StemCell Therapy

The "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" report has been added to ResearchAndMarkets.com's offering.

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included in this Europe Cell and Gene Therapy Market Report

The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 2021-2026.

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually.

Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand. In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes.

There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

Europe Cell and Gene Therapy Market Segmentation

The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields.

The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

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Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies.

While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures. Hospitals are growing at a significant rate due to the increasing target population in Europe.

Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patient's quality of life. Cell and gene therapies for certain types of cancers have been promising results.

The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving. CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer.

Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

KEY QUESTIONS ANSWERED

1. What is the Europe cell and gene therapy market size and growth rate during the forecast period?

2. What are the factors driving the demand for CAR-T therapy in the European region?

3. How are strategic acquisitions aiding in market growth of cell and gene therapy products?

4. Which segments are expected to generate the highest revenues during the forecast period?

5. Who are the leading vendors in the European cell and gene therapy market?

INSIGHTS BY VENDORS

Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors.

While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

Prominent Vendors

Novartis

Spark Therapeutics

Amgen

Gilead Sciences

Organogenesis

Other Prominent Vendors

APAC Biotech

AVITA Medical

bluebird bio

CHIESI Farmaceutici

CollPlant

CO.DON

Human Stem Cells Institute PJSC (HSCI)

Medipost

NuVasive

Nipro

Orchard Therapeutics

RMS Regenerative Medical System

Orthocell

Osiris Therapeutics

Sibino GeneTech

Shanghai Sunway Biotech

Takeda Pharmaceutical Company

Terumo

Vericel

Emerging Investigational Vendors In Europe

Adaptimmune Therapeutics

AgenTus Therapeutics

Autolus

Cellecits

Celyad

CombiGene

EUKARYS

Freeline Therapeutics

Innoskel

PsiOxus Therapeutics Ltd

SparingVision

uniQure

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Europe Cell and Gene Therapy Market Industry Outlook and Forecast Report 2021-2026 with Data-driven Insights on the Impact of COVID-19 -...

Close to a year after Maryland-based CDMO Vigene Biosciences cut the ribbon on its headquarters, spiking demand for cell and gene therapy has prompted the company to lay out a major manufacturing upgrade in its home state.

Vigene is picking up a lease for 52,000-square-feet of manufacturing space in Montgomery County, Maryland, situated near its existing headquarters in Rockville. The expansion is set to bring the company's total lab and production space up to 110,000 square feet and, by 2025, will see up to 245 new hires join Vigene's current workforce of 125.

The new facility, located at 14200 Shady Grove Road, will complement existing R&D and manufacturing operations at Vigene's home base as the company faces growing demand for its cell and gene therapy products. Vigene's expansion has snared some financial perks from the state, too, including a $1,225,000 loan from the Maryland Department of Commerce, which is contingent on job creation and capital investment.

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Join patients and their families, legislators, industry experts, advocates and sponsors to discuss education initiatives, recent advancements and the future promise of cell and gene medicine, and current patient experiences with these therapies.

RELATED: Cognate beefs up cell, gene therapy manufacturing with new plants in U.S., EU

The company is keeping its own spending on the site under wraps, Jeffrey Hung, Ph.D., chief commercial officer of Vigene, said over email. "It suffices to say that we are going to invest heavily on the facility to qualify and commission it for commercial production purpose," he added.

With the new site, Vigene will add five more GMP suites to the 10 it operates now, Hung said. Specifically, the company plans to commission and set up two 2,000-liter single-use bioreactor suites, where upstream and downstream production trains will be located on the same floor for commercial viral vector production. Another floor will house multiple large-scale fermenters for commercial plasma production, he said.

Formed in 2012, Vigene specializes in gene therapies for patients with cancers and serious genetic disorders. It develops, manufactures and distributes adeno-associated viruses, lentiviruses, retroviruses, adenoviruses and plasmid viral vectors for gene delivery.

RELATED: Fujifilm continues CMDO expansion spree with $76M in funding for new Boston site

The company has checked into the COVID-19 fight, too, signing on to produce clinical materials for Maryland compatriot Altimmune's nasal vaccine candidate. Vigene in July agreed to churn out both drug substance and drug product for studies on the vaccine, which registered for a phase 1 trial in late December.

On Dec. 23, Altimmune revealed the FDA had slapped the investigational new drug application for its vaccine, AdCOVID, with a clinical hold, citing the need for protocol modifications and additional chemistry, manufacturing and control data. The company responded to the hold and, at the time, said it didn't expect the move to significantly disrupt its clinical timeline.

Altimmune has also added Swiss CDMO Lonza as a production partner on its nasal vaccine, and it previously set the goal to crank out at least 100 million AdCOVID doses in 2021.

Meanwhile, Vigene's expansion comes shortly after the christening of its Rockville HQ. It was just a year ago that we cut the ribbon at Vigenes new custom-built headquarters and already the growing demand for its gene and cellular therapy products requires additional physical expansion, Benjamin Wu, CEO and president of the company, said in a release.

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CDMO Vigene plots cell and gene therapy manufacturing expansion, adding 245 new jobs along the way - FiercePharma

LONDON and LAUPHEIM, Germany, Feb. 11, 2021 (GLOBE NEWSWIRE) -- The Cell and Gene Therapy Catapult (CGT Catapult), an independent centre of excellence in innovation advancing the UKs cell and gene therapy industry, and Rentschler Biopharma SE, a leading global contract development and manufacturing organisation (CDMO) for biopharmaceuticals, have announced today that Rentschler Biopharma will establish their manufacturing capability in Advanced Therapy Medicinal Products (ATMPs), including Adeno-Associated Virus (AAV) Vectors for clinical trial supply, at the CGT Catapult site in Stevenage.

Under the terms of the agreement, Rentschler Biopharma will make a significant investment at the site over the next five years to set up their manufacturing capabilities, benefitting from the expertise and unique collaborative model provided by the CGT Catapult. The companys investment is expected to make a major contribution to meeting the demand from UK and international researchers for suitable manufacturing capability. This development will further strengthen the UK ecosystem through the addition of Rentschler Biopharmas more than 40 years of experience and solid reputation in the development and manufacturing of biologics for both clinical and commercial supply. The company will leverage the CGT Catapults expertise in ATMP manufacturing setup and technology development, as well as the cell and gene therapy cluster and ecosystem that has developed around Stevenage and across the UK.

Dr. Frank Mathias, CEO of Rentschler Biopharma, said:We are excited to take this next big step in our evolution and address the growing industry demand for ATMP manufacturing capacity and viral vector supply. With the largest industry cluster for cell and gene therapies outside the US, the UK is an ideal location for us to establish our Center of Excellence for cell and gene therapy. We look forward to working with the CGT Catapult as we invest in this growing field. They are well established in this important market, enabling us to immediately tap into the organisations network and utilisethe UKs strong expertise and supply chain in cell and gene therapy manufacturing.

Matthew Durdy, CEO of the Cell and Gene Therapy Catapult, commented:We are very pleased that Rentschler Biopharma, a global CDMO, has chosen to build their ATMP capacity in the UK, bringing in their expertise and investment. This will build new capacity to benefit the international ATMP supply chain and meet growing academic and commercial demand across the industry. As more companies from around the globe come to the UK, it demonstrates and enhances the attractiveness of its cell and gene therapy ecosystem as a place to develop new technologies and capabilities.

The investment in the UK cell and gene therapy industry announced today is expected to further accelerate the development of the vital infrastructure and skilled jobs needed to meet the rising demand for manufacturing capacity in the UK and globally, as well as streamline the supply chain for these advanced therapies. Currently, 27% of European ATMP companies are operating in the UK, and there are more than 90 advanced therapy developers. The last year has also seen a 50% increase in the number of ATMP clinical trials being run in the UK, accounting for 12% of global ATMP clinical trials, and these numbers are predicted to increase further.

The CGT Catapult manufacturing centre has been backed by over 75m of funding, including investment from the UK Governments Industrial Strategy Challenge Fund, the Department for Business, Energy and Industrial Strategy, Innovate UK and from the European Regional Development Fund. Since it was announced, there has been over 1.1bn of investment in the ATMP industry in its vicinity.

About Rentschler Biopharma SE

Rentschler Biopharma is a leading contract development and manufacturing organization (CDMO), focused exclusively on client projects. The company offers process development and manufacturing of biopharmaceuticals as well as related consulting activities, including project management and regulatory support. Rentschler Biopharma's high quality is proven by its long-standing experience and excellence as a solution partner for its clients. A high-level quality management system, a well-established operational excellence philosophy and advanced technologies ensure product quality and productivity at each development and manufacturing step. In order to offer best-in-class formulation development along the biopharmaceutical value chain, the company has entered into a strategic alliance with Leukocare AG. Rentschler Biopharma is a family-owned company with about 1,000 employees, headquartered in Laupheim, Germany, with a second site in Milford, MA, USA. In Stevenage, UK, Rentschler Biopharma launched a company dedicated to cell and gene therapies, Rentschler ATMP Ltd.

