The Gene Therapy report provides independent information about the Gene Therapy industry supported by extensive research on factors such as industry segments size & trends, inhibitors, dynamics, drivers, opportunities & challenges, environment & policy, cost overview, porters five force analysis, and key companies profiles including business overview and recent development.

Gene Therapy MarketLatest Research Report 2020:

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In this report, our team offers a thorough investigation of Gene Therapy Market, SWOT examination of the most prominent players right now. Alongside an industrial chain, market measurements regarding revenue, sales, value, capacity, regional market examination, section insightful information, and market forecast are offered in the full investigation, and so forth.

Scope of Gene Therapy Market: Products in the Gene Therapy classification furnish clients with assets to get ready for tests, tests, and evaluations.

Major Company Profiles Covered in This Report

Pfizer Inc.,Novartis AG,Bayer AG,Sanofi,GlaxoSmithKline plc.,Amgen Inc.,Boehringer Ingelheim International GmbH,uniQure N.V.,bluebird bio, Inc.,Celgene Corporation

Gene Therapy Market Report Covers the Following Segments:

Application: By Disease Indication, Cancer, Genetic disorders, Cardiovascular diseases, Ophthalmology, Neurological conditions, Others,,By Type of Vectors, Viral vectors, Non-viral vectors,,By Type of Cells, Somatic cells, Germline cells

North America

Europe

Asia-Pacific

South America

Center East and Africa

United States, Canada and Mexico

Germany, France, UK, Russia and Italy

China, Japan, Korea, India and Southeast Asia

Brazil, Argentina, Colombia

Saudi Arabia, UAE, Egypt, Nigeria and South Africa

Market Overview:The report begins with this section where product overview and highlights of product and application segments of the global Gene Therapy Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company:Here, the competition in the Worldwide Gene Therapy Market is analyzed, By price, revenue, sales, and market share by company, market rate, competitive situations Landscape, and latest trends, merger, expansion, acquisition, and market shares of top companies.

Company Profiles and Sales Data:As the name suggests, this section gives the sales data of key players of the global Gene Therapy Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the global Gene Therapy Market.

Market Status and Outlook by Region:In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the global Gene Therapy Market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User:This section of the research study shows how different end-user/application segments contribute to the global Gene Therapy Market.

Market Forecast:Here, the report offers a complete forecast of the global Gene Therapy Market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Research Findings and Conclusion:This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

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We publish market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students.

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Gene Therapy Market Size Analysis and Growth (2020-2025) Owned - Owned

Global Tissue Engineering Gene Therapy Market: Past, Current, and Future Market Analysis, Trends, and Opportunities, 2016-2026

The new report published by theMarket Research StoreglobalTissue Engineering Gene Therapy marketis slated for a rapid growth in the coming years. The research study projects that the market is expected to grow at a good CAGR of XX% during the forecast period. The valuation for the Tissue Engineering Gene Therapy market made by our research analysts is around USD XX Million in 2019 and anticipates USD XX Million by the end of 2026.

Request a sample copy of this report@http://www.marketresearchstore.com/report/global-tissue-engineering-gene-therapy-industry-market-report-643176#RequestSample

The competitive landscape evaluation of the Tissue Engineering Gene Therapy market players includeOrthocell, Histogenics, Epithelix Sarl, Biostage, BioAxone Biosciences, Histogen, Admedus, Osiris Therapeutics. The information that is profiled for each of the market player will include their primary foundation business model as well as their current business strategy, SWOT analysis, their market share, revenue, pricing, gross margin , and the recent developments.

Tissue Engineering Gene Therapy Market Report Insights

Overview of the Tissue Engineering Gene Therapy market, its scope, and target audience. In-depth description about the market drivers, restraints, future market opportunities, and challenges. Details about the advanced technologies, including big data & analytics, artificial intelligence, and social media platforms used by the global Tissue Engineering Gene Therapy Market Primary legislations that will have a great impact on the global platform. Comprehensive analysis about the key players in the global Tissue Engineering Gene Therapy market. Recent developments, mergers and acquisitions, collaborations, R&D projects are mentioned in the Tissue Engineering Gene Therapy market report.

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Tissue Engineering Gene Therapy Market Segmentation

Global Tissue Engineering Gene Therapy market: By Type Analysis

Autologous cell therapy, Allogeneic cell therapy

Global Tissue Engineering Gene Therapy market: By Application Analysis

Implantable Grafts, Tissue Healing and Remodeling, Wound Healing and Wound Care, 3D Organoids, Dental Tissue Engineering, Organ Engineering

Global Tissue Engineering Gene Therapy market: By Regional Analysis North America Europe Asia Pacific Latin America Middle East and Africa

If Any Inquiry of Tissue Engineering Gene Therapy Report:http://www.marketresearchstore.com/report/global-tissue-engineering-gene-therapy-industry-market-report-643176#InquiryForBuying

In the segmentation part of the report a thorough research of each and every segment is done. For in-depth information some of the major segments have been segregated into sub-segments. In the regional segmentation also our research analysts have not only concentrated on the major regions but have also included the country-wise analysis of the Tissue Engineering Gene Therapy market.

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Global Tissue Engineering Gene Therapy Market 2020 Coronavirus (COVID-19) Updated Analysis By Product (Autologous cell therapy, Allogeneic cell...

NEW YORK, July 27, 2020 /PRNewswire/ -- Report Scope: The scope of this study encompasses an investigation of the markets cell and gene therapy tools such as GMP proteins, media, cell separation and activation reagents, viral and non-viral, cytokine release syndrome monitoring products, GMP antibodies, leukapheresis instrumentation, immunoassays (multiplex and singleplex) and bioreactors.The analyst examines each tool type, determines its current market status, examines its impact on future markets, and presents forecasts of growth over the next five years.

Read the full report: https://www.reportlinker.com/p05938974/?utm_source=PRN

Technological issues, including the latest trends, are discussed. The analyst examined the industry on a worldwide basis, from both application and demand perspectives, in the major regions of the world.

Report Includes: - 77 data tables - An overview of the global market for immunoassay-based cell and gene therapy tools, and reagents within the life sciences industry - Analyses of the global market trends, with data corresponding to market size for 2017-2019, and projections of compound annual growth rates (CAGRs) through 2024 - Information about in vitro diagnostics (IVD), its background and importance of IVD testing - Discussion on prevalence of infectious disease, metabolic disorders and chronic ailments as drivers of immunoassay testing market - Description of recent technologies such as chemiluminescent immunoassays (CLIA), fluorescent immunoassays, multiplex assays and rapid diagnostic technologies - Evaluation of market potential for immunoassay-based cell and gene therapy tools and reagents, current market size and market forecast, and market share analysis of the leading players engaged - Patent review and new developments, R&D efforts, industrial changes with emphasis on recent investments, and current state of the immunoassay technology - Company profiles of the key market participants, including Bio-Rad Laboratories, Cytiva Life Sciences, Luminex Corp., Millipore Sigma, Thermo Fisher Scientific and STEMCELL Technologies

Reasons for Doing this Study: Gene and cell therapy are emerging as important tools to treat human health.Techniques such as CAR-T therapy have emerged as key ways of treating many different types of cancers.

The promise of gene therapy using technologies such as CRISPR is starting to be realized in clinical trials, and markets are scaling up to treat other diseases as well, particularly rare gene- based diseases.As these therapies are coming to the fore, a new market for tools to develop these therapies using standard methodologies is emerging.

This report will cover what those tools are, how they impact the larger life science tools market, and how they will evolve over the next five years.

Read the full report: https://www.reportlinker.com/p05938974/?utm_source=PRN

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Cell and Gene Therapy Tools, and Reagents: Global Markets - Yahoo Finance

Six months after departing Abeona, Tim Miller, Ph.D., is on his next chapter: Forge Biologics, a gene therapy biotech thats providing manufacturing and development services to other companies while working on its own pipeline. To fuel this dual mission, Forge picked up $40 million from the Perceptive Xontogeny Venture Fund and Drive Capital.

Forge is the brainchild of Miller and a confluence of gene therapy experts who saw a widening gap between the growth of the industrys gene therapy pipeline and its capacity to actually manufacture the treatments, Miller told Fierce Biotech.

Those experts include Jaysson Eicholtz, who previously oversaw gene therapy manufacturing for Nationwide Childrens Hospital; Erandi De Silva, Ph.D., who led program management at Myonexus Therapeutics; and Maria Escolar, M.D., the University of Pittsburgh professor who developed Forges lead program. The trio are Forges chief operating officer, chief strategy officer and chief medical officer, respectively.

RELATED: Novartis, Sarepta tap Dyno to unearth new gene therapy vectors

We came together with the idea that if we did this hybrid play, what you end up with is a development engine that helps secure the needs of additional clients, serving as an end-to-end solution provider, but also being able to do that for ourselves and build out our pipeline through that engine capacity, Miller said.

Like other companies with a hub-and-spoke model, Forge comprises a core that offers vector design know-how, adeno-associated virus (AAV) vector manufacturing and an experienced management team, and will set up subsidiary entities around therapeutics, Miller said.

Forges 175,000-square-foot manufacturing site is already up and running, but the series A funding will boost its capacity and enable cGMP manufacturing by the middle of 2021.

The cash will also propel Forges lead program, FBX-101, which combines an AAV gene therapy with an umbilical cord bone marrow transplant. Forge is developing it for infants with Krabbe disease, a neurodegenerative disorder that destroys myelin, the fatty substance that coats and protects nerve fibers in the brain and nervous system. The combination approach could address a challenge in using AAVs to deliver gene therapies: Some people have developed a natural immunity to this type of virus, rendering an AAV gene therapy ineffective.

RELATED: ElevateBio reels in $170M to push 6 cell, gene therapy programs

The transplant is solving for two problems at the same time, Miller said. Youre not going to see an immune response against the protein expressed from the gene therapy approach. And youre able to remove a lot of the immune response to AAV capsids overall.

Beyond Krabbe disease, Forge has two more AAV gene therapies, but its not divulging which diseases they are for. And it could pick up more programs as it signs on more clients.

Those clients could be biopharma companies looking for a manufacturing partner, but they could just as easily be an academic center with programs its looking to spin out or a disease foundation that wants to use its proceeds to advance a gene therapy program, Chris Garabedian, manager of the PXV Fund, told Fierce Biotech.

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'Gene therapy engine' Forge debuts with $40M and Tim Miller at the helm - FierceBiotech

Precision gene therapy company Encoded Therapeutics, Inc. (Encoded) announced it has raised $135 million in an oversubscribed Series D round of funding. And the company also announced that its lead asset ETX101 was granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation by the U.S. Food and Drug Administration (FDA) for the treatment of SCN1A+ Dravet Syndrome.

Dravet syndrome is a rare, severe genetic disorder that occurs in about 1 in 16,000 births worldwide. The disorder is characterized by uncontrolled seizures, ataxia, significant developmental delays, and an increased risk of early mortality due to sudden unexpected death in epilepsy (SUDEP). The majority of Dravet Syndrome cases are caused by loss-of-function mutations in the SCN1A gene. And current treatments reduce seizures but do not address the underlying cause of the disorderSCN1A haploinsufficiency.

GV (formerly Google Ventures) led the Series D round of funding with participation from Matrix Capital Management, ARCH Venture Partners, Illumina Ventures, RTW Investments, Boxer Capital, Nolan Capital, HBM Genomics, Menlo Ventures, Meritech Capital, Farallon Capital Management, SoftBank Vision Fund 21, and several additional unnamed Encodeds discovery engine combines biological and computational approaches for identifying and screening human DNA sequences known as regulatory elements at a high throughput scale. And the resulting multi-dimensional large scale datasets are leveraged to design optimal gene therapy expression cassettes which more precisely control transgene expression.

By recapitulating natural patterns of gene expression, the resulting gene therapy vectors are able to provide maximal therapeutic benefit, minimize off-target expression, and address genetic disorders that were previously Encoded also announced that ETX101 was granted Orphan Disease and Rare Pediatric Disease Designation by FDA. Both programs offer incentives for the development of therapeutics for underserved populations.

The proceeds of the Series D round of funding will be used to conduct clinical trial activities including a natural history study to better understand the progression of SCN1A+ Dravet Syndrome as well as first-in-human trials for ETX101. And the funds will support progression of the companys pipeline of gene therapies being evaluated for additional pediatric CNS disorders.

David Schenkein, M.D., general partner and co-leader of GVs life sciences team, will join the Encoded Board of Directors as an observer in connection with the funding round.

KEY QUOTES:

I am incredibly proud of the progress our organization has made over the past year. We have built an outstanding team, further advanced ETX101 towards the clinic, applied our technology to a series of pipeline programs, and expanded our capabilities to become a fully integrated organization. We are grateful to our investors for supporting our vision to transform patients lives with cell type-selective genetic medicines.

Encoded co-founder and chief executive officer Kartik Ramamoorthi, Ph.D.

Since 2019, our company has attracted some of gene therapys most experienced executive leaders to prepare ETX101 for the clinic and beyond. This well-rounded team has made significant advances in manufacturing, clinical development, and regulatory affairs for ETX101. By coupling these with an innovative discovery engine, Encoded is advancing its preclinical pipeline of gene therapies to patients suffering from devastating pediatric CNS diseases.

Encoded board chairman Sean Nolan.

Encodeds cutting edge platform has the potential to impact a broad range of diseases across different organ systems. Importantly, Encoded has translated this innovation into a one-time, first-in-class precision gene therapy candidate for the treatment of SCN1A+ Dravet Syndrome, a devastating pediatric disease with significant unmet medical need. I am enthusiastic to work with this top-tier team in their mission to treat these underserved patients and advance a portfolio of innovative medicines.

Dr. Schenkein

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Encoded Therapeutics Closes $135 Million In Series D Funding - Pulse 2.0

Biomarin Pharmaceutical, a California company that makes what could become the first gene therapy for hemophilia, says its drug's price tag might be $3 million per patient. Maciej Frolow/Getty Images hide caption

Biomarin Pharmaceutical, a California company that makes what could become the first gene therapy for hemophilia, says its drug's price tag might be $3 million per patient.

Jack Grehan, who was born with hemophilia, used to inject himself every couple of days with a protein he needs for his blood to clot. But not anymore.

"It's been absolutely brilliant and life-changing for me," says Grehan, 26, of Billinge in North West England. He received an experimental gene therapy in 2017 that, at least for now, has eliminated his need for regular injections. "I can just go about my day and not have to worry."

Based on experiences like Grehan's, the company that developed the therapy is seeking approval in Europe and the United States to start selling the first gene therapy for hemophilia. That's generating excitement among patients, patient advocates and doctors.

"Not to have to worry about hemophilia any longer I think it's essentially transformational for many patients," says Dr. John Pasi of the Royal London Hospital and the London School of Medicine and Dentistry. Pasi led the recently published study Grehan took part in.

Jack Grehan, of Billinge, U.K., in North West England, is one recipient of BioMarin's still experimental gene therapy to treat hemophilia. He says his quality of life has improved dramatically since getting an infusion of the drug three years ago. Jack Grehan hide caption

Others are more cautious.

"This is really exciting, but also raises a lot of questions," says Meg Bradbury, director of research at the Hemophilia Federation of America, a patient advocacy group.

One of the biggest questions is the possible cost. BioMarin Pharmaceutical Inc. of San Rafael, Calif., the company that developed the gene therapy, says the treatment could cost as much as $3 million per patient, which would make it the most expensive drug ever approved.

"It's just outrageous," says Peter Bach, who studies drug prices at Memorial Sloan Kettering Cancer Center in New York.

Company officials defend the possible price, however. It currently costs hundreds of thousands of dollars a year to treat each hemophilia patient. The gene therapy would, researchers hope, be a one-time treatment that lasts a lifetime.

"The context is this gigantically expensive disease to treat," says Jeff Ajer, BioMarin's executive vice president and chief commercial officer. "It's likely that our gene therapy would save a lot of money millions, perhaps many millions."

Hemophilia is a relatively rare condition. Hemophilia A, the most common form of the disease, occurs in 1 of about every 5,000 boys. (Though girls can be born with hemophilia, too, that's much rarer.) People born with the condition are missing a crucial protein that their blood needs to clot. So doing things as simple as walking can cause dangerous bleeding in their joints that eventually can be crippling.

"It was quite difficult, because I was always getting these bleeds in the ankles," Grehan says. "So walking around was becoming more and more troublesome."

Grehan has a severe form of hemophilia A, which causes a deficiency in a clotting protein called factor VIII.

"It's the microbleeds that just sort of wear your down not even physically but mentally," Grehan says.

And Grehan knew that propensity to bleed could cause even more serious, possibly fatal, complications if the hemorrhage every happened in his brain or other parts of his body.

"Internally, there could have been a lot worse situations," Grehan says. "I consider myself quite lucky in that respect."

Before anything like that happened to Grehan, he learned that doctors were testing the gene therapy.

"When I first heard about the trial I thought it was unbelievable that we were in this situation that this even existed," Grehan says.

So he volunteered three years ago to let doctors infuse trillions of neutralized viruses that had been genetically engineered to carry the healthy gene he needed into his liver.

Within a couple of weeks, Grehan could stop injecting himself with the clotting protein that he had previously needed.

In fact, the treatment dramatically cut bleeding in all 13 of the patients who got the effective dose of gene therapy determined by the study Grehan was in.

"I think it's amazing data actually," Pasi says. "It's been the Holy Grail for years to be able to treat hemophilia with a gene therapy treatment. And maybe we're beginning to see that that wasn't a pipe dream that this is a realistic option."

It's the latest promising development for gene therapy, which has finally started producing effective treatments for a variety of diseases after decades of setbacks.

Several other experimental forms of gene therapy are also showing promise for hemophilia, including another type of the condition, known as hemophilia B.

So far, nearly 150 patients have been treated with BioMarin's gene therapy as part of a larger study, and the results continue to be encouraging, according to the company. Some patients having been followed for as long as four years.

