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RSS FeedPosted by Michael Wonder on 15 Oct 2020
Taysha Gene Therapies receives rare paediatric disease designation and orphan drug designation for TSHA-102 as a treatment for Rett syndrome
14 October 2020 - Program leverages novel miRARE platform technology used to control transgene expression on a cellular basis. ...
Posted by Michael Wonder on 11 Oct 2020
Axovant Gene Therapies receives rare paediatric disease designation for AXO-AAV-GM1 for GM1 gangliosidosis
9 October 2020 - Company on-track to report data from on-going Phase 1/2 clinical study in Q4 2020. ...
Posted by Michael Wonder on 04 Oct 2020
BioMarin receives FDA fast track designation for investigational gene therapy, BMN 307
2 October 2020 - Second investigational gene therapy in clinic, potential third therapy in PKU franchise. ...
Posted by Michael Wonder on 02 Oct 2020
Pfizer receives FDA fast track designation for Duchenne muscular dystrophy investigational gene therapy
1 October 2020 - Pfizer today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy ...
Posted by Michael Wonder on 24 Sep 2020
FDA grants Spirovant Sciences orphan drug and rare paediatric disease designations for SPIRO-2101 for treatment of cystic fibrosis
24 September 2020 - Spirovant Sciences today announced that the U.S. FDA has granted orphan drug and rare paediatric disease ...
Posted by Michael Wonder on 22 Sep 2020
Gyroscope Therapeutics granted FDA fast track designation for GT005, an investigational gene therapy for dry age-related macular degeneration
22 September 2020 - Gyroscope Therapeutics today announced that the U.S. FDA has granted fast track designation to GT005 for the ...
Posted 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. ...