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    • Spark Therapeutics

    - 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

    Pfizer’s sickle cell disease therapy Oxbryta recommended by NICE for NHS use

    About 4,000 people in England with the genetic blood disorder are expected to benefit from the decision

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    EC approves Vertex/CRISPR’s Casgevy gene therapy for inherited blood disorders

    There are now more than 8,000 patients who are potentially eligible for treatment with Casgevy

    - 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

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    CHMP recommends gene therapy for beta thalassemia and sickle cell disease

    If approved, Casgevy will be the only genetic therapy in the EU to treat these conditions

    - PMLiVE

    FDA approves two cell-based gene therapies for sickle cell disease

    Vertex/CRISPR and bluebird bio’s gene therapies have been approved to treat patients aged 12 years and older

    - PMLiVE

    MHRA authorises world-first gene therapy for two inherited blood disorders

    Casgevy is now the first licensed treatment that uses the gene-editing tool CRISPR

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