Category Archives: Gene therapy

SOLANA BEACH, Calif., May 20, 2022 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. ( CLPT) (the Company), a global therapy-enabling platform company providing navigation and delivery to the brain, today congratulates partner PTC Therapeutics for receiving a positive opinion from the European Medicines Agencys (EMA) Committee for Medicinal Products for Human Use (CHMP) recommending its gene therapy treatment Upstaza (eladocagene exuparvovec), formerly PTC-AADC, for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. The use of ClearPoint Neuros proprietary CE Marked SmartFlow Neuro Ventricular Cannula for minimally invasive infusion of the gene therapy is included in the submission for administration of Upstaza.

This is a significant milestone for PTC Therapeutics, for ClearPoint and for biologics researchers everywhere as it marks the very first positive CHMP recommendation for direct injection to the brain of a gene therapy to treat a severe, highly morbid and fatal neurological disorder, commented Joe Burnett, President and CEO of ClearPoint Neuro. This milestone is incredibly important for a couple of reasons. First, it provides a potential path for other therapies to follow and sets an important precedent for regulatory approval of direct administered gene therapies to the brain. And second, we see this as a validation of ClearPoints biologics partnership strategy and our rigorous bench, preclinical and clinical experience. We believe this announcement will give our 45 existing and future pharmaceutical, academic and biotech partners the confidence that SmartFlow is able to pass through the necessary regulatory scrutiny and can de-risk the commercialization efforts of many different drugs to multiple targets in the brain.

About aromatic L-amino acid decarboxylase (AADC) deficiency

AADC deficiency is a fatal, rare genetic disorder that typically causes severe disability and suffering from the first months of life, affecting every aspect of life physical, mental, and behavioral. The suffering of children with AADC deficiency may be exacerbated by episodes of distressing seizure-like oculogyric crises, which can happen daily and last for hours, causing the eyes to roll up in the head, frequent vomiting, behavioral problems, difficulty sleeping, and life-threatening complications such as respiratory infections and gastrointestinal problems.

There is no disease-modifying treatment approved for AADC deficiency, and the lives of affected children are severely impacted, and shortened, with the use of many different medications to help manage symptoms. Ongoing physical, occupational and speech therapy, and interventions, including surgery, to manage potentially life-threatening complications such as infections, severe feeding, and breathing problems also are often required.

About the SmartFlow Cannula

With over 5,000 cannulas sold to date, SmartFlow is the only co-labeled device to gain approval by a regulatory agency for delivery of an approved gene therapy to the brain. The industry-leading cannula is used by many of ClearPoint Neuros 45 pharmaceutical, academic, and biotech partners to bypass the blood brain barrier and deliver therapeutics to regions of interest using Convection Enhanced Delivery (CED) under direct image guidance. The SmartFlow cannula has received 510(k) clearance from the FDA for use in the United States for the aspiration of cerebrospinal fluid or injection of the chemotherapy drug Cytarabine into the ventricles. It has also been CE marked to deliver approved fluids into the brain or aspiration of CSF. SmartFlow is being utilized in approved clinical and preclinical studies for various research and drug trials. This device is not intended for implant and is intended for single patient use only.

About ClearPoint Neuro

ClearPoint Neuros mission is to improve and restore quality of life to patients and their families by enabling therapies for the most complex neurological disorders with pinpoint accuracy. Applications of the Companys current product portfolio include deep brain stimulation, laser ablation, biopsy, neuro-aspiration, and delivery of drugs, biologics, and gene therapy to the brain. The ClearPoint Neuro Navigation System has FDA clearance, is CE-marked, and is installed in over 60 active sites in the United States, Canada, and Europe. ClearPoint Neuro is partnered with approximately 45 biologics/pharmaceutical companies and academic centers, providing solutions for direct CNS delivery of therapeutics in preclinical studies and clinical trials worldwide. To date, more than 5,000 cases have been performed and supported by the Companys field-based clinical specialist team, which offers support and services to our customers and partners worldwide. For more information, please visit http://www.clearpointneuro.com.

Forward-Looking Statements

Statements herein concerning the Companys plans, growth and strategies may include forward-looking statements within the context of the federal securities laws. Statements regarding the Company's future events, developments and future performance, as well as management's expectations, beliefs, plans, estimates or projections relating to the future, are forward-looking statements within the meaning of these laws. Uncertainties and risks may cause the Company's actual results to differ materially from those expressed in or implied by forward-looking statements. Particular uncertainties and risks include those relating to: the impact of the COVID-19 pandemic and the measures adopted to contain its spread; future revenue from sales of the Companys ClearPoint Neuro Navigation System products; the Companys ability to market, commercialize and achieve broader market acceptance for the Companys ClearPoint Neuro Navigation System products; and risks inherent in the research and development of new products. More detailed information on these and additional factors that could affect the Companys actual results are described in the Risk Factors section of the Companys Annual Report on Form 10-K for the year ended December 31, 2021, and the Companys Quarterly Report on Form 10-Q for the three months ended March 31, 2022, both of which have been filed with the Securities and Exchange Commission.

