2 April 2024 - Positive, previously disclosed results from the global Phase 1/2 trial demonstrated genetic and phenotypic correction combined ...
23 February 2024 - The EMA has recommended granting a marketing authorisation in the European Union for a new therapy ...
13 February 2024 - Over 8,000 patients 12 years of age and older with severe sickle cell disease or transfusion-dependent ...
15 December 2023 - If approved by the European Commission, patients 12 years of age and older with severe sickle cell ...
13 October 2023 - Intellia Therapeutics today announced that the EMA has granted Priority Medicine (PRIME) designation to NTLA-2002 for ...
31 May 2023 - Rocket Pharmaceuticals today announced that the EMA has granted Priority Medicines (PRIME) designation to RP-A501, the ...
3 May 2023 - Gene therapies hold promise for treating a wide range of diseases. Innovative companies such as bluebird bio ...
20 April 2023 - GenSight Biologics today announces that the Committee for Advanced Therapies (CAT) of the CHMP of the EMA ...
7 March 2023 - Genethon today announced that the EMA has granted PRIME (Priority Medicines) status to the gene therapy, ...
20 February 2023 - Hemgenix underscores CSL's promise to deliver life-changing innovations that have the potential to help patients lead ...
17 January 2023 - FBX-101 is granted priority medicines (PRIME) designation by the EMA after review of Phase 1/2 RESKUE clinical ...
16 December 2022 - Regulatory milestone moves CSL one step closer to bringing the promise of gene therapy for haemophilia ...
5 December 2022 - EMA acceptance follows FDA's acceptance of tofersen new drug application earlier this year. ...
20 September 2022 - Amvuttra demonstrated halting or reversal in neuropathy impairment with subcutaneous administration once every three months. ...
24 August 2022 - Maintains orphan drug designation in the EU providing 10 years of market exclusivity. ...