23 August 2022 - GTP-506, a potential single-dose gene editing therapy designed to restore metabolic function in patients suffering with OTC ...
13 June 2022 - First and only FDA approved treatment demonstrating reversal in neuropathy impairment with subcutaneous administration once every three ...
24 May 2022 - If approved, etranacogene dezaparvovec would be the first gene therapy option for people living with haemophilia B. ...
17 May 2022 - The US FDA's top biologics regulator said the use of a “playbook” or platform approach ...
10 May 2022 - Amicus Therapeutics today announced that the U.S. FDA has extended the review period by 90 days ...
29 March 2022 - ASC Therapeutics has received from the U.S. FDA the fast track designation for ASC618, a second-generation gene ...
16 March 2022 - The U.S. FDA has issued two draft guidances addressing the development of human gene therapy products that ...
16 February 2022 - Initiation of Phase 1/2 clinical trial expected in the second half of 2022. ...
24 January 2022 - Interim data submitted to the FDA show majority of pre-symptomatic babies treated with Evrysdi for at least ...
18 January 2022 - FDA PDUFA goal dates for both therapies extended by three months. ...
10 January 2022 - 4D Molecular Therapeutics announced that the U.S. FDA has granted fast track designation for 4D-125 for treatment ...
17 December 2021 - If approved, eli-cel will be the first and only gene therapy for the treatment of cerebral adrenoleukodystrophy, ...
22 November 2021 - FDA set PDUFA date of 20 May 2022. ...
3 November 2021 - AVROBIO today announced that the U.S. FDA has granted rare paediatric disease designation to AVR-RD-05, its ...
27 October 2021 - The U.S. FDA, the National Institutes of Health, 10 pharmaceutical companies and five non-profit organisations have partnered ...