"For a good fraction of these people, they don't have to even think about having hemophilia anymore," says Henry Fuchs, president of research and development at BioMarin.

The Food and Drug Administration has designated the treatment a "breakthrough" therapy and accepted the company's application to give the gene therapy priority status for evaluation, representatives of BioMarin say. That makes it the first gene therapy for hemophilia the agency has agreed to consider.

According to the company, the FDA has signaled it will make its decision by Aug. 21 about whether to approve the treatment for sale.

Longer studies will be need to determine if the treatment is, in fact, a one-time, therapy for lifelong effect, experts say.

"It seems to be working very well, but we are only at three years," says Dr. W. Keith Hoots, director of the division of blood diseases and resources at the National Heart, Lung and Blood Institute. "We need to know for sure whether it will extend for their entire life. And only time will tell for that."

Nevertheless, Bach agrees the treatment appears very promising. Still, he questions the price tag, which would be just the latest in what appears to be an ongoing rise in the cost of the new wave of genetic therapies.

"The clinical breakthrough is prodigious. We should be thrilled by it," Bach says.

"But the greatest innovation by the pharmaceutical industry is not the biologic breakthroughs they're making," he says. "It's their ability to extract money from society that we could put into other things like better benefits in Medicare, lower out-of-pocket costs for poor people, dental coverage and things like that."

The prices of the drugs already used to treat hemophilia are inflated, Bach argues.

Bradbury, from the Hemophilia Federation of America, agrees cost is a concern.

"We need to make sure all those who are eligible would have access to it," Bradbury says.

Ajer says the company has already been negotiating with insurance companies and government programs to cover the costs.

"Our work is not done here, but my expectation is that most of the patients who need access to therapy would be able to get it, in not a terribly long time, through their insurance system," Ajer says.

As someone who has had to deal with hemophilia all his life, Grehan thinks the price is reasonable.

"I think $3 million for this is very cheap because it is life-changing," Grehan says. "And if you're going to spend hundreds of thousands of dollars a year over a lifetime, that seems worth it to me."

Continued here:

Gene Therapy Drug For Hemophilia May Be Priced As High As $3 Million Per Patient : Shots - Health News - NPR

Janssen has unveiled six-month data from an ongoing Phase I/II trial of its investigational gene therapy for the treatment of inherited retinal disease X-linked retinitis pigmentosa (XLRP).

The interim data show that low and intermediate doses of the investigational adeno-associated virus retinitis pigmentosa GTPase regulator (AAV-RPGR), jointly developed with MeiraGTx, were generally well-tolerated and indicated significant improvement in vision.

In patients with XLRP, the photoreceptors in the eye responsible for converting light into signals sent to the brain don't function as they should, leading to degeneration of the retina and legal blindness in adulthood.

The companies AAV-RPGR gene therapy is being investigated to treat the most common and severe forms of XLRP caused by mutations in the RPGR gene by preserving and improving vision and slowing retinal degeneration.

In the dose escalation phase of the trial, at six months, the low and intermediate dose cohorts demonstrated significant improvement from baseline in retinal sensitivity after treatment, evident when assessed with two perimetry approaches and three analysis metrics.

Currently, there are no approved treatments for this condition.

There is an urgent need to deliver a transformational therapy for people living with XLRP who experience progressive visual loss from childhood with eventual blindness in early adulthood, said Michel Michaelides, trial investigator, consultant ophthalmologist at Moorfields Eye Hospital and Professor of Ophthalmology at University College London.

We have learned valuable safety and efficacy information from this Phase I/II trial and look forward to applying those learnings in our next phase of study.

Continued here:

Janssen/MeiraGTx sight loss gene therapy hits targets - PharmaTimes

Robert Millman co-founded and led Semma Therapeutics as CEO while also a managing director of MPM Capital. By the time he left the stem cell therapy pioneer two years before it would be sold to Vertex he had left VC life behind.

Instead, he went around scouting new technologies, visiting with tech transfer offices and academics in the Boston/Cambridge area to find worthy ideas that could benefit from his IP expertise.

And he found quite a few.

One of them is Vesigen Therapeutics, which is launching with $28.5 million to turn a new class of extracellular vesicles into packaging and delivery tools for a variety of cargos mRNA, Cas9, base editors, PROTACs, you name it.

The barrier to being a drug company right now is not technology or targets, he told Endpoints News. Theres plenty of targets, theres plenty of technology, but theres no way of getting the two of those into a patient.

Vesigens platform stems from scientific co-founder and Harvard professor Quan Lus work on ARMMs, or arrestin domain-containing protein 1 [ARRDC1]-mediated microvesicles. As he wrote in Nature Communications in March 2018, ARMMs are distinct from exosomes, in that they lend themselves to more controlled production and cargos that can be recruited into the vesicle during the process of manufacturing rather than put together after the fact.

I dont understand really why a whole lot of venture firms didnt jump on it at the time, Millman said. I think its because most were focused on exosomes and just thought that this was another exosome company, and there was already Codiak and Evox.

But Lu showed in his paper delivery of function p53 protein to an p53 deficient animal and restoring p53 activity: To me it was revolutionary.

And compared to the current workhorses of genetic medicines such as viral vectors and lipid nanoparticles, ARMMs promise to package bigger and more varied payloads, get into more organs, and stay in circulation longer without triggering toxicity.

Faced with the Willy Wonka-like range of possibilities, Millman said the top order right now for his team of seven is to figure out a manufacturing setup that would load the cargos efficiently and be cleared by regulators.

In the three years that it will likely take to get to the cusp of the clinic, Vesigen is also collaborating with researchers to show how ARMMs can get into tissues that no one can go to with implications in ocular and neurological diseases as well as cancer.

The Series A, co-led by Leaps by Bayer and Morningside Ventures and joined by Linden Lake Ventures and Alexandria Venture Investments, should fund the company until 2025, when Millman expects to have built out the team to 30.

Its exactly the kind of projects that his group likes to invest in early, Leaps by Bayer head Jrgen Eckhardt said, especially as big believers in the cell and gene therapy space who see delivery as the missing link. For Vesigen, it also opens up opportunities to find partners within the pharma giants network down the road.

While Millman is keen not to lose focus, he also isnt hiding ambitions to make this the next big one.

I saw all of the problems Alnylam I was there for three years, we couldnt get delivery. I worked very early at Celera and established collaborations with Ionis and again delivery was limited. I was helping set up Verve Therapeutics and again delivery of Cas9 was a problem, he said. And here was an eloquent solution. I was just pleased to be at the right place at the right time.

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'Missing link': Bayer, Morningside help catapult a new kind of delivery tech to cell and gene therapy - Endpoints News

Based on FDA feedback, Applied Genetic Technologies (NASDAQ:AGTC) has revised the development plan for its gene therapy for X-linked retinitis pigmentosa, an inherited disorder affecting the retina that leads to vision loss.

Enrollment in the current 30-subject Phase 1/2 trial will be expanded. About 20 patients will be dosed, beginning in Q4, in two masked arms to collect additional functional data, including a mobility test added as a supplemental endpoint.

The agency indicated that a change in visual sensitivity of at least 7 decibels in at least 5 loci would be considered clinically meaningful. The company previously reported visual sensitivity as a mean over an entire treated area. Multiple participants in the Phase 1/2 study would meet the new criteria.

The planned Phase 2/3 trial will include two masked active arms in addition to a control arm. The primary endpoint will be visual sensitivity. Supplemental endpoints will include the mobility test. The study should launch in Q1 2021.

The company will provide additional information in its 10-K filing for fiscal 2020.

Shares up6%premarket on light volume.

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Applied Genetic Tech to expand study of gene therapy for vision loss disorder - Seeking Alpha

With reliable and impactful research methodologies, PMR provides critical information pertaining to the growth of the global CNS Gene Therapy market. Our team of analysts monitor the ongoing developments within the CNS Gene Therapy space and provide an unbiased assessment of the global CNS Gene Therapy market. The data included in the report are procured from reliable and trustworthy primary and secondary sources.

According to the findings of the report, the value of the global CNS Gene Therapy market in 2018 was ~US$ XX (Mn/Bn) and expected to attain a value of ~US$ XX (Mn/Bn) by the end of 2029. In addition, the report reveals that the global CNS Gene Therapy market is likely to grow at a CAGR of XX% during the forecast period (2019-2029).

