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RSS FeedPosted by Michael Wonder on 28 Aug 2020
Taysha Gene Therapies receives orphan drug designation and rare paediatric disease designation for TSHA-101 for GM2 gangliosidosis
27 August 2020 - TSHA-101 anticipated to advance into the clinic by the end of 2020. ...
Posted by Michael Wonder on 19 Aug 2020
BioMarin receives complete response letter from FDA for valoctocogene roxaparvovec gene therapy for severe haemophilia A
19 August 2020 - FDA introduces new recommendation for 2 year annualised bleeding rate as primary outcome for on-going Phase 3 ...
Posted by Michael Wonder on 13 Aug 2020
4D Molecular Therapeutics receives FDA fast track designation for 4D-310 gene therapy for treatment of Fabry disease
13 August 2020 - 4D Molecular Therapeutics announced that the U.S. FDA has granted fast track designation to 4D-310 for ...
Posted by Michael Wonder on 24 Jul 2020
Sarepta Therapeutics receives fast track designation for SRP-9001 micro-dystrophin gene therapy for the treatment of Duchenne muscular dystrophy
24 July 2020 - Sarepta Therapeutics today announced that the U.S. FDA has granted fast track designation to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). ...
Posted by Michael Wonder on 20 Jul 2020
Gene therapy shows promise for haemophilia, but could be most expensive U.S. drug ever
20 June 2020 - Jack Grehan, who was born with hemophilia, used to inject himself every couple of days with ...
Posted by Michael Wonder on 11 Jun 2020
Cell and gene therapies: FDA official on COVID-19 impact
10 June 2020 - Speaking at a session on cell and gene therapies at BIO Digital on Monday, a top US ...
Posted by Michael Wonder on 22 May 2020
Passage Bio receives rare paediatric disease designation for PBGM01 for patients with GM1 gangliosidosis
21 May 2020 - PBGM01 to enter the clinic in fourth quarter of 2020. ...
Posted by Michael Wonder on 26 Feb 2020
Translate Bio receives FDA fast track designation for MRT5005 for the treatment of cystic fibrosis
26 February 2020 - First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose portion of ...
Posted by Michael Wonder on 25 Feb 2020
Lysogene receives FDA fast track designation for LYS-SAF302 gene therapy in MPS IIIA
25 February 2020 - 17th patient dosed in the ongoing phase 2/3 AAVance study. ...
Posted by Michael Wonder on 21 Feb 2020
BioMarin's biologics license application for valoctocogene roxaparvovec accepted for priority review by FDA with review action date of 21 August 2020
20 February 2020 - No advisory committee meeting currently planned to review the application. ...
Posted by Michael Wonder on 13 Feb 2020
NDA accepted for filing by the FDA for antisense oligonucleotide viltolarsen
7 February 2020 - Nippon Shinyaku announced today that the U.S. FDA has accepted the filing of our new drug ...
Posted by Michael Wonder on 05 Feb 2020
Orsini Pharmaceutical Services now providing FDA approved Vyondys 53 (golodirsen) injection for Duchenne muscular dystrophy
5 February 2020 - Commercial distribution of Vyondys 53 in the U.S. is currently underway. ...
Posted by Michael Wonder on 30 Jan 2020
ProQR receives rare paediatric disease designation from FDA for QR-421a
30 January 2020 - Designation is for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 ...
Posted by Michael Wonder on 29 Jan 2020
With new rules, FDA aims to spark competition, lower prices for expensive gene therapies
28 January 2020 - The FDA released a slew of gene therapy policies Tuesday aimed at encouraging drug makers to ...
Posted by Michael Wonder on 28 Jan 2020
FDA continues strong support of innovation in development of gene therapy products
28 January 2020 - This is a pivotal time in the field of gene therapy as the FDA continues its ...