Blog
RSS FeedPosted by Michael Wonder on 11 Jan 2022
4D Molecular Therapeutics announces FDA fast track designation granted to 4D-125 for the treatment of X-linked retinitis pigmentosa
10 January 2022 - 4D Molecular Therapeutics announced that the U.S. FDA has granted fast track designation for 4D-125 for treatment ...
Posted by Michael Wonder on 18 Dec 2021
bluebird bio announces FDA priority review of biologics license application for eli-cel gene therapy for cerebral adrenoleukodystrophy in patients without a matched sibling donor
17 December 2021 - If approved, eli-cel will be the first and only gene therapy for the treatment of cerebral adrenoleukodystrophy, ...
Posted by Michael Wonder on 22 Nov 2021
bluebird bio Announces FDA priority review of biologics license application for beti-cel gene therapy for patients with β-thalassemia who require regular red blood cell transfusions
22 November 2021 - FDA set PDUFA date of 20 May 2022. ...
Posted by Michael Wonder on 03 Nov 2021
AvroBio receives rare paediatric disease designation from the U.S. FDA for AVR-RD-05, a gene therapy for mucopolysaccharidosis type II or Hunter syndrome
3 November 2021 - AVROBIO today announced that the U.S. FDA has granted rare paediatric disease designation to AVR-RD-05, its ...
Posted by Michael Wonder on 27 Oct 2021
FDA, NIH and 15 private organisations join forces to increase effective gene therapies for rare diseases
27 October 2021 - The U.S. FDA, the National Institutes of Health, 10 pharmaceutical companies and five non-profit organisations have partnered ...
Posted by Michael Wonder on 23 Oct 2021
Sio Gene Therapies announces granting of FDA fast track designation for investigational AXO-AAV-GM1 (AAV9-GLB1) gene therapy in patients with GM1 gangliosidosis
21 October 2021 - Sio Gene Therapies today announced that the U.S. FDA has granted fast track designation to AXO-AAV-GM1, its ...
Posted by Michael Wonder on 12 Oct 2021
GenSight Biologics announces FDA grant of fast track designation for optogenetic therapy GS030 as treatment for retinitis pigmentosa
12 October 2021 - GenSight Biologics today announced that the U.S. FDA has granted fast track designation to GS030, which combines ...
Posted by Michael Wonder on 21 Sep 2021
bluebird bio submits biologics license application to FDA for betibeglogene autotemcel (beti-cel) gene therapy for patients with β-thalassaemia who require regular red blood cell transfusions
21 September 2021 - Biologics license application submission based on data from Phase 1/2 and Phase 3 Northstar studies, which ...
Posted by Michael Wonder on 10 Sep 2021
Decibel Therapeutics receives orphan drug and rare paediatric disease designations for DB-OTO for the treatment of otoferlin related congenital hearing loss
9 September 2021 - Decibel Therapeutics today announced that the U.S. FDA has granted both orphan drug designation and rare paediatric ...
Posted by Michael Wonder on 08 Jul 2021
Lysogene announces FDA fast track designation for LYS-GM101 gene therapy for the treatment of GM1 gangliosidosis
8 July 2021 - Lysogene today announced that the U.S. FDA has granted fast track designation to its LYS-GM101 program, which ...
Posted by Michael Wonder on 28 Jun 2021
AskBio receives FDA fast track designation for LION-101, a novel investigational AAV gene therapy for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)
28 June 2021 - LGMD2I/R9 is a rare form of muscular dystrophy with no approved therapies. ...
Posted by Michael Wonder on 22 Jun 2021
Targovax receives fast track designation for ONCOS-102 in melanoma
22 June 2021 - Targovax today announces that its lead clinical candidate ONCOS-102 has received fast track designation in PD-1-refractory ...
Posted by Michael Wonder on 19 May 2021
FDA: flexibility okay for gaps in gene therapy trials
18 May 2021 - The US FDA is not amenable to changing study outcome measures or sample sizes for gene therapy ...
Posted by Michael Wonder on 14 May 2021
BridgeBio Pharma receives FDA fast track designation for investigational gene therapy for congenital adrenal hyperplasia
14 May 2021 - BridgeBio Pharma today announced that the U.S. FDA granted fast track designation to BBP-631, an investigational adeno-associated ...
Posted by Michael Wonder on 20 Apr 2021
LEXEO Therapeutics announces FDA fast track designation granted to LX1001 for the treatment of APOE4 associated Alzheimer’s disease
20 April 2021 - LX1001 is the first investigational gene therapy being evaluated to address the underlying genetics of Alzheimer’s disease. ...