Blog
RSS FeedPosted by Michael Wonder on 24 Jul 2024
This gene therapy may not work. So why did the FDA fully approve it?
22 July 2024 - The agency has repeatedly neglected its obligation to ensure that drugs are effective. ...
Posted by Michael Wonder on 13 Jul 2024
Innorna announces US FDA rare paediatric disease designation granted to IN016 for the treatment of progressive familial intrahepatic cholestasis
11 July 2024 - Innorna today announced that the US FDA has granted rare paediatric disease designation to IN016, one of ...
Posted by Michael Wonder on 29 Jun 2024
Rocket Pharmaceuticals provides regulatory update on Kresladi (marnetegragene autotemcel)
28 June 2024 - Rocket Pharmaceuticals today announced a regulatory update for Kresladi (marnetegragene autotemcel), a lentiviral vector-based gene therapy ...
Posted by Michael Wonder on 23 Jun 2024
Top FDA official Peter Marks overruled staff, review team to approve Sarepta gene therapy
20 June 2024 - For a third time, Sarepta Therapeutics has convinced a top FDA official to overrule the prevailing ...
Posted by Michael Wonder on 20 Jun 2024
FDA expands approval of gene therapy for patients with Duchenne muscular systrophy
20 June 2024 - Today, the FDA expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment ...
Posted by Michael Wonder on 10 Jun 2024
SpliSense receives FDA fast track designation for SPL84 for the treatment of cystic fibrosis
29 May 2024 - SpliSense today announced that the US FDA has granted fast track designation to SPL84 for cystic ...
Posted by Michael Wonder on 06 Jun 2024
Neurogene announces NGN-401 gene therapy for Rett syndrome selected by FDA for START pilot program
3 June 2024 - NGN-401 is one of only three CBER programs chosen by FDA. ...
Posted by Michael Wonder on 05 Jun 2024
uniQure receives FDA regenerative medicine advanced therapy designation for investigational gene therapy AMT-130 in Huntington’s disease
3 June 2024 - Designation based on 24 month interim Phase 1/2 clinical data for AMT-130 announced in December 2023. ...
Posted by Michael Wonder on 21 May 2024
Ractigen Therapeutics secures FDA fast track designation for RAG-01, a first in class saRNA therapy
21 May 2024 - Ractigen Therapeutics proudly announces that its flagship program, RAG-01, has been granted fast track designation by ...
Posted by Michael Wonder on 15 May 2024
PTC Therapeutics announces FDA acceptance and priority review of the BLA for Upstaza
14 May 2024 - PDUFA target action date of 13 November 2024. ...
Posted by Michael Wonder on 08 May 2024
First patient begins newly approved sickle cell gene therapy
6 May 2024 - On Wednesday, Kendric Cromer, a 12 year old boy from a suburb of Washington, became the ...
Posted by Michael Wonder on 07 May 2024
iECURE receives FDA fast track designation for ECUR-506 for the treatment of neonatal onset ornithine transcarbamylase deficiency
6 May 2024 - iECURE announced today that it has received fast track designation from the US FDA for ECUR-506, ...
Posted by Michael Wonder on 06 May 2024
Taysha Gene Therapies announces regenerative medicine advanced therapy designation granted by US FDA for TSHA-102 in Rett syndrome
2 May 2024 - RMAT designation follows FDA’s review of available safety and efficacy data from the first three patients dosed ...
Posted by Michael Wonder on 27 Apr 2024
Use evidence to support early coverage of gene therapy after accelerated approval
23 April 2024 - Gene editing and therapy to replace missing or defective genes is one of the most exciting ...
Posted by Michael Wonder on 26 Apr 2024
US FDA approves Pfizer’s Beqvez (fidanacogene elaparvovec-dzkt), a one-time gene therapy for adults with hemophilia B
26 April 2024 - Pfizer announced today that the US FDA has approved Beqvez (fidanacogene elaparvovec-dzkt) for the treatment of adults ...