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RSS FeedPosted by Michael Wonder on 09 Jun 2023
FDA accepts biologics license applications for exagamglogene autotemcel (exa-cel) for severe sickle cell disease and transfusion-dependent beta thalassaemia
8 June 2023 - FDA grants priority review for severe sickle cell disease and standard review for transfusion-dependent beta thalassemia. ...
Posted by Michael Wonder on 08 Jun 2023
Rocket Pharmaceuticals receives FDA fast track and orphan drug designations for RP-A601 gene therapy for PKP2 arrhythmogenic cardiomyopathy
8 June 2023 - Rocket Pharmaceuticals today announced that the US FDA has granted fast track and orphan drug designations ...
Posted by Michael Wonder on 01 Jun 2023
Arcturus Therapeutics receives US FDA fast track designation for ARCT-810, mRNA therapeutic candidate for ornithine transcarbamylase deficiency
1 June 2023 - Arcturus Therapeutics today announced that the US FDA has granted fast track designation to ARCT-810, the Company’s ...
Posted by Michael Wonder on 01 Jun 2023
Rocket Pharmaceuticals receives EMA Priority Medicines (PRIME) designation for RP-A501 gene therapy for Danon disease
31 May 2023 - Rocket Pharmaceuticals today announced that the EMA has granted Priority Medicines (PRIME) designation to RP-A501, the ...
Posted by Michael Wonder on 24 May 2023
Sarepta Therapeutics announces update on regulatory review of SRP-9001
24 May 2023 - New regulatory action date is 22 June 2023. ...
Posted by Michael Wonder on 23 May 2023
RegenxBio receives FDA regenerative medicine advanced therapy designation for RGX-121 gene therapy for Hunter syndrome
23 May 2023 - CAMPSIITE trial is enrolling mucopolysaccharidosis type II patients as part of a pivotal program that incorporates ...
Posted by Michael Wonder on 23 May 2023
Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy designation for RP-L301 gene therapy for pyruvate kinase deficiency
23 May 2023 - Rocket Pharmaceuticals today announced that the US FDA has granted regenerative medicine advanced therapy designation to ...
Posted by Michael Wonder on 22 May 2023
Sangamo Therapeutics receives US FDA fast track designation for isaralgagene civaparvovec for the treatment of Fabry disease
22 May 2023 - Sangamo Therapeutics today announced that the US FDA has granted fast track designation to isaralgagene civaparvovec, or ...
Posted by Michael Wonder on 19 May 2023
FDA approves first topical gene therapy for treatment of wounds in patients with dystrophic epidermolysis bullosa
19 May 2023 - Today, the US FDA approved Vyjuvek, a herpes-simplex virus type 1 vector-based gene therapy, for the treatment ...
Posted by Michael Wonder on 16 May 2023
FDA grants SiSaf’s innovative siRNA therapy SIS-101-ADO orphan drug designation and rare paediatric disease designation for the treatment of autosomal dominant osteopetrosis
15 May 2023 - SiSaf announces that SIS-101-ADO, its siRNA therapeutic, has been granted orphan drug designation and rare paediatric disease ...
Posted by Michael Wonder on 04 May 2023
Gene therapies are still hampered by substantial delays between approval and launch
3 May 2023 - Gene therapies hold promise for treating a wide range of diseases. Innovative companies such as bluebird bio ...
Posted by Michael Wonder on 25 Apr 2023
FDA grants accelerated approval for Qalsody (tofersen) for SOD1-ALS, a major scientific advancement as the first treatment to target a genetic cause of ALS
25 April 2023 - FDA granted accelerated approval of Qalsody based on a reduction of neurofilament, a marker of neurodegeneration. ...
Posted by Michael Wonder on 25 Apr 2023
bluebird bio submits biologics license application to FDA for lovotibeglogene autotemcel (lovo-cel) for patients with sickle cell disease 12 years and older with a history of vaso-occlusive events
24 April 2023 - BLA submission based on data from the largest and most mature clinical development program for any ...
Posted by Michael Wonder on 21 Apr 2023
GenSight Biologics withdraws its EMA application for Lumevoq
20 April 2023 - GenSight Biologics today announces that the Committee for Advanced Therapies (CAT) of the CHMP of the EMA ...
Posted by Michael Wonder on 11 Apr 2023
RegenxBio receives FDA fast track designation for RGX-202, a novel gene therapy candidate for the treatment of Duchenne muscular dystrophy
11 April 2023 - RGX-202 is a potential one-time AAV therapeutic for the treatment of Duchenne and includes an optimised transgene ...