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Some of the leading companies profiled in the market study include:

The CNS Gene Therapy market report provides an extensive analysis of the different product types including:

The presented market study includes a brief introduction of the CNS Gene Therapy market to enhance the reading experience of our users. Further, a thorough quantitative and qualitative analysis of each of these segments is provided in the report along with graphs, tables, and figures to support the data.

key players and product offerings

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Key information drawn from the CNS Gene Therapy market study

The market report aims to address the following queries:

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CNS Gene Therapy Market Forecasted To Surpass The Value Of US$ XX Mn/Bn By 2018 2028 - Bulletin Line

Preclinical Data Demonstrate Potential of Prevails AAV Gene Therapy Approach to Slow or Halt Progression in Multiple Neurodegenerative Diseases

Company Provides Overview of Planned Phase 1/2 PR006 PROCLAIM Clinical Trial for FTD-GRN Patients

Company to Host a Panel Discussion and Q&A Session on FTD-GRNwith Jonathan Rohrer, Ph.D., MRCP, an Expert in Frontotemporal Dementia (FTD) and its Genetic Causes

NEW YORK, July 22, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced three upcoming poster presentations at the Alzheimers Association International Conference (AAIC) 2020. These data underscore the robust preclinical evidence in support of Prevails AAV-based gene therapy approach, and highlight the Companys strategy to validate these data in the planned PROCLAIM clinical trial evaluating PR006 for the treatment of frontotemporal dementia patients with GRN mutations (FTD-GRN). The conference will be held virtually July 27-31, 2020.

Our novel gene therapy candidates have the potential to transform the treatment of patients with FTD-GRN and other devastating neurodegenerative diseases, said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. An increased understanding of genetically defined forms of neurodegenerative diseases, including the role that GRN mutations can play in frontotemporal dementia, has opened exciting possibilities to stop or slow disease progression using a gene therapy approach.

PR001

Prevail will highlight preclinical data demonstrating the effect of PR001 treatment on key disease biomarkers and functional parameters. These data provide the basis for the Companys clinical trials for Type 2 neuronopathic Gaucher disease (nGD) patients and Parkinsons disease with GBA1 mutations (PD-GBA) patients.

Poster title: PR001 Gene Therapy Improved Phenotypes in Models of Parkinsons Disease with GBA1 MutationSession date and time: Monday, July 27, 12:00 a.m. - 11:59 p.m. CDT

PR006

Prevail will present the design of the PROCLAIM Phase 1/2 clinical trial for the treatment of FTD-GRN patients and preclinical data demonstrating the effect of PR006 treatment on progranulin expression, lysosomal dysfunction and inflammation in the CNS.

Poster title: Preclinical Development of PR006, a Gene Therapy for the Treatment of Frontotemporal Dementia with Progranulin Mutations Session date and time: Monday, July 27, 12:00 a.m. - 11:59 p.m. CDT

Poster title: Design of a Phase 1/2 Study of an AAV9-Based Gene Therapy for Fronto-Temporal Dementia Patients with Pathogenic GRN Mutations (PROCLAIM Trial)Session date and time: Wednesday, July 29, 12:00 a.m. - 11:59 p.m. CDT

FTD-GRN Panel Discussion and Q&A SessionIn addition to its presentations at AAIC, on Wednesday, July 29 at 2:30 p.m. EDT, management will be hosting a panel discussion and Q&A session on FTD-GRN by Jonathan Rohrer, Ph.D., MRCP, an expert in frontotemporal dementia (FTD) and its genetic causes. A live webcast of the event and replay following its conclusion will be available on the Events and Presentations section of the Company's website at https://ir.prevailtherapeutics.com/events.

About Prevail TherapeuticsPrevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The Company is developing PR001 for patients with Parkinsons disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinsons with GBA and OrbiMed, and is headquartered in New York, NY.

Forward-Looking Statements Related to PrevailStatements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Examples of these forward-looking statements include statements concerning the potential for Prevails gene therapy candidates to transform the treatment of patients with, and slow or halt the progression of, FTD-GRN and other neurodegenerative diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Prevails novel approach to gene therapy makes it difficult to predict the time, cost and potential success of product candidate development or regulatory approval; Prevails gene therapy programs may not meet safety and efficacy levels needed to support ongoing clinical development or regulatory approval; the regulatory landscape for gene therapy is rigorous, complex, uncertain and subject to change; the fact that gene therapies are novel, complex and difficult to manufacture; and risks relating to the impact on our business of the COVID-19 pandemic or similar public health crises. These and other risks are described more fully in Prevails filings with the Securities and Exchange Commission (SEC), including the Risk Factors section of the Companys Quarterly Report on Form 10-Q for the period ended March 31, 2020, filed with the SEC on May 14, 2020, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Prevail undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Media Contact:Mary CarmichaelTen Bridge Communications [emailprotected] 617-413-3543

Investor Contact:[emailprotected]

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Prevail Therapeutics to Highlight Multiple Gene Therapy Programs for Neurodegenerative Diseases at the Virtual 2020 Alzheimer's Association...

There are good investments and there are great investments. CRISPR Therapeutics (NASDAQ:CRSP) stock certainly qualifies as the latter.

This is a company that, four years after it went public, has only been profitable for one year, yet its future is so promising that its stock is trading for 540% more than it did in 2016.

What's the big deal about CRISPR Therapeutics? The biopharmaceutical company says it is using breakthrough gene-altering therapies to treat diseases such as sickle cell disease (SCD) and beta-thalassemia, both of which are inherited blood disorders that don't have a cure and require frequent blood transfusions. The company is also working on gene therapies to treat type 1 diabetes, muscular dystrophy, cystic fibrosis, multiple myeloma, and solid tumors in pancreatic cancer and lung cancer.

Image source: Getty Images.

CRISPR Therapeutics is actually named for the technology it uses: CRISPR stands for "clusters of regularly interspaced short palindromic repeats." The gene-editing technology uses Cas9 proteins to locate a sequenceof DNA within a cell and alter it.

Though CRISPR Therapeutics has yet to bring a product to market, some of its clinical trials have had amazing results. In June, the Swiss company announced that in a joint trial with Vertex Pharmaceuticals (NASDAQ:VRTX), five patients with beta-thalassemia and two patients with sickle cell disease were treated with gene therapy CTX001.

Two of the early beta-thalassemia patients are now transfusion independent 15 months afterward. The first SCD patient in the trial is transfusion independent and free of vaso-occlusive crises (VOCs), a painful condition when blood vessels are blocked by sickled red blood cells, seven months after his dose of CTX001.

Beta-thalassemia, which reduces the body's production of hemoglobin, is extremely rare. Sickle cell anemia is common among African Americans but also can affect Latinos and people of Indian, Asian, Mediterranean backgrounds. The U.S. Department of Health and Human Services says 300,000 babies worldwide are born every year with the disease and 100,000 people in the United States are currently living with the disease.

While the results are quite promising, the study is still in its infancy. .

If you had invested $100 in CRISPR Therapeutics stock when it went public in 2016, how much would that be worth today?

By the end of the day on Oct. 19, 2016, the day CRISPR Therapeutics went public, the stock was trading at $14.09, you would have bought seven shares. Assuming you bought at that price, your original $98.63 investment would be worth $632.45 as of the close of trading Friday when the stock went for $90.35 per share. That's a gain of 541%.

CRISPR Therapeutics isn't the only biotech to use CRISPR-Cas9 technology. Others include Editas Medicine (NASDAQ:EDIT) and Intellia Therapeutics (NASDAQ:NTLA), both of which went public in 2016. Neither of those has done quite as well as CRISPR Therapeutics since its IPO.

Compared to a biotech ETF such as the iShares NASDAQ Biotechnology ETF (NASDAQ:IBB), CRISPR has shined. The ETF closed at $89.16 on the day of CRISPR's IPO. As of Friday's close, IBB was at $136.41. If you had invested $100 on Oct. 19, 2016, you would have a return of only 36%.

CRSP data by YCharts

Clinical-stage biotech stocks are inherently risky plays because there are so many hurdles these companies have to clear before they can make money. The therapies have to be approved by the Food and Drug Administration (FDA) and many therapies look promising only to fail in late-stage clinical trials.

CRISPR Therapeutics looks like a solid bet, though, both from a cash standpoint and in its strength of pipeline. The company has four candidates already in trials.It is already making money, though not a lot of it. Last year, CRISPR Therapeutics generated net income of $66.8 million, thanks to $289.5 million in collaboration revenue.

The other major question for clinical-stage biotechs is whether they have enough capital to pay for research and development until the therapies pay off. Last quarter, the company burned through $54 million in cash. However, CRISPR Therapeutics says it has more than $900 million left, so it can continue that burn rate for more than four years. The possibility that CRISPR Therapeutics' technology could actually cure diseases, not just treat them, easily makes this a risk worth taking.

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If You Invested $100 in CRISPR Therapeutics' IPO, This Is How Much Money You'd Have Now - Motley Fool

SAN DIEGO and BOSTON, July 24, 2020 /PRNewswire/ --Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento") and SmartPharm Therapeutics, Inc. ("SmartPharm") announced today the signing of a letter of intent for Sorrento to acquire SmartPharm, a gene-encoded therapeutics company developing non-viral DNA and RNA gene delivery platforms for COVID-19 and rare diseases, with broad potential for application in enhancing antibody-centric therapeutics, including against COVID-19. The transaction is expected to close in August 2020. Financial terms of the deal were not disclosed.

Sorrento and SmartPharm previously announced a research and development collaboration to encode and express in vivo Sorrento's proprietary SARS-CoV-2 neutralizing monoclonal antibodies utilizing SmartPharm's Gene Mab plasmid nanoparticle platform.