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ClearPoint Neuro Congratulates Partner PTC Therapeutics on Receiving Positive CHMP Opinion for Gene Therapy to Treat AADC Deficiency - GuruFocus.com

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a biotechnology company with a mission to discover, develop and deliver curative therapies that address the underlying causes of heart disease, today announced that it has completed the build-out and operational launch of its Genetic Medicines Manufacturing Center in Union City, California. Tenaya is advancing a pipeline of therapeutic candidates, including several adeno-associated virus (AAV) gene therapies, for the potential treatment of both rare and prevalent forms of heart disease.

Tenaya made an early, strategic commitment to internalize several core capabilities to optimize the safety, efficacy, and supply of our product candidates on behalf of patients. With todays announcement we have made a big leap forward on that commitment by establishing end-to-end in-house manufacturing capabilities for our pipeline of AAV-based gene therapies, said Faraz Ali, Chief Executive Officer of Tenaya. The operational launch of Tenayas Genetic Medicines Manufacturing Center represents an important milestone as we prepare to advance our robust pipeline of potentially first-in-class cardiovascular therapies into initial clinical studies.

Tenayas Genetic Medicines Manufacturing Center is designed to meet regulatory requirements for production of AAV gene therapies from discovery through commercialization under Current Good Manufacturing Practice (cGMP) standards. Initial production efforts will support first-in-human studies of Tenayas lead gene therapy, TN-201. TN-201 is being developed for the treatment of genetic hypertrophic cardiomyopathy (HCM) due to MYBPC3 gene mutations. Tenaya plans to submit an Investigational New Drug (IND) application for TN-201 to the U.S. Food and Drug Administration (FDA) in the second half of this year. The facility will also support cGMP production for TN-401, Tenayas gene therapy program being developed for the treatment of genetic arrhythmogenic right ventricular cardiomyopathy (ARVC) due to PKP2 gene mutations, for which the company plans to submit an IND to the FDA in 2023.

The investment in our own world-class manufacturing facility provides Tenaya with greater control over product attributes, quality, production timelines and costs, which we believe will ultimately translate into better treatments for patients, said Kee-Hong Kim, Ph.D., Chief Technology Officer of Tenaya Therapeutics. Tenayas Genetic Medicines Manufacturing Center complements our established internal genetic engineering and drug discovery capabilities and is designed to meet our near- and long-term needs such that we can readily scale and expand as our pipeline matures and evolves.

Tenaya completed customization of approximately half of the 94,000 square foot facility to incorporate manufacturing suites and labs, office space and storage. Utilizing a modular design, the state-of-the-art facility is now fully operational with initial capacity to produce AAV-based gene therapies at the 1000L scale, utilizing Tenayas proprietary baculovirus-based production platform and suspension Sf9 cell culture system. The excess space and modular design of the Genetic Medicines Manufacturing Center is intended to provide Tenaya with considerable flexibility to expand manufacturing capacity by increasing both the number and the scale of bioreactors to meet future clinical and commercial production needs.

The Union City location, approximately 30 miles from Tenayas South San Francisco headquarters, is expected to enable the seamless transition of Tenayas science from early research through commercial manufacturing. The selection of this location is intended to foster a culture of close collaboration across teams at all stages of developing and testing novel AAV capsids, de-risk the translation from research to process development and create opportunities for improvements in production processes. The Genetic Medicines Manufacturing Center is staffed by a growing in-house team with expertise in all aspects of gene therapy manufacture, including process development, analytical development, quality assurance and quality control.

About Tenaya Therapeutics

Tenaya Therapeutics is a biotechnology company committed to a bold mission: to discover, develop and deliver curative therapies that address the underlying drivers of heart disease. Founded by leading cardiovascular scientists from Gladstone Institutes and the University of Texas Southwestern Medical Center, Tenaya is developing therapies for rare genetic cardiovascular disorders, as well as for more prevalent heart conditions, through three distinct but interrelated product platforms: Gene Therapy, Cellular Regeneration and Precision Medicine. For more information, visit http://www.tenayatherapeutics.com.

Forward Looking Statements

This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Words such as potential, will, plans, believe, expected, and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, statements regarding the therapeutic potential of Tenayas pipeline of therapeutic candidates; Tenayas plan to use the cGMP manufacturing facility for the production of TN-201 and TN-401; Tenayas belief that its cGMP manufacturing facility will enable seamless transition from early research through commercial manufacturing and translate into better treatments for patients; the expected timing for submission of IND applications for TN-201 and TN-401; and statements by Tenayas chief executive officer and chief technology officer. The forward-looking statements contained herein are based upon Tenayas current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including but not limited to: risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early stage company; Tenayas ability to successfully manufacture product candidates in a timely and sufficient manner that is compliant with regulatory requirements; Tenayas ability to develop, initiate or complete preclinical studies and clinical trials, and obtain approvals, for any of its product candidates; the timing, progress and results of preclinical studies for TN-201, TN-401 and Tenayas other programs; Tenayas ability to raise any additional funding it will need to continue to pursue its business and product development plans; negative impacts of the COVID-19 pandemic on Tenayas manufacturing and operations, including preclinical studies and planned clinical trials; the timing, scope and likelihood of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; Tenayas manufacturing, commercialization and marketing capabilities and strategy; the loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenayas reliance on third parties; Tenayas ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled Risk Factors in documents that Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

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Tenaya Therapeutics Launches Operations of New Genetic Medicines Manufacturing Center to Support the Development of Potentially First-In-Class...

