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RSS FeedPosted 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 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 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 ...
Posted 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 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 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 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 30 Nov 2022
How CSL priced the world’s most expensive drug
1 December 2022 - When blood products giant CSL announced last week it had been given US regulatory approval for ...
Posted by Michael Wonder on 28 Nov 2022
Sarepta Therapeutics announces that US FDA has accepted for filing and granted priority review for the biologics license application for SRP-9001, Sarepta’s gene therapy for the treatment of ambulant individuals with Duchenne muscular dystrophy
28 November 2022 - Regulatory action date of 29 May 2023. ...