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RSS FeedPosted 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. ...
Posted by Michael Wonder on 23 Nov 2022
BioMarin announces advancements in FDA review of Roctavian (valoctocogene roxaparvovec) for adults with severe haemophilia A
23 November 2022 - FDA no longer plans to hold an advisory committee meeting, as previously planned, to discuss the biologics ...
Posted by Michael Wonder on 23 Nov 2022
CSL lands world first gene therapy with $5.3 million haemophilia treatment
23 November 2022 - Australian biotech giant CSL will bring the first gene therapy for haemophilia B to market in ...
Posted by Michael Wonder on 23 Nov 2022
FDA approves first gene therapy to treat adults with haemophilia B
22 November 2022 - Today, the US FDA approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment ...
Posted by Michael Wonder on 08 Nov 2022
BioMarin announces incremental progress on biologics license application review for valoctocogene roxaparvovec AAV gene therapy for adults with severe haemophilia A program
7 November 2022 - FDA requests submission of upcoming 3 year data analysis from Phase 3 GENEr8-1 trial. ...
Posted by Michael Wonder on 07 Nov 2022
Sensorion announces US FDA grants rare paediatric disease designation to OTOF-GT for the treatment of otoferlin gene-mediated hearing loss
7 November 2022 - OTOF-GT targets the restoration of hearing in people living with otoferlin deficiency. ...
Posted by Michael Wonder on 05 Nov 2022
FDA finalises umbrella trial guidance for cell and gene therapies
4 November 2022 - The US FDA laid out its recommendations for sponsors to study multiple versions of a cellular or ...
Posted by Michael Wonder on 27 Oct 2022
AvroBio receives rare paediatric disease designation from the US FDA for first in class gene therapy for Gaucher disease
27 October 2022 - AVR-RD-02 has previously received fast track status from FDA, orphan drug designation in the US and EU, ...