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    Lilly and MeiraGTx partner in $475m ophthalmology deal

    The companies will develop and commercialise therapies for genetic conditions

    - PMLiVE

    FDA unveils new voucher programme to accelerate drug review process

    The programme is designed to reduce the review time for companies supporting US national interests

    The (clinical) trials and tribulations of gene therapy – why meaningful patient engagement strategies matter

    By Sarah Mohamad and Lauren Coleman-Bennett

    Amiculum

    What do people look for in gene therapy materials? Results from our survey of patients and relatives

    By Sam Fraser

    Amiculum

    - PMLiVE

    FDA approves Bavarian Nordic’s chikungunya vaccine for individuals aged from 12 years

    The mosquito-borne viral disease has been reported across many popular travel destinations

    - PMLiVE

    Roche’s Evrysdi tablet formulation granted FDA approval for spinal muscular atrophy

    The progressive neuromuscular disease affects approximately one in every 10,000 babies globally

    - PMLiVE

    CSL Behring announces four-year results for haemophilia B gene therapy Hemgenix

    The genetic bleeding disorder affects an estimated one per every 19,283 male births in the US

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    NICE approves Vertex’s Casgevy one-time gene therapy for sickle cell disease

    About 15,000 people in England are living with the inherited blood disorder

    - PMLiVE

    Roche/Sarepta announce two-year results for Duchenne muscular dystrophy gene therapy

    The muscle-wasting disorder is estimated to affect one in every 5,000 male births worldwide

    - PMLiVE

    FDA accepts updated protocol for Annovis’ phase 3 Alzheimer’s disease study

    The company hopes the revised protocol will accelerate the development timeline of buntanetap

    - PMLiVE

    MHRA approves Italfarmaco’s Duvyzat to treat Duchenne muscular dystrophy

    The severe neuromuscular disease affects approximately 2,500 people in the UK

    - PMLiVE

    NICE recommends Santhera’s Duchenne muscular dystrophy drug Agamree

    Approximately 1,700 patients with the muscle-wasting disorder in England are expected to benefit from the decision

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