Blog
RSS FeedPosted by Michael Wonder on 09 Jul 2024
A new gene therapy, transformative cystic fibrosis medicines and an obesity jab included in guidance recommended during pre-election period
8 July 2024 - More than 20 pieces of draft and final guidance on a range of medicines and medical conditions ...
Posted by Michael Wonder on 05 Jul 2024
CSL Behring announces first two patients treated with Hemgenix (etranacogene dezaparvovec) gene therapy for haemophilia B in Europe
4 July 2024 - CSL Behring today announced that two haemophilia B patients were treated with the gene therapy Hemgenix ...
Posted by Michael Wonder on 29 Jun 2024
NICE recommends CSL Behring’s haemophilia B gene therapy Hemgenix
28 June 2024 - NICE has recommended CSL Behring’s haemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) for immediate reimbursement by ...
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 03 Jun 2024
Biogen receives European Commission approval for Qalsody (tofersen), the first therapy to treat a rare, genetic form of ALS
30 May 2024 - Qalsody is Biogen’s third rare disease therapy to be approved in the EU, demonstrating the company’s commitment ...
Posted by Michael Wonder on 01 Jun 2024
New gene therapy treatment for haemophilia B
31 May 2024 - The EMA has recommended granting a conditional marketing authorisation in the European Union for Durveqtix (fidanacogene ...
Posted by Michael Wonder on 31 May 2024
Highlights from the 27-30 May 2024 CHMP meeting
31 May 2024 - The EMA’s CHMP recommended 14 medicines for approval at its May 2024 meeting. ...
Posted by Michael Wonder on 23 May 2024
Advancing access to cell and gene therapies in Medicaid
22 May 2024 - The Biden administration’s stated priority to increase access to novel cell and gene therapeutics while controlling their ...
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. ...