with Pfizer and is in phase 3 trials. The company also has 4 candidates in the phase 1/2 stage and 13 in the Preclinical stage. [3]

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The Sell Off In Gene Editing Stocks Continues. Time To Buy? - Forbes

Today, its clear that our genes not only cause many diseases, but also hold potential cures. But that wasnt always the case. It wasnt until 1949 that scientists first found the molecular culprit of a disease its roots in the genetic code. The disease was the blood disorder known as sickle cell disease, an inherited disorder that causes severe and debilitating pain. Now, nearly 75 years later, researchers are developing gene therapies to cure it.

Sickle cell disease results from a change in a key protein in hemoglobin, which helps transport oxygen in red blood cells. Hemoglobin normally allows red blood cells to be very floppy and pliable, and slip and slide through the blood vessels easily, says pediatrician Erica Esrick. But a mutation in a single gene, the HBB gene, makes hemoglobin stack in long strings inside blood cells, giving them an inflexible, sickle shape. Instead of being squishy, the stiff red blood cells get stuck inside blood vessels, blocking blood flow.

Sickle cell affects millions of people around the world, particularly those whose ancestors come from sub-Saharan Africa, parts of the Middle East and Southeast Asia. In the United States, for instance, approximately 100,000 people live with the disease, most of them Black or Latino. People with sickle cell disease have a shortened life expectancy, living only into their late 40s on average, in large part due to strokes or organ damage from blocked blood vessels. Esrick, of Boston Childrens Hospital and Harvard Medical School, and others are trying to fight the disease through gene therapy.

Gene therapies seek to manipulate the very information of life by replacing, inactivating or fixing missing or broken genes and so curing patients. But the journey to todays handful of approved gene therapies, including for diseases like severe combined immunodeficiency syndrome, or SCID, certain blood cancers and spinal muscular atrophy, has been rocky. Early clinical trials in the 1990s werent effective, and the 2000s brought unintended and sometimes deadly consequences, including a leukemia-like illness.

Despite gene therapys challenges, many researchers believe sickle cell is a good target because the molecular pathways are well understood and straightforward. Whats more, every copy of the gene doesnt need to be mended to have an effect. (Individuals who inherit the mutated gene from only one parent, for example, dont develop sickle cell disease.)

Esrick is co-leading a clinical trial testing a gene therapy that attempts to encourage the body to make more of a healthy type of hemoglobin produced by fetuses and young babies but not adults called fetal hemoglobin. DNA for making a short string of genetic material called a microRNA is delivered by a virus into cells from a patients bone marrow. The virus, called a vector, permanently inserts the DNA into the cells genetic blueprint. The microRNA then interferes with the production of a protein that prevents fetal hemoglobin from being made. Once that protein is blocked, fetal hemoglobin production turns back on. Like turning on a faucet, a steady stream of the healthy hemoglobin can flow into the bloodstream, making up for the faulty form.

Preliminary data released in January 2021 showed that the treatment helped six sickle cell patients make fetal hemoglobin, Esrick and colleagues reported in the New England Journal of Medicine. During the follow-up period, ranging from several months to more than two years, the patients symptoms were reduced or eliminated. The team has expanded the trial to include more patients and further test the treatment.

Scientists are testing other ways to tackle sickle cell via gene therapy, too. A biotechnology company called bluebird bio is testing an approach that delivers a functional copy of the HBB gene to patients. Another team is preparing to begin a trial that will edit that gene directly using CRISPR/Cas9.

Science News staff writer Erin Garcia de Jess spoke with Esrick about the ongoing fetal hemoglobin clinical trial, including the hurdles and the hope. The conversation has been edited for length and clarity.

Garcia de Jess: What tools do we currently have to treat sickle cell?

Esrick: The only curative treatment is a bone marrow transplant. The bone marrow is like the factory for the blood cells. If you can get bone marrow from somebody who doesnt have sickle cell disease, then you can grow your own healthy red blood cells that dont sickle. But that is a major procedure, and its really only standard if you have whats called a matched sibling [a brother or sister without sickle cell whose key white blood cell proteins match yours].

Less than 20 percent of people with sickle cell have a matched sibling available. If a matched sibling is available, then thats a really good potential treatment option, but it is still a risky procedure. It comes along with some up-front risk of mortality and a lot of potential side effects, such as graft-versus-host disease and a higher risk of infection because of immunosuppressive drugs.