ImmunoACT, an IIT-Bombay spin-off backed by Hyderabad-based Laurus Labs, is testing a new gene-therapy treatment for cancer, which they claim would not only cure the disease, but also costs one-tenth of the global price.

We are working with CD19 CAR T Cell for leukemia and lymphoma. What we do is we take out some blood and plasma from the cancer patient, and then isolate the T-cells (which are immune cells) from this sample. These cells are then genetically modified so that they eliminate cancer cells. Then this is transfused back to the patient, explains Rahul Purwar, founder and chairman of ImmunoACT. The product is named H-CAR T-19.

He claims that the results start showing in around one weeks time. Moreover, this is a one-time treatment that would cost around Rs 20-30 lakh per patient, as compared to Rs 4 crore or so in the US, Purwar adds.

It takes around two weeks to prepare the customised cells to be transfused into the patient, and thereafter this is like a live drug working inside the patients body, he says.

Are there any side-effects of this treatment? Purwar says none like what we witness in chemotherapy, but sometimes there is cytokine release syndrome because of dead cancer cells in the body, which can be managed. So far, we have not seen any patient with an uncontrolled cytokine release syndrome.

The therapy has completed its phase 1 or safety trials, and is now all set to go into phase two trials here. ImmunoACT expects that it would be ready for a market launch in around 18-months time subject to regulatory approvals.

Laurus Labs has picked up 26 per cent stake in this biotech start-up, and its promoters have picked up around 5-6 per cent in personal capacity, informed Laurus Labs CEO Satyanarayana Chava.

He said that as a part of policy, they have decided to pump in 10 per cent of their annual profits into such healthcare start-ups with breakthrough technology.

I feel there is a lot of promise this therapy offers in India, and especially the price is way below the global standards. We have spoken to several oncologists and they are waiting for this tool to be available to them. Several NGOs also work in this space that help patients with treatment costs, Chava said.

In the US, the insurance companies cover immunotherapy costs. In India once this product is available, one would have to work out these nuances.

ImmunoACT is also working on two more cancer products using this technology one for neuroblastoma and another for brain cancer.

Purwar says that the technology has no limitations and can be adapted for various kinds of cancers. At the moment they have set up a GMP facility in Mumbai, which can process H-CAR T -19 for 100 patients a month or around 1,200 a year.

As demand rises, they plan to add capacity at the existing site as well as add sites in new locations. There are approximately 50,000 leukemia patients diagnosed in India every year, and many of them are put on oral drugs and chemotherapy. The success rate of chemotherapy is approximately 60 percent, Purwar adds.

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Gene therapy to cure leukemia at one-tenth global costs in the works - Business Standard

Wilmington, Delaware, United States: According to the report, the global gene therapy market was valued at US$ 1.3 Bn in 2020 and is projected to expand at a CAGR of 19.8% from 2017 to 2028. Gene therapy is a way of fixing genetic disorders by introducing a normal and healthy gene in place of the defective one in a cell to prevent or cure different types of genetic and chronic disorders for which no cure has been developed. Gene therapy is considered an important mode of treatment, as it helps eliminate the usage of drugs, surgery, or other procedures, which can have side effects. Several clinical research studies are being conducted to develop effective gene therapy products for the treatment of diseases related to ophthalmology, oncology, and ADA-SCID.

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Gene therapy in ophthalmology includes the injection of fully functional genes into the retinal cells in order to allow restoration of normal gene expression. Different gene therapy approaches are being studied in the fields of oncology, such as pro-drug activating suicide gene therapy, anti-angiogenic gene therapy, oncolytic virotherapy, and gene therapy-based immune modulation. The adenosine deaminase (ADA)deficient severe combined immunodeficiency (SCID) is a very rare form of congenital disorder of the immune system.

Promising Therapeutic Outcomes to Drive Market

Gene therapy has considerable potential to eliminate and prevent several genetic disorders and numerous life-threatening disorders, especially cancer, heart diseases, AIDS, cystic fibrosis, and age-related disorders. Gene therapy provides a complete cure to patients affected with genetic disorders, rather than ease symptoms with other therapeutic treatments. The first gene therapy product Gendicine was developed by Shenzhen, China-based SiBiono GeneTech Co. Ltd. in 2003 and was commercialized in 2004 for the treatment of head and neck cancer. Since 2004, more than 30,000 patients have been treated with Gendicine in China. Gendicine has exhibited notable safety results, and when this gene therapy is combined with other treatment forms, such as chemotherapy and radiotherapy, the overall outcome outperforms other standard therapies alone.

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Patients treated with Novartis AGs gene therapy product Kymriah have exhibited a remission rate of 81% with three months of treatment, and all the patients who exhibit a positive response to treatment were not detected with any minimal residual disease. Hence, the overall promising results of gene therapy fuels the demand for gene therapy products.

