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Archive for the ‘Gene therapy’ Category

Orchard Therapeutics to Present at Barclays Gene Editing & Gene Therapy Summit – Yahoo Finance

Tuesday, November 10th, 2020

BOSTON and LONDON, Nov. 09, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company is scheduled to present at the Barclays Gene Editing & Gene Therapy Summit on November 16, 2020 at 11:15 AM ET.

A live webcast of the presentation will be available under "News & Events" in the Investors & Media section of the company's website at http://www.orchard-tx.com. A replay of the webcast will be archived on the Orchard website following the presentation.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

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Contacts

Investors

Renee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

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Orchard Therapeutics to Present at Barclays Gene Editing & Gene Therapy Summit - Yahoo Finance

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Increase in the Adoption of Cell and Gene Therapy to Propel the Growth of the Cell and Gene Therapy Market Between 2020-2027 – Eurowire

Tuesday, November 10th, 2020

Final Report will add the analysis of the impact of COVID-19 on this industry.

November 2020:

The global Cell and Gene Therapy Market research report offers all the vital data in the domain. The latest report assists new bees as well as established market participants to analyze and predict the Cell and Gene Therapy market at the regional as well as global level. It covers the volume [k MT] as well as revenues [USD Million] of the global Cell and Gene Therapy market for the estimated period. Numerous key players Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST. are dominating the global Cell and Gene Therapy market. These players hold the majority of share of the global Cell and Gene Therapy market.

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The data presented in the global Cell and Gene Therapy market offers budding opportunities, which help users to make strategic moves and prosper their business. The report highlights the impact of numerous factors that might result in obstructing or propelling the Cell and Gene Therapy market at global as well as local level. The global Cell and Gene Therapy market research report offers the summary of key players dominating the market including several aspects such as their financial summary, business strategy, and most recent developments in these firms.

Detailed Segmentation:

By Therapy Type:Cell TherapyStem CellsT CellsDendritic CellsNK CellsTumor CellsGene TherapyGlobal Cell and Gene Therapy Market, By Indication:Cardiovascular DiseaseCancerGenetic DisorderInfectious DiseaseNeurological DisordersOthers

By Region

North America (U.S., Canada, Mexico)

Asia Pacific (India, China, Japan, South Korea, ASEAN, Rest of Asia Pacific)

Europe (Italy, Germany, France, Spain, Central & Eastern Europe, Rest of Europe)

Middle East & Africa (GCC, Turkey, Rest of the Middle East & Africa)

South America (Brazil, Argentina, Rest of South America)

The study encompasses profiles of major companies operating in the Cell and Gene Therapy Market. Key players profiled in the report includes: Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

Key points of the global Cell and Gene Therapy market

The oretical analysis of the global Cell and Gene Therapy market stimulators, products, and other vital facets

Recent, historical, and future trends in terms of revenue and market dynamics are reported

Pin-point analysis of the competitive market dynamics and investment structure is predicted to grow

Future market trends, latest innovations, and various business strategies are reported

Market dynamics include growth influencers, opportunities, threats, challenges, and other crucial facets

The global Cell and Gene Therapy market research report offers users with an all-inclusive package of market analysis that includes current market size, expansion rate, and value chain analysis. The global Cell and Gene Therapy market is segmented on a regional basis Europe, North America, Latin America, Asia Pacific, and Middle East & Africa as well. To offer a comprehensive view and competitive outlook of the global Cell and Gene Therapy market, our review team employs numerous methodological procedures, for instance, Porters five forces analysis.

This research report includes the analysis of various Cell and Gene Therapy market segments. The bifurcation of the global market is done based on its present and prospective inclinations. The regional bifurcation involves the present market scenario in the region along with the future projection of the global Cell and Gene Therapy market. The global market report offers an overview of expected market conditions due to changes in the technological, topographical, and economic elements.

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Questions answered in the report include

What is the expected market size by the end of the forecast period?

What are the major factors initiating the global Cell and Gene Therapy market growth?

What are the latest developments and trending market strategies that are influencing the growth of the Cell and Gene Therapy market?

What are the key outcomes of the Cell and Gene Therapy market developments?

Who are the key players in the market?

What are the opportunities and challenges faced by the key players?

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Link:
Increase in the Adoption of Cell and Gene Therapy to Propel the Growth of the Cell and Gene Therapy Market Between 2020-2027 - Eurowire

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Gene Therapy Market to Witness Marginal decline in Near Term amid COVID-19 Pandemic, Projects Fact.MR 2020 2026 – The Cloud Tribune

Tuesday, November 10th, 2020

The global Gene Therapy market is rife with several new opportunities for market growth. This study provides a comprehensive description of the various propensities and trends that are aiding the growth of this market. The review takes a holistic approach in analyzing and decrypting the various forces of market growth. The all-inclusive review covers multiple insights on the historic trends and future projections that have shaped the current fettle of the market. It is worthwhile to note that the review is written with careful evaluation of various outliers and exceptions to market growth. In order to reconcile the exceptions, the projections made in the review leave scope for scaling and optimization.

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The global Gene Therapy Market is projected to reach a value of US$ XX Mn/Bn by the end of the forecast period. These projections are based on evidence-based research and analysis of the various parameters of market growth.

The global Gene Therapy market consists of the following key players:

The report takes a cognitive approach to decode the business strategies of each of the leading players. Besides, the report also gauges the impact of recent market developments on the growth trajectory of the global Gene Therapy market. The insights presented in the report are written after taking due cognizance of the various trends that currently exist in the industry.

The declaration of the coronavirus disease as a global health emergency, and successively a pandemic, created formidable challenges for the vendors within the global Gene Therapy market. The COVID-19 crisis caused a global economic downturn, aggravated by industrial shutdowns and supply chain disruptions. The vendors operating in the global Gene Therapy market are expected to make relentless efforts to minimize the damage caused by the pandemic over the coming years. Companies that deployed strategies of business continuity management and damage control were able to weather the storm. Therefore, COVID-19 pandemic has been a subjective crisis for various industries and players.

The report covers the following key regions:

Based on products, the Gene Therapy market can be segmented into the following categories:

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The report gives an objective and pragmatic outlook on key trends pertaining to:

The report answers, in detail, the following key questions pertaining to market expansion:

The Gene Therapy market makes projections for the following phases:

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Link:
Gene Therapy Market to Witness Marginal decline in Near Term amid COVID-19 Pandemic, Projects Fact.MR 2020 2026 - The Cloud Tribune

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Gene therapy for autism-linked condition weakened legs, robbing two people of ability to walk – Science Magazine

Saturday, November 7th, 2020

A new gene therapy may lessen traits of Angelman syndrome, but its makers may need to limit the dose.

By Giorgia Guglielmi, SpectrumNov. 5, 2020 , 9:30 AM

Originally published onSpectrum

A small clinical trial of a gene therapy for Angelman syndromea rare genetic condition related to autismis on hold after two participants temporarily lost the ability to walk. The safety issue is important to resolve, experts say, given that the therapy otherwise appears to be effective, and the trial could guide treatment strategies for similar brain conditions.

Biopharmaceutical companyUltragenyxin Novato, California, in collaboration with Florida-based biotech startupGeneTx, launched the trial in February to assess the safety of a therapy forAngelman syndrome, a neurodevelopmental condition characterized by intellectual disability, balance and motor problems, seizures, sleep problems and, in some cases, autism.

