For the upcoming gene therapy candidates getting ready for BLA submission in the US, the first on the list is Krystal Biotech’s beremagene geperpavec, a topicalCOL7A1-expressing gene therapy for the treatment of dystrop...
For that reason, I’ve made and kept updated a comprehensive list of FDA Approved Cell and Gene Therapies, which is the focus of today’s post. These include FDA-approved stem cell therapies. I’ve updated the list now in 2025 to includeCasgevyandLyfgenia, approved cell and gene therapies...
“Today we celebrate the decades of work by many individuals to bring gene therapy from science fiction to clinical reality for patients with inherited retinal disease. Gene therapy will now join the list of innovative therapies used routinely at Mass. Eye and Ear to treat patients, said Joan ...
Here’s my list of the 85 granted RMATs that I could find in the public domain. Abeona Therapeutics(EB-101 Gene Therapy in Epidermolysis Bullosa) Abeona Therapeutics(ABO-102 Gene Therapy in MPS IIIA) AdaptImmune(ADP‑A2M4 for the Treatment of Synovial Sarcoma) AdaptImmune(ADP-A2M4CD8 for t...
the CGT field has undergone significant advancements since then. Recognizing this evolution, the FDA recently released a draft guidance titled "Potency Assurance for Cellular and Gene Therapy Products" in December 2023. This new document moves beyond just focusing on potency tests and adopts a more ...
Tab.1 List of Companies with over than 10 Approved Orphan Drugs 在700个获批孤儿药中,开发策略2类的药物最多,有587个,占83.9%。以非罕见病适应证拓展的第3类药物最少,仅33个,但其中有3个被列入了2022年畅销药物前10名,年销售额均超过了90亿美元。例如,修美乐(阿达木单抗)是2022年孤儿药中销售额最高...
Among some of the reasons I’m so optimistic about our shared future are new scientific opportunities, like gene therapy and regenerative medicine that give us plausible hope that we might be able to actually cure many more diseases...
Gene therapy products. Human gene therapy/gene transfer is the administration of nucleic acids, viruses, or genetically engineered microorganisms that mediate their effect by transcription and/or translation of the transferred genetic material, and/or by integrating into the host genome. Cells may be ...
This guidance excludes all vaccines, whole cells, whole blood and plasma, blood and plasma derivatives (plasma fractionation), and gene therapy APIs. However, it does include APIs that are produced using blood or plasma as raw materials. Note that cell substrates (mammalian, plant, insect or ...
2022 FDA Drug Approval List, 2022 Biological Approvals and Approved Cellular and Gene Therapy Products Posted in cell-based therapy, Drug Discovery Chemistry, FDA, FDA, CE Mark & Global Regulatory Affairs: process management and strategic planning - GCP, GLP, ISO 14155, Pharmaceutical Discovery, ...