Then there are medications to treat sickle cell. The most well-established and long-lasting is called hydroxyurea. It increases fetal hemoglobin. In many people, it increases the fetal hemoglobin by a lot; thats why it works so well. Its been available since the 90s, and has been moving gradually to younger and younger ages.

Now it is a very clear recommendation that essentially every child with sickle cell should be on it. But not everyone has access to specialized hematology care, and its a medication that has to be taken daily. Some people have adverse effects and cant take it. It also doesnt work for everybody.

Garcia de Jess: How many people are in your teams trial and what results have you seen so far?

Esrick: Nine patients have been treated. We anticipate the 10th patient will be treated soon. The preliminary data from the first six patients was published about a year ago. Additional data from subsequent patients has been largely quite similar except for one patient whose fetal hemoglobin response was unfortunately not as robust.

Garcia de Jess: What is the process like for the trial participants?

Esrick: Patients have to get their cells collected [the cells live in the bone marrow and give rise to blood cells], which takes a three-day hospital admission and sometimes has to be repeated a few times. Its through IV, basically. Then the cells get taken off to the lab.

When we get word from the lab, OK, we have a good product [meaning the virus got the DNA into enough cells], then the patient comes back and is admitted to the hospital for a month or so. Its a long and arduous hospital admission because they need to receive chemotherapy.

The reason they need chemotherapy is because the bone marrow cells that havent been collected need to get nearly wiped out in order to give the advantage to the cells that are being given back [also through IV] to set up shop and produce.

Chemotherapy comes with a lot of the side effects and risks associated with gene therapy, including acute short-term risks like hearing loss and nausea. And it also comes with some of the long-term risks, including infertility and a risk of blood cancers.

Garcia de Jess: Why choose gene therapy over a bone marrow transplant if both require chemotherapy?

Esrick: With gene therapy, theres no issue with immunosuppression, because its your own cells. People who get a transplant from another person have to be on immunosuppressive medications for a period of months after the transplant. Theres a risk of graft rejection because of the mismatch between the donor and the recipient.

The other risk in a bone marrow transplant from another person is graft-versus-host disease, where the graft and donated cells reject the recipient. That can cause severe disease. With gene therapy, thats not a risk at all.

Garcia de Jess: Last year, a clinical trial run by a company called bluebird bio announced that a trial participant developed leukemia. Cancer is obviously a huge concern and has thwarted previous gene therapy trials. What do we know so far about that?

Esrick: This was, of course, of major concern to the field. It was actually the second case of leukemia in that trial. The first one was published a couple of years ago as a case report.

If theres ever a case of leukemia or any preleukemia in a gene therapy trial, we always ask: Was it caused because the vector stuck a gene into a spot that was dangerous?

It does not look like thats the case. In the first patient in the bluebird bio trial who developed leukemia, the leukemia cells didnt even have the transferred gene in them. So, the thought was that was probably just an example of chemotherapy causing leukemia, which we know can happen in a small percent of people who receive chemotherapy.

But the second case, in February 2021, really raised a red flag. Why is that happening two times in a trial of only 40-something patients? Its still not exactly clear. There are some studies that suggest that people with sickle cell disease may have an increased risk of leukemia. But the [U.S. Food and Drug Administration] placed the bluebird bio trial on hold while some investigations were done. When it became pretty clear that it wasnt directly related to the vector, the trial was allowed to reopen.

Our trial, which has many similarities to the bluebird bio trial, was not put on hold by the FDA but was put on hold by our funder, the National Heart, Lung and Blood Institute while they looked at the data. That hold was recently lifted.

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Garcia de Jess: Have there been any cases of leukemia in your teams trial?

Esrick: Fortunately, no.

But you know when anything like that happens in the field, its a big deal. I called all of the patients who we had treated in our trial to let them know. [The bluebird bio cases] happened in patients who had been treated three and five years prior. The longest-treated patient in our trial was almost three and a half years ago, and the most recently treated was about eight or nine months ago. I hope we see no concerning signs for any new development like that, but its too early to say.

Garcia de Jess: What are some of the biggest challenges that sickle cell has had to overcome?

Esrick: For the longest time, there were no new therapies at all. These technologies took a long time because they are based on basic science discoveries that were being worked on. But also, the patient population with sickle cell is a population that has historically been underserved and without a lot of power.

