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...
BioMarin’s gene therapy candidate, valoctocogene roxaparvovec, is also an AAV5-based gene therapy preparing for BLA filing this year. The therapy contains a B-domain-deletedFVIIIgene and a liver-specific promoter, for...
33.https://www.fda.gov/drugs/resources-information-approved-drugs/fda-disco-burst-edition-fda-approvals-talvey-talquetamab-tgvs-relapsed-or-refractory-multiple 34.https://go.drugbank.com/drugs/DB15766 35.https://www.drugs.com/newdrugs/fda-approves-...
图1 批准孤儿药的年度数量(自1983-01-04至2023-06-30) Fig.1 Annual Numbers of Approved Orphan Drugs(from 1983-01-04 to 2023-06-30) 表1 获批孤儿药数量超过10个的企业名单 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,...
a condition that leads to vision loss and complete blindness. The treatment, Luxturna (voretigene neparvovec-rzyl), is the first and only FDA-approved gene therapy treatment for an inherited disease. It wasapproved last Decemberwith the inaugural procedure taking place on March 20, 2018, on...
Depending on severity, a hemophilia B patient can require multiple infusions of factor IX on a weekly basis to prevent a bleed. However, after the one-time Hemgenix® infusion, the gene therapy is expected to enable the individu...
Approved Cellular and Gene Therapy Products Below is a list of licensed products from the Office of Tissues and Advanced Therapies (OTAT). Approved Products ABECMA (idecabtagene vicleucel)Celgene Corporation, a Bristol-Myers Squibb Company ADSTILADRINFerring Pharmaceuticals A/S ALLOCORD (HPC, Cord...
List of Cleared or Approved Companion Diagnostic Devices (In Vitro and Imaging Tools) Drug Trade Name (Generic Name) Erbitux (cetuximab); BLA 125084; DAKO EGFR PharmDx Kit P030044S001-S002 Dako North America, Inc. The EGFR pharmDx? assay is a qualitative immunohistochemical (IHC) kit system ...
Production and laboratory control records of noncritical process steps can be reviewed by qualified production personnel or other units following procedures approved by the quality unit(s). 在一批原料药放行或分发之前,关键工序的批生产记录和实验室控制记录应当由质量部门审核和批准。非关键性工序的生产和...
The idea of gene therapy has been discussed in the medical literature since as early as the 1970s. In 1972, Friedman and Roblin proposed that it was theoretically possible to introduce “good” DNA to replace defective DNA.1 Over the years, a number of gene therapy clinical trials emerged ...