For further information, please visit http://www.rentschler-biopharma.com. Follow Rentschler Biopharma on LinkedIn and Facebook.

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 330 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK.

For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

About the European Regional Development Fund

This project has received 3.36m of funding from the England European Regional Development Fund as part of the European Structural and Investment Funds Growth Programme 2014-2020. The Ministry of Housing, Communities and Local Government (and in London the intermediate body Greater London Authority) is the Managing Authority for European Regional Development Fund. Established by the European Union, the European Regional Development Fund helps local areas stimulate their economic development by investing in projects which will support innovation, businesses, create jobs and local community regenerations. For more information visit https://www.gov.uk/european-growth-funding.

About the Industrial Strategy Challenge Fund

This project has received 12m of funding from the Industrial Strategy Challenge Fund, part of the governments modern Industrial Strategy. The Industrial Strategy Challenge Fund is a four-year, 1 billion investment in cutting-edge technology designed to create jobs and improve living standards, built on guidance from business and the academic community. Healthcare and Medicine is one of three core areas for investment under the programme.

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Rentschler Biopharma to build new cell and gene therapy capabilities in the UK - BioSpace

WhiteLab Genomics, a specialist in artificial intelligence applied to gene and cell therapies, has signed a partnership agreement with Genethon, a pioneering research center in the field of gene therapy.The alliance will harness the power of artificial intelligence to accelerate development of innovative gene therapies.

As part of this partnership, Genethon teams will use WhiteLab Genomics CatalystTM platform to develop new capsids, or vectors, which are essential components for gene therapy products.

While several gene therapy products have already obtained market authorization for the treatment of rare and common diseases, which demonstrates the efficacy of this approach for conditions considered to be incurable, development of these complex therapies continues to face major scientific and technical hurdles. Many vectors used in gene therapy are derivatives of adeno-associated viruses (AAV). Their use has limitations: natural immunization of 30% to 40% of the population and difficulty targeting a specific tissue. As a result, extremely large quantities of vectors are necessary. In this context, the use of artificial intelligence solutions stands out as a deciding factor to overcome these obstacles and produce optimized vectors that better target the relevant tissues, thus making it possible to inject smaller quantities of product while maximizing the effect of the therapy.

Turning to AI for faster development of optimized vectors

Genethons teams will use WhiteLab Genomics CatalystTM platform to accelerate select research programs.

Thanks to its Machine Learning algorithms, the WhiteLab Genomics platform will help researchers develop next-generation gene therapy vectors, with a view to enhancing their precision with regard to the tissues to be treated while reducing their immunogenic qualities.

"The tools developed by WhiteLab will make it possible for us to review thousands of sequences and devise new and innovative combinations. We aim to develop a new generation of more specific AAV vectors, contributing to the emergence of original treatments for neuromuscular disorders," said Dr. Ronzitti, who is managing the collaboration for Genethon.

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"We are thrilled to be working together with worldwide trailblazers and experts in the area of gene therapy," stated David Del Bourgo, CEO and co-founder of WhiteLab Genomics. "France is a prime source of innovation in this field, and we look forward to helping research teams, in France and abroad, to make practical use of these extremely complex biological datasets, while also providing assistance to accelerate the development of optimized products."

About White Lab Genomics

Founded in 2019 by David Del Bourgo, Julien Cottineau and Lucia Cinque, WhiteLab Genomics is a French start-up specializing in artificial intelligence solutions dedicated to biotherapies, such as gene and cell therapies. The companys proprietary technology allows for multi-parameter analysis of complex biological data to optimize these treatments while reducing development costs. WhiteLab Genomics provides this unique technology to its clients via the Catalyst platform, available in SaaS mode. Today, the start-up has locations at the Evry Gnopole Frances first biocluster and at Station F. WhiteLab Genomics was recently ranked among Station Fs "Future 40," an index of the 40 most promising companies within Europes largest start-up incubator. https://www.whitelabgx.com

About Genethon

Created by AFM-Telethon, Genethon is a non-profit research and development center dedicated to creating gene therapy products for rare diseases, from initial research to clinical validation. Genethon has several programs underway, in clinical, pre-clinical and research phases, to treat rare muscular, blood, immune system and liver disorders. Today, a product incorporating technologies developed thanks to Genethons pioneering research is available on the market in the United States, Europe and Japan to treat spinal muscular atrophy. Ten other products created through Genethon R&D, alone or in collaboration with partners, are at the clinical trial stage, while many more are slated to begin clinical trials in 2021 and 2022. Genethon.fr

View source version on businesswire.com: https://www.businesswire.com/news/home/20210210005707/en/

Contacts

Press

Beyond Communication Najette Chaib nchaib@beyondcom.fr +33 6 18 38 11 14

WhiteLab Genomics David Del Bourgo - contact@whitelabgx.com

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Genethon and WhiteLab Genomics Join Forces to Enhance Gene Therapy Through Artificial Intelligence - Yahoo Finance

TipRanks

We are indeed living in interesting times and in many ways, thats a good thing. Take the automotive industry, for example. Technology is changing a rapid pace, and when it settles, it will dramatically change the way we drive. In 2030, our concept of car will likely be unrecognizable to drivers from 1980. The biggest changes are coming from power systems and artificial intelligence. AI will bring autonomous tech to our cars, making self-driving vehicles a reality. But the power systems changes will hit us first. In fact, electric-drive vehicles are already on our roads, and electric vehicle (EV) companies are proliferating rapidly. For the moment, there are several roads to potential success in the EV market. Companies are working to position themselves as leaders in battery tech, or electric power trains, or to maximize their range and performance per charge. Its a fact-paced industry environment, offering both opportunity and excitement for investors. Smart investors will look for companies capable of meeting scaling demands, once they have settled on marketable models. Investment firm Morgan Stanley has been watching the EV industry, seeking out innovative new design and production companies that are positioning themselves for gains as the market matures. The firms automotive analyst, Adam Jonas, has selected two stocks that investors should seriously consider buying into, saying As we survey the EV/battery startup landscape, we are prioritizing highly differentiated technology and/or business models with a path to scale at a reasonable level of risk. Opening up the TipRanks database, weve pulled up the details on both of Jonas picks to see whether they could be a good fit for your portfolio. Fisker (FSR) First up, Fisker, is based in Southern California, the epicenter of so much of our ground-breaking tech industries. Fiskers focus is on solid-state battery tech, a growing alternative to the lithium-ion batteries that most EVs depend on. While more expensive that the older lithium-based systems, solid state batteries are safer and offer higher energy densities. Fisker has been busy patenting its moves into solid-state batteries, a sound strategy to lock in its advances in this field. For EVs, solid-state batteries offer faster charging times, longer range per charge, and potentially lower battery weight all important factors in vehicle performance. Every car company needs a flagship model, and Fisker has the Ocean an EV SUV with a mid-range price ($37,499) and a long-range power system (up to 300 miles). The vehicle features stylish design and room mounted solar panels to supplement the charging system, and is scheduled to enter serial production for the markets in 2022. The stylish design reflects the sensibilities of the companys founder, Henrik Fisker, known for his work on the BMW Z8 and the Aston Martin DB9. Fisker entered the public markets through a SPAC merger agreement last fall. Since completing the SPAC transaction on October 29, shares in FSR are up 112%. Morgan Stanleys Jonas is impressed by this company, describing the value proposition of Fisker as design, time to market, clean sheet user experience and management expertise, and saying that the 4Q22 launch schedule for the Ocean is likely to be met. Fisker is specifically targeting the personal owned/passenger car business as opposed to commercial oriented end markets, where emotive design and user experience matter more. Additionally, the company wants to create an all-digital experience from the website to the app to the HMI in the car and continued customer engagement through its flexible lease product, Jonas added. In line with his upbeat outlook on the company (and the car), Jonas rates Fisker an Overweight (i.e. Buy), and sets a $27 price target suggesting an upside of 42% for the coming year. (To watch Jonas track record, click here) Turning to the TipRanks data, weve found that Wall Streets analysts hold a range of views on Fisker. The stock has a Moderate Buy analyst consensus rating, based on 7 reviews, including 4 Buys, 2 Holds, and 1 Sell. Shares are currently priced at $18.99, and the $21.20 average price target implies a one-year upside of ~12%. (See FSR stock analysis on TipRanks) QuantumScape (QS) Where Fisker is working on solid-state batteries in the context of vehicle production, QuantumScape is setting itself up as a leader in EV battery technology and a potential supplier of the next generation of battery and power systems for the EV market. QuantumScape designs and builds solid-state lithium-metal batteries, the highest energy density battery system currently available. The key advantages of the technology are in safety, lifespan, and charging times. Solid-state batteries are non-flammable; they last longer than lithium-ion batteries, with less capacity loss at the anode interface; and their composition allows faster charging, of 15 minutes or less to reach 80% capacity. QuantumScape is betting that these advantages will outweigh the technologys current higher cost, and create a new standard in EV power systems. The companys strongest tie to the EV production field is its connection with Volkswagen. The German auto giant put $100 million into QuantumScape in 2018, and an additional $200 million in 2020. The two companies are using their partnership to prepare for mass-scale development and production of solid-state batteries. Like Fisker, QuantumScape went public through a SPAC agreement late last year. The agreement, which closed on November 27, put the QS ticker in the public markets where it promptly surged above $130 per share. While the stock has since slipped, it remains up 47% from its NYSE opening. For Morgan Stanleys Jonas, involvement in QS stock comes with high risk, but also high potential reward. In fact, the analyst calls it, "The Biotech of Battery Development." "We believe their solid state technology addresses a very big impediment in battery science (energy density) that, if successful, can create extremely high value to a wide range of customers in the auto industry and beyond. The risks of moving from a single layer cell to a production car are high, but we think these are balanced by the commercial potential and the role of Volkswagen to help underwrite the early manufacturing ramp," Jonas explained. Noting that QS is a stock for the long haul, Jonas rates the shares an Overweight (i.e. Buy), and his $70 price target indicates confidence in an upside of 28% for one-year time horizon. Granted, not everyone is as enthusiastic about QS as Morgan Stanly. QS's Hold consensus rating is based on an even split between Buy, Hold, and Sell reviews. The shares are priced at $54.64 and their recent appreciation has pushed them well above the $46.67 average price target. (See QS stock analysis on TipRanks) To find good ideas for EV stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analyst. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Global Gene Therapy Partnering Deals Terms and Agreements Directory 2010-2020: Company A-Z, Deal Value, Phase of Development, Deal Type, and Therapy...