Sorrento plans to accelerate the development of multiple candidates for in vivo gene-encoded expression of Sorrento's antibodies, starting with Sorrento's previously announced STI-1499, or COVI-GUARD, which is currently moving through preclinical and manufacturing requirements with an IND submission targeted for August 2020. The initial clinical trial for STI-1499 is expected to be in ICU patients to ensure safety and potentially allow a preliminary look at efficacy. In vitro results so far have demonstrated STI-1499's ability to completely neutralize SARS-CoV-2 infection at low doses, making STI-1499 Sorrento's lead candidate for potential cost-effective passive protection against COVID-19.

SmartPharm's Gene Mab platform delivers to muscles a novel low-immunogenic DNA plasmid encoded with a therapeutic antibody for long-lasting expression in vivo. If clinical trials are successful, a single administration of STI-1499dpi (DNA plasmid injection) could allow the recipient's own muscle cells to produce the antibody for a prolonged period of time after a single injection, potentially providing extended protection against COVID-19 for periods of time that might provide an alternative to vaccines. Manufacturing of DNA plasmids (in bacterial fermenters) can be done with Sorrento's in-house cGMP capabilities for a fraction of the cost associated with traditional antibody manufacturing.

"We are very encouraged by the preclinical data generated thus far by our STI-1499 neutralizing antibody against COVID-19," said Henry Ji, Ph.D., CEO of Sorrento Therapeutics. "We are excited about the prospect of leveraging SmartPharm's Gene Mab platform in combination with our G-MAB library to produce next-generation gene-encoded antibody candidates against a host of pathogens and cancer cell types. Being able to stimulate the body to produce in vivo our most potent antibodies at optimized manufacturing costs will offer an additional competitive advantage as Sorrento transitions from product development to full scale manufacturing and commercialization."

About SmartPharm Therapeutics

SmartPharm Therapeutics, Inc. is a privately held, development stage biopharmaceutical company focused on developing next-generation, non-viral gene therapies for the treatment of serious or rare diseases with the vision of creating "Biologics from Within." SmartPharm is currently developing a novel pipeline of non-viral, gene-encoded proteins for the treatment of conditions that require biologic therapy such enzyme replacement and tissue restoration. SmartPharm commenced operations in 2018 and is headquartered in Cambridge, MA, USA. For more information, please visit http://www.smartpharmtx.com.

About Sorrento Therapeutics, Inc.

Sorrento is a clinical stage, antibody-centric, biopharmaceutical company developing new therapies to treat cancers. Sorrento's multimodal, multipronged approach to fighting cancer is made possible by its extensive immuno-oncology platforms, including key assets such as fully human antibodies ("G-MAB library"), clinical stage immuno-cellular therapies ("CAR-T", "DAR-T"), antibody-drug conjugates ("ADCs"), and clinical stage oncolytic virus ("Seprehvir", "Seprehvec"). Sorrento is also developing potential antiviral therapies and vaccines against coronaviruses, including COVIDTRAP, ACE-MAB, COVI-MAB, COVI-GUARD, COVI-SHIELD and T-VIVA-19.

Sorrento's commitment to life-enhancing therapies for patients is also demonstrated by our effort to advance a first-in-class (TRPV1 agonist) non-opioid pain management small molecule, resiniferatoxin ("RTX"), and ZTlido (lidocaine topical system) 1.8% for the treatment of post-herpetic neuralgia. RTX is completing a phase IB trial for intractable pain associated with cancer and a phase 1B trial in osteoarthritis patients. ZTlido was approved by the FDA on February 28, 2018.

For more information visit http://www.sorrentotherapeutics.com

Forward-Looking Statements

This press release and any statements made for and during any presentation or meeting contain forward-looking statements related toSorrento Therapeutics, Inc., under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995 and subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include statements regarding the proposed acquisition of SmartPharm; the potential effects that the acquisition of SmartPharm may have on Sorrento's business and product candidate pipeline; the expected timing for the closing of the transaction; the initiation and completion of ongoing studies for COVID-19 using antibodies and gene-encoded antibodies, and data read-outs related thereto; the potency and potential blocking capabilities of STI-1499 and STI-1499dpi and their respective impact on SARS-CoV-2; the expected length of any antiviral protection provided by STI-1499 and STI-1499dpi; the potential administration and applications of STI-1499 and STI-1499dpi, alone or in combination; the timeline and status of preclinical testing for STI-1499 and STI-1499dpi; the expected timing of an IND submission for STI-1499; expectations regarding the initial clinical trial for STI-1499; the potential safety and efficacy of STI-1499 and STI-1499dpi; the therapeutic potential of STI-1499 and STI-1499dpi for SARS-CoV-2 and COVID-19; the potential costs associated with manufacturing STI-1499dpi and other DNA plasmids; Sorrento's ability to produce antibody candidates against pathogens and cancer cells; Sorrento's ability to transition from product development to full scale manufacturing and commercialization; and Sorrento's potential position in the anti-viral immunity industry. Risks and uncertainties that could cause our actual results to differ materially and adversely from those expressed in our forward-looking statements, include, but are not limited to: risks related to Sorrento's technologies and prospects with newly acquired technologies, including the proposed acquisition of SmartPharm and the utilization of SmartPharm's Gene-Encoded Therapeutics (GET) platforms for the treatment and prevention of coronavirus infections and other pathogens and cancer cells; risks related to seeking regulatory approvals and conducting clinical trials; the clinical and commercial success of the treatment and prevention of coronavirus infections using monoclonal antibodies and gene-encoded antibodies; the viability and success of using monoclonal antibodies and gene-encoded antibodies for treatments in anti-viral therapeutic areas, including coronavirus; clinical development risks, including risks in the progress, timing, cost and results of clinical trials and product development programs; risk of difficulties or delays in obtaining regulatory approvals; risks that prior study and trial results may not be replicated in future studies and trials; risks that clinical study results may not meet any or all endpoints of a clinical study and that any data generated from such studies may not support a regulatory submission or approval; risks related to seeking regulatory approvals and conducting clinical trials; risks of manufacturing drug product; risks related to leveraging the expertise of its employees, subsidiaries, affiliates and partners to assist the company in the execution of its strategies; risks related to the global impact of COVID-19 and other risks that are described in Sorrento's most recent periodic reports filed with theSecurities and Exchange Commission, including Sorrento's Annual Report on Form 10-K for the year endedDecember 31, 2019, and subsequent Quarterly Reports on Form 10-Q filed with theSecurities and Exchange Commission, including the risk factors set forth in those filings. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release and we undertake no obligation to update any forward-looking statement in this press release except as required by law.

Media and Investor Relations

Contact: Dani FrankEmail: [emailprotected]

Sorrento and the Sorrento logo are registered trademarks of Sorrento Therapeutics, Inc.G-MAB, COVI-GUARD, COVI-SHIELD, COVIDTRAP, T-VIVA-19, COVI-MAB, ACE-MAB, COVI-TRACK, Saving-Life and Improving-Life are trademarks of Sorrento Therapeutics, Inc.

ZTlido is a trademark owned by Scilex Pharmaceuticals Inc.All other trademarks are the property of their respective owners. 2020 Sorrento Therapeutics, Inc. All Rights Reserved.

SOURCE Sorrento Therapeutics, Inc.

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Sorrento Enters Into Letter Of Intent To Acquire SmartPharm And Develop Pipeline Of Gene-Encoded Therapeutic Antibodies, Starting With Neutralizing...

This article is an overview of the financial results of Johnson & Johnson, Novartis, Roche, Biogen and selected other healthcare companies who have reported their earnings since July 16.

Many of the top healthcare companies reporting second-quarter earnings over the past week have seen COVID-19 continue to impact revenues. But some are cautiously optimistic for the second half of the year, as they learn to better navigate the pandemic.

The biggest drug- and device-maker by market cap, Johnson & Johnson reported a 35.3% decline in its year-over-year earnings, driven in part by a 33.9% fall in medical device sales. CFO Joseph Wolk said on a July 16 earnings call that the company had previously expected to wait until the fourth quarter for a rebound in these sales, but the turnaround began earlier than expected resulting in the company raising its full-year guidance. While CEO Alex Gorsky expressed concern about a second wave of COVID-19, he pointed out that better-prepared hospitals mean the global impact is unlikely to reach levels seen in the spring.

Novartis CEO Vas NarasimhanSource: Novartis

Swiss pharmaceutical company Novartis AG reported relatively unchanged earnings year over year for the quarter, with the pandemic's effect felt most keenly on its ophthalmology and dermatology businesses, as well as therapies requiring hospital stays. This was partially offset by sales of heart disease drug Entresto, gene therapy Zolgensma and blockbuster psoriasis medicine Cosentyx.

The Basel, Switzerland-based company will not join the race to find a coronavirus vaccine, CEO Vas Narasimhan said on a July 21 earnings call. Instead, the company is focused on pivotal studies in more than 20 medicines, as well as researching inhibitors against both the current and future coronaviruses. In addition, Narasimhan expects strong uptake in Europe for nuclear therapy Lutathera and the drug's expansion in the U.S.

