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    - PMLiVE

    Lilly and MeiraGTx partner in $475m ophthalmology deal

    The companies will develop and commercialise therapies for genetic conditions

    - PMLiVE

    Vertex’s triple combination cystic fibrosis therapy Alyftrek recommended by NICE

    The rare inherited disease affects approximately 11,000 people in the UK

    - PMLiVE

    Vertex Pharmaceuticals’ cystic fibrosis therapy Alyftrek granted EC approval

    The rare inherited disease affects more than 109,000 people worldwide

    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

    Vertex’s triple combination cystic fibrosis therapy Alyftrek approved by MHRA

    Over 11,000 people in the UK are affected by the rare genetic disease

    - 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

    NICE recommends Vertex/CRISPR’s Casgevy gene therapy for beta thalassaemia

    An estimated 460 patients in England with severe cases of the blood disorder could be eligible for Casgevy

    - 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

    Vertex and Genomics expand collaboration to accelerate precision medicines

    The partners have been working together since 2018 to develop treatments for serious diseases

    - PMLiVE

    Scientists discover new form of gene editing to recombine and rearrange DNA

    The bridge RNA system allows researchers to edit, delete and convert DNA recombinases without using CRISPR

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