PARIS--(BUSINESS WIRE)--Regulatory News:

Lysogene (FR0013233475 LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announces that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for LYS-GM101, the companys gene therapy candidate for the treatment of GM1 gangliosidosis, a serious, pediatric, life threatening disease. LYS-GM101 builds on Lysogenes extensive experience in direct to CNS adeno-associated viral vector (AAV)-based gene therapy clinical development.

The IND clearance follows the recent clinical trial authorization granted by the MHRA in the United Kingdom. Lysogene intends to initiate its global, multi-center, single-arm, two-stage, adaptive-design clinical trial of LYS-GM101 in patients with a diagnosis of early or late infantile GM1 gangliosidosis. The clinical trial will include a safety phase and a confirmatory efficacy phase. The company intends to dose a total of 16 patients, with dosage of the first patient expected in the first half of 2021.

We are very pleased to receive this IND clearance for LYS-GM101 which completes the MHRA approval received a few weeks ago. It represents a major milestone that marks our second CNS gene-therapy program to enter into a global clinical trial said Karen Aiach, Founder Chairman and Chief Executive Officer of Lysogene. This IND clearance once again demonstrates our quality and timely execution, and our strong determination to bring new therapeutic solutions for diseases that currently have no treatment.

Christine Waggoner, President and Co-Founder of Cure GM1 Foundation added: Children with GM1 gangliosidosis represent a clear unmet medical need and we are thrilled to see a new therapeutic option entering the clinic, as it brings tremendous hope to families and the entire GM1 gangliosidosis community.

LYS-GM101 (adeno-associated viral vector serotype rh.10 expressing beta-galactosidase) received orphan drug designation for the treatment of GM1 gangliosidosis in the European Union and in the US in 2017, as well as Rare Pediatric Disease designation in the US in 2016.

Leading international gene therapy and Lysosomal Storage Disease centers plan to participate in the clinical trial (NCT04273269).

Lysogene is also funding a GM1 gangliosidosis natural history study being conducted by Casimir Trials to collect prospective and/or retrospective videos of children doing certain everyday tasks and behaviors in infantile and juvenile GM1 gangliosidosis (NCT04310163).

About LysogeneLysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS). The Company has built a unique capability to enable delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing. An adaptative clinical trial in GM1 gangliosidosis is in preparation. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism. http://www.lysogene.com.

Forward Looking StatementThis press release may contain certain forward-looking statements, especially on the Companys progress of its clinical trials and cash runway. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice, (ii) factors beyond the Companys control, (iii) clinical trial results, (iv) increased manufacturing costs and (v) potential claims on its products. These statements may include, without limitation, any statements preceded by, followed by or including words such as target, believe, expect, aim, intend, may, anticipate, estimate, plan, objective, project, will, can have, likely, should, would, could and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Companys control that could cause the Companys actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. A further list and description of these risks, uncertainties and other risks can be found in the Companys regulatory filings with the French Autorit des Marchs Financiers, including in the 2019 universal registration document, registered with the French Markets Authorities on April 30, 2020, under number D.20-0427, and future filings and reports by the Company. Furthermore, these forward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.

This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede.

Excerpt from:
Lysogene Receives FDA Clearance of Investigational New Drug Application to Initiate the Gene Therapy Clinical Trial in the US with LYS-GM101 for the...

DUBLIN--(BUSINESS WIRE)--The "Adeno-Associated Virus (AAV) Vectors in Gene Therapy - Market Insight, Epidemiology and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.

This report delivers an in-depth understanding of the AAV Vector Based Gene Therapy, historical and forecasted epidemiology as well as the AAV Vector Based Gene Therapy market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan.

The AAV Vector Based Gene Therapy market report provides emerging drugs, AAV Vector Based Gene Therapy market share of the individual diseases, current and forecasted AAV Vector Based Gene Therapy market size from 2017 to 2030 segmented by seven major markets. The Report also covers current AAV Vector Based Gene Therapy market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses underlying potential of the market.

The AAV Vector Based Gene Therapy epidemiology division provides the insights about historical and current AAV Vector Based Gene Therapy patient pool and forecasted trend for each seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of The report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

The disease epidemiology covered in the report provides historical as well as forecasted AAV Gene Therapies epidemiology [segmented as Total cases of AAV Gene Therapy Targeted Indications, Total diagnosed indication-specific cases, Number of Patients Eligible for AAV Gene Therapy and Total treated cases] scenario of AAV Gene Therapy in the 7MM covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2017 to 2030.

Country Wise- AAV Vector Based Gene Therapy Epidemiology

Estimates show that the highest cases of AAV vector based gene therapy in the 7MM were in the United States, followed by Germany, Japan, France, the United kingdom, Italy, and Spain in 2017.

AAV vector based gene therapy Drug Chapters

Drug chapter segment of the AAV vector based gene therapy report encloses the detailed analysis of AAV vector based gene therapy marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the AAV vector based gene therapy clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Scope of the Report

AAV Vector Based Gene Therapy Report Insights

AAV Vector Based Gene Therapy Report Key Strengths

AAV Vector Based Gene Therapy Report Assessment

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Companies Mentioned

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Global Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market to 2030 - Insight, Epidemiology and Forecasts - ResearchAndMarkets.com - Business...

bluebird bios beta-thalassaemia gene therapy betibeglogene autotemcel (beti-cel) has not been recommended by the UKs National Institute of Health and Care Excellence (NICE) for use on the NHS.

Beti-cel, marketed as Zynteglo in Europe, is a gene therapy intended for the treatment of transfusion-dependent beta-thalassaemia (TDT) in people aged 12 years and older who do not have a beta0/beta0 genotype.

It is indicated for TDT patients when haematopoietic stem cell transplantation (HSCT) is appropriate but there is no suitable donor.

TDT is the most severe form of thalassaemia, a condition wherein an inherited faulty gene leads to the inability of the body to produce normally functioning haemoglobin.

People living with TDT require life-long blood transfusions every two to five weeks.

In its draft recommendations, NICE commented that the follow-up on people included in clinical trials of beti-cel was not very long, adding that the population included was small.

NICE also determined that there were uncertainties around the cost-effectiveness of beti-cel, with the estimate for the gene therapy considerably higher than what it usually deems an acceptable use of NHS resources.