Angelman syndrome results from the mutation or absence of a gene calledUBE3A. People inherit two copies of UBE3A. Typically, only the maternal copy is active in neurons and the paternal copy is silent. But in people with Angelman syndrome, the maternal copy is mutated or missing, so their brain cells express no active UBE3A protein.

The drug developed by Ultragenyx and GeneTx, called GTX-102, is a short snippet of RNA called an antisense oligonucleotide thatactivates the paternal copy of UBE3Aand aims to restore the protein to typical levels. Three other companiesRoche, Biogen, and Ionisare pursuing similar therapies for the syndrome.

On 26 October, Ultragenyx and GeneTx reported that the clinical trial hadenrolled five individualswith Angelman syndrome, aged 5 to 15. The plan had been to administer to each participant a dose of GTX-102 once a month over four months. Researchers injected the drug directly into the nutrient-rich solution that envelops the brain and spinal cord through a site in the lower back.

The participants were to receive increasing doses, but all started with different amounts: Two began at the lowest dose, two started with the second-lowest dose, and one started at the second-highest dose. The final dose was about 10 times higher than the lowest dose.

After a single dose at the second-highest level, one participant developed leg weakness. The other four participants experienced the same adverse effect after taking the highest dose. The symptoms emerged one to four weeks after the participants last dose.

Two of the patients were not able to support themselves to walk and three were, but they were weaker, saysElizabeth Berry-Kravis, professor of child neurology at Rush University Medical Center in Chicago, Illinois, where the five children were treated.

The side effects appear to be a result of inflamed nerves where the drug was injected, perhaps due to accumulation of the drug in that area. In animal studies, the drug didnt cause similar adverse effects, says Ultragenyx chief executive officer,Emil Kakkis. We do know, though, that antisense oligonucleotides are known to have local toxic effects if given at high concentrations.

The participants all recovered after they received drugs that decrease inflammation, Berry-Kravis says. Even those who couldnt support themselves on their legs are walking around finethey actually are somewhat more coordinated now than they were before the study.

When the researchers evaluated the participants at day 128, all five showed significant improvements in some traits, including communication, sleep, and motor skills, Berry-Kravis says. Within weeks of the initial doses, parents and caregivers reported that the participants had acquired new words and gestures.

Were seeing things like using a fork independently for the first time ever, learning to swim on their own, using their augmentative communication device, being able to play an interactive game with the family, Berry-Kravis says. But, she adds, you cant go on with an adverse event.

Going forward, the companies plan to limit the maximum dose to a range in which the drug appears to improve traits without causing leg weakness. They also intend to change how they administer the drug so it cannot accumulate at the site of injection. The drug solution will be given to the patient with their head down to allow the drug to flow toward the brain more efficiently, Kakkis says.

Before resuming the study, the companies will seek approval from the U.S. Food and Drug Administration, says Scott Stromatt, chief medical officer of GeneTx. We hope dosing will start in the next one to two months, he says. Parents are pretty excited to resume because of the positive changes theyve observed in their children.

All drugs have a side effect at some point, saysMark Zylka, professor of cell biology and physiology at the University of North Carolina at Chapel Hill, who was not involved in the study. It seems like theyre just going to need to dial in the dosage better.

Zylka is working on atherapy for Angelman syndromethat uses the gene-editing technology CRISPR to unmute the paternal copy of UBE3A. The rapid improvement observed in the trial participants is encouraging, he says. It suggests that this idea of turning on the dads copy of the gene really has the potential to help individuals with Angelman.

Others are excited about what the trial results might mean for other brain conditions. One of the biggest questions in the field is how long the therapeutic window remains open in neurodevelopmental disorders like Angelman syndrome, saysTimothy Yu, assistant professor of pediatrics at Harvard University. The preliminary findings from the Ultragenyx and GeneTx trial suggest that the therapy can work even in teenagers.

Its still early days, and we have to be careful, Yu says. But if this result continues to hold true, thats going to be really game-changing.

This article was reprinted with permission fromSpectrum,the home of autism research news and analysis.

Go here to see the original:
Gene therapy for autism-linked condition weakened legs, robbing two people of ability to walk - Science Magazine

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Gene therapy trial for autism-linked condition is put on hold – Spectrum

Saturday, November 7th, 2020

Stand by: A new gene therapy may lessen traits of Angelman syndrome, but its makers may need to limit the dose.

Courtesy of Ultragenyx

A small clinical trial of a gene therapy for Angelman syndrome a rare genetic condition related to autism is on hold after two participants temporarily lost the ability to walk. The safety issue is important to resolve, experts say, given that the therapy otherwise appears to be effective, and the trial could guide treatment strategies for similar brain conditions.

Biopharmaceutical company Ultragenyx in Novato, California, in collaboration with Florida-based biotech startup GeneTx, launched the trial in February to assess the safety of a therapy for Angelman syndrome, a neurodevelopmental condition characterized by intellectual disability, balance and motor problems, seizures, sleep problems and, in some cases, autism.

Angelman syndrome results from the mutation or absence of a gene called UBE3A. People inherit two copies of UBE3A. Typically, only the maternal copy is active in neurons and the paternal copy is silent. But in people with Angelman syndrome, the maternal copy is mutated or missing, so their brain cells express no active UBE3A protein.

The drug developed by Ultragenyx and GeneTx, called GTX-102, is a short snippet of RNA called an antisense oligonucleotide that activates the paternal copy of UBE3A and aims to restore the protein to typical levels. Three other companies Roche, Biogen and Ionis are pursuing similar therapies for the syndrome.

On 26 October, Ultragenyx and GeneTx reported that the clinical trial had enrolled five individuals with Angelman syndrome, aged 5 to 15. The plan had been to administer to each participant a dose of GTX-102 once a month over four months. Researchers injected the drug directly into the nutrient-rich solution that envelops the brain and spinal cord through a site in the lower back.

The participants were to receive increasing doses, but all started with different amounts: Two began at the lowest dose, two started with the second-lowest dose, and one started at the second-highest dose. The final dose was about 10 times higher than the lowest dose.

After a single dose at the second-highest level, one participant developed leg weakness. The other four participants experienced the same adverse effect after taking the highest dose. The symptoms emerged one to four weeks after the participants last dose.

Two of the patients were not able to support themselves to walk and three were, but they were weaker, says Elizabeth Berry-Kravis, professor of child neurology at Rush University Medical Center in Chicago, Illinois, where the five children were treated.

The side effects appear to be a result of inflamed nerves where the drug was injected, perhaps due to accumulation of the drug in that area. In animal studies, the drug didnt cause similar adverse effects, says Ultragenyx chief executive officer, Emil Kakkis. We do know, though, that antisense oligonucleotides are known to have local toxic effects if given at high concentrations.

The participants all recovered after they received drugs that decrease inflammation, Berry-Kravis says. Even those who couldnt support themselves on their legs are walking around fine they actually are somewhat more coordinated now than they were before the study.

When the researchers evaluated the participants at day 128, all five showed significant improvements in some traits, including communication, sleep and motor skills, Berry-Kravis says. Within weeks of the initial doses, parents and caregivers reported that the participants had acquired new words and gestures.

Were seeing things like using a fork independently for the first time ever, learning to swim on their own, using their augmentative communication device, being able to play an interactive game with the family, Berry-Kravis says. But, she adds, you cant go on with an adverse event.

