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 ...

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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 ...

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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. ...

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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 ...

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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 ...

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FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation

12 August 2020 - Today, the U.S. FDA granted accelerated approval to Viltepso (viltolarsen) injection for the treatment of Duchenne muscular ...

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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).  ...

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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 ...

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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 ...

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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. ...

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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 ...

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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. ...

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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. ...

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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 ...

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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. ...

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