We are extremely disappointed with NICES decision not to recommend betibeglogene autotemcel as a treatment option in the UK, said Romaine Maharaj, executive director, UK Thalassaemia Society.

We also feel disheartened that our patient experts were misquoted and used out of context and feel that NICE needs to rectify this. Having an option and the access to a potentially curable treatment is vital and should be offered to patients, she added.

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bluebird bio's beta-thalassaemia gene therapy rejected by NICE - PharmaTimes

Having raised $150 million, Orchard hopes to expand its therapy pipeline into larger indications and says its HSC approach is not limited to the vector it uses.

The firm has said the funding will also aid the launch of gene therapy Libmeldy in Europe. Last year, the firm received approval for the one-time treatment which is used to treat children with metachromatic leukodystrophy (MLD).

Though often viewed as a single technology, the delivery mechanism, the vector, and the type of cell modified in gene therapy can conjure different approaches. Libmeldy is an ex vivo Hematopoietic Stem Cell (HSC) gene therapy. The company told us HSCs are central to Orchards other products in its pipeline.

Image/iStock: CIPhotos

It also asserted that despite the use of a lentiviral vector, HSC is the facilitating technology in its gene therapy as it is not limited to the vector used.

We insert a working copy of the gene into the genome of HSCs, and once engrafted, these genetically modified cells can lead to multiple corrected cell types in the blood stream including white blood cells, red blood cells, platelets, and tissue macrophages, SVP, Leslie Meltzer told BioProcess Insider. Importantly, the progeny of HSCs can migrate into multiple organ systems including the brain and GI tract.

The firm uses a lentiviral vector where genes can be inserted, deleted, or modified. Orchard told us lentiviral vectors are an appealing choice because of its ability to stably integrate the genome and be passed on to all the progeny.

HSCs are particularly appealing because of their intrinsic ability to self-renew which means that these cells serve as the repository of stem cells is expected for the lifetime of the individual, said Meltzer.

To expand into larger indications, Chemistry Manufacturing and Controls (CMC) will be used to improve efficiency and the firm tout manufacturing as a critical step to transforming the capabilities of HSC.

We are focused on improving the HSC gene therapy manufacturing process through important technology innovations, including a scalable stable vector producing cell line, transduction enhancing compounds and a fully closed, automated drug product process.

With growing clinical data available, the firm is confident HSC gene therapy has the potential to make a durable impact in devastating disorders of the central nervous system.

Continue reading here:
Orchard Therapeutics talks the benefits of HSC therapy - BioProcess Insider

Patients with Bacillus Calmette-Guerin (BCG)unresponsive, non-muscle invasive bladder cancer (NMIBC) carcinoma in-situ (CIS) who were treated with N-803 (also known as ALT-803) added to BCG Vaccine experienced promising response rates and a tolerable safety profile, according to data presented at the 2021 Genitourinary Cancers Symposium.1

The findings, taken from cohort A of the phase 2/3 QUILT-3.032 trial (NCT03022825), showed that at a median follow-up of 10.7 months, 71% of evaluable patients (n = 51/72) achieved a complete response (CR) at any time (95% CI, 59%-81%), meeting the primary end point of the study.

Moreover, investigators reported a 56% probability of maintaining a CR with this approach at 12 months (95% CI, 38.8%-70.3%). Among responders, the estimated median duration of CR was 19.2 months (95% CI, 7.3not reached [NR]). Notably, the majority of patients, or 87.5%, (n = 70/80) have not progressed to radical cystectomy.

N-803 is an IL-15 superagonist antibody cytokine fusion protein with significant binding capacity to the IL-2R beta and gamma and increased half-life of the Fc receptor, Karim Chamie, MD, MSHS, an associate professor in residence of urology and Society of Urologic Oncology Fellowship Director at the University of California, Los Angeles, explained during a presentation on the findings. This results in significant accrual of T cells and natural killer cells without upregulation of regulatory T cells.

Results from the phase 1 trial examining N-803 plus BCG indicated that all patients (n = 9) with high-risk NMIBC experienced a durable CR or no recurrence. All patients were disease free at 24 months. Based on these data, the FDA granted N-803 a fast track therapy designation in December 2019; this led to the launch of the phase 2/3 trial to further examine this approach.2

To be eligible for enrollment on the trial, patients had to have histologically-confirmed BCG-unresponsive CIS with or without Ta or T1. Investigators defined BCG-unresponsive disease as persistent or recurrent CIS within 12 months of being treated with BCG; at least 5 of 6 doses of initial induction course with at least 2 of 3 doses of maintenance treatment or at least 2 of 6 doses of a second induction course were required.

Eighty patients were enrolled to cohort A and they received 50 mg of BCG in combination with 400 g of N-803 intravesically weekly x6 induction or single re-induction x3 plus maintenance.

The primary end point of the study is CR at any time with a lower bound 95% confidence interval of 20% or greater. At least 24 of the 80 patients enrolled needed to have experienced a CR to meet this end point. Key secondary end points included duration of CR and CR rate at 6 and 12 months. Other end points included progression-free survival, time to cystectomy, cystectomy rate, overall survival, and CR rate at any time per Central Pathology Review.

The mean age of patients enrolled on the study was 72.5 years, with 84% of patients aged 65 years or older. The majority of patients were male (86%), and most patients were white (90%). Eighty-two percent of patients had an ECOG performance status of 0, while 18% had a status of 1.

The median time from last previous BCG dose to study entry was 6.2 months and the mean time was 8.0 months. Additionally, the mean time from last prior BCG dose to first detected recurrence was 3.6 months with a median time of 2.7 months. Moreover, the median time from first detected recurrence to study entry was 2.3 months with a mean time of 4.6 months. The majority of patients had CIS at first recurrence (69%), with 21% had CIS/Ta and 9% had CIS/T1.

This is a heavily pretreated cohort with the median number of transurethral resection of the bladder tumor being 5.0 [and] a median number of prior BCG instillations being 16.2, said Chamie. One hundred percent of patients received prior BCG. In addition to that, 78% of patients received additional therapy with BCG, including checkpoint inhibitors [3%], chemotherapy [59%], interferon [13%], and vicinium [3%].

N-803 demonstrated promising results compared with other FDA-approved agents or investigational therapeutics in the space, such as pembrolizumab (Keytruda), which yielded a 41% CR rate at any time and a median duration of response of 16.2 months in responders.3 Similarly, nadofaragene firadenovec (rAd-IFNa/Syn3) elicited a 53% CR rate at any time with a median duration of response of 9.7 months.4

N-803 had a smaller cohort, however, the CR rate at any time was much higher, at 71% compared with 41% and 53% for pembrolizumab and nadofaragene, [respectively], Chamie reported. Our median duration was also favorable, although this must be taken within the context of a shorter median follow-up of 10.7 months. The cystectomy-free rate of 88% also compares favorably, with only 1 of 10 patients demonstrating extravesical disease.

The most common any-grade treatment-related adverse effects (TRAEs) included dysuria (18%), hematuria (15%), and pollakiuria (14%). Although grade 3 or higher TRAEs were observed during the study, with grade 3 arthralgia, myalgia, and extremity pain each reported in 1% of patients. Serious AEs were also rare but included cardiac disease (1%), hematuria (1%), and adenocarcinoma of the colon (1%).

Notably, no treatment-related serious AEs, no immune-related systemic AEs, and no grade 5 TRAEs were reported. Moreover, no TRAEs led to treatment discontinuation.

This is an administration profile that is quite familiar to urologists and favorable as an intravesical agent, concluded Chamie.

References:

1. Chamie K, Chang S, Gonzalgo M, et al. Phase II/III clinical results of IL-15RFb superagonist N-803 with BCG in BCG-unresponsive non-invasive bladder cancer (NMIBC) carcinoma in-situ (CIS) patients (cohort A). J Clin Oncol. 2021;39(suppl 6):510. doi:10.1200/JCO.2021.39.6_suppl.510

2. ImmunityBio granted FDA breakthrough therapy designation for N-803 IL-15 superagonist in non-muscle invasive bladder cancer. News release. BioSpace. December 4, 2019. Accessed February 13, 2021. http://bit.ly/2ZgGAvM

3. Balar AV, Kamat AM, Kilkarni GS, et al. Pembrolizumab (pembro) for the treatment of patients with Bacillus Calmette-Gurin (BCG) unresponsive, high-risk (HR) nonmuscle-invasive bladder cancer (NMIBC): over two years follow-up of KEYNOTE-057. J Clin Oncol. 2020;38(suppl 15):5041-5041. doi:10.1200/JCO.2020.38.15_suppl.5041

4. Boorjian SA, Alemozaffar M, Konety BR, et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Lancet. 2021;22(1):P107-117. doi:10.1016/S1470-2045(20)30540-4

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Addition of N-803 Results in Efficacy, Safety in NMIBC CIS Unresponsive to BCG - Cancer Network

A gene therapy costing 16 crore is the only shot of life for nearly 200 children with Spinal Muscular Atrophy (SMA) Type 1, a rare genetic disease, in Karnataka.

