bluebird bio’s LentiGlobin for sickle cell disease gene therapy (bb1111) granted priority medicines (PRIME) designation by European Medicines Agency

23 September 2020 - EMA’s PRIME program designed to optimise development and expedite evaluation of innovative medicines for patients with high ...

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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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GenSight Biologics submits EU marketing authorisation application for Lumevoq gene therapy to treat vision loss due to Leber hereditary optic neuropathy

15 September 2020 - First marketing authorisation application for a gene therapy treating a mitochondrial disease. ...

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EU review extended for BioMarin's gene therapy Valrox

10 September 2020 - European regulators have requested more data to review the marketing application for BioMarin's haemophilia A gene ...

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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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TGA approves new gene therapy from Novartis

13 August 2020 - Luxturna has been for the treatment of patients with inherited retinal dystrophy caused by pathological biallelic RPE65 ...

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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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AveXis receives EC approval and activates “Day One” access program for Zolgensma, the only gene therapy for spinal muscular atrophy

19 May 2020 - Zolgensma (onasemnogene abeparvovec) is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy ...

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