New Product Approvals & Commercialization to Propel Market

The years 2016, 2017, and 2018 were key milestones in the history of the gene therapy market in the U.S. and Europe because around four gene therapy products have been approved and commercialized. These products are currently in a nascent stage of commercialization, and have exhibited highly positive therapeutic outcomes. Increase in competition among the leading biopharmaceutical companies to approve and commercialize gene therapy products in different areas of unmet medical needs to gain the first mover advantage is likely to boost the growth of the gene therapy market during the forecast period.

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Increase in Number of Gene Therapy Treatment Centers to Augment Market

New gene therapy product approvals and potential success rates of gene therapy have led to an increased demand for more number of treatment centers in the U.S. and Europe. The companies, after initial success rates of gene therapy, strive to increase the number of treatment centers in order to gain access to a large patient pool requiring gene therapy treatments.

The gene therapy product Kymriah, which was approved for the treatment of patients up to the age of 25 years affected with large B-cell lymphoma, has only 41 treatment centers in the U.S., which limits a large number of patients from receiving gene therapy treatment.

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Promising Product Pipeline and High Investment in R&D

Significant investments in gene-related research & development by various biopharmaceutical companies, governments, and research institutes presents an opportunity to gain the first mover advantage in the gene therapy market. A large number of gene therapy products are under different stages of clinical studies, and the number of gene therapy candidates is projected to rise during the forecast period. High investment in gene therapy research and large number of late stage gene therapy candidates in the oncology domain and other genetic disorders are projected to fuel the demand for gene therapy products during the forecast period.

Competition Landscape

The global cell culture market is consolidated in terms of number of players. Key players in the global market includes Novartis AG, Orchard Therapeutics Limited, Celgene Corporation, Spark Therapeutics, Inc., Gilead Sciences, Inc., Sibiono GeneTech Co. Ltd., and Other prominent players.

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Gene Therapy Market is Estimated to Reach US$ 5.3 Bn By 2028, Increase in Incidence of Cancer to Drive the Market - BioSpace

Two-stage collaboration aims to accelerate development of high-capacity manufacturing processes for AAV-based products and secure Coave's infrastructure needs to set up its process development capabilities

PARIS and STRASBOURG, France, June 9, 2022 /PRNewswire/ -- Coave Therapeutics ('Coave'), a clinical-stage biotechnology company focused on developing life-changing gene therapies for CNS (Central Nervous System) and eye diseases, and ABL, a pure play contract development and manufacturing organization (CDMO) specialized in the development and manufacturing of viruses for vaccine candidates, gene and cancer therapies, today announce that they have entered into a strategic collaboration to develop gene therapy manufacturing processes. The financial terms are not disclosed.

Under the two-stage collaboration both companies will initially combine their complementary expertise to co-develop manufacturing technologies for AAV-based gene therapy products. ABL and Coave's process development teams will work jointly in ABL's state-of-the-art GMP facility in Lyon, France.

The second stage of the collaboration provides Coave with an exclusive option to secure process development capacity and laboratory space within ABL's facility. This will enable Coave to further develop and scale-up manufacturing technologies for AAV-based products, including its proprietary next-generationAAV-Ligand Conjugate vectors (ALIGATER) platform. The deal will also strengthen Coave's ability to generate large-scale and high-quality gene therapy products based on this new generation of advanced AAV vectors.

"Our collaboration with ABL, a best-in-class and complementary partner for Coave, is a major step in our strategy towards the vertical integration of our R&D capacities, which will be crucial in enabling us to control the development and manufacture of our pipeline products in an end-to-end manner. The manufacturing processes developed through this partnership will be critical in the path to achieving our future clinical and commercial development milestones, in particular for our CNS programs addressing large patient populations," said Rodolphe Clerval, CEO, Coave Therapeutics.

Patrick Mahieux, General Manager ABL Europe, said: "We are delighted to join forces with Coave, a fellow French company. This exclusive partnership aims to bring together our knowledge and expertise to co-develop a state-of-the-art manufacturing process for viral vectors used in cell and gene therapies. We are excited to welcome Coave's team of expert scientists to our facilities in Lyon to jointly accelerate the development of an AAV manufacturing platform. We look forward to a long-term partnership enabling the development and manufacturing of innovative cell and gene therapy treatments in France."

About Coave Therapeutics

Coave Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for CNS (Central Nervous System) and eye diseases.

Coave Therapeutics' next-generation AAV-Ligand Conjugate ('ALIGATER') platform enables targeted delivery and enhanced gene transduction to improve the effectiveness of advanced gene therapies for rare diseases.

The company is advancing a pipeline of novel therapies targeting CNS and eye diseases where targeted gene therapy using chemically-modified AAVs has the potential to be most effective.

Coave Therapeutics, which is headquartered in Paris (France), is backed by leading international life science and strategic investors Seroba Life Sciences, Tha Open Innovation, eureKARE, Fund+, Omnes Capital, V-Bio Ventures, Kurma Partners, Idinvest, GO Capital and Sham Innovation Sant/Turenne.