Going forward, the companies plan to limit the maximum dose to a range in which the drug appears to improve traits without causing leg weakness. They also intend to change how they administer the drug so it cannot accumulate at the site of injection. The drug solution will be given to the patient with their head down to allow the drug to flow toward the brain more efficiently, Kakkis says.

Before resuming the study, the companies will seek approval from the U.S. Food and Drug Administration, says Scott Stromatt, chief medical officer of GeneTx.

We hope dosing will start in the next one to two months, he says. Parents are pretty excited to resume because of the positive changes theyve observed in their children.

All drugs have a side effect at some point, says Mark Zylka, professor of cell biology and physiology at the University of North Carolina at Chapel Hill, who was not involved in the study. It seems like theyre just going to need to dial in the dosage better.

Zylka is working on a therapy for Angelman syndrome that uses the gene-editing technology CRISPR to unmute the paternal copy of UBE3A. The rapid improvement observed in the trial participants is encouraging, he says. It suggests that this idea of turning on the dads copy of the gene really has the potential to help individuals with Angelman.

Others are excited about what the trial results might mean for other brain conditions. One of the biggest questions in the field is how long the therapeutic window remains open in neurodevelopmental disorders like Angelman syndrome, says Timothy Yu, associate professor of pediatrics at Harvard University. The preliminary findings from the Ultragenyx and GeneTx trial suggest that the therapy can work even in teenagers.

Its still early days, and we have to be careful, Yu says. But if this result continues to hold true, thats going to be really game-changing.

See the article here:
Gene therapy trial for autism-linked condition is put on hold - Spectrum

Read More...

Hemophilia Gene Therapy Market: Growing prevalence of diseases related to blood clotting or bleeding to drive the market – BioSpace

Saturday, November 7th, 2020

Global Hemophilia Gene Therapy Market: Overview

The global hemophilia gene therapy market is predicted to increase at a stellar pace in the years to follow. The medical fraternity has shown immense resilience in dealing with bleeding disorders, backed by the research excellence of the microbiology sector. The growing incidence of diseases related to genetic dysfunctions has out the healthcare industry on its toes. Research related to genetic mutations have given an impetus to the healthcare sector. There has been a renewed interest in genetic studies amongst researchers and scientists. The commendable results achieved from next-generation genetic studies has created a myriad of opportunities for growth within healthcare. The aforementioned factors are expected to drive sales across the global hemophilia gene therapy market.

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This review on the global hemophilia gene therapy market gives a concise description of the leading factors responsible for market expansion. The review analyses the growing relevance of gene therapies to make forecasts related to market expansion. The trends and propensities exhibited by the healthcare sector have also been enunciated herein. Henceforth, the review is an evidence-based commentary on the factors that can drive market demand over the near future. The COVID-19 crisis has had adverse impacts on several markets and industries, save the healthcare sector. Therefore, the review also points to the marked increase in revenues across this market due to the pandemic.

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Global Hemophilia Gene Therapy Market: Competitive Dynamics

Next-generation DNA sequencing and genetic mutations have played a vital role in enhancing healthcare treatments across a range of specializations. For this reason, leading players in the hemophilia gene therapy market are upping their game with genetic research. The ability of these vendors to produce visible results in patient care shall be a sound driver of market demand. A number of companies are also assisting patients in defining their own clotting factor with the help of new aids and technologies.

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Some of the market vendors have acquired visible expertise in the use of a modified virus for treatment of blood clotting. This virus is different from the one that caused the disease, enabling it to help in encoding the clotting factor. Market players can grow at the back of these advancements and distinctions over the next decade. Some of the leading vendors operating in the global hemophilia gene therapy market are Spark Therapeutics, UniQure NV, Pfizer, Inc., Ultragenyx Pharmaceutical, and Shire PLC Sangamo Therapeutics, Inc.

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Global Hemophilia Gene Therapy Market: Key Trends

The presence of a seamless industry for studying and analysing diseases related to blood clotting or bleeding has given an impetus to market growth. The next decade is expected to witness a surge in demand for hemophilia treatments. The proven excellence of gene therapy in preventing excessive bleeding in ailing individuals shall also play a vital role in market growth and maturity.

Prolonged external or internal bleeding can also be a cause of several other diseases and disorders. This has also led to increased investments in treatment of diseases related to blood clots. The next decade is expected to usher an era of advancements in gene therapy, giving a thrust to growth across the global market.

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Global Hemophilia Gene Therapy Market: Regional Landscape

On the basis of regions, the global hemophilia gene therapy market can be segmented into North America, Asia Pacific, South America, Europe, and the Middle East and Africa. The excellence of the US and Canada in gene therapy has given a thrust to the growth of the North America hemophilia gene therapy market.

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Originally posted here:
Hemophilia Gene Therapy Market: Growing prevalence of diseases related to blood clotting or bleeding to drive the market - BioSpace

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Bringing Flexion’s OA Gene Therapy to Market Starts with Cost and Benefits in Mind – BioSpace

Saturday, November 7th, 2020

As more and more gene therapies are approved, the elements that determine their success depend, increasingly, on the business decisions made early on.

There is nuance to every program but, (across-the-board) the cost of production is a big challenge, Mike Clayman, M.D., CEO and co-founder of Flexion, told BioSpace.

Flexions lead candidate, FX201 (humantakinogene hadenovec), is a case in point. This is an intra-articular gene therapy product for osteoarthritis (OA). It uses a helper-dependent adenovirus (HDAd) vector whose genetic construct the payload is a gene that expresses the interleukin-1 receptor antagonist (IL-1Ra) in the joint to control inflammation.

Inflammation is a known cause of pain, and chronic inflammation is believed to play a key role in the progression of OA, he explained.

FX201 is taken up rapidly by cells lining the joint, converting them into factories to make IL-1Ra. It quells the inflammation and then turns off the gene construct, Clayman said. It has the potential to provide long-term pain relief, functional improvement, and to actually delay the progression of the disease.

By injecting the product directly into the approximately 5 ml volume of the knee joint, he continued, The absolute amount of protein you need to create an effective intra-articular concentration is far less than that which would be needed to achieve systemic therapeutic levels. That translates into what we expect will be much lower doses of FX201, which would reduce manufacturing cost and give us flexibility to charge a price that would still be proportional to value delivered.

The product entered Phase I, a single ascending dose study, this year. Preclinical work suggests that once injected, there is no significant distribution outside the joint.

It is a non-integrating, non-replicating vector, Clayman said. There is preclinical evidence that it delivers meaningful improvement for at least one year, with the slowing of disease progression.

Company scientists believe a single injection FX201 has the potential to provide at least 6 to 12 months of pain relief and functional improvement in addition to possibly slowing disease progression, Clayman said. We believe this product profile, if realized, would represent a transformative new treatment for knee OA.

Currently, Flexion is actively pursuing the possibility of identifying sub-populations of OA patients who may differentially benefit, Clayman said.

The initial study is open broadly to OA patients to evaluate safety and tolerability. Once those data are analyzed, he said subsequent studies will consider and, if possible, incorporate biomarker, phenotypic or genotypic criteria to focus on a more restricted population.

There may be a marker perhaps a serum marker to identify a patient subset that may most benefit from the therapy, Clayman said. With 15 million patients in the U.S. with knee symptoms of OA, and 8 million annual injections to relieve knee pain, it behooves companies to identify a focal population that can most benefit.

