20 June 2023 - Hemgenix, the first and only FDA approved gene therapy for haemophilia B, has been proven to ...
15 June 2023 - The US FDA should reject the accelerated approval of the gene therapy SRP-9001 (Sarepta Therapeutics) for ...
8 June 2023 - FDA grants priority review for severe sickle cell disease and standard review for transfusion-dependent beta thalassemia. ...
8 June 2023 - Rocket Pharmaceuticals today announced that the US FDA has granted fast track and orphan drug designations ...
1 June 2023 - Arcturus Therapeutics today announced that the US FDA has granted fast track designation to ARCT-810, the Company’s ...
24 May 2023 - New regulatory action date is 22 June 2023. ...
23 May 2023 - CAMPSIITE trial is enrolling mucopolysaccharidosis type II patients as part of a pivotal program that incorporates ...
23 May 2023 - Rocket Pharmaceuticals today announced that the US FDA has granted regenerative medicine advanced therapy designation to ...
22 May 2023 - Sangamo Therapeutics today announced that the US FDA has granted fast track designation to isaralgagene civaparvovec, or ...
19 May 2023 - Today, the US FDA approved Vyjuvek, a herpes-simplex virus type 1 vector-based gene therapy, for the treatment ...
15 May 2023 - SiSaf announces that SIS-101-ADO, its siRNA therapeutic, has been granted orphan drug designation and rare paediatric disease ...
25 April 2023 - FDA granted accelerated approval of Qalsody based on a reduction of neurofilament, a marker of neurodegeneration. ...
24 April 2023 - BLA submission based on data from the largest and most mature clinical development program for any ...
11 April 2023 - RGX-202 is a potential one-time AAV therapeutic for the treatment of Duchenne and includes an optimised transgene ...
3 April 2023 - EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review ...