For more information, please visit http://www.coavetx.comor follow us on LinkedIn

About ABL, an Institut Mrieux company

ABL is a pure play contract development and manufacturing organization (CDMO) specialized in the development and manufacturing of virus for vaccine candidates, gene and cancer therapies. ABL's mission is to provide GMP viral vectors from early-stage to market, contributing to the success of its clients' immunotherapy innovations. ABL's CDMO services include bulk drug substance, fill/finish of drug product, process and assay development, and bioanalytical testing.

ABL is a subsidiary of the Institut Mrieux and operates from various locations in Europe and in the US.

http://www.abl-biomanufacturing.com

CONTACTS

Coave TherapeuticsRodolphe Clerval, CEO[emailprotected]

MEDiSTRAVA ConsultingSylvie Berrebi, Eleanor Perkin, Mark Swallow PhD[emailprotected] Tel: +44 203 928 6900

ABLJustine Chabrol, Head of Communications & CSR[emailprotected]

Andrew Lloyd & AssociatesEmilie Chouinard Saffiyah Khalique [emailprotected] [emailprotected] Tel: +44 1273 952 481@ALA_Group

SOURCE Coave Therapeutics and ABL

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Coave Therapeutics and ABL enter into strategic collaboration to develop gene therapy manufacturing processes and create joint capabilities for...

DUBLIN--(BUSINESS WIRE)--The "Adeno-Associated Virus Vectors in Gene Therapy - Market Insight, Epidemiology and Market Forecast -2032" report has been added to ResearchAndMarkets.com's offering.

The Adeno-Associated Virus Vectors in Gene Therapy market report provides current treatment practices, emerging drugs, Adeno-Associated Virus Vectors in Gene Therapy market share of the individual therapies, current and forecasted Adeno-Associated Virus Vectors in Gene Therapy market Size from 2019 to 2032 segmented by seven major markets.

The Report also covers current Adeno-Associated Virus Vectors in Gene Therapy treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses the underlying potential of the market.

Adeno-Associated Virus Vectors in Gene Therapy Disease Understanding and Treatment Algorithm

The Adeno-Associated Virus Vectors in Gene Therapy market report gives a thorough understanding of the Adeno-Associated Virus Vectors in Gene Therapy by including details such as disease definition, symptoms, causes, pathophysiology, diagnosis and treatment.

Diagnosis

This segment of the report covers the detailed diagnostic methods or tests for Adeno-Associated Virus Vectors in Gene Therapy.

Treatment

It covers the details of conventional and current medical therapies available in the Adeno-Associated Virus Vectors in Gene Therapy market for the treatment of the condition. It also provides Adeno-Associated Virus Vectors in Gene Therapy treatment algorithms and guidelines in the United States, Europe, and Japan.

Adeno-Associated Virus Vectors in Gene Therapy Epidemiology

The Adeno-Associated Virus Vectors in Gene Therapy epidemiology division provide insights about historical and current Adeno-Associated Virus Vectors in Gene Therapy patient pool and forecasted trend for every 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.

The disease epidemiology covered in the report provides historical as well as forecasted Adeno-Associated Virus Vectors in Gene Therapy epidemiology scenario in the 7MM covering the United States, EU5 countries (Germany, Spain, Italy, France, and the United Kingdom), and Japan from 2019 to 2032.

Adeno-Associated Virus Vectors in Gene Therapy Drug Chapters

Drug chapter segment of the Adeno-Associated Virus Vectors in Gene Therapy report encloses the detailed analysis of Adeno-Associated Virus Vectors in Gene Therapy marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the Adeno-Associated Virus Vectors in 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.

Marketed Drugs

The report provides the details of the marketed product available for Adeno-Associated Virus Vectors in Gene Therapy treatment.

Adeno-Associated Virus Vectors in Gene Therapy Emerging Drugs

The report provides the details of the emerging therapies under the late and mid-stage of development for Adeno-Associated Virus Vectors in Gene Therapy treatment.

Adeno-Associated Virus Vectors in Gene Therapy Market Outlook

The Adeno-Associated Virus Vectors in Gene Therapy market outlook of the report helps to build the detailed comprehension of the historic, current, and forecasted Adeno-Associated Virus Vectors in Gene Therapy market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.

This segment gives a thorough detail of Adeno-Associated Virus Vectors in Gene Therapy market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to the publisher, Adeno-Associated Virus Vectors in Gene Therapy market in 7MM is expected to change in the study period 2019-2032.

Adeno-Associated Virus Vectors in Gene Therapy Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the Adeno-Associated Virus Vectors in Gene Therapy market or expected to get launched in the market during the study period 2019-2032. The analysis covers Adeno-Associated Virus Vectors in Gene Therapy market uptake by drugs; patient uptake by therapies; and sales of each drug.

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Adeno-Associated Virus Vectors in Gene Therapy Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase II, and Phase III stage. It also analyses Adeno-Associated Virus Vectors in Gene Therapy key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing, patent details and other information for Adeno-Associated Virus Vectors in Gene Therapy emerging therapies.

Reimbursement Scenario in Adeno-Associated Virus Vectors in Gene Therapy

Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In a report, we take reimbursement into consideration to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy.

