21 October 2021 - Sio Gene Therapies today announced that the U.S. FDA has granted fast track designation to AXO-AAV-GM1, its ...
12 October 2021 - GenSight Biologics today announced that the U.S. FDA has granted fast track designation to GS030, which combines ...
21 September 2021 - Biologics license application submission based on data from Phase 1/2 and Phase 3 Northstar studies, which ...
9 September 2021 - Decibel Therapeutics today announced that the U.S. FDA has granted both orphan drug designation and rare paediatric ...
8 July 2021 - Lysogene today announced that the U.S. FDA has granted fast track designation to its LYS-GM101 program, which ...
28 June 2021 - LGMD2I/R9 is a rare form of muscular dystrophy with no approved therapies. ...
22 June 2021 - Targovax today announces that its lead clinical candidate ONCOS-102 has received fast track designation in PD-1-refractory ...
18 May 2021 - The US FDA is not amenable to changing study outcome measures or sample sizes for gene therapy ...
14 May 2021 - BridgeBio Pharma today announced that the U.S. FDA granted fast track designation to BBP-631, an investigational adeno-associated ...
20 April 2021 - LX1001 is the first investigational gene therapy being evaluated to address the underlying genetics of Alzheimer’s disease. ...
27 March 2021 - The U.S. FDA approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple ...
9 March 2021 - Enrollment complete in Phase 1/2 clinical trial of RP-L201 for the treatment of LAD-I. ...
8 March 2021 - FDA designation will facilitate development and expedited review of company’s lead gene therapy product candidates for ...
8 March 2021 - RMAT designation granted by FDA during bleeding disorders awareness month. ...
25 February 2021 - Today, the U.S. FDA granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular ...