18 September 2023 - PDUFA date set for 18 March 2024. ...
12 September 2023 - Ferring launches ABLE-41 Real World Evidence Study, a patient registry to explore early use, experiences and outcomes ...
29 August 2023 - Bloomsbury Genetic Therapies Limited announced today that the US FDA has granted rare paediatric disease designation for ...
24 August 2023 - TSHA-102 has also received orphan drug and rare paediatric disease designations from the US FDA and ...
23 August 2023 - Comanche Biopharma today announced that they have received fast track designation from the US FDA for ...
7 August 2023 - HG004 is a one time, direct to RPE treatment of inherited retinal disease caused by mutations in ...
20 July 2023 - EBT-101 is a potentially curative, one time CRISPR based treatment which makes two cuts in integrated retroviral ...
11 July 2023 - Synlogic today announced that the US FDA has granted fast track designation to labafenogene marselecobac (previously known ...
7 July 2023 - NS Pharma announced today the US FDA has granted rare paediatric disease designation to NS-089/NCNP-02 (brogidirsen) an ...
1 July 2023 - After quite the regulatory process, with Roctavian (valoctocogene roxaparvovec) BioMarin has a potential blockbuster. On 29 ...
29 June 2023 - FDA issues simultaneous approval of Roctavian (valoctocogene roxaparvovec-rvox), BioMarin's gene therapy for severe haemophilia A. ...
29 June 2023 - Roctavian's approval was based on durability, efficacy and safety results from the largest and longest Phase 3 ...
27 June 2023 - Submissions based on positive Phase 3 data from BENEGENE-2 trial. ...
22 June 2023 - Today, the US FDA approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 ...
21 June 2023 - PDUFA date set for 20 December 2023. ...