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RSS FeedPosted by Michael Wonder on 03 Apr 2023
Vertex and CRISPR Therapeutics complete submission of rolling biologics license applications to the US FDA for exa-cel for the treatment of sickle cell disease and transfusion-dependent beta thalassaemia
3 April 2023 - EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review ...
Posted by Michael Wonder on 21 Mar 2023
Top FDA official: agency needs to start using accelerated approval for gene therapies
20 March 2023 - A top FDA official said Monday that the agency needs to start using accelerated approval, a ...
Posted by Michael Wonder on 08 Mar 2023
Genethon given PRIME status by EMA for gene therapy to treat Crigler-Najjar syndrome, a rare liver disease
7 March 2023 - Genethon today announced that the EMA has granted PRIME (Priority Medicines) status to the gene therapy, ...
Posted by Michael Wonder on 07 Mar 2023
BioMarin provides update on FDA review of Roctavian (valoctocogene roxaparvovec) gene therapy for adults with severe haemophilia A
7 March 2023 - FDA extends PDUFA target action date to 30 June 2023. ...
Posted by Michael Wonder on 21 Feb 2023
Alnylam announces US FDA acceptance of supplemental new drug application for Onpattro (patisiran) for the treatment of the cardiomyopathy of ATTR amyloidosis
21 February 2023 - PDUFA date set for 8 October 2023. ...
Posted by Michael Wonder on 21 Feb 2023
First gene therapy for haemophilia B, CSL's Hemgenix, approved by the European Commission
20 February 2023 - Hemgenix underscores CSL's promise to deliver life-changing innovations that have the potential to help patients lead ...
Posted by Michael Wonder on 15 Feb 2023
FDA gene therapy head raises concern on accelerated approval use
14 February 2023 - More than 1,000 cell and gene therapy clinical trials registered. ...
Posted by Michael Wonder on 14 Feb 2023
Top FDA official interested in ‘Project Orbis’ for cell and gene therapies
13 February 2023 - A top official from the US FDA said he wants to see something similar to the agency’s ...
Posted by Michael Wonder on 07 Feb 2023
Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy designation for RP-A501 gene therapy for Danon disease
7 February 2023 - Rocket Pharmaceuticals today announced that the US FDA has granted regenerative medicine advanced therapy designation to ...
Posted by Michael Wonder on 30 Jan 2023
Nanoscope Therapeutics receives fast track designation by the FDA for MCO-010 for the treatment of Stargardt disease
30 January 2023 - Nanoscope Therapeutics today announced that the US FDA has granted fast track designation to MCO-010, an ...
Posted by Michael Wonder on 18 Jan 2023
Forge Biologics receives PRIME designation from the EMA for novel gene therapy FBX-101 for the treatment of patients with Krabbe disease
17 January 2023 - FBX-101 is granted priority medicines (PRIME) designation by the EMA after review of Phase 1/2 RESKUE clinical ...
Posted by Michael Wonder on 18 Dec 2022
FDA approves first gene therapy for the treatment of high risk, non-muscle invasive bladder cancer
16 December 2022 - Today, the US FDA approved Adstiladrin (nadofaragene firadenovec-vncg), a non-replicating adenoviral vector based gene therapy indicated for ...
Posted by Michael Wonder on 16 Dec 2022
CSL receives positive CHMP opinion for etranacogene dezaparvovec – gene therapy for adults with haemophilia B
16 December 2022 - Regulatory milestone moves CSL one step closer to bringing the promise of gene therapy for haemophilia ...
Posted by Michael Wonder on 06 Dec 2022
Ionis announces European Medicines Agency accepts marketing authorisation application of tofersen to treat rare, genetic form of ALS
5 December 2022 - EMA acceptance follows FDA's acceptance of tofersen new drug application earlier this year. ...
Posted by Michael Wonder on 03 Dec 2022
Orchard Therapeutics announces Swissmedic validation of the marketing authorisation application for Libmeldy (atidarsagene autotemcel)
1 December 2022 - Orchard Therapeutics today announced its marketing authorisation application for Libmeldy (atidarsagene autotemcel) has been accepted for ...