Roche Holding AG, another Swiss pharmaceutical giant and one of the world's biggest cancer drug manufacturers, reported a 6% decline in first-half earnings compared to the same period in 2019, with the pandemic and generic competition of its former blockbuster cancer therapies Herceptin, Avastin and MabThera/Rituxan having a combined impact on sales. However, the company expects the outlook to improve later in the year, with Roche's diagnostics unit's CEO Thomas Schinecker noting that an influx of orders for its coronavirus diagnostic tests from governments worldwide means that even after quadrupling production capacity, the company is still struggling to keep up with demand.

Cambridge, Mass.-based Biogen Inc. reported a 12% increase in second-quarter earnings, with rare disease therapy Spinraza remaining a key seller. The company announced plans to conduct a two-year phase 4 clinical study of the drug combined with Novartis' competitor therapy Zolgensma to treat infants and children with spinal muscular atrophy. GlobalData analysts said Biogen could maintain dominance in the market by demonstrating that Spinraza benefits patients who also receive the Novartis gene therapy.

Impact of reduced elective care

The reduction in elective surgeries, a key strategy to limit the initial spread of the coronavirus, took a heavier toll on healthcare equipment suppliers notably Intuitive Surgical Inc., which reported a $1.01 billion drop in revenue compared to 2019's second quarter. The surgical-robot maker's CFO, Marshall Mohr, said in a July 21 earnings call that "the recovery tail of surgery will be a long one, likely to last many quarters."

Similarly, Abbott Laboratories saw its second-quarter EPS drop 30.5% year over year, but its diabetes line remained resilient. President and CEO Robert Ford said he was "very bullish" about the opportunity for the Libre 2 blood sugar monitoring system approved in the U.S. in June and the Abbott Park, Ill.-based healthcare equipment company reissued its 2020 guidance of mid-single-digits growth.

The pause in elective procedures to slow the pandemic has impacted surgical equipment suppliers.Source: Pixel

HCA Healthcare Inc. blamed a continued drop in admissions and surgery volumes for a 12% decline in its revenue for the second quarter, but the healthcare services provider was already able to point to a rebound in May and June. The company brought in about $1.1 billion in second-quarter income although more than half came from economic relief funds.

CEO Samuel Hazen was hesitant to project into the second half of the year but said the company has gained "muscle memory" to deal with current and future COVID-19 outbreaks. RBC Capital Markets analysts expect HCA to weather the "significant challenges" of the pandemic, noting that "underlying demand in its markets remains strong, despite the likelihood for continued ebb and flow in patient census as reopenings progress and adjust to flare-ups."

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After another tough quarter, healthcare companies pin hopes on brighter H2'20 - S&P Global

QY Research has Published Latest Trending Report on Global Gene Therapy Market

Los Angeles, United State, The report titledGlobal Gene Therapy Marketis one of the most comprehensive and important additions to QY Researchs archive of market research studies. It offers detailed research and analysis of key aspects of the global Gene Therapy market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Gene Therapy market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global Gene Therapy market is carefully analyzed and researched about by the market analysts.

Request Sample Report and Full Report TOC:https://www.qyresearch.com/sample-form/form/1436547/global-gene-therapy-market

The Essential Content Covered in the GlobalGene Therapy Market Report:

* Top Key Company Profiles.* Main Business and Rival Information* SWOT Analysis and PESTEL Analysis* Production, Sales, Revenue, Price and Gross Margin* Market Share and Size

Global Gene Therapy Market is estimated to reach xxx million USD in 2020 and projected to grow at the CAGR of xx% during 2020-2026. According to the latest report added to the online repository of QY Research the Gene Therapy market has witnessed an unprecedented growth till 2020. The extrapolated future growth is expected to continue at higher rates by 2025.

Top Players of Gene Therapy Market are Studied: Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical Inc., Advantagene

The report provides a 6-year forecast (2020-2026) assessed based on how the Gene Therapy market is predicted to grow in major regions likeUSA, Europe, Japan, China, India, Southeast Asia, South America, South Africa, Others.

Segmentation by Type:Ex vivoIn Vivo

Segmentation by Application:CancerMonogenicInfectious diseaseCardiovascular diseaseOther

Reasons to Buy this Report:

Table of Contents

Table of Contents1 Report Overview

1.1 Study Scope

1.2 Key Market Segments

1.3 Players Covered: Ranking by Gene Therapy Revenue

1.4 Market Analysis by Type

1.4.1 Global Gene Therapy Market Size Growth Rate by Type: 2020 VS 2026

1.4.2 Ex vivo

1.4.3 In Vivo

1.5 Market by Application

1.5.1 Global Gene Therapy Market Share by Application: 2020 VS 2026

1.5.2 Cancer

1.5.3 Monogenic

1.5.4 Infectious disease

1.5.5 Cardiovascular disease

1.5.6 Other 1.6 Study Objectives 1.7 Years Considered2 Global Growth Trends by Regions

2.1 Gene Therapy Market Perspective (2015-2026)

2.2 Gene Therapy Growth Trends by Regions

2.2.1 Gene Therapy Market Size by Regions: 2015 VS 2020 VS 2026

2.2.2 Gene Therapy Historic Market Share by Regions (2015-2020)

2.2.3 Gene Therapy Forecasted Market Size by Regions (2021-2026) 2.3 Industry Trends and Growth Strategy 2.3.1 Market Top Trends 2.3.2 Market Drivers

2.3.3 Market Challenges

2.3.4 Porters Five Forces Analysis

2.3.5 Gene Therapy Market Growth Strategy

2.3.6 Primary Interviews with Key Gene Therapy Players (Opinion Leaders)3 Competition Landscape by Key Players

3.1 Global Top Gene Therapy Players by Market Size

3.1.1 Global Top Gene Therapy Players by Revenue (2015-2020)

3.1.2 Global Gene Therapy Revenue Market Share by Players (2015-2020)

3.1.3 Global Gene Therapy Market Share by Company Type (Tier 1, Tier 2 and Tier 3)

3.2 Global Gene Therapy Market Concentration Ratio

3.2.1 Global Gene Therapy Market Concentration Ratio (CR5 and HHI)

3.2.2 Global Top 10 and Top 5 Companies by Gene Therapy Revenue in 2019

3.3 Gene Therapy Key Players Head office and Area Served

3.4 Key Players Gene Therapy Product Solution and Service

3.5 Date of Enter into Gene Therapy Market

3.6 Mergers & Acquisitions, Expansion Plans4 Breakdown Data by Type (2015-2026)

4.1 Global Gene Therapy Historic Market Size by Type (2015-2020)

4.2 Global Gene Therapy Forecasted Market Size by Type (2021-2026)5 Gene Therapy Breakdown Data by Application (2015-2026)

5.1 Global Gene Therapy Market Size by Application (2015-2020)

5.2 Global Gene Therapy Forecasted Market Size by Application (2021-2026)6 North America

6.1 North America Gene Therapy Market Size (2015-2020)

6.2 Gene Therapy Key Players in North America (2019-2020)

6.3 North America Gene Therapy Market Size by Type (2015-2020)

6.4 North America Gene Therapy Market Size by Application (2015-2020)7 Europe

7.1 Europe Gene Therapy Market Size (2015-2020)

7.2 Gene Therapy Key Players in Europe (2019-2020)

7.3 Europe Gene Therapy Market Size by Type (2015-2020)

7.4 Europe Gene Therapy Market Size by Application (2015-2020)8 China

8.1 China Gene Therapy Market Size (2015-2020)

8.2 Gene Therapy Key Players in China (2019-2020)

8.3 China Gene Therapy Market Size by Type (2015-2020)

8.4 China Gene Therapy Market Size by Application (2015-2020)9 Japan

9.1 Japan Gene Therapy Market Size (2015-2020)

9.2 Gene Therapy Key Players in Japan (2019-2020)

9.3 Japan Gene Therapy Market Size by Type (2015-2020)

9.4 Japan Gene Therapy Market Size by Application (2015-2020)10 Southeast Asia

10.1 Southeast Asia Gene Therapy Market Size (2015-2020)

10.2 Gene Therapy Key Players in Southeast Asia (2019-2020)

10.3 Southeast Asia Gene Therapy Market Size by Type (2015-2020)

10.4 Southeast Asia Gene Therapy Market Size by Application (2015-2020)11 India

11.1 India Gene Therapy Market Size (2015-2020)

11.2 Gene Therapy Key Players in India (2019-2020)

11.3 India Gene Therapy Market Size by Type (2015-2020)

11.4 India Gene Therapy Market Size by Application (2015-2020)12 Central & South America

12.1 Central & South America Gene Therapy Market Size (2015-2020)

12.2 Gene Therapy Key Players in Central & South America (2019-2020)

12.3 Central & South America Gene Therapy Market Size by Type (2015-2020)

12.4 Central & South America Gene Therapy Market Size by Application (2015-2020)13Key Players Profiles

13.1 Bluebird Bio

13.1.1 Bluebird Bio Company Details

13.1.2 Bluebird Bio Business Overview and Its Total Revenue

13.1.3 Bluebird Bio Gene Therapy Introduction

13.1.4 Bluebird Bio Revenue in Gene Therapy Business (2015-2020))

13.1.5 Bluebird Bio Recent Development

13.2 Sangamo

13.2.1 Sangamo Company Details

13.2.2 Sangamo Business Overview and Its Total Revenue

13.2.3 Sangamo Gene Therapy Introduction

13.2.4 Sangamo Revenue in Gene Therapy Business (2015-2020)

13.2.5 Sangamo Recent Development

13.3 Spark Therapeutics

13.3.1 Spark Therapeutics Company Details

13.3.2 Spark Therapeutics Business Overview and Its Total Revenue

13.3.3 Spark Therapeutics Gene Therapy Introduction

13.3.4 Spark Therapeutics Revenue in Gene Therapy Business (2015-2020)

13.3.5 Spark Therapeutics Recent Development

13.4 Dimension Therapeutics

13.4.1 Dimension Therapeutics Company Details

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Gene Therapy Market 2020-2026: Key Vendor Landscape By Regional Output, Demand By Countries And Future Growth|Bluebird Bio, Sangamo, Spark...

