Blog
RSS FeedPosted by Michael Wonder on 19 May 2021
FDA: flexibility okay for gaps in gene therapy trials
18 May 2021 - The US FDA is not amenable to changing study outcome measures or sample sizes for gene therapy ...
Posted by Michael Wonder on 14 May 2021
BridgeBio Pharma receives FDA fast track designation for investigational gene therapy for congenital adrenal hyperplasia
14 May 2021 - BridgeBio Pharma today announced that the U.S. FDA granted fast track designation to BBP-631, an investigational adeno-associated ...
Posted by Michael Wonder on 22 Apr 2021
TGA publishes AusPAR for Zolgensma
22 April 2021 - Rapid publication following approval. ...
Posted by Michael Wonder on 20 Apr 2021
LEXEO Therapeutics announces FDA fast track designation granted to LX1001 for the treatment of APOE4 associated Alzheimer’s disease
20 April 2021 - LX1001 is the first investigational gene therapy being evaluated to address the underlying genetics of Alzheimer’s disease. ...
Posted by Michael Wonder on 27 Mar 2021
FDA approves first cell-based gene therapy for adult patients with multiple myeloma
27 March 2021 - The U.S. FDA approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple ...
Posted by Michael Wonder on 10 Mar 2021
TGA approves Zolgensma
10 March 2021 - Onasemnogene abeparvovec is a new gene therapy for children with spinal muscular atrophy. ...
Posted by Michael Wonder on 09 Mar 2021
Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy designation for RP-L201 gene therapy for leukocyte adhesion deficiency-I
9 March 2021 - Enrollment complete in Phase 1/2 clinical trial of RP-L201 for the treatment of LAD-I. ...
Posted by Michael Wonder on 08 Mar 2021
U.S. FDA grants fast track designation to three Passage Bio gene therapy candidates targeting rare CNS disorders
8 March 2021 - FDA designation will facilitate development and expedited review of company’s lead gene therapy product candidates for ...
Posted by Michael Wonder on 08 Mar 2021
BioMarin announces FDA regenerative medicine advanced therapy designation granted to valoctocogene roxaparvovec, investigational gene therapy for haemophilia A
8 March 2021 - RMAT designation granted by FDA during bleeding disorders awareness month. ...
Posted by Michael Wonder on 25 Feb 2021
FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation
25 February 2021 - Today, the U.S. FDA granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular ...
Posted by Michael Wonder on 26 Jan 2021
MeiraGTx announces AAV-CNGA3 granted fast track designation by U.S. FDA for treatment of achromatopsia
26 January 2021 - MeiraGTx today announced that the U.S. FDA has granted fast track designation to its AAV-CNGA3 gene therapy ...
Posted by Michael Wonder on 19 Jan 2021
Taysha Gene Therapies receives rare paediatric disease and orphan drug designations for TSHA-105 for the treatment of epilepsy caused by SLC13A5 deficiency
19 January 2021 - Designations reinforce unmet need for treatment options for patients with rare form of genetic epilepsy. ...
Posted by Michael Wonder on 21 Dec 2020
Sesen Bio submits completed biologics license application to the FDA for Vicineum and has requested priority review
21 December 2020 - BLA supported by strong Phase 3 VISTA trial data and positive analytical comparability data. ...
Posted by Michael Wonder on 18 Dec 2020
Novartis receives complete response letter from U.S. FDA for inclisiran
18 December 2020 - The U.S. FDA has not raised any concerns related to the efficacy or safety of inclisiran. The ...
Posted by Michael Wonder on 16 Dec 2020
Health Canada approves Zolgensma, the one-time gene therapy for paediatric patients with spinal muscular atrophy
16 December 2020 - Zolgensma is designed to address the genetic root cause of SMA by replacing the missing or defective ...