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    - PMLiVE

    Lilly and MeiraGTx partner in $475m ophthalmology deal

    The companies will develop and commercialise therapies for genetic conditions

    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

    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

    - PMLiVE

    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

    Vertex and Orum partner on gene editing conditioning drugs in deal worth $945m

    The agreement gives Vertex the rights to use Orum’s targeted protein degradation technology

    - PMLiVE

    FDA publishes final industry guidance for CAR-T and gene therapies

    The new guidance can also be applied to other genetically modified lymphocyte products, including  CAR NK cells

    - PMLiVE

    AstraZeneca and Cellectis enter gene therapy partnership worth over $2.2bn

    The deal gives AZ access to Cellectis’ gene editing technologies and manufacturing capabilities

    Biomarin

    BioMarin announces first patient dosed with haemophilia A gene therapy in Europe

    The therapy was granted conditional marketing authorisation by the EC in August 2022

    regeneron headquarters

    Regeneron to acquire Decibel Therapeutics in deal worth up to $213m

    The transaction expands the company’s gene therapy and hearing loss pipeline

    - PMLiVE

    NICE publishes draft guidance not recommending CSL’s haemophilia B gene therapy

    The company has been seeking approval for use of the therapy in adults with severe or moderately severe haemophilia B without a history of factor IX inhibitors

    - PMLiVE

    FDA approves BioMarin’s Roctavian as first gene therapy for severe haemophilia A

    Approximately 2,500 patients are expected to be eligible to receive the one-time therapy

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