Biogen is planning to trial its Spinraza spinal muscular atrophy (SMA) drug in patients who have not responded properly to Novartis gene therapy Zolgensma, combining the two ultra-expensive therapies to treat the rare muscle-wasting disease.

In the US, a single shot of Zolgensma (onasemnogene abeparvovec) costs $2.1 million, while Spinraza (nusinersen) costs $750,000 in its first year.

Biogen said it aims to begin a phase IV study as in a long-term follow-up study of Novartis drug it has been reported that some patients have been subsequently treated with Spinraza.

The phase IV RESPOND study aims to test efficacy and safety of Spinraza with a suboptimal clinical response to Zolgensma.

So far four out of 10 patients in a long-term study of Zolgensma have been subsequently treated with Spinraza, Biogen said.

Based on the planned study design, RESPOND will be a two-year, open-label study to evaluate the efficacy and safety of Spinraza in SMA patients previously treated with Zolgensma to further inform treatment decisions.

Efficacy will be assessed by change from baseline on motor function measures and additional clinical outcomes such as swallowing and caregiver burden.

Neurofilament levels, an exploratory endpoint, will also be evaluated as a marker of biological disease activity.

The primary study group aims to include 40 infants aged nine months or younger at the time of the first dose of Spinraza, who have two copies of SMN and are likely to develop type 1 disease and received Zolgensma at six months old or younger.

A second study group will include 20 children and will generate data in patients with a broader age range, up to three years old at the time of the first Spinraza dose.

After a screening period, participants will receive the approved 12 mg dose of Spinraza, which is four loading doses followed by maintenance doses every four months, over the two-year study period.

If approved by regulators the company aims to begin enroling patients in the first quarter of next year.

While Zolgensma is a gene therapy that aims to correct the genetic defect that causes the disease, Spinraza works by increasing the amount of full-length SMS protein that is critical to maintaining motor neurons.

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Biogen plans trial of Spinraza in patients not responding to SMA gene therapy - - pharmaphorum

PALM BEACH, FL, July 22, 2020 /PRNewswire/ -- The rising incidence of liver cancer and growing research activities for developing novel treatments plus the availability of newer drugs and treatments coupled with rising awareness among consumer/patients regarding liver cancer, are the factors that are driving the market. A report from Fior Markets said that the global liver cancer treatment market is expected to reach USD 2,186.63 Million by 2025, at a CAGR of 19.85% from 2018 to 2025. The report said that: "Hepatic or liver cancer is one of the most commonly prevalent types of cancer It is known for its aggressive nature and poor survival rate. Certain medical conditions such as Hepatitis B and C viral infections, type 2 diabetes, excess body weight, and liver cirrhosis are known to be the leading causes for the onset of liver cancer. Consumption of alcohol, tobacco is acknowledged to exponentially increase the risk of liver cancer. Thus, increasing research activities in the development of the novel drug and therapeutic systems are anticipated to propel the global liver cancer treatment market. Hepatocellular carcinoma, or primary liver cancer, tends to grow and expand at a fast rate if it is not detected early. This means that people who have been diagnosed with it may not survive longer than 10 to 11 months. The studies show that in the United States, hepatocellular carcinoma is the ninth leading cause of cancer-related deaths. To treat it, specialists will usually prescribe therapy with a drug called "sorafenib." But unfortunately, this drug can typically prolong survival by only 3 months, and it can have numerous adverse effects." Active biotech and pharma companies in the markets this week include: Q BioMed Inc. (OTCQB: QBIO), OPKO Health, Inc. (NASDAQ: OPK), Midatech Pharma plc (NASDAQ: MTP), Genprex, Inc. (NASDAQ: GNPX), Onconova Therapeutics, Inc. (NASDAQ: ONTX).

The report continued: "Factors such as rising incidence of liver cancer and growing research activities for developing novel treatments coupled with increasing government funding for the cancer treatment research are driving the global liver cancer treatment market. Growing use of immunotherapy drugs in the combination drug treatments is leading major drug manufacturers to develop and launch the newer medicines Growth government funding has provided a fillip for the cancer treatment research activities." It also said that the due to its increasing adoption in the combination drug treatments the immunotherapy segment is projected to grow at a CAGR of 21.63% over the forecast period."

Q BioMed Inc. (OTCQB: QBIO) BREAKING NEWS: Q BIOMED TO SCALE UP PRODUCTION OF UTTROSIDE-B, A NOVEL CHEMOTHERAPEUTIC FOR LIVER CANCER, IN PREPARATION FOR IND FILING - Q BioMed and Chemveda Life Sciences are very pleased to continue their collaboration on Uttroside-B, a chemotherapeutic that has shown remarkable potential efficacy as a treatment for liver cancer.

While focused on its commercial rollout of Strontium89, this drug development program will advance another important asset in the Company's portfolio towards monetization. The efficacy of Uttroside-B, a potent saponin, against liver cancer was demonstrated in a preclinical study published in the November 2016 issue of Scientific Reports, a Nature journal.

In the study, researchers showed that in animal models, Uttroside-B was ten times more cytotoxic to the HepG2 liver cancer cell line than sorafenib, the only drug approved by the Food and Drug Administration for liver cancer at the time, and the current first line treatment for hepatocellular carcinoma. Uttroside-B drastically shrunk tumors in mice bearing human liver cancer xenografts. In addition, in pre-clinical experiments Uttroside-B induced cytotoxicity in all liver cancer cell lines, and researchers were also able to confirm its biological safety, both by in vitro and in vivo studies.

Denis Corin, Q BioMed CEO said, "Having successfully completed a very challenging synthesis program, we are pleased to be able to advance this important asset towards the clinic and the patients we hope to treat. Liver cancer incidence rates have more than tripled since 1980, while the death rates have more than doubled during this time. More than 800,000 people are diagnosed with this cancer each year throughout the world and it accounts for more than 700,000 deaths annually. New, more effective treatments for these patients is vital and we are encouraged by the early data we have seen with our molecule."

Q BioMed and its partners in the project, the Oklahoma Medical Research Foundation and The Rajiv Gandhi Centre for Biotechnology (RGCB),will now advance the most promising candidate into preclinical testing and validation over the next few months in anticipation of an Orphan Drug application and an Investigational New Drug (IND) application for a clinical program.

The Uttroside B technology is covered by a provisional patent application. To see the full Scientific Reports study, go to:http://www.nature.com/articles/srep36318 Read this entire release and more news for QBIO at: https://www.financialnewsmedia.com/news-qbio/

Other recent developments in the biotech industry include:

OPKO Health, Inc. (NASDAQ: OPK) BioReference Laboratories, Inc., an OPKO Health company, recently announced it will accept an Indefinite Delivery Indefinite Quantity (IDIQ) contract award to provide Commercial Surge Capacity Testing for COVID-19 Emergency Response to the Centers for Disease Control and Prevention (CDC).

Under the contract with the CDC, BioReference will perform antibody testing to determine COVID-19 seroprevalence, and will provide results with key demographic information and analysis in collaboration with the CDC. The agreement's period of performance beganJuly 20, 2020, and is ongoing throughNovember 19, 2020.

Midatech Pharma plc (NASDAQ: MTP) a drug delivery technology company focused on improving the bio-delivery and bio-distribution of medicines recently announced that electronic copies of its Annual Report and Accounts for the year ended 31 December 2019 are now available from the Company's investor relations website.

Midatech Pharma PLC (dual listed on LSE AIM: MTPH; andNASDAQ: MTP) is an R&D company focused on 'Making Medicines Better' by improving delivery of drugs in the body. The Company combines existing medications with its proprietary and innovative drug delivery technologies to provide compelling oncology and rare disease products that have the potential to powerfully impact the lives of patients undergoing treatment for life threatening diseases.

