Blog
RSS FeedPosted by Michael Wonder on 08 Aug 2018
The FDA is poised to approve the first-ever drug that mutes disease-causing genes
7 August 2018 - After a decades-long wait, the FDA is on the brink of approving a landmark rare disease ...
Posted by Michael Wonder on 29 Jul 2018
Ultragenyx announces first patient dosed in Phase 1/2 study of DTX401, a gene therapy for the treatment of glycogen storage disease Type Ia; FDA grants fast track designation to gene therapy program
26 July 2018 - Ultragenyx today announced that the first patient has been dosed in the Phase 1/2 study of DTX401, ...
Posted by Michael Wonder on 11 Jul 2018
Statement from FDA Commissioner on agency’s efforts to advance development of gene therapies
11 July 2018 - Once just a theory, gene therapies are now a therapeutic reality for some patients. ...
Posted by Michael Wonder on 23 Jun 2018
Voyager Therapeutics announces FDA regenerative medicine advanced therapy designation granted for VY-AADC for the treatment of Parkinson’s Disease
21 June 2018 - Voyager Therapeutics today announced that the U.S. FDA granted regenerative medicine advanced therapy designation for Voyager’s VY-AADC ...
Posted by Michael Wonder on 17 Jun 2018
Nightstar Therapeutics receives regenerative medicine advanced therapy designation for NSR-REP1 in choroideremia
14 June 2018 - First gene therapy RMAT designation for an inherited retinal disease. ...
Posted by Michael Wonder on 13 Jun 2018
RegenxBio receives FDA fast track designation for RGX-111 gene therapy for the treatment of mucopolysaccharidosis type I
12 June 2018 - Phase I clinical trial expected to enroll children and adults with MPS I. ...
Posted by Michael Wonder on 25 May 2018
U.S. FDA grants fast track designation for Krystal Biotech’s KB103 for the treatment of dystrophic epidermolysis bullosa
24 May 2018 - Krystal Biotech today announces that the U.S. FDA has granted fast track designation to KB103 for ...
Posted by Michael Wonder on 24 May 2018
FDA grants breakthrough therapy designation to Lenti-D for the treatment of cerebral adrenoleukodystrophy
23 May 2018 - bluebird bio today announced that the U.S. FDA has granted breakthrough therapy designation to Lenti-D for ...
Posted by Michael Wonder on 23 May 2018
Gene therapies that could transform diseases get easier FDA path
23 May 2018 - Haemophilia treatments could be first under proposed method. ...
Posted by Michael Wonder on 17 May 2018
Myonexus Therapeutics receives FDA rare paediatric drug designation for pioneering treatment of limb girdle muscular dystrophy Type 2E
16 May 2018 - Rare paediatric disease designation for MYO-101 program reflects compelling data and enables priority eeview voucher eligibility. ...
Posted by Michael Wonder on 06 May 2018
Ferring signs global agreement to commercialise novel gene therapy for bladder cancer patients
3 May 2018 - The US FDA has granted fast track and breakthrough therapy designations to rAd-IFN/Syn3. ...
Posted by Michael Wonder on 03 May 2018
RegenxBio receives FDA fast track designation for RGX-121 gene therapy for the treatment of mucopolysaccharidosis Type II
2 May 2018 - Phase I/II clinical trial expected to enrol children with MPS II. ...
Posted by Michael Wonder on 23 Apr 2018
Abeona announces FDA grants RMAT designation to ABO-102 gene therapy in MPS IIIA
23 April 2018 - First gene therapy using AAV approach granted regenerative medicine advanced therapy designation. ...
Posted by Michael Wonder on 23 Apr 2018
MeiraGTx announces AAV-RPGR granted fast track designation by U.S. FDA for treatment of X-linked retinitis pigmentosa due to RPGR deficiency
23 April 2018 - MeiraGTx Limited today announced that the U.S. FDA has granted fast track designation for AAV-RPGR for the ...
Posted by Michael Wonder on 16 Mar 2018
Abeona Therapeutics receives FDA rare paediatric disease designation for ABO-202 gene therapy program in CLN1 disease
15 March 2018 - Company’s fourth gene therapy program to receive rare paediatric disease designation, enabling priority review voucher. ...