Last month, the therapy Zolgensma was offered free to a 14-month-old baby from Bhatkal (Uttara Kannada) who was the lucky winner of a lottery through a compassionate access programme by Novartis, the Swiss drugmaker. This lottery is held once in two weeks for SMA children across the world and doctors at Baptist Hospital, that has a dedicated Paediatric Neuromuscular Service, are hoping more children will benefit.

The therapy is a one-time infusion that takes about an hour, Ann Agnes Mathew, Consultant Paediatric Neurologist and Neuromascular Specialist, at Baptist Hospital told The Hindu. The therapy was approved by U.S. regulators in May 2019 and has since then turned into a miracle drug for this rare disorder that destroys a babys muscle control.

SMA is a disease caused by loss of nerve cells, which carry electrical signals from the brain to the muscles. The protein needed for this signalling is coded by a gene for which everyone has two copies - one from the mother and the other from the father. A child develops this disorder only if both the copies are faulty. Without treatment, this disease is ultimately fatal, said Dr. Mathew. The disease as it progresses, makes it extremely difficult for the babies to carry out basic activities like sitting up, lifting their head or swallowing milk.

Pointing out that the current treatment options range from medicines, which increase these proteins, to replacing the faulty gene, the doctor said, Zolgensma is a revolutionary treatment, which works by supplying a healthy copy of the faulty gene, which allows nerve cells to then start producing the needed protein. That halts deterioration of the nerve cells and allows the baby to develop more normally.

The drug has a 14-day shelf life and when it was sent from U.S. for the Bhatkal baby, it was stuck with customs for three days in mid-January making doctors jittery. Dr. Mathew said she had to personally meet the Customs officials to get it released. When we explained the situation to them, they immediately released it. Any further delay would have been risky. The parents have taken a house on rent and are staying near the hospital for follow up. The baby is doing fine now, she said.

Pointing out that 38 babies had succumbed to the rare disease in Karnataka in over one-and-a-half years, Dr. Mathew said most families have given up hope as they cannot afford the treatment.

The Paediatric Neuromuscular Service at Baptist Hospital is a pioneering centre in the country with a multidisciplinary team of a paediatric neurologist, paediatric neuromuscular specialist, paediatric geneticist, paediatric pulmonologist, paediatric intensivist, paediatric cardiologist and paediatric endocrinologist providing comprehensive care under one roof. This service is run in collaboration with Organisation for Rare Diseases India, a NGO.

A Bengaluru-based couple - Naveen Kumar and Jyothi - have taken to crowdfunding on ImpactGuru.com, a crowdfunding platform, to cover the cost of Zolgensma therapy for their 10-month-old baby Janish who was diagnosed with SMA.

Mr. Kumar, who works as an insurance surveyor and barely earns 30,000 a month, cannot afford the expensive treatment.

The couple were counting their babys milestones after his birth in February 2020. They caught his first smile and his first laugh but baby Janish never went past his first two milestones. The parents then rushed him to a pediatrician and from there the baby was referred to Baptist Hospital, said Dr. Ann Agnes Mathew, who has been treating the baby for the last five months.

Piyush Jain, co-founder and CEO, ImpactGuru.com, said over 22 lakh has been raised so far for baby Janish from over 1,500 donors.

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16 crore drug is the hope for SMA patients - The Hindu

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dyno Therapeutics, a biotech company applying artificial intelligence (AI) to gene therapy, today announced a publication in Nature Biotechnology that demonstrates the use of artificial intelligence to generate an unprecedented diversity of adeno-associated virus (AAV) capsids towards identifying functional variants capable of evading the immune system, a factor that is critical to enabling all patients to benefit from gene therapies. The research was conducted in collaboration with Google Research, Harvards Wyss Institute for Biologically Inspired Engineering and the Harvard Medical School laboratory of George M. Church, Ph.D., a Dyno scientific co-founder. The publication is entitled Deep diversification of an AAV capsid protein by machine learning.

It is estimated that up to 50-70% of the human population have pre-existing immunity to natural forms of the AAV vectors currently being using to deliver gene therapies. This immunity renders a large portion of patients ineligible to receive gene therapies which rely upon these capsids as the vector for delivery. Overcoming the challenge of pre-existing immunity to AAV vectors is therefore a major goal for the gene therapy field.

The approach described in the Nature Biotechnology paper opens a radically new frontier in capsid design. Our study clearly demonstrates the potential of machine learning to guide the design of diverse and functional sequence variants, far beyond what exists in nature, said Eric Kelsic, Ph.D., Dynos CEO and co-founder. We continue to expand and apply the power of artificial intelligence to design vectors that can not only overcome the problem of pre-existing immunity but also address the need for more effective and selective tissue targeting. At Dyno, we are making rapid progress to design novel AAV vectors that overcome the limitations of current vectors, improving treatments for more patients and expanding the number of diseases treatable with gene therapies.

The Nature Biotechnology paper describes the rapid production of a large library of distinct AAV capsid variants designed by machine learning models. Nearly 60% of the variants produced were determined to be viable, a significant increase over the typical yield of <1% using random mutagenesis, a standard method of generating diversity.

The more we change the AAV vector from how it looks naturally, the more likely we are to overcome the problem of pre-existing immunity, added Sam Sinai, Ph.D., Dyno co-founder and Machine Learning Team Lead. Key to solving this problem, however, is also ensuring that capsid variants remain viable for packaging the DNA payload. With conventional methods, this diversification is time- and resource-intensive, and results in a very low yield of viable capsids. In contrast, our approach allows us to rapidly unlock the full potential diversity of AAV capsids to develop improved gene therapies for a much larger number of patients.

This research builds upon previous work published in Science in which a complete landscape of single mutations around the AAV2 capsid was generated followed by evaluation of the functional properties important for in vivo delivery. In parallel with these works, Dyno has established collaborations with leading gene therapy companies Novartis, Sarepta Therapeutics, Roche and Spark Therapeutics to develop next-generation AAV gene therapy vectors with a goal of expanding the utility of gene therapies for ophthalmic, muscle, central nervous system (CNS) and liver diseases.

About CapsidMap for Designing Optimized AAV Gene Therapies

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dynos proprietary CapsidMap platform overcomes the limitations of todays gene therapies on the market and in development. Todays treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery efficiency, immunity, payload size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology to engineer capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dynos massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the design of novel capsids optimized for gene therapy.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experiments to gene therapy. The companys proprietary CapsidMap platform rapidly discovers and systematically optimizes Adeno-Associated Virus (AAV) capsid vectors that significantly outperform current approaches for in vivo gene delivery, thereby expanding the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts. Visit http://www.dynotx.com for additional information.

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Dyno Therapeutics Announces Publication in Nature Biotechnology Demonstrating Use of Artificial Intelligence to Generate Unprecedented Diversity of...

Global Cancer Gene Therapy Market Report Provides Future Development Possibilities By Key Players, Key Drivers, Competitive Analysis, Scope, And Key Challenges Analysis. The Reports Conjointly Elaborate The Expansion Rate Of The Industry Supported The Highest CAGR And Global Analysis. This Report Providing An In Depth And Top To Bottom Analysis By Market Size, Growth Forecast By Applications, Sales, Size, Types And Competitors For The Creating Segment And The Developing Section Among The Global Cancer Gene Therapy Market . Market Expansion Worldwide With Top Players Future Business Scope and Investment Analysis Report

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Cancer gene therapy market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to account to USD 6407.88 million by 2027 growing with the CAGR of 32.54% in the above-mentioned forecast period. The high success rate of cancer gene therapy along with clinical trial and preclinical trial is gaining popularity among the patient which is leading towards the market.

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Cancer gene therapy market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to cancer gene therapy market.

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Increase in funding of research and development in the activities of cancer gene therapy along with rise in prevalence of cancer is likely to accelerate the growth of the cancer gene therapy market in the forecast period of 2020-2027. On the other hand, the favourable government regulations for therapy is further going to boost various opportunities that will lead to the growth of the cancer gene therapy market in the above mentioned forecast period.

High cost involved in gene therapy along with unwanted immune responses wills likely to hamper the growth of the cancer gene therapy market in the above mentioned forecast period.