KOL-Views

To keep up with current market trends, we take KOLs and SME's opinion working in Adeno-Associated Virus Vectors in Gene Therapy domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Adeno-Associated Virus Vectors in Gene Therapy market trend. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

The publisher performs Competitive and Market Intelligence analysis of the Adeno-Associated Virus Vectors in Gene Therapy Market by using various Competitive Intelligence tools that include - SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies etc. The inclusion of the analysis entirely depends upon the data availability.

Key Topics Covered:

1. Key Insights

2. Executive Summary of Adeno-Associated Virus Vectors in Gene Therapy

3. Competitive Intelligence Analysis for Adeno-Associated Virus Vectors in Gene Therapy

4. Adeno-Associated Virus Vectors in Gene Therapy: Market Overview at a Glance

4.1. Adeno-Associated Virus Vectors in Gene Therapy Total Market Share (%) Distribution in 2019

4.2. Adeno-Associated Virus Vectors in Gene Therapy Total Market Share (%) Distribution in 2032

5. Adeno-Associated Virus Vectors in Gene Therapy: Disease Background and Overview

5.1. Introduction

5.2. Sign and Symptoms

5.3. Pathophysiology

5.4. Risk Factors

5.5. Diagnosis

6. Patient Journey

7. Adeno-Associated Virus Vectors in Gene Therapy Epidemiology and Patient Population

7.1. Epidemiology Key Findings

7.2. Assumptions and Rationale: 7MM

7.3. Epidemiology Scenario: 7MM

7.3.1. Adeno-Associated Virus Vectors in Gene Therapy Epidemiology Scenario in the 7MM (2019-2032)

7.4. United States Epidemiology

7.5. EU-5 Country-wise Epidemiology

7.5.1. Germany Epidemiology

7.5.2. France Epidemiology

7.5.3. Italy Epidemiology

7.5.4. Spain Epidemiology

7.5.5. United Kingdom Epidemiology

7.5.6. Japan Epidemiology

8. Treatment Algorithm, Current Treatment, and Medical Practices

8.1. Adeno-Associated Virus Vectors in Gene Therapy Treatment and Management

8.2. Adeno-Associated Virus Vectors in Gene Therapy Treatment Algorithm

9. Unmet Needs

10. Key Endpoints of Adeno-Associated Virus Vectors in Gene Therapy Treatment

11. Marketed Products

12. Emerging Therapies

13. Adeno-Associated Virus Vectors in Gene Therapy: Seven Major Market Analysis

13.1. Key Findings

13.2. Adeno-Associated Virus Vectors in Gene Therapy Market Size in 7MM

13.3. Adeno-Associated Virus Vectors in Gene Therapy Market Size by Therapies in the 7MM

14. Attribute analysis

15. 7MM: Market Outlook

15.1. United States: Market Size

15.1.1. Adeno-Associated Virus Vectors in Gene Therapy Total Market Size in the United States

15.1.2. Adeno-Associated Virus Vectors in Gene Therapy Market Size by Therapies in the United States

15.2. EU-5 countries: Market Size and Outlook

15.3. Germany Market Size

15.4. France Market Size

15.5. Italy Market Size

15.6. Spain Market Size

15.7. United Kingdom Market Size

15.8. Japan Market Outlook

15.8.1. Japan Market Size

16. Access and Reimbursement Overview of Adeno-Associated Virus Vectors in Gene Therapy

17. KOL Views

18. Market Drivers

19. Market Barriers

20. Appendix

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/t0ddt0

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Adeno-Associated Virus Vectors in Gene Therapy Research Report 2022: Market Insight, Epidemiology and Market Forecasts 2019-2032 -...

Dublin, June 08, 2022 (GLOBE NEWSWIRE) -- The "Investor Series: Opportunities in the Cell and Gene Therapy Market" report has been added to ResearchAndMarkets.com's offering.

The report provides detailed information on the cell and gene therapy industry, covering both core and peripheral products, and affiliated services.

One of the key objectives of the report was to evaluate the current opportunity and the future potential of cell and gene therapies over the coming decades. We have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2021-2035.

It offers a technical and financial perspective on how the opportunity in this domain is likely to evolve, in terms of future business success, over the coming decade. The information in this report has been presented across multiple deliverables, featuring MS Excel sheets (some of which include interactive elements) and an MS PowerPoint deck, which summarizes the key takeaways from the project, and insights drawn from the curated data.

Contemporary medical science has traced thousands of clinical conditions to a genetic cause. Cancer, a life-threatening disease, also has genetic origins, and is considered among the leading causes of death across the globe. In fact, the World Health Organization (WHO) has reported that close to 10 million cancer related deaths annually, across the world.

Moreover, experts believe that there are over 7,000 different types of rare diseases (including some rare forms of cancer), most of which originate as a consequence of genetic anomalies. The majority of the aforementioned conditions are still considered incurable. As a result, these disease areas are characterized by a significant unmet need for curative interventions; and therefore, considered among the most lucrative opportunity areas for biopharmaceutical developers.