But, he acknowledged, The genetics of OA are very complex. Its clearly a multigene disease with additional contributions from environmental and other external factors. Its not as though there are clearly defined genetic subtypes of the disease.

FX201 isnt the first OA product Flexion has developed. Zilretta (triamcinolone acetonide extended-release injectable suspension), an extended-release, intra-articular corticosteroid, was approved by the FDA in 2017 to relieve OA-related knee pain. In Phase III trials, it significantly reduced knee pain for 12 weeks and, for some patients, 16 weeks or more. Since approval, it has completed a Phase IIIb trial for synovitis.

Flexion also is developing FX301 (funapide, a selective sodium channel blocker, formulated in a thermosensitive gel) for acute post-operative pain.

This isnt a gene therapy, Clayman said. This is a peripheral nerve block that is expected to enter the clinic in 2021. The preclinical data so far suggest it may durably prevent post-op pain while preserving motor function.

The FDA and EMA are up-to-speed on gene therapy now. At Claymans meetings with the FDA, We had very robust scientific exchanges. I left impressed with the sophistication that was applied to the discussion. The EMA was similarly sophisticated, he said.

Coincidentally, the criteria for RMAT designation comparable to breakthrough designation for other products illustrating the points for clinical conditions, used OA as the example.

This indicates the FDA is thinking about creative approaches to complex diseases, Clayman said. If we have compelling data, we would not hesitate to pursue RMAT designation.

Flexion already has begun its health economics and outcomes research, modeling this therapeutic in terms of its ability to delay the need for knee replacement (which costs $25,000 to $50,000), and quality of life.

Whether payers will reimburse for gene therapies often depends as much on the price as on delivered value.

Our dose likely will give us manufacturing costs that will allow us to price FX201 much lower than a systemic therapy, which puts us in a better position, Clayman said. Of course, We still must generate compelling data.

Read more from the original source:
Bringing Flexion's OA Gene Therapy to Market Starts with Cost and Benefits in Mind - BioSpace

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The City of Biotech Love – Science Magazine

Saturday, November 7th, 2020

Known as the City of Brotherly Love, Philadelphia, Pennsylvania, now has a new monickerCellicon Valleyto honor the cell and gene therapies that companies such as Spark Therapeutics are bringing to it.

Science@Work

Nov. 6, 2020 , 2:00 PM

Affectionally named Cellicon Valley, Philadelphia is the birthplace of cell and gene therapy and home to more than 30 companies dedicated to cell and gene therapy research. Philadelphia-based Spark Therapeutics became the first to bring a U.S. Food and Drug Administration (FDA)-approved gene therapy to market and has been expanding its footprint in this biotech hotspot for gene and cell therapy ever since, according to Chief Scientific Officer Federico Mingozzi, Ph.D.

Headquartered in Philadelphia, Pennsylvania, Spark Therapeutics is helping the city become a premier spot for cell and gene therapy.

In 2013, Spark Therapeutics was founded as a result of the technological and scientific know-how developed at the Childrens Hospital of Philadelphia (CHOP). Now part of the Roche Group, the company is committed to further growing Philadelphia as a global hub of cell and gene therapy. With ambitious growth plans, the company remains focused on extending its scientific platform and unique competencies to evaluate and select a portfolio of gene therapies targeting diseases of the retina, hemophilia A, lysosomal storage disorders, and neurodegenerative diseases. They currently have four programs in clinical trials advancing therapies to treat hemophilia A, Pompe disease, and choroideremia.

Chief Scientific Officer Federico Mingozzi, Ph.D.

With Roches worldwide reach and resources, we are closer than weve ever been before to fulfilling our vision of living in a world where no life is limited by genetic disease, Mingozzi says. Although many gene therapy studies focus on genetic diseases, Mingozzi notes, there is an appetite to expand gene therapy to more common indications.

Despite the challenges of 2020, there has been tremendous growth at Spark. The company now employs more than 500 people and has a growing footprint in West Philadelphia. Spark has hired more than 130 employees since the start of 2020 and does not intend to slow down as the company heads into the upcoming year. Part of that expansion brought Senior Research Associate Phillip Price, Ph.D. to Spark in February. Drawn by the companys passion and commitment to treating genetic diseases, Price recalls thinking, I knew I wanted to be part of this.

At the time he was hired by Spark, Price had recently completed his doctorate in neuroscience at Emory University and was serving as a visiting researcher at the Mayo Clinic in Jacksonville, Florida. His strong conviction about the need to bring more treatments to more patients living with genetic disease, in tandem with a seamless interviewing process, propelled Price to make the leap from Florida to Pennsylvania.

Senior Research Associate Phillip Price, Ph.D.

Despite working virtually, Price says he was able to get plugged into the company quickly and find a strong relationship between the employer and the employees, who encouraged both personal and professional growth. Price mentions that the company also made significant efforts to understand and respond to the changing dynamics resulting from the pandemic, such as working remotely. He has benefitted from the strong lines of communication from Sparks leadership andits unique employee programs such as half days every other Friday, which have enabled him to step away from work and relax while learning more about Philadelphia.

Although Price experienced some hesitancy in transitioning from academia to industry, that disappeared when he found that throughout the Spark workforce people are dedicated to the learning, listening, and innovation needed to bring the best treatments to patients. Price appreciates that high level of motivation. Gene therapy is not just about therapeutic targets of the future, he notes. This is here now, and the door is open! At Spark, he finds even more excitement since being a part of Roche provides a platform to reach globally.

With experience in studying spinal muscular atrophy, an inherited disorder, Price fits right in with Sparks central nervous system (CNS) team, which has treatments in its pipeline for other genetic diseases, including Huntingtons disease and Batten disease. Already, he describes the environment as very dynamic and based on beneficial processes that help cultivate ideas. Plus, he quickly felt like part of the team, where he says, everyone has a voice.

This is the hope: Philadelphia will continue to attract and grow the community of biotechnology experts and become one of the most important hubs for gene and cell therapy in the world.

The thriving life sciences industry in Philadelphia also appealed to Price. More than 42,000 Philadelphians work in life sciences and the city is now rated the fifth largest research and development (R&D) hub in the nation, boasting $10.5 billion spent annually in R&D, according to statistics provided by Life Sciences Pennsylvania. Plus, international information firm EqualOcean reports, More than 700 life science companies have chosen to come to Philadelphia to develop their products.

In addition to CHOP, the region is home to a number of other world-class life sciences institutions, including the University of Pennsylvania Perelman School of Medicine, Temple University, and the Drexel University College of Medicine. The combination of Philadelphias academic institutions with the thriving environment it provides for biotech start-ups has resulted in an excellent system for networking and collaborating, according to Price.

Assay development scientist Stephanie Kutza at work in Sparks labs.

Mingozzi agrees. He added that Spark is one of a cluster of many active companies in gene and cell therapy that are creating a vibrant biotech center in the city. Having a hub attracts talent from other cities and retains the talent that is already here, Mingozzi says. For example, he notes that Philadelphia is a place where people come for training and then stay for work. Overall, its an incredible success story for Philadelphia, and the field of gene and cell therapy is driving this success, he says.

Still, Mingozzi sees this as just the beginning for Philadelphia. Youll see that the driver of this hub will be a couple of companies, like Spark, and that people trained in Philadelphia will start new companies here, he says. Then gene therapy will become a major contributor for the local economy.

This diverse city, however, offers much more than science. Price lives downtown, which he describes as a very lively and inspiring atmosphere. As a history buff, he enjoys exploring Philadelphia with its many important sites, including Independence Hall, the Liberty Bell and the Betsy Ross House. Thinking of the architecture, museums, and parks, Price has nothing but appreciation for the City of Brotherly Love, saying its really quite beautiful here.

Notwithstanding its beauty, even more biotech growth lies ahead in Philadelphias future. As Mingozzi sees it, This is the hope: Philadelphia will continue to attract and grow the community of biotechnology experts and become one of the most important hubs for gene and cell therapy in the world.

Spark ranks among the top employers in Science Careers 2020 Top Employer survey. Read more

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Citing pandemic, Sarepta defers much-anticipated DMD gene therapy PhIII, will head to regulators after 10-person study – Endpoints News

Saturday, November 7th, 2020

Roger Perlmutter has a few more moves to make before he exits the top job in Merck R&D.

The pharma giant is buying VelosBio for $2.75 billion in cash, grabbing a promising ROR1 antibody drug conjugate that is now in Phase I and Phase II studies for hematologic malignancies and solid tumors.

Perlmutter, who will leave at the end of the year after building Keytruda into a monster drug franchise, clearly likes the data hes seen from the biotech. And the oncology group sees plenty of opportunities for the rest of the pipeline at VelosBio, which raised a crossover round of $137 million just a few months ago in an apparent move toward an IPO.

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Ocugen Provides Business Update and Third Quarter 2020 Financial ResultsFirst Gene Therapy Candidate OCU400 On Track to Enter the Clinic in 2021 -…

Saturday, November 7th, 2020

MALVERN, Pa., Nov. 06, 2020 (GLOBE NEWSWIRE) --Ocugen, Inc.. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing and commercializing transformative therapies to cure blindness diseases, today reported third quarter 2020 financial results along with a general business update.

I am proud of the advancements we have made this quarter in developing our modifier gene therapy and novel biologic product candidates. We remain on-track to achieve multiple near and mid-term milestones with a plan to initiate four Phase 1/2a trials during 2021 and 2022. Additionally, I am pleased that we have raised approximately $28million of gross proceeds in 2020, which have extended our cash runway for continued development of our product candidates, said Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen.

Business Highlights:

Third Quarter 2020 Financial Results:

About Ocugen, Inc.

Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug one to many and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. For more information, please visit http://www.ocugen.com.

Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the SEC), including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.

Corporate Contact:Ocugen, Inc.Sanjay SubramanianChief Financial OfficerIR@Ocugen.com

Media Contact:LaVoieHealthScienceKatie Gallagherkgallagher@lavoiehealthscience.com+1 617-792-3937

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Roadmap to Market for Cell and Gene Therapies, Upcoming Webinar Hosted by Xtalks – PR Web

Saturday, November 7th, 2020

Is your team confident in handling the complicated logistics that come with cell and gene trials?

TORONTO (PRWEB) November 06, 2020

Nothing is standard about the study design, study conduct or the regulatory process in cell and gene therapy trials. Every cell and gene therapy is different due to the specific characteristics of each product, with a complicated regulatory approval process and unique therapeutic challenges.

Do you have confidence in your trials endpoints? Are you pursuing an accelerated pathway or special designation? Will you need to plan for a centralized site model or remote monitoring? Is your team confident in handling the complicated logistics that come with cell and gene trials?

In this webinar, participants will learn an end-to-end approach to bringing a cell and gene therapy to market. You will receive advice from experts responsible for supporting over 100 advanced cell and gene therapy programs. They will share each step of this process covering the planning, execution, submission and post-marketing for trials in the US and EU. The information provided will help participants determine their next step in the development of their cell and gene therapy product.

Join Ewan Campbell, Advanced Therapy & Biotech Director, Veristat; Kevin Hennegan, Senior Regulatory Strategist, Veristat; and Rachel Smith, Project Director, Veristat in a live webinar on Friday, November 20, 2020 at 11am EST (4pm GMT/UK).

For more information, or to register for this event, visit Roadmap to Market for Cell and Gene Therapies.

ABOUT XTALKS

Xtalks, powered by Honeycomb Worldwide Inc., is a leading provider of educational webinars to the global life science, food and medical device community. Every year, thousands of industry practitioners (from life science, food and medical device companies, private & academic research institutions, healthcare centers, etc.) turn to Xtalks for access to quality content. Xtalks helps Life Science professionals stay current with industry developments, trends and regulations. Xtalks webinars also provide perspectives on key issues from top industry thought leaders and service providers.

To learn more about Xtalks visit http://xtalks.comFor information about hosting a webinar visit http://xtalks.com/why-host-a-webinar/

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Accelerate Cell and Gene Therapy Development and Increase Manufacturing Capacity with Higher Titer LV and AAV Transfection Platforms – BioProcess…

Saturday, November 7th, 2020

This webcast features:Leisha Kopp, Applications Scientist, Mirus Bio

Cell and gene therapies show unparalleled promise to improve the human condition by eradicating cancer and rectifying genetic disorders. However, low viral titers and capacity constraints in viral vector manufacturing processes can dramatically hinder the progress of these transformative therapies.

TheTransIT-VirusGEN Transfection Reagent from Mirus Bio offers a simplified, cost-effective workflow for recombinant lentivirus (LV) and adenoassociated virus (AAV) generation.TransIT-VirusGEN Transfection Reagent is also effective in both adherent and suspension HEK 293derived cell types, making it the superior reagent choice for most large-scale production platforms. In addition, Mirus developed LV and AAV enhancers to increase functional virus titers two to three fold over previously optimized conditions and offer up to 10-fold titer improvements over polyethylenimine (PEI)-based formulations.

Just fill out the form below to watch the recorded webcast now.

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Homology Medicines Announces Upcoming Oral Presentation on pheNIX Gene Therapy Clinical Trial for Adults with PKU – GlobeNewswire

Saturday, November 7th, 2020

Homology Webcast / Conference Call Scheduled for Friday, November 6 at 4:30 p.m. ET

BEDFORD, Mass., Nov. 05, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the upcoming oral presentation of data from the dose-escalation portion of the Companys ongoing Phase 1/2 pheNIX gene therapy clinical trial with HMI-102 for adults with phenylketonuria (PKU), the worlds first PKU gene therapy clinical trial. The data will be presented by pheNIX principal investigator Olaf Bodamer, M.D., Ph.D., FACMG, FAAP, Park Gerald Chair in Genetics & Genomics and Associate Chief of Genetics & Genomics at Boston Childrens Hospital, a premier center for metabolic disorders and the first site to enroll a patient in the pheNIX trial. The pheNIX trial results will be featured at the virtual New England Consortium of Metabolic Programs (NECMP) annual meeting, which is focused on new research in metabolic disorders, including PKU, on Friday, November 6. NECMP includes metabolic clinics, healthcare providers, patient organizations and others dedicated to increasing knowledge of metabolic disorders and improving delivery of healthcare to patients.

We are pleased that additional data from our pheNIX trial, the first-ever gene therapy trial for PKU, will be shared for the first time by a respected leader in the field of metabolic disorders and directly with the PKU community as part of a featured, peer-reviewed scientific session, stated Gabe Cohn, M.D., Chief Medical Officer of Homology Medicines. The oral presentation by Dr. Bodamer will include data from three dose cohorts in the dose-escalation phase of our pheNIX trial, the results of which are informing the upcoming expansion phase of our study.

