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cadth.ca/dv/ieht/gene-therapy-overview-approved-and-pipeline-technologies

Gene therapy has been an active area of research for at least the past two decades. 13 As of January 28, 2018, ten gene therapy products have been approved for marketing in at least one country in the world.

globalforum.diaglobal.org/issue/january-2019/us-fda-recommendations-for-gene...

Human gene therapy (GT) is a treatment approach that seeks to modify or manipulate a person’s genes to treat or cure disease. GT can work in several ways including by (1) replacing a disease-causing gene with a healthy copy of the gene; (2) inactivating a disease-causing gene that is not functioning properly; or (3) introducing a new or modified gene into the body to help treat a disease. GT ...

www.sciencedirect.com/science/article/abs/pii/S0734975019302022

Consequently, this review summarizes the gene therapy drugs approved worldwide from 1998 to 2019 in details, including names, indications, dates of approval, companies, vectors, the applied technologies and mechanisms of gene therapy drugs, etc. Furthermore, the gene therapy drugs were classified and addressed in accordance with the employed ...

govzilla.com/blog/2020/09/pharma-pcoming-webinar-cmc-considerations-for-cell...

Cell and gene therapy products have only been around a short time. Yet the market for these products continues to climb exponentially. In fact, currently, 18 cell and gene therapy products have been approved FDA and over 37,000 clinical trials for cell therapies are listed on clinicaltrials.gov.

www.frontiersin.org/articles/10.3389/fphar.2019.00921

Advanced therapy medicinal products (ATMPs) are a fast-growing field of innovative therapies. The European Union (EU) and the United States (US) are fostering their development. For both regions, ATMPs fall under the regulatory framework of biological products, which determines the legal basis for their development. Sub-classifications of advanced therapies are different between regions, while ...

www.asgct.org/ASGCT/media/about/Approved-Vatsan-ASGCT-2019.pdf

Managing Post-Approval Changes In Gene Therapy Products Ramjay S. Vatsan, Ph.D., CQA Gene Therapy Team Leader Division of Cellular and Gene Therapies Office of Tissue and Advanced Therapies Center for Biologics Evaluation and Research/FDA Post-Approval Commercialization Workshop Post-Approval Change Management and Overall Impact on ...

www.liebertpub.com/doi/abs/10.1089/humc.2018.201

The EMA withdrew the authorization for two tissue engineering products, one cell therapy and one gene therapy, and New Zealand lapsed approval of one cell therapy. Most products were first authorized after 2010, including 10 (83.3%) gene therapies, 13 (72.2%) cell therapies, and 13 (56.5%) tissue-engineered products.

www.cbsnews.com/news/gene-therapy-drug-rpe65-blindness-luxturna-spark-therapeutics

The injection, called Luxturna, is the first gene therapy approved by the Food and Drug Administration in which a corrective gene is given directly to patients. The gene mutation interferes with ...

www.risingtidebio.com/gene-therapy-clinical-trials

Zolgensma was then approved in 2019 to treat Spinal Muscular Atrophy. There are now many additional companies moving aggressively to develop their pipelines of gene therapy products. The specifics of each gene therapy is slightly different, however the main challenge is to replace a missing or non-functional protein with a functional protein.

www.reviewofophthalmology.com/article/fda-approves-firstever-retinal-gene-therapy

“This is the second gene therapy approved by the FDA,” she notes, “but it’s the first one to target a genetic disease, and the first in which the gene is delivered directly into the person, rather than first being delivered into a cell in a dish. It’s also the first gene therapy approved worldwide for a retinal disease.