Genprex, Inc. (NASDAQ: GNPX) a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes, recently announced that Dr. George K. Gittes, MD of the University of Pittsburg, the lead researcher that developed the Company's potentially curative diabetes gene therapy, was awarded a grant of $2.59 million from the NIH National Institute of Diabetes and Digestive and Kidney Diseases. The grant will assist Dr. Gittes's development for his research project titled, "Alpha Cell Conversion to Beta Cells in Non-human Primates" and will build upon his accumulating groundbreaking gene therapy work toward finding a cure for diabetes. In this project, Dr. Gittes' research team will conduct important proof-of-principle studies in non-human primates as the last steps in preparation for human gene therapy clinical trials. This technology is the subject of an exclusive license agreement entered into between Genprex and the University of Pittsburgh in February of 2020.

"We are excited to receive this funding to support our research in diabetic primates as we move toward human clinical trials," said Dr. George Gittes, Co-Scientific Director and Professor of Surgery at the UPMC Children's Hospital of Pittsburgand the lead researcher behind the diabetes gene therapy. "We saw encouraging data in our preclinical mice studies, where the gene therapy reprogrammed pancreatic cells to restore normal blood glucose levelsin diabetic mice for approximately four months, which could translate to decades in humans. More recently, preliminary results in non-human primates (monkeys) has also been very promising."

Onconova Therapeutics, Inc. (NASDAQ: ONTX) recently announced the e-publication of results from a Phase 1 company-sponsored study of oral rigosertib in combination with standard dose azacitidine in the treatment of patients diagnosed with either higher-risk myelodysplastic syndrome (HR-MDS) or acute myeloid leukemia (AML) in the international hematological malignancy journalLeukemia Research.

"A key strategy emerging in the treatment of MDS is the identification of safe and effective combinations, particularly those involving oral agents. The results from this Phase 1 study represent Onconova's first efforts to explore oral rigosertib in combination with azacitidine to address the unmet medical need in patients with MDS and AML. We anticipate meeting with the FDA, in conjunction with the pivotal data readout from the INSPIRE Trial, for alignment with the agency on a registration trial for the combination of oral rigosertib plus azacitidine in HMA-nave HR-MDS," said Steven M. Fruchtman, M.D., President and CEO of Onconova.

DISCLAIMER: FN Media Group LLC (FNM), which owns and operates FinancialNewsMedia.com and MarketNewsUpdates.com, is a third party publisher and news dissemination service provider, which disseminates electronic information through multiple online media channels. FNM is NOT affiliated in any manner with any company mentioned herein. FNM and its affiliated companies are a news dissemination solutions provider and are NOT a registered broker/dealer/analyst/adviser, holds no investment licenses and may NOT sell, offer to sell or offer to buy any security. FNM's market updates, news alerts and corporate profiles are NOT a solicitation or recommendation to buy, sell or hold securities. The material in this release is intended to be strictly informational and is NEVER to be construed or interpreted as research material. All readers are strongly urged to perform research and due diligence on their own and consult a licensed financial professional before considering any level of investing in stocks. All material included herein is republished content and details which were previously disseminated by the companies mentioned in this release. FNM is not liable for any investment decisions by its readers or subscribers. Investors are cautioned that they may lose all or a portion of their investment when investing in stocks. For current services performed FNM has been compensated forty six hundred dollars for news coverage of the current press releases issued by Q BioMed Inc by the company. FNM HOLDS NO SHARES OF ANY COMPANY NAMED IN THIS RELEASE.

This release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E the Securities Exchange Act of 1934, as amended and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. "Forward-looking statements" describe future expectations, plans, results, or strategies and are generally preceded by words such as "may", "future", "plan" or "planned", "will" or "should", "expected," "anticipates", "draft", "eventually" or "projected". You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements, including the risks that actual results may differ materially from those projected in the forward-looking statements as a result of various factors, and other risks identified in a company's annual report on Form 10-K or 10-KSB and other filings made by such company with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and FNM undertakes no obligation to update such statements.

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Orphan Drug Development to Target Increasing Incidence of Liver Cancer - PRNewswire

Global Gene Therapy for Age-related Macular Degeneration Market 2020 by Manufacturers, Countries, Type and Application, Forecast to 2025 launched by MarketsandResearch.biz aims to unveil key opportunities available in the global market to help players in securing a strong market position in the market. The report provides a detailed analysis of the market status, investment plans, production and consumption, price trends, and analysis by the market player, by region, by type, by the application. The report comprises verified and reliable market forecasts for the overall size of the global Gene Therapy for Age-related Macular Degeneration market in terms of revenue. The report discusses all major market aspects with an expert opinion on current market status along with historic data. A comprehensive analysis of the market standard, geographical regions, market key vendors, end-user applications, and products are studied in this study report.

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Influential players covered up in the report: RetroSense Therapeutics, REGENXBIO, AGTC,

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Global Gene Therapy for Age-related Macular Degeneration Market 2020 with COVID-19 Impact Analysis and Forecast by 2025 - NJ MMA News

Gene Therapy for Ovarian Cancer Market report involves all together a different chapter on COVID 19 Impact. The Covid-19 (coronavirus) pandemic is impacting society and the overall economy across the world. The impact of this pandemic is growing day by day as well as affecting the supply chain. The COVID-19 crisis is creating uncertainty in the stock market, massive slowing of supply chain, falling business confidence, and increasing panic among the customer segments. The overall effect of the pandemic is impacting the production process of several industries including Life Science, and many more. Trade barriers are further restraining the demand- supply outlook. nicolas.shaw@cognitivemarketresearch.com or call us on +1-312-376-8303.Download The report Copy form the webstie: https://cognitivemarketresearch.com/medical-devicesconsumables/gene-therapy-for-ovarian-cancer-market-report

The major players profiled in this report include: Takara Bio, VBL Therapeutics, CELSION, Targovax

Market segment by type can be split into: Intravenous, Intratumoral, Intraperitoneal

Market segment by the application can be split into: Ovarian Cancer (unspecified), Recurrent Ovarian Epithelial Cancer, Platinum Resistant Ovarian Cancer

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As government of different regions have already announced total lockdown and temporarily shutdown of industries, the overall production process being adversely affected; thus, hinder the overall Gene Therapy for Ovarian Cancer globally. This report on Gene Therapy for Ovarian Cancer provides the analysis on impact on Covid-19 on various business segments and country markets. The report also showcases market trends and forecast to 2027, factoring the impact of COVID-19 situation.

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Objectives of Gene Therapy for Ovarian Cancer Market Report:To justifiably share in-depth info regarding the decisive elements impacting the increase of industry (growth capacity, chances, drivers and industry specific challenge and risks)To know the Gene Therapy for Ovarian Cancer Market by pinpointing its many sub segmentsTo profile the important players and analyze their growth plansTo endeavor the amount and value of the Gene Therapy for Ovarian Cancer Market sub-markets, depending on key regions (various vital states)To analyze the Global Gene Therapy for Ovarian Cancer Market concerning growth trends, prospects and also their participation in the entire sectorTo inspect and study the Global Gene Therapy for Ovarian Cancer Market size form the company, essential regions/countries, products and applications, background information and also predictions to 2027Primary worldwide Gene Therapy for Ovarian Cancer Market manufacturing companies, to specify, clarify and analyze the product sales amount, value and market share, market rivalry landscape, SWOT analysis and development plans for the next coming yearsTo examine competitive progress such as expansions, arrangements, new product launches and acquisitions on the market

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Drivers and Restraints of Gene Therapy for Ovarian Cancer Market Research Report 2020 Top Compnaies Include, Takara Bio, VBL Therapeutics, CELSION,...

Encoded Therapeutics, Inc., a South Francisco, Calif.-based precision gene therapy company, raised $135m in Series D financing.

The round was led by GV (formerly Google Ventures) with participation from Matrix Capital Management, ARCH Venture Partners, Illumina Ventures, RTW Investments, Boxer Capital, Nolan Capital, HBM Genomics, Menlo Ventures, Meritech Capital, Farallon Capital Management, SoftBank Vision Fund 21, and additional unnamed investors. In conjunction with the funding, David Schenkein, M.D., general partner and co-leader of GVs life sciences team, will join the Encoded Board of Directors as an observer.

Led by co-founder and chief executive officer, Kartik Ramamoorthi, Ph.D., Encoded is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. It is advancing its lead asset, ETX101, for the treatment of SCN1A+ Dravet Syndrome by leveraging a discovery engine that combines biological and computational approaches to identify and screen human DNA sequences known as regulatory elements at a high throughput scale.

The company intends to use the funds to advance its lead asset, ETX101, which was granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation by the U.S. Food and Drug Administration (FDA) for the treatment of SCN1A+ Dravet Syndrome.

Proceeds from the Series D will be used to conduct clinical trial activities including a natural history study to better understand the progression of SCN1A+ Dravet Syndrome as well as first-in-human trials for ETX101. Additionally, the funds will support progression of the companys pipeline of gene therapies being evaluated for additional pediatric CNS disorders.

FinSMEs

23/07/2020

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Encoded Therapeutics Raises $135M in Series D Financing - FinSMEs