Webcast/Conference Call The NECMP annual meeting is for members of the Consortium. Homology will host a conference call and webcast following the meeting on Friday, November 6 at 4:30 p.m. ET. The webcast will be accessible on Homologys website in the Investors section, and the webcast replay will be available on the website for 90 days following the presentation. To access using the conference call line, dial (866) 244-8091 (U.S./Canada toll-free) or (602) 563-8623, with Conference ID 7394503.

About HMI-102HMI-102 is an investigational gene therapy in clinical development for the treatment of phenylketonuria (PKU) in adults. HMI-102 is designed to encode the PAH gene, which is mutated in people with PKU, delivered via the liver-tropic AAVHSC15 vector. Homology has received Fast Track Designation and orphan drug designation for HMI-102 from the U.S. Food and Drug Administration (FDA), and orphan drug designation from the European Medicines Agency (EMA).

About Phenylketonuria (PKU)PKU is a rare inborn error of metabolism caused by a mutation in thePAHgene. PKU results in a loss of function of the enzyme phenylalanine hydroxylase, which is responsible for the metabolism of phenylalanine (Phe), an amino acid obtained exclusively from the diet. If left untreated, toxic levels of Phe can accumulate in the blood and result in progressive and severe neurological impairment. Currently, there are no treatment options for PKU that target the underlying genetic cause of the disease. According to the National PKU Alliance, PKU affects nearly 16,500 people in the U.S. with approximately 350 newborns diagnosed each year. The worldwide prevalence of PKU is estimated to be 50,000 people.

About Homology Medicines, Inc. Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicinesin vivoeither through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visitwww.homologymedicines.com.

Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans and timing for the release of clinical data from the Phase 1/2 pheNIX trial; our position as a leader in the development of genetic medicines; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarterly period endedJune 30, 2020and our other filings with theSECcould cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

Company ContactsTheresa McNeely Chief Communications Officer and Patient Advocate tmcneely@homologymedicines.com781-301-7277

Media Contact: Cara Mayfield Senior Director, Patient Advocacy and Corporate Communications cmayfield@homologymedicines.com781-691-3510

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Homology Medicines Announces Upcoming Oral Presentation on pheNIX Gene Therapy Clinical Trial for Adults with PKU - GlobeNewswire

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CRISPR, Vertex show CRISPR/Cas9 gene-editing therapy works in more patients – FierceBiotech

Saturday, November 7th, 2020

CRISPR Therapeutics and Vertex have presented updated data on their CRISPR/Cas9 gene-editing therapy in sickle cell disease and beta thalassemia, showing patients continue to benefit from the drug. Yet, hemoglobin levels fell in two of the seven patients in the latest update, raising early questions about the durability of CTX001.

One year ago, CRISPR and Vertex linked CTX001 to improvements in two hemoglobinopathy patients. The results were a milestone for gene editing, but CTX001 needs to build on the early success if it is to emerge as a viable rival to bluebird bios LentiGlobin gene therapy, which is now set to be submitted for FDA approval in late 2022.

The LentiGlobin delays create an opportunity for CTX001. The question is whether CRISPR and Vertex can take it. Ahead of the American Society of Hematology's (ASH's) annual meeting, the partners shared new data that shed light on the potential prospects of CTX001.

Fetal hemoglobin levels rose in all seven patients after administration of CTX001 and have remained elevated for up to 15 months of follow-up. The five beta thalassemia patients last needed packed red blood cells in the first month or two after receiving CTX001, having previously more than 10 units a year, and neither of the sickle cell patients have suffered vaso-occlusive crises since being treated.

There are potential points of concern in the data, though, particularly given the likelihood of CTX001 facing competition. Hemoglobin fell from 12.5 to 11.6 in one beta thalassemia patient between the fifth and sixth months. The drop followed a decrease from 12.9 to 12.5 between the fourth and fifth months.

Investigators also tracked a decline in hemoglobin levels in one of the sickle cell patients. Levels fell from 11.8 to 10.3 between month nine and month 12.

The declines happened in two of the three patients with the longest follow-up, raising the question of whether similar trends will play out in other subjects in the coming months. Those concerns are countered by data on the first beta thalassemia patient, whose hemoglobin level is higher than ever 15 months after treatment with CTX001.

Safety is another concern. In an update earlier this year, CRISPR and Vertex revealed they had seen seven serious adverse events across three patients, suggesting bluebirds LentiGlobin may have the edge in terms of safety. The ASH update discussed a new patient who suffered four serious adverse events that may be related to CTX001.

Such details could matter, given CTX001 is still likely to cede a head start to LentiGlobin. Yet, CRISPR sees CTX001 as a best-in-class treatment and, with bluebird stumbling, thinks the drug is well placed to capture a sizable slice of the market.

CRSP has mentioned they are working on bridging the development timeline gap (~1-2 yrs now) w/ BLUE, and they expect BLUE's earlier launch and investment in developing+educating the mkt could benefit CRSP w/o loss of a bolus of pts, analysts at Jefferies wrote in a note to investors.

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Researchers Explore Respiratory Impact of Novel Therapies in SMA – AJMC.com Managed Markets Network

Saturday, November 7th, 2020

Short-term pulmonary outcomes of these medications are published and longitudinal monitoring is active, they wrote. Most studies have demonstrated favorable responses in overall muscle strength compared to natural history, but the pulmonary outcomes vary.

Studies included in the review revolved around 3 recently approved treatments:

Nusinersen

Although nusinersen has demonstrated efficacy on respiratory muscle function, the effect varies by SMA subtype, according to the researchers. They cited a study that found the treatment had little impact on patients with SMA1 A and B and had better response in patients with SMA1 C when it came to ventilatory and thoracoabdominal patterns.

Onasemnogene abeparvovec-xioi

In the pivotal phase 1 START trial for the gene therapy, survival data was striking, with an event-free survival of 100% without need for permanent ventilation at 20 months compared with the 8% event-free survival in natural history studies. Throughout the study, 83% (n = 10) of patients required at least 1 hospitalization for respiratory illness with increased length of noninvasive ventilation support, although no hospitalizations surpassed 8 days. Patients in the study had 1.4 respiratory-related hospitalizations per year, with a mean length of stay of 6.7 days.

According to the researchers, the study data suggest that overall pulmonary morbidity and consequent healthcare utilization may have decreased, but some patients may still require escalation of respiratory support during illnesses.

Other data, coming from the phase 3 STRIVE trial, the ongoing SPR1NT study, and real-world experience with the treatment in 3 tertiary centers in Ohio, have made the case for improved respiratory and nutritional outcomes with the gene therapy as researchers await long-term data.

Risdiplam

In the multicenter FIREFISH trial, 90% of the 21 patients were alive without permanent ventilation and rarely required hospitalized after 12 months of treatment. After 23 months, 81% were alive without permanent ventilation. All patients were able to swallow safely and most (95%) were able to be fed orally or with supplemental gastrostomy feeds.

Promising data have also emerged from the SUNFISH and JEWELFISH trials, both of which included patients with varying subtypes of SMA. The researchers also highlighted the RAINBOWFISH study, which is actively recruiting patients with SMA type 1. According to the researchers, the results of the study could be transforming to those with a neonatal diagnosis of SMA type 1.

