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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Human Rights Commission grants case against PHARMAC for failing to fund rare muscular disorder drug

16 September 2020 - Newshub can reveal a ground-breaking Human Rights Commission case has been taken against Pharmac for failing ...

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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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Stranded mum with dying daughter makes desperate plea to Jacinda Ardern

13 September 2020 - A distraught mum stuck in New Zealand with her dying daughter Stella amid the COVID pandemic ...

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Kiwi mum pleads with PHARMAC to fund life-changing drug Spinraza for her two children

11 September 2020 - The mother of two young boys with a rare disease is begging PHARMAC to fund a ...

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NICE widens its Zolgensma appraisal due to European Marketing Authorisation

11 September 2020 - England’s NICE is expanding its appraisal of Zolgensma, a gene therapy for spinal muscular atrophy, according ...

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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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Employers are planning how to blunt the cost of gene therapies, pricey new specialty drugs

27 August 2020 - As the pharmaceutical industry develops increasingly expensive medicines – notably, those costing seven figures – a ...

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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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Family battles PHARMAC and eyes Australian move to get 'miracle drug'

15 August 2020 - When Lani McLeod found out her toddler would never walk, she was gutted. ...

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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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