For example, ZOLGENSMA, a blockbuster product developed by Novartis, and indicated for the treatment of spinal muscular atrophy, generated net revenues of approximately USD 1.35 billion in 2021 alone. The first gene therapy trial was conducted in 1990, and it took almost three decades for the first of such interventions to enter the market.

Given recent developments in genetic manipulation, cell biology and molecular targeting, a number of highly specific interventions have been developed against prominent types of cancers and certain rare genetic conditions. Currently, there are over 20 cell and gene therapies approved for use in the United States alone.

During the COVID-19 pandemic, the pace of R&D in this field slowed down - a consequence of complex manufacturing protocols, extensive logistical considerations and supply chain-related concerns. However, the field still witnessed a considerable inflow of capital, with over USD 21 billion invested into various companies since the start of the pandemic.

With over 1,200 product candidates in various stages of development, experts suggest that, by 2025, the US FDA may start approving around 10 to 20 cell and gene therapy products, on an annual basis. It is likely that, over the next two decades, gene therapies facilitate the evolution of medical practice from a treatment-based paradigm to a prevention-focused approach.

Despite the fact that niche startups are spearheading the innovation in this domain, several big pharma players are also actively acquiring capabilities related to upcoming advanced therapy medical products (ATMPs). Prominent players in the field, such as Juno Therapeutics, AveXis, and Kite Pharma, have been acquired as a consequence of the rapid expertise building efforts of more established pharma companies.

Moreover, gene therapy-focused businesses that have gone public, have experienced considerable growth in share value as their respective products / product candidate progressed through the various stages of development. Taking into consideration both the historical and contemporary scenario, the cell and gene therapies market continues to present lucrative investment opportunities for both short- and long-term investors.

The report features the following details:

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

Link:
Global Cell and Gene Therapy Market Investor/Opportunities Report 2022: Evaluate the Current/Future Potential Over the Coming Decades - GlobeNewswire

This article was originally published here

Expert Rev Cardiovasc Ther. 2022 Jun 2. doi: 10.1080/14779072.2022.2085686. Online ahead of print.

ABSTRACT

INTRODUCTION: To date, the treatment option for tachyarrhythmia is classified into drug therapy, catheter ablation, and implantable device therapy. However, the efficacy of the antiarrhythmic drugs is limited. Although the indication of catheter ablation is expanding, several fatal tachyarrhythmias are still refractory to ablation. Implantable cardioverter-defibrillator increases survival, but it is not a curable treatment. Therefore, a novel therapy for tachyarrhythmias refractory to present treatments is desired. Gene therapy is being developed as a promising candidate for this purpose, and basic research and translational research have been accumulated in recent years.

AREAS COVERED: This paper reviews the current state of gene therapy for arrhythmias, including susceptible arrhythmias, the route of administration to the heart, and the type of vector to use. We also discuss the latest progress in the technology of gene delivery and genome editing.

EXPERT OPINION: Gene therapy is one of the most promising technologies for arrhythmia treatment. However, additional technological innovation to achieve safe, localized, homogeneous, and long-lasting gene transfer is required for its clinical application.

PMID:35655364 | DOI:10.1080/14779072.2022.2085686

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Gene therapy to terminate tachyarrhythmias - DocWire News

Dublin, May 30, 2022 (GLOBE NEWSWIRE) -- The "Cell and Gene Therapy Global Market Report 2022: By Product, By Application, By End-User" report has been added to ResearchAndMarkets.com's offering.

The global cell and gene therapy market is expected to grow from $6.58 billion in 2021 to $8.57 billion in 2022 at a compound annual growth rate (CAGR) of 30.2%.

The growth is mainly due to the companies resuming their operations and adapting to the new normal while recovering from the COVID-19 impact, which had earlier led to restrictive containment measures involving social distancing, remote working, and the closure of commercial activities that resulted in operational challenges.

The market is expected to reach $21.33 billion in 2026 at a CAGR of 25.6%.

Major players in the cell and gene therapy market are Gilead Sciences, Bristol-Myers Squibb, Novartis AG, Amgen, Merck, Organogenesis Holdings, Dendreon, Vericel, Bluebird Bio and Fibrocell Science.

The cell and gene therapy market consist of sales of cell and gene therapies by entities (organizations, sole traders and partnerships) that develop cell and gene therapies. Cell therapy refers to the transfer of intact, live cells that are originated from autologous or allogenic sources and gene therapy refers to the introduction, removal, or change in the genome for treating diseases. The market consists of revenue generated by the companies developing cell and gene therapy products by the sales of these products.

The main types of products in cell and gene therapy are cell therapy and gene therapy. Gene therapy is a branch of medicine that focuses on modifying the genetic material of cells to achieve a therapeutic effect or to heal disease by repairing or reconstructing damaged genetic material. The different applications include oncology, dermatology, musculoskeletal, others and involves various sectors such as hospitals, ambulatory surgical centers, cancer care centers, wound care centers, others.

Steady investment and consolidation in cell and gene therapies contributed to the growth of the cell and gene therapy (CGT) market. After recognizing the potential of the CGT market, 16 out of the 20 largest biopharma companies by revenue, added CGT products to their portfolio.

