20 March 2024 - A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a ...
19 March 2024 - BLA submitted to FDA for Upstaza for the treatment of AADC deficiency - ...
18 March 2024 - Today, the US FDA approved Lenmeldy (atidarsagene autotemcel), the first FDA approved gene therapy indicated for the ...
13 March 2024 - PepGen today announced that the US FDA granted both orphan drug and rare paediatric disease designations for ...
14 February 2024 - In the Phase 1/2A clinical trial for SRD-001, an adeno-associated virus mediated first in human gene therapy ...
16 February 2024 - Priority review granted, with a review goal date of 21 June 2024. ...
13 February 2024 - New Prescription Drug User Fee Act date of 30 June 2024. ...
8 February 2024 - Ionis Pharmaceuticals announced today that the US FDA has granted fast track designation to Ionis and ...
29 January 2024 - Ascidian Therapeutics today announced that the US FDA has granted fast track ...
16 January 2023 - Atsena Therapeutics today announced the US FDA has granted rare paediatric disease designation to ATSN-101, the company’s ...
16 January 2024 - Approximately 1,000 patients in the US 12 years of age and older are now eligible for ...
13 November 2023 - New medicines to treat sickle cell disease and beta thalassaemia, two genetic blood disorders, will make headlines ...
22 December 2023 - Sarepta has also submitted the EMBARK post-marketing requirement to the FDA seeking conversion of the Elevidys accelerated ...
21 December 2023 - US FDA approval based on NEURO-TTRansform Phase 3 results showing Wainua demonstrated consistent and sustained benefit improving ...
19 December 2023 - Ocugen today announced that the FDA has granted regenerative medicine advanced therapy designation to Ocugen’s investigational product ...