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    Home > Biochemistry News > Biotechnology News > Gene therapy phase 3 clinical results achieved key results, 67% of patients' skin wounds healed completely

    Gene therapy phase 3 clinical results achieved key results, 67% of patients' skin wounds healed completely

    • Last Update: 2022-01-12
    • Source: Internet
    • Author: User
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    On November 29, 2021, Krystal Biotech announced that a pivotal phase 3 clinical trial of Vyjuvek (beremagene geperpavec, B-VEC), a topical topical gene therapy under investigation, has achieved positive top-line results
    In patients with dystrophic epidermolysis bullosa (DEB), the trial reached the primary endpoint of complete wound healing at 6 months of treatment and the secondary endpoint of complete wound healing at 3 months of treatment.
    The drug showed Good tolerance

    According to the test results, the company plans to submit Vyjuvek's Biological Products Licensing Application (BLA) to the US FDA in the first quarter of 2022


    DEB is a rare and serious single-gene chronic genetic disease.
    It is caused by mutations in the COL7A1 gene encoding type VII collagen (COL7), which causes the skin to lack collagen, which leads to the separation of the epidermis and the dermis

    The patient’s skin and mucous membrane tissues are extremely fragile.
    Minor friction or trauma can cause blisters and tears.
    Open wounds can cause skin infections and fibrosis, which may fuse fingers and toes, and ultimately increase the risk of squamous cell carcinoma.
    Time can be fatal

    There are currently no approved treatments


    Different from the current standard treatment, Vyjuvek aims to treat DEB at the molecular level by providing a template for the production of normal COL7 protein in the skin cells of patients
    Vyjuvek uses the engineered HSV-1 virus vector to directly deliver two copies of the COL7A1 gene with normal functions to the divided and undivided skin cells of the patient

    Previously, the FDA has granted the drug the orphan drug designation for the treatment of DEB and the Regenerative Medicine Advanced Therapy (RMAT) designation


    ▲ Mechanism of action of Vyjuvek (picture source: Krystal Biotech official website)

    31 DEB patients were enrolled in this phase 3 clinical trial.
    The main key results are as follows:

    At 6 months of treatment, compared with placebo (22%), 67% of patients in the Vyjuvek group had wounds healed completely (p<0.

    At 3 months of treatment, compared with placebo (20%), 71% of patients in the Vyjuvek group had their wounds healed completely (p<0.

    Vyjuvek is well tolerated, and no serious adverse events related to the drug have been reported or the drug has been discontinued due to treatment
    The immunogenic characteristics of Vyjuvek (measured by anti-HSV-1 and anti-COL7 antibodies) are consistent with previous studies


    ▲Efficacy results of Vyjuvek's Phase 3 clinical trial (picture source: Krystal official website)

    Reference materials:

    [1] Krystal Biotech Announces Positive Topline Results from GEM-3 Pivotal Trial of VYJUVEK™ in Patients with Dystrophic Epidermolysis Bullosa.
    Retrieved November 29, 2021, from https://ir.
    com/news-releases/news-release- details/krystal-biotech-announces-positive-topline-results-gem-3-pivotal

    (The original text has been deleted)

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