18 May 2021 - The US FDA is not amenable to changing study outcome measures or sample sizes for gene therapy ...
14 May 2021 - BridgeBio Pharma today announced that the U.S. FDA granted fast track designation to BBP-631, an investigational adeno-associated ...
22 April 2021 - Rapid publication following approval. ...
20 April 2021 - LX1001 is the first investigational gene therapy being evaluated to address the underlying genetics of Alzheimer’s disease. ...
27 March 2021 - The U.S. FDA approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple ...
10 March 2021 - Onasemnogene abeparvovec is a new gene therapy for children with spinal muscular atrophy. ...
9 March 2021 - Enrollment complete in Phase 1/2 clinical trial of RP-L201 for the treatment of LAD-I. ...
8 March 2021 - FDA designation will facilitate development and expedited review of company’s lead gene therapy product candidates for ...
8 March 2021 - RMAT designation granted by FDA during bleeding disorders awareness month. ...
25 February 2021 - Today, the U.S. FDA granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular ...
26 January 2021 - MeiraGTx today announced that the U.S. FDA has granted fast track designation to its AAV-CNGA3 gene therapy ...
19 January 2021 - Designations reinforce unmet need for treatment options for patients with rare form of genetic epilepsy. ...
21 December 2020 - BLA supported by strong Phase 3 VISTA trial data and positive analytical comparability data. ...
18 December 2020 - The U.S. FDA has not raised any concerns related to the efficacy or safety of inclisiran. The ...
16 December 2020 - Zolgensma is designed to address the genetic root cause of SMA by replacing the missing or defective ...