16 April 2024 - Lexeo Therapeutics today announced the US FDA has granted fast track designation to LX2006, the company’s AAVrh.10hFXN ...
2 April 2024 - Positive, previously disclosed results from the global Phase 1/2 trial demonstrated genetic and phenotypic correction combined ...
1 April 2024 - Exa-cel is the first CRISPR-based gene-edited therapy to be submitted for Health Canada review. ...
1 April 2024 - Site initiations scheduled for April; patient dosing expected to begin in Q2 ,2024. ...
19 March 2024 - Health Canada regulatory decision on tofersen new drug submission expected in early 2025. ...
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 ...
23 February 2024 - The EMA has recommended granting a marketing authorisation in the European Union for a new therapy ...
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. ...
13 February 2024 - Over 8,000 patients 12 years of age and older with severe sickle cell disease or transfusion-dependent ...
8 February 2024 - Ionis Pharmaceuticals announced today that the US FDA has granted fast track designation to Ionis and ...