In the United States, its primarily Black and Latino patients, and across the board those populations have suffered from health inequality. I think that if there were a disease that caused this degree of morbidity and mortality and pain in other parts of the population, it may have been speedier.

Garcia de Jess: What gives you hope? What do you find exciting?

Esrick: I find myself bending over backwards to make sure that Im not coming across as, We have a cure! But that said, it is really exciting that this is a treatment that is theoretically possible for everyone without needing to find a [bone marrow] match. Thats a huge difference from classic bone marrow transplants.

The speed at which new [gene therapy] treatments are being developed is amazing. I think the horizon is very bright in terms of one or maybe many of these therapies being really effective and safe. Ive talked to so many patients and families who have reached out interested in our trial or other trials. Theres such a huge unmet need. The fact that there are a lot of these new treatments that are being developed is an encouragement to these families.

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Gene therapies for sickle cell disease come with hope and challenges - Science News Magazine

Six researchers from Washington University in St. Louis have been named senior members of the National Academy of Inventors (NAI).

Richard Axelbaum, PhD, at the McKelvey School of Engineering, along with five researchers at the School of Medicine David T. Curiel, MD, PhD; James W. Janetka, PhD; Gregory M. Lanza, MD, PhD; Robi D. Mitra, PhD; and Jennifer N. Silva, MD are being recognized for their success in patents, licensing and commercialization, and for producing technologies that have the potential to have a significant impact on the welfare of society.

They are among 83 new senior members who will be honored in June at the NAI annual meeting in Phoenix.

Axelbaum, a professor of energy, environmental and chemical engineering and the Stifel & Quinette Jens Professor of Environmental Engineering Science, researches combustion in its many forms and in different environments including in microgravity on the International Space Station. He uses his understanding of fossil fuel combustion and its resulting pollutants to address concerns over carbon dioxide emissions, notably by developing novel approaches to carbon capture and storage. Axelbaum holds 10 patents and founded the startup company AP Material Inc., which commercialized a flame-synthesis technology to manufacture high-purity nanopowders.

Curiel, a professor of radiation oncology and director of the Biologic Therapeutics Center at the School of Medicine, has harnessed gene therapy and viral vectors to develop therapeutics and vaccines for a number of diseases, including cancer, genetic disorders and COVID-19. More recently he has pioneered gene editing strategies toward gene therapy cures. He has co-founded a number of biotechnology startups, including Altimmune, DNAtrix, Unleash Immuno Oncoloytics and Precision Virologics. Most recently, he co-developed a nasal vaccine against COVID-19 that is in phase 3 human clinical trials in India.

Janetka, a professor of biochemistry and molecular biophysics, is a medicinal chemist who is developing small-molecule therapeutics to treat cancer and infectious diseases. He has expertise in rational, structure-based drug design. Janetkas team is working on novel drugs for treatment of a broad scope of infections caused by bacteria, parasitic worms, toxoplasma and viruses. His team has discovered broad-spectrum new anticancer and antiviral drugs for the treatment of COVID-19. He is a co-founder of two pharmaceutical startups, Fimbrion Therapeutics and ProteXase Therapeutics.

Lanza, a cardiologist and professor of medicine, of biomedical engineering, and of biology and biomedical sciences, has developed nanotechnologies with wide applications in medicine, from cardiovascular imaging to cancer therapy. He was the co-founder, chief scientific officer and a board member of Kereos Inc., a biotechnology startup focused on developing molecular imaging agents and therapeutics for cardiovascular disease and cancer. He co-founded and is chief scientific officer of Capella Imaging Inc., a startup focused on biomedical imaging, and is in partnership with NorthStar Medical Radioisotopes, with a particular focus on detecting blood clots in the heart and in operating ventricular assist devices.

Mitra, a professor of genetics and the Alvin Goldfarb Distinguished Professor of Computational Biology, develops new technologies for analyzing the genome. He and his lab have pioneered new methods for efficient DNA sequencing, analyzing the binding of transcription factors, studying single-molecule proteomics, analyzing single-cell genomics and capturing specific regions of the genome. Mitra played key roles in the founding of a number of genomic resources for the School of Medicine, including the universitys Genomic Technology Access Center, the Genomics and Pathology Services Lab, and the Genome Engineering and iPSC Core facility.

Silva, a pediatric cardiologist and professor of pediatrics, is an electrophysiologist who treats children with disorders of the heart, including those that cause life-threatening arrhythmias. She has developed a 3D imaging system using virtual-reality technology to help cardiologists better visualize the electrical circuits that are misfiring. She co-founded the startup SentiAR to develop a headset that can display a hologram of a patients heart and show a real-time 3D map of what is happening as the patient undergoes a catheter ablation procedure, in which the tissue causing the arrhythmia is burned to stop the erratic signals.

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Six innovators named National Academy of Inventors senior members - The Source - Washington University in St. Louis - Washington University in St....

PARIS, FRANCE (February 8, 2022) Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.

"These results confirm the stability and good tolerance of the lentiviral vector as a tool for gene transfer into blood stem cells, said Anne Galy, Ph.D., Inserm Research Director at Genethon. Our teams have carried this project from translational research to a clinical trial by working with the best clinical and research teams internationally. We are delighted with the results of this trial which now show the long-term efficacy and safety of this approach for this rare and severe immune deficiency.

Frederic Revah, Ph.D., CEO of Genethon, added, I would like to congratulate Anne Galy and her team who have been working on this project for more than 15 years in the service of patients and their families. This trial for Wiskott-Aldrich syndrome was the first international trial launched by our laboratory, and today 12 clinical trials are being conducted worldwide for products stemming from our R&D. From basic research to clinical development, Genethon has developed a unique expertise in the field of gene therapy for different families of rare diseases.

The long-term results of the WAS clinical trial, sponsored by Genethon and conducted by colleagues in France and England, were published in Nature Medicine, in a paper titled Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for WiskottAldrich syndrome. The vector used in the study was designed, developed and manufactured by Genethon.

WAS, a rare and severe complex immune deficiency, is caused by a mutation in the WAS gene in hematopoietic progenitor cells, which are blood forming cells. The inherited disease affects only boys and results in hemorrhages, repeated severe infections, severe eczema and, in some patients, autoimmune reactions and development of cancers. The only treatment currently available is bone marrow transplantation, which requires a compatible donor and can cause serious complications. Symptoms of the disease emerge at 6 months old and life expectancy for severe forms is 3.5 years without treatment.

Genethons gene therapy involves extracting from patients the blood stem cells carrying the genetic abnormality, correcting them in the laboratory with a healthy WAS gene and transplanting the cells back into the patients. Initial results from the clinical trial, published in the Journal of the American Medical Association in 2015 showed safety and efficacy along with stabilized engraftment of the blood cells 9 months to 42 months after the treatment.

Results from the longer term follow-up of eight patients for a median of 7.6 years confirm the stability of the transplanted genetically modified cells and their safety and efficacy. The gene therapy corrected major disease symptoms, improved or eliminated bleeding and signs of autoimmunity, and restored T-cell (or immune system) function. In addition, a 30-year-old patient was treated in the trial, demonstrating efficacy in adult patients whose thymus gland, which makes T-cells, was thought to be low- to non-functioning after many years of illness. Platelet levels remain low but gene therapy alleviates the need for platelet transfusions and prevents the occurrence of spontaneous hemorrhages.

The WAS gene therapy clinical trial was sponsored by Genethon and conducted in collaboration with Inserm (the French National Institute of Health and Medical Research) and NeckerEnfants Malades Hospital in France; and the University College of London, Great Ormond Street Hospital and Royal Free London Hospital in England.

About Genethon

A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a unique non-profit organization created by a patient association, the AFM-Telethon. A first gene therapy drug, to which Genethon contributed, has obtained marketing for spinal muscular atrophy. With 200+ scientists and professionals, Genethon is pursuing its mission to bring life-changing therapies to patients suffering from rare genetic diseases. 12 products resulting from Genethons research are in clinical trials for eye, liver, blood, immune system and muscle diseases. A further 7 products are in the preparation phase for clinical trials over the next five years. Find out more: genethon.com

Contacts:

Dan Eramian

Opus Biotech Communicationshttp://opusbiotech.com/425-306-8716

danieleramian@comcast.net

Charles Craig

Opus Biotech Communications

http://opusbiotech.com/404-245-0591

charles.s.craig@gmail.com

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Genethon's Lentiviral vector-based gene therapy demonstrates long-term safety and efficacy for Wiskott-Aldrich Syndrome - EurekAlert