For instance, Merck invested $109 million in viral vector and gene therapy manufacturing in April 2020. Moreover, 12% of industrial clinical pipeline products and at least 16% of preclinical pipeline products consist of CGT. Steady investment and consolidation in CGT production capacity led to an increase in production capacity and also contributed to the growth of the market.

Limited reimbursements preventing patients from receiving treatments are expected to limit the growth of cell and gene therapy (CGT market. In 2019, Trinity Life Sciences, a life sciences solution provider, researched national and large regional commercial health insurance plans in the US.

It found that the confluence of increasing price, patient volume and number of CGTs on the market is likely to change the reimbursement model for CGTs and impact payer budgets by 5-10%. Payers realize that financing needs to be generated for cost management due to the uncertainty surrounding reimbursement of ancillary costs. Limited reimbursements and uncertain insurance plans are preventing patients from receiving high-cost CGT, which is expected to limit market growth.

Chimeric antigen receptor (CAR) T-cell therapy is shaping the cell and gene therapy (CGT) market. (CAR) T-cell therapy is a combination of cell and gene therapy in which T cells are collected from the patient's blood and are genetically engineered to produce modified receptors at their surface, known as chimeric antigen receptors (CARs). These modified T cells with special structures (receptors) are reinfused into the patient.

Then, the modified receptors of T cell help in targeting the surface antigen of the cancer cell that ultimately results in the killing of tumor cells in patients. In 2020, the US-FDA approved Bristol-Myers Squibb's two CAR-T cell therapies to treat lymphoma and multiple myeloma and is set to be launched.

Currently, FDA approved CAR-T cell therapy treatments like Tisagenlecleucel for the treatment of B-cell precursor acute lymphoblastic leukemia (ALL) in children and Axicabtagene ciloleucel for the treatment of adult patients with relapsed or refractory large B-cell lymphoma.

Key Topics Covered:

1. Executive Summary

2. Cell and Gene Therapy Market Characteristics

3. Cell and Gene Therapy Market Trends And Strategies

4. Impact Of COVID-19 On Cell and Gene Therapy

5. Cell and Gene Therapy Market Size And Growth5.1. Global Cell and Gene Therapy Historic Market, 2016-2021, $ Billion 5.1.1. Drivers Of The Market 5.1.2. Restraints On The Market 5.2. Global Cell and Gene Therapy Forecast Market, 2021-2026F, 2031F, $ Billion 5.2.1. Drivers Of The Market 5.2.2. Restraints On the Market

6. Cell and Gene Therapy Market Segmentation6.1. Global Cell and Gene Therapy Market, Segmentation By Product, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

6.2. Global Cell and Gene Therapy Market, Segmentation By Application, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

6.3. Global Cell and Gene Therapy Market, Segmentation By End User, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

7. Cell and Gene Therapy Market Regional And Country Analysis7.1. Global Cell and Gene Therapy Market, Split By Region, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion 7.2. Global Cell and Gene Therapy Market, Split By Country, Historic and Forecast, 2016-2021, 2021-2026F, 2031F, $ Billion

Companies Mentioned

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Global Cell and Gene Therapy Markets, Analysis & Forecasts, 2016-2021, 2021-2026F, 2031F - Benzinga - Benzinga

Dive Brief:

Treatment of SMA has transformed over the six years since Biogen first gained approval for Spinraza. The disease was previously considered fatal for many babies born with the mutation that causes it. Even for those with less severe forms of the disorder, there were lifelong disabilities that often required use of a wheelchair or mechanical ventilation.

Until Evrysdi's latest approval, Spinraza was the only drug approved for all patients. Novartis' Zolgensma, which replaces the defective genes to stimulate production of an important muscle protein, is approved for children younger than 2 years old. The company has faced setbacks in trying to complete the clinical studies necessary to win approval in patients older than that.

With SMA's incidence estimated as once per every 10,000 births, the three drugmakers are all targeting a small group of patients with their treatments. While oral and therefore more convenient, Evrysdi must be taken every day. Spinraza, by comparison, is administered once every four months after a two-month loading period through the more invasive intrathecal injection. Zolgensma is intended to be a one-time treatment, although with the long-term safety of gene therapy still unclear, some parents could be more cautious about using it for their babies.

Spinraza is the biggest seller, with $473 million in sales in the first quarter of 2022, compared to $363 million for Zolgensma and about $236 million for Evrysdi. When the two Swiss drugmakers reported earnings, Novartis disclosed that Zolgensma had been used in about 2,000 patients worldwide and Roche said Evrysdi had been used in more than 5,000. (Roche's number includes those treated in clinical trials and compassionate use, however.)

Sales of both Zolgensma and Evrysdi are growing, at the apparent expense of Spinraza, sales of which shrank by 9% in the first quarter compared to the same period last year.

With all three drugs now approved for use in the youngest infants and two of the three available more widely, a new question is how these treatments might be used in sequence. All three, however, are costly: Zolgensma's one-dose list price is around $2 million, while Spinraza's list price is $375,000 a year at the maintenance dose and Evrysdicosts $340,000 annually.

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Roche SMA drug approved in youngest infants, challenging Novartis and Biogen - BioPharma Dive