This cancer gene therapy market report provides details of new recent developments, trade regulations, import export analysis, production analysis, value chain optimization, market share, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographical expansions, technological innovations in the market. To gain more info on Cancer gene therapy market contactData Bridge Market Researchfor anAnalyst Brief, our team will help you take an informed market decision to achieve market growth.

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Cancer Gene Therapy Market Country Level Analysis

Cancer gene therapy market is analysed and market size insights and trends are provided by country, therapy and end user as referenced above.

The countries covered in the cancer gene therapy market report are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

North America dominates the cancer gene therapy market due to the advanced healthcare infrastructure along with rise in R & D expenditure, while Asia-Pacific is expected to grow with the highest growth rate in the forecast period of 2020 to 2027 due to the improving healthcare infrastructure and government initiatives.

The country section of the cancer gene therapy market report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as consumption volumes, production sites and volumes, import export analysis, price trend analysis, cost of raw materials, down-stream and upstream value chain analysis are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.

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Healthcare Infrastructure Growth Installed Base and New Technology Penetration

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Global Cancer Gene Therapy Market Insights, Size Estimation, Research Insights, COVID-19 Impact and Future Trends By 2028 KSU | The Sentinel...

LAUPHEIM, Germany and STEVENAGE, United Kingdom, Feb. 11, 2021 (GLOBE NEWSWIRE) -- Rentschler Biopharma SE, a leading global contract development and manufacturing organization (CDMO) for biopharmaceuticals, today announced that the Company is establishing a Center of Excellence for cell and gene therapy, which will be located in Stevenage, UK. With the newly formed UK subsidiary, Rentschler ATMP Ltd., the CDMO will establish development and manufacturing capability in Advanced Therapy Medicinal Products (ATMPs). The Companys UK site is being set up at an already existing facility run by the Cell and Gene Therapy Catapult (CGT Catapult) through a unique collaborative model. Over the next five years, Rentschler Biopharma plans to make a significant investment at the site to set up state-of-the-art manufacturing of viral vectors for clinical supply and expects to be ready for cGMP manufacturing by the first half of 2022.

Dr. Frank Mathias, CEO of Rentschler Biopharma, said: With more than 1000 cell and gene therapies currently in clinical development, the demand for manufacturing capabilities and support is ever increasing. Leveraging our specific expertise in process development and manufacturing for challenging molecules with highest quality and within demanding timelines, we are ideally suited to implement this knowledge in the field of viral vector production for new modalities. Our well-planned move into this growing area of biopharmaceuticals fits well with our strategic plans to expand internationally ensuring proximity to our clients and important biopharma hubs.

Rentschler Biopharma will continue its well-established practice of forming true partnerships with its clients. At this new Center of Excellence, the company plans to partner with entrepreneurial players to enable them to transform their ideas into real products with the potential to treat and even cure patients with serious and life-threatening diseases. Rentschler Biopharma utilizes a highly flexible business model that addresses the specific needs of each client.

About Rentschler Biopharma SE

Rentschler Biopharma is a leading contract development and manufacturing organization (CDMO), focused exclusively on client projects. The company offers process development and manufacturing of biopharmaceuticals as well as related consulting activities, including project management and regulatory support. Rentschler Biopharma's high quality is proven by its long-standing experience and excellence as a solution partner for its clients. A high-level quality management system, a well-established operational excellence philosophy and advanced technologies ensure product quality and productivity at each development and manufacturing step. In order to offer best-in-class formulation development along the biopharmaceutical value chain, the company has entered into a strategic alliance with Leukocare AG. Rentschler Biopharma is a family-owned company with about 1,000 employees, headquartered in Laupheim, Germany with a second site in Milford, MA, USA. In Stevenage, UK, Rentschler Biopharma launched a company dedicated to cell and gene therapies, Rentschler ATMP Ltd.

For further information, please visit http://www.rentschler-biopharma.com. Follow Rentschler Biopharma on LinkedIn and Facebook.

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Rentschler Biopharma establishes Center of Excellence for cell and gene therapy in the UK - GlobeNewswire

Feb. 10, 2021 08:00 UTC

AMSTERDAM--(BUSINESS WIRE)-- VectorY, a fully integrated gene therapy company focused on the development of innovative vectorized antibodies for muscular and neurodegenerative disorders, today announces its official launch. The Company has been operational since October 2020 and has established its laboratories and offices at the Amsterdam Science Park in The Netherlands.

VectorY develops proprietary and partnered programs based on a novel AAV platform and innovative antibody-based targeted degradation technologies. The Company is creating a pipeline of innovative vectorized antibodies targeting muscular and CNS diseases, aimed at overcoming limitations of current therapies, specifically by improving delivery, durability and accessibility of targeted tissues and cells. These novel viral vectors will have enhanced properties of non-immunogenicity, cell type specificity and expression in selected sub-cellular compartments, thus unlocking new treatment opportunities in these disease areas.

This year, the Company is setting up its own state-of-the-art GMP manufacturing facilities in Amsterdam. Its technology platform will be developed for scalability and yield, with the aim of significantly lowering the costs of advanced gene therapies.

VectorY has already negotiated its first corporate collaboration agreement, gaining access to a highly relevant neuro-inflammation target to develop a vectorized antibody therapy for treatment of neurological disorders. VectorY will continue to consider further collaborations in parallel to progressing its proprietary programs.

The highly experienced management team will be led by Marco Boorsma, General Partner at Forbion, who is joining the management team as Interim CEO. Co-founders Carlo Incerti, Pavlina Konstantinova, Anthony Newcombe, and Barbara Sanders all bring significant longstanding experience in gene therapy, vector development, and biopharmaceutical manufacturing expertise. Carlo Incerti, Operating Partner at Forbion and previously Chief Medical Officer of Sanofi Genzyme, will join VectorYs board as Chairman, together with Sander Slootweg, Managing Partner at Forbion, as board member.

VectorY was seeded by Forbion, following previous investments in successful gene vector companies including bluebird bio (then named Genetix), Biovex, Hookipa, EnGene, Amsterdam Molecular Therapeutics, uniQure and Replimune.

Carlo Incerti, Co-Founder and Chairman of the Board of VectorY, said: I have been working in the gene therapy field for over 30 years, and, from experience, know that accurately targeting the right cells with the transgene has always been of paramount importance. Using viral vectors developed at VectorY to deliver the genetic sequence for therapeutic antibody expression in target cells has the potential to address some of the key shortcomings of intravenously injected molecules. Our aim is to create novel treatments for patients.

Marco Boorsma, Interim CEO of VectorY, said: VectorY is developing innovative technologies to create novel, potentiated vectorized antibodies that will target muscle and CNS disorders, for which substantial unmet medical needs exist. Our aim is to bring new, powerful treatments to patients suffering from serious diseases. The Company is hitting the ground running, capitalizing on the extensive experience of the co-founders, and the support and expertise from Forbion as a longstanding gene therapy investor.

-Ends-

Notes to Editors

About VectorY VectorY combines the therapeutic potential of antibodies and gene therapy to develop long-lasting therapeutic solutions for muscular and neurodegenerative diseases with high unmet medical need.

Founded in August 2020, and based in the Amsterdam Science Park, VectorY is a fully integrated gene therapy company focused on the development of innovative therapeutics based on a novel AAV gene therapy platform, antibody-based targeted degradation technologies, and proprietary manufacturing technology.

VectorY develops proprietary & partnered programs and product candidates are based on new technologies that will enable the next generation of highly scalable manufacturing processes within VectorYs own manufacturing facilities. VectorYs manufacturing capabilities will include a state-of-the-art multi-product GMP facility in the Netherlands, with the capability to deliver suspension based AAV viral vector manufacturing of up to 2000L for both clinical and commercial supply.

About Forbion Forbion is a dedicated life sciences venture capital firm with offices in The Netherlands, Germany and Singapore. Forbion invests in life sciences companies that are active in the (bio-) pharmaceutical space.

Forbion manages well over EUR 1.7 billion across multiple fund strategies that cover all stages of (bio)pharmaceutical drug development. Forbions current team consists of 20 life sciences investment professionals that have built an impressive performance track record since the late nineties with successful investments in over 69 companies.

The firm is a signatory to the United Nations Principles for Responsible Investment. Besides financial objectives, Forbion selects investments that will positively affect the health and well-being of patients.

Its investors include the EIF, through its European Recovery Programme (ERP), LfA, Dutch Venture Initiative (DVI), AMUF and EFSI facilities and KfW Capital through the Programme, ERP Venture Capital Fonds investments. Forbion operates a joint venture with BGV, the manager of seed and early-stage funds, especially focused on Benelux and Germany.