Looking forward

Looking toward the future, the researchers of the review noted that monitoring of short- and long-term outcomes is still critical, as its still too early to determine sustained improvement in pulmonary outcomes. They add that currently available data come from a limited number of patients and emphasize the need for more multicenter trials to better validate the impact of novel treatments.

Reference

Paul G, Gushue C, Kotha K, Shell R. The respiratory impact of novel therapies for spinal muscular atrophy. Pediatr Pulmonol. Published online October 24, 2020. doi:10.1002/ppul.25135

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Orchard Therapeutics’ gene therapy Strimvelis linked to a leukemia case – FiercePharma

Tuesday, November 3rd, 2020

The potential of gene therapies as cures for some hard-to-treat genetic diseases can be very attractive. But one such product is now suspected of causing a serious safety problem.

Friday, Orchard Therapeutics said its Strimvelis treatment, approved by European authorities in 2016 to treatthe rare inherited condition ADA-SCID, has been linked to a patients leukemia.

Preliminary findings suggest this diagnosis may be attributable to an insertional event related to treatment with Strimvelis, the company said. Its now investigating whether theres indeed a causal relationship.

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Since its 2016 EU approvalwhen it was owned by original developer GlaxoSmithKlineonly 16 patients have been treated with Stimvelis. The patient who developed leukemia had apparently been treated under a GSK compassionate use program in 2016.

No more patients will get the therapy before the investigation is complete, Orchard said. The drug was never approved in the U.S.

ADA-SCIDis a condition marked by a mutation in the gene making the adenosine deaminase (ADA) enzyme, which is essential for maintaining normal white blood cells. ADA-SCID patients, with a dysfunctional immune system, have less than two years to live without effective intervention.

RELATED:Orchard licenses gene therapy tech from GSK

Strimvelis, originally developed by GSK and bought by Orchard in 2018, offers an option for patients who cant find a matched stem cell donor. It works by editing the patients own hematopoietic stem cells with the functional ADA gene. The cells arethen transferred back into the patient's bone marrow to mature and produce the normal ADA protein.

The therapy uses a gammaretrovirus as the vector to carry the gene. Problem is, retrovirus can incorporate their own genetic information into the human genome, causing unintended changes that can give rise to cancer. It is a known risk factor of gammaretroviral vector-based gene therapy and has been described as one of the important potential risks for Strimvelis in its EU approval.

Besides Strimvelis, Orchard is also developing OTL-101, which uses a lentivirus to insert a functional copy of the ADA gene into a patients cells. The drug is currently undergoing a registrational trial and has won breakthrough and orphan drug designations from the FDA.

All the gene therapy candidates in Orchards pipeline use lentiviral vectors that have been specifically designed to avoid insertional oncogenesis after administration, Orchard said, adding that no dangerous gene insertion has been reported around lentiviral vector-based stem cell gene therapy in any indication.

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Gene therapy may be able to restore vision – Genetic Literacy Project

Tuesday, November 3rd, 2020

In a normal eye, opsins are expressed by the rod and cone photoreceptors in the retina. When activated by light, the photoreceptors pulse and send a signal through other retinal neurons, the optic nerve, and on to neurons in the brain.

A variety of common eye diseases, including age-related macular degeneration and retinitis pigmentosa, damage the photoreceptors, impairing vision. But while the photoreceptors may no longer fully function, other retinal neurons (bipolar cells) remain intact. The team identified a way for bipolar cells to take on some of the work of damaged photoreceptors.

The beauty of our strategy is its simplicity, said Samarendra Mohanty, PhD, Nanoscope founder and corresponding author of a report on the mouse study that appears today in Nature Gene Therapy. Bipolar cells are downstream from the photoreceptors, so when the MCO1 opsin gene is added to bipolar cells in a retina with nonfunctioning photoreceptors, light sensitivity is restored.

The strategy could overcome challenges plagued by other approaches to retinal regeneration, according to the researchers. Gene replacement therapy has thus far worked principally in rare diseases that leave photoreceptors intact.

Under a best-case scenario, the therapy could help patients achieve 20/60 vision, according to the researchers; however, no one knows how the restored vision will compare to normal vision.

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Avantor Discusses Cell & Gene Therapy Production Trends Through the Lens of COVID-19 – PRNewswire

Tuesday, November 3rd, 2020

Avantor executives joined other industry leaders at virtual Cell & Gene Therapy Bioprocessing & Commercialization Conference

Panels discussed critical topics including process efficiency and scaling solutions

RADNOR, Pa., Nov. 2, 2020 /PRNewswire/ --Executives from Avantor Inc. (NYSE: AVTR), a leading global provider of mission-critical products and services to customers in the life sciences, advanced technologies and applied materials industries, recently provided expert insight at the Cell & Gene Therapy Bioprocessing & Commercialization Virtual Conference. Cell and gene therapy (C>) are two of the most revolutionary applications driving the biopharmaceutical industry.

In separate panel discussions with other global industry leaders, Dr. Ger Brophy, EVP, Biopharma Production at Avantor and Claudia Berrn, SVP, Business Development and Commercial Operations at Avantor, each addressed novel considerations and challenges facing the biopharmaceutical industry, including the impact of COVID-19. They highlighted innovation to help drive the creation and commercialization of life-changing, personalized C> treatments.

In a panel on the evolution of C> and the path toward scalability and manufacturability of these treatments, Dr. Brophy gave his perspective on this growing need for the bioprocessing industry.

"Genuine progress is being made in the long-standing battle to effectively treat and control disease, and cell & gene therapies will only continue to unlock new frontiers in medicine," said Dr. Brophy. "We're beginning to see more clearly the issues that need to be addressed, and know that if products are to scale and become more accessible to patients worldwide, there needs to be efficiency in operations. There is an absolute requirement for automation, both to reduce variability and to generate process efficiencies. At Avantor, we are ready to make the essential materials and technologies available to companies leading the charge and treating patients. Innovation and agility are central to how we're partnering with the industry to resolve these issues."

On a separate panel, Ms. Berrn highlighted how the industry is navigating the challenges and impact of the global pandemic, from the fragmentation of supply chains to clinical trial disruption.

"In this critical moment for the industry and the world, it is more important than ever for leaders in the cell & gene therapy community to come together to strategize and share ideas," said Ms. Berrn. "Avantor is actively working with the world's leading pharmaceutical and biotechnology companies to accelerate the production of novel treatments. Collectively, our goal is to mitigate any challenges in the process of taking a groundbreaking treatment from its initial scientific discovery to delivery in treating patients."

Avantor provides products for biopharma production workflows, including cell and gene therapy offering. Search 'Avantor biopharma' in your browser.

About AvantorAvantor, a Fortune 500 company, is a leading global provider of mission-critical products and services to customers in the biopharma, healthcare, education & government, and advanced technologies & applied materials industries. Our portfolio is used in virtually every stage of the most important research, development and production activities in the industries we serve. One of our greatest strengths comes from having a global infrastructure that is strategically located to support the needs of our customers. Our global footprint enables us to serve more than 225,000 customer locations and gives us extensive access to research laboratories and scientists in more than 180 countries.We set science in motion to create a better world. For information, visit avantorsciences.com and find us on LinkedIn, Twitter and Facebook.

Robert DonohoeSenior Director, Corporate CommunicationsAvantorM: 484-688-4730[emailprotected]

Source: Avantor and Financial News

SOURCE Avantor and Financial News

http://avantorsciences.com

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Avantor Discusses Cell & Gene Therapy Production Trends Through the Lens of COVID-19 - PRNewswire

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Euro Convergence: Early consultation critical in overcoming gene therapy hurdles – Regulatory Focus

Tuesday, November 3rd, 2020

When it comes to advancing gene therapy products in the United States and Europe, the key is early consultation with regulators and identification of potential problems, experts said at the Euro Convergence 2020 virtual meeting.

It is better to do it earlier and establish a collaborative and educational approach to discussions, advised Mridula Shukla, director of global regulatory affairs at Arcutis Biotherapeutics in Palo Alto, Calif. Patrick Celis, PhD, head of the scientific secretariat of the Committee for Advanced Therapies at the European Medicines Agency (EMA), echoed that advice.

Please talk to EMA and National Authorities early and frequently so that we can help you as much as possible to get these innovative products to the patients as soon as possible, Celis said.

There are currently more than 10 gene therapy products approved globally and momentum has been building in this area since around 2015, Shukla said.

Gene therapy seems to have potential as it offers the possibility of an actual cure instead of chronic treatment for rare diseases, hereditary diseases and cancer, Shukla said.

Despite the promise of the therapies, more than half of gene therapy clinical trials are in phase 1. Shukla said this is due to a combination of factors including the complexity of the therapies, the rare indications, the high price of the therapies and reimbursement issues, unique manufacturing and supply chain challenges and controversies surrounding the ethnics of the techniques.

There is guidance from the US Food and Drug Administration (FDA) to assist developers. In July 2018, FDA issued draft guidance on Human Gene Therapy for Rare Diseases, which was finalized in January 2020. The document offers advice on some common manufacturing challenges, including encouraging companies to establish critical process parameters (CPP) early on and establish and qualify a potency assay prior to conducting clinical trials.

The FDA recommends communication with its Office of Tissues and Advanced Therapies early in product development, even before submission of an investigational new drug (IND) application. The agency also offers an Initial Targeted Engagement for Regulatory Advice on CBER Products (INTERACT) meeting, which can be used to discuss specific product issues, Shukla said.

Overall, the recommended approach is to front-load as much development as possible, preferably prior to Phase 1, and to hold early interactions and frequent interactions with FDA or EMA, Shukla said.

In the US, there are also multiple options for expedited review of products, including:

These expedited processes are similar to programs available for advanced therapy medicinal products (ATMPs) in Europe, such as the priority medicines (PRIME) scheme, which offers early consultation and the potential for accelerated assessment.

Since 2009, the EMA has approved 17 ATMPs, of which 10 are gene therapy products, Celis said.

The common issues that come up with ATMP applications include a lack of experience with commercial manufacturing processes, finding a relevant potency assay, ensuring product consistency and challenges with consistent starting materials, according to Celis. Other problems that can lead to delays in authorization include a lack of randomization in trial design, an indication that doesnt reflect the patients in the trial, and limited data on safety and durability of response.

The nature of gene therapies and the small number of patients in clinical trials mean that sponsors often have difficulty showing long-term safety and efficacy in a robust patient population. While regulators dont expect the same level of data as with other traditional medicines, there is a need for early planning of post-authorization studies, Celis said.

The EMA is asking for a detailed post-authorization plan that can include follow-up of patients in the clinical trials, post-marketing trials, and post-authorization registry-based studies.

If a sponsor is using a registry, Celis said it is better to go with an established disease registry and to contact the registry early to find out what data is collected. Additionally, registries may not be a good basis for safety reporting, Celis said.

There are support mechanisms in place for ATMP developers including scientific advice, a meeting with the EMAs Innovation Task Force, and the PRIME program for medicines addressing unmet needs.

We can help, Celis said. Make sure that you make best use of what we can do.

For sponsors looking to market gene therapies globally, Celis said that the EMA and FDA are in frequent contact and are seeing many of the same issues as they review products. He encouraged developers to make use of this interaction between European and US regulators either formally, through parallel scientific advice, or informally.

Euro Convergence 2020

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Euro Convergence: Early consultation critical in overcoming gene therapy hurdles - Regulatory Focus

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ELEVECTA for AAV gene therapy vector production – Bioprocess Insider – BioProcess Insider

Tuesday, November 3rd, 2020

Pharmaceutical companies with gene therapies in their pipeline need to establish scalable production processes that can keep up with the increasing demand in terms of volume, quality and robustness, says CEVEC.

Gene therapies are seen as one of the most promising approach to treat life-threatening diseases with no therapeutic options and high medical needs. In addition, gene therapies are now advancing also into indications with larger numbers of patients, including Alzheimers and Parkinsons, thereby dramatically increasing the demand for viral vectors. As a result, pharmaceutical companies with gene therapies in their pipeline need to establish scalable production processes that can keep up with the increasing demand in terms of volume, quality and robustness.

CEVEC Pharmaceuticals, a German biotechnology company focusing on viral vector technologies, has developed and commercially launched ELEVECTA, a unique technology that enables fully scalable AAV manufacturing providing unprecedented process robustness and consistency in vector yield and quality.

One of the major bottlenecks in AAV vector production is volumes. Standard technologies have proven to be very hard or impossible to scale, as they are often based on adherent cell lines or require transient transfections or helper viruses.

While suspension cell lines are getting more and more common now, most processes still rely on transient transfection to bring the different AAV genes into the producer cells. The typical transient transfection process is complex and requires three different GMP-grade plasmids driving up the production costs. This is where ELEVECTA stableAAV producer cell lines come into play.

Figure 1: ELEVECTA, a revolutionary technology that enables fully scalable, stable viral vector manufacturing with unprecedented process robustness and consistency in vector yield and quality.

Based on a stable human suspension cell line, ELEVECTA enables full scalability in all types and formats of suspension bioreactors, from laboratory to commercial scale. ELEVECTA Producer Cell Lines reach titers of 1*1014 vg/l and more during a typical single-use suspension bioreactor run. CEVEC has now successfully scaled-up from 15 ml to 50 L bioreactors in a single step without any productivity loss, demonstrating the scalability and robustness of the process.

ELEVECTA Producer Cells contain all components required for AAV stably integrated into the cell. There is no need for cGMP plasmids, special transfection reagents and difficult-to-scale-up transfection protocols. Just one cell line which, after propagation and expansion to the desired cell titers, produces AAV vectors at high yields a process that resembles well-established production methods for recombinant proteins and monoclonal antibodies.

Figure 2: Cryo TEM picture from AAV particles derived from ELEVECTA Producer Cell Lines. The viral particles display an approximate size of 22 nm. Full particles are indicated by a black arrow, empty particles by a white arrow

AAV vectors can have different surface structures, so called serotypes, which in nature allow the virus to target specific cells in a body. For gene therapy applications viral serotypes can further be engineered to improve their tissue-specificity and safety. Every ELEVECTA producer cell line is custom-made and employs a specific natural or engineered capsid gene and the therapeutic gene of interest.

With this newly introduced technology, CEVEC is the leading technology provider for stable AAV producer cell lines. Its inherent scalability, process robustness and consistency in yield and quality provide the features that are needed to pave the way for the success of modern gene therapies in a broad range of indications.

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ELEVECTA for AAV gene therapy vector production - Bioprocess Insider - BioProcess Insider

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