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

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Launch of Novel Gene Therapy Company, VectorY, to Develop Innovative Vectorized Antibodies - BioSpace

New York, Feb. 10, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" - https://www.reportlinker.com/p06021776/?utm_source=GNW

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually. Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand. In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes. There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

The following factors are likely to contribute to the growth of the Europe cell and gene therapy market during the forecast period: CMOs Offering Vector Manufacturing Services for Cell and Gene Therapy Companies Robust Cell & Gene Therapies in the Pipeline Increase in Strategic Acquisitions Regulatory Support for Cell and Gene Therapy Products

The study considers the present scenario of the Europe cell and gene therapy market and its market dynamics for the period 2020?2026. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.

Europe Cell and Gene Therapy Market Segmentation The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields. The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies. While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures. Hospitals are growing at a significant rate due to the increasing target population in Europe. Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patients quality of life. Cell and gene therapies for certain types of cancers have been promising results. The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving. CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer. Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

Product Cell Therapies Gene Therapies End-user Hospitals Cancer Care Centers Wound Care Centers Others Application Oncology Dermatology Musculoskeletal Others

INSIGHTS BY GEOGRAPHY Germany, France, the UK, Italy, and Spain play a significant role in the Europe cell and gene therapy market. Clinical trials and the number of manufacturing facilities are increasing slowly in the European region. The region has become a major R&D destination for several vendors as the funding for cell & gene therapies is increasing. Europe has supported collaborative efforts in gene transfer and gene therapy research. In addition, the target patient population is increasing across Europe; there were an estimated 3.9 million new cases of cancer and 1.9 million cancer deaths in Europe in 2018. In addition, the prevalence surveys in the UK and Denmark indicate that there are 34 people with one or more wounds per 1,000 people. Favorable government support in terms of product approvals, reimbursement and coverage, and high R&D funding to academic institutes that are involved in the development of cell and gene therapies are expected to boosting the market in Europe.

Geography Europe o UK o Germany o France o Italy o Spain o Switzerland o Netherlands

INSIGHTS BY VENDORS Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors. While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

Prominent Vendors Novartis Spark Therapeutics Amgen Gilead Sciences Organogenesis

Other Prominent Vendors APAC Biotech AVITA Medical bluebird bio CHIESI Farmaceutici CollPlant CO.DON Human Stem Cells Institute PJSC (HSCI) Medipost NuVasive Nipro Orchard Therapeutics RMS Regenerative Medical System Orthocell Osiris Therapeutics Sibino GeneTech Shanghai Sunway Biotech Takeda Pharmaceutical Company Terumo Vericel

Emerging Investigational Vendors In Europe Adaptimmune Therapeutics AgenTus Therapeutics Autolus Cellecits Celyad CombiGene EUKARS Freeline Therapeutics Innoskel PsiOxus Therapeutics Ltd SparingVision uniQure

KEY QUESTIONS ANSWERED 1. What is the Europe cell and gene therapy market size and growth rate during the forecast period? 2. What are the factors driving the demand for CAR-T therapy in the European region? 3. How are strategic acquisitions aiding in market growth of cell and gene therapy products? 4. Which segments are expected to generate the highest revenues during the forecast period? 5. Who are the leading vendors in the European cell and gene therapy market?Read the full report: https://www.reportlinker.com/p06021776/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 20212026 - GlobeNewswire

ALISO VIEJO, Calif.--(BUSINESS WIRE)--As gene therapies and editing technologies rapidly advance, it is more urgent than ever to provide updates and information to the rare disease community on how these technologies can be applied across multiple diseases. Global Genes, a leading rare disease patient advocacy organization, is pleased to announce they will be publishing a multimedia series, titled Platforms of Hope: Advances in Gene Therapy and Gene Editing, throughout 2021 regarding upcoming data announcements and information on gene therapy and editing technology advances with thought leaders from the National Center for Advancing Translational Sciences (NCATS), the NIH Common Funds Somatic Cell Genome Editing (SCGE) program and other leading voices in these fields.

In addition to ongoing coverage through Global Genes videos, online publication RARE Daily and RARECast podcast, the organization will be publishing a special report at the end of 2021 on gene therapy and gene editing innovation. This will address a wide range of topics, with a focus on efforts to accelerate the translation of discoveries into genetic medicines that benefit patients with rare diseases.

With more than 7,000 rare diseases, there is an urgent need to keep the rare disease community abreast of developments in the rapidly changing fields of gene therapy and genome editing, said P.J. Brooks, program director at the Office of Rare Diseases Research at the NCATS. In this collaboration, NIH will help Global Genes identify the ideas, technologies and advances that have broad implications for many patients and families affected by rare diseases and provide information that could positively impact their lives and care in the future.

This collaboration will bring visibility into cutting-edge science at the frontier of genetic medicines and provide the rare disease community with insights into emerging technologies and therapies in development for rare diseases, said Christian Rubio, vice president, strategic advancement at Global Genes. Its critically important to educate the rare disease community on these rapidly evolving events.

For more information, visit http://www.globalgenes.org/media-hub.

About Global Genes

Global Genes is a 501(c)(3) nonprofit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. In pursuit of our mission, we connect, empower, and inspire the rare disease community to stand up, stand out, and become more effective on their own behalf -- helping to spur innovation, meet essential needs, build capacity and knowledge, and drive progress within and across rare diseases. We serve the more than 400 million people around the globe and nearly one in 10 Americans affected by rare diseases. If you or someone you love has a rare disease, or are searching for a diagnosis, contact Global Genes at 949-248-RARE, or visit our resource hub.

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Global Genes Announces New Multimedia Series Focused on Advances in Gene Therapy and Editing, in Collaboration with the National Institutes of Health...

Shares of AVROBIO wereup nearly 20% in premarket trading after the company posted positive clinical data from its gene therapy trials in three different rare lysosomal diseases, Fabry, Gaucher type 1 and cystinosis.

The data from the Phase II study assessing AVR-RD-01, an investigational ex vivo lentiviral gene therapy for Fabry disease, was particularly promising. This morning, Cambridge, Mass.-based Avrobio said a second kidney biopsy conducted on the first patient dosed with AVR-RD-01 showed 100% clearance of the toxic substrate Gb3.

Kidney substrate reduction is the primary endpoint of the Phase II study and has been a cornerstone for evaluating and approving treatments in Fabry disease, Avrobio noted. Avrobio said the patient came in with significant toxic buildup in his kidneys, which is quite common with Fabry disease. One year after the gene therapy treatment was administered in the trial, two independent pathologists foundzero markersof toxic substrate across all the 99 biopsy slides each evaluated.

The first biopsy conducted on the patient showed an 87% clearance of the substrate. In addition to the substrate clearance in the Fabry disease study, AvroBio reported continued strong and durable results in other key metrics across all nine Phase I and Phase II Fabry patients. All patients are now producing the functional enzyme they need to clear toxic substrate from their cells and seeing a concurrent drop in plasma substrate. The farthest patient is out 3.5 years, the company said.

Avrobio Chief Executive Officer Geoff MacKay hailed the data and said it was a thrilling way to begin 2021. The data announced this morning builds on the breadth of strong clinical data weve reported across our leading lysosomal disorder pipeline of single-dose gene therapies, he added.

The Fabry disease study wasnt the only positive news from Avrobio. The company also announced six-month data from the first patient dosed in the Phase I/II study of AVR-RD-02, an investigational ex vivo lentiviral gene therapy for Gaucher disease type 1. That data showed plasma chitotriosidase and the toxic metabolite lyso-Gb1, which are key biomarkers of Gaucher disease, had both dropped 49% below the patients baseline levels that had been achieved on enzyme replacement therapy (ERT) before gene therapy was administered. Also, Lyso-Gb1, the toxic metabolite that builds up in cells throughout the body in Gaucher, is down 44% below the patients ERT baseline. Avrobio said this is an early sign of efficacy.

Based on the data observed to date, we believe lentiviral gene therapy drives down toxic metabolites below levels of ERT, supporting our view that gene therapy has the potential to prevent, halt or even reverse progression of these devastating diseases with a single infusion, MacKay said.

For the cystinosis study, Avrobio said three patients who are taking part in the study are now off of standard-of-care treatment. The first study patient has had sharp reductions in crystal density in the eyes and skin and a marked improvement in photophobia, which is an extreme sensitivity to light that is associated with the disease.

With 13 patients dosed across three clinical programs, we have observed sustained and potentially transformative improvements in key biomarkers and functional metrics, with data from our Fabry disease program out 3 years after dosing. Additionally, enrollment activities for our Fabry disease trial are accelerating, giving us added confidence in our efforts to meet our goal of having dosed a cumulative 30 patients across all our clinical programs by the end of the year. With this strong momentum, we look forward to clarifying the regulatory pathway with regulatory agencies, MacKay said in a statement.

Full data from the studies will be presented later this week and WORLDSymposium, an annual meeting dedicated to lysosomal disorders.

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Avrobio Gene Therapy Shows Early Promise in Fabry, Other Rare Lysosomal Diseases - BioSpace

Amicus Optimized Transgene Show Greater Substrate Reduction than Wild Type Construct Across All Tissues and Doses

Further Validates Combining Amicus-Engineered Transgenes with Penns AAV Gene Therapy Technologies to Develop Next Generation Gene Therapies

PHILADELPHIA, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced initial preclinical data from its investigational adeno-associated viral (AAV) gene therapy program for Fabry disease in mice. The results are featured in a virtual poster presentation at the 17th Annual WORLDSymposium 2021, being held February 8-12, 2021. The poster is also available in the Events and Presentations section of the Amicus Therapeutics corporate website.

Fabry disease is an inherited lysosomal disorder caused by deficiency of the enzyme alpha-galactosidase A (GLA). Reduced or absent levels of GLA lead to accumulation of disease substrate leading to cellular disfunction and organ damage, which results in the clinical manifestations of Fabry disease. Amicus, in collaboration with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania (Penn), is developing a novel gene therapy for Fabry disease that combines the Amicus protein-engineering expertise and deep knowledge and experience in Fabry disease with Penns adeno associated virus (AAV) gene transfer technologies.

This initial preclinical study assessed a range of single doses of AAV in Gla knockout (KO) mice with either natural unmodified hGLA (wildtype hGLA) or Amicus/Penn engineered hGLA transgenes (engineered hGLA). The Amicus/Penn engineered hGLAs are designed for improved stability which is believed to provide a larger window for the enzyme to stay active while in circulation prior to being taken up into the target tissues and for additional stabilization after cell uptake. The lead Amicus/Penn engineered hGLA declared as an IND candidate is designated as AT-GTX-701.

Preclinical Poster Highlights for Amicus/Penn AAV Gene Therapy for Fabry Disease:

Hung Do, Ph.D., Chief Science Officer of Amicus Therapeutics, stated, These very important preclinical results validate our capabilities to develop engineered proteins via a gene therapy that can result in superior substrate reduction compared with a wildtype transgene. This is the second program in our collaboration with Penn that has demonstrated the potential advantages of optimizing the target protein in these disorders, and may be applicable to other lysosomal disorders as we continue to combine our understanding of the molecular basis of these diseases and expertise in protein engineering, together with Penns vector engineering expertise, to develop novel gene therapies.

Amicus is currently developing AAV gene therapies in collaboration with Penn for Pompe disease, Fabry disease, CDD, CLN1, MPS IIIB, a next generation program in MPS IIIA, as well as Angelman Syndrome. The agreement between Amicus and Penn is a Research, Collaboration and License Agreement, providing funding to Penn to advance the preclinical research programs in the Wilson Lab and to license certain technologies invented under the funded Research Collaboration.

About Fabry DiseaseFabry disease is an inherited lysosomal disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb3). Lipids that can be degraded by the action of alpha-Gal A are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including the central nervous system, heart, kidneys, and skin. Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease, and stroke. The symptoms can be severe, differ from patient to patient, and begin at an early age. All Fabry disease is progressive and may lead to irreversible organ damage regardless of the time of symptom onset.

About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at http://www.amicusrx.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to initial preclinical data from its investigational adeno-associated viral (AAV) gene therapy program for Fabry disease in mice and the potential implications of these data for the future advancement and development of a gene therapy for Fabry disease and other lysosomal disorders and development of potential platform technologies. Words such as, but not limited to, look forward to, believe, expect, anticipate, estimate, intend, "confidence," "encouraged," potential, plan, targets, likely, may, will, would, should and could, and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the preliminary data reported before completion of the study will not be predictive of future results, that results of additional preliminary data or data from the completed study or any future study will not yield results that are consistent with the preliminary data presented, that later study results will not support further development, or even if such later results are favorable, that the Company will not be able to successfully complete the development of, obtain regulatory approval for, or successfully commercialize. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended December 31, 2019 and the Quarterly Report filed on Form 10-Q for the quarter ended September 30, 2020. As a consequence, actual results may differ materially from those set forth in this press release. You are cautioned not to place undue reliance on these forward looking statements, which speak only of the date hereof. All forward looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise this press release to reflect events or circumstances after the date hereof.

CONTACTS:

Investors:Andrew FaughnanSr. Director, Investor Relationsafaughnan@amicusrx.com(609) 662-3809

Media:Diana MooreHead of Global Corporate Communicationsdmoore@amicusrx.com(609) 662-5079

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Amicus Therapeutics Presents Positive Preclinical Fabry Disease Gene Therapy Data at the 17th Annual WORLDSymposium 2021 - GlobeNewswire

The "Adeno-Associated Virus (AAV) Vectors in Gene Therapy - Market Insight, Epidemiology and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.

This report delivers an in-depth understanding of the AAV Vector Based Gene Therapy, historical and forecasted epidemiology as well as the AAV Vector Based Gene Therapy market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan.

The AAV Vector Based Gene Therapy market report provides emerging drugs, AAV Vector Based Gene Therapy market share of the individual diseases, current and forecasted AAV Vector Based Gene Therapy market size from 2017 to 2030 segmented by seven major markets. The Report also covers current AAV Vector Based Gene Therapy market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses underlying potential of the market.

The AAV Vector Based Gene Therapy epidemiology division provides the insights about historical and current AAV Vector Based Gene Therapy patient pool and forecasted trend for each seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of The report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

The disease epidemiology covered in the report provides historical as well as forecasted AAV Gene Therapies epidemiology [segmented as Total cases of AAV Gene Therapy Targeted Indications, Total diagnosed indication-specific cases, Number of Patients Eligible for AAV Gene Therapy and Total treated cases] scenario of AAV Gene Therapy in the 7MM covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2017 to 2030.

Country Wise- AAV Vector Based Gene Therapy Epidemiology

Estimates show that the highest cases of AAV vector based gene therapy in the 7MM were in the United States, followed by Germany, Japan, France, the United kingdom, Italy, and Spain in 2017.

Story continues

AAV vector based gene therapy Drug Chapters

Drug chapter segment of the AAV vector based gene therapy report encloses the detailed analysis of AAV vector based gene therapy marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the AAV vector based gene therapy clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Scope of the Report

The report covers a detailed overview of the development of AAV vectors and gene therapies based on them across the various therapeutic areas and several selected indications, explaining its various serotypes, benefits, and challenges for future

Comprehensive insight has been provided into the AAV Vector Based Gene Therapy epidemiology and treatment in the 7MM.

Additionally, an all-inclusive account of both the current and emerging therapies for AAV Vector Based Gene Therapy is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.

A detailed review of AAV Vector Based Gene Therapy market; historical and forecasted is included in the report, covering drug outreach in the 7MM.

The report provides an edge while developing business strategies, by understanding trends shaping and driving the global AAV Vector Based Gene Therapy market.

AAV Vector Based Gene Therapy Report Insights

Patient Population

Therapeutic Approaches

AAV Vector Based Gene Therapy Pipeline Analysis

AAV Vector Based Gene Therapy Market Size and Trends

Market Opportunities

Impact of upcoming Therapies

AAV Vector Based Gene Therapy Report Key Strengths

AAV Vector Based Gene Therapy Report Assessment

SWOT Analysis

Current Treatment Practices

Unmet Needs

Pipeline Product Profiles

Conjoint Analysis

Market Attractiveness

Market Drivers and Barriers

Reasons to Buy

The report will help in developing business strategies by understanding trends shaping and driving the AAV Vector Based Gene Therapy market.

To understand the future market competition in the AAV Vector Based Gene Therapy market and Insightful review of the key market drivers and barriers.

Organize sales and marketing efforts by identifying the best opportunities for AAV Vector Based Gene Therapy in the US, Europe (Germany, France, Italy, Spain, and the United Kingdom) and Japan.

Identification of strong upcoming players in market will help in devising strategies that will help in getting ahead of competitors.

Organize sales and marketing efforts by identifying the best opportunities for AAV Vector Based Gene Therapy market.

To understand the future market competition in the AAV Vector Based Gene Therapy market.

Companies Mentioned

For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/p3sdwo

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ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.com

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Global Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market to 2030 - Insight, Epidemiology and Forecasts - ResearchAndMarkets.com - Yahoo...