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    Home > Active Ingredient News > Blood System > Express | US$900 million to help develop CRISPR gene editing therapy, Vertex renews cooperation

    Express | US$900 million to help develop CRISPR gene editing therapy, Vertex renews cooperation

    • Last Update: 2021-05-10
    • Source: Internet
    • Author: User
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    ▎The content team editor of WuXi AppTec today, Vertex Pharmaceuticals and CRISPR Therapeutics announced that the two companies have updated their cooperation agreement for the joint development of the therapeutic CTX001 under investigation.

    CTX001 is a gene editing therapy based on CRISPR/Cas9 technology and is being developed to treat sickle cell anemia (SCD) and transfusion-dependent β-thalassemia syndrome (TDT).

    It is currently one of the fastest clinically progressing CRISPR gene editing therapies.

    CRISPR-Cas9 gene editing technology uses artificially designed guide RNA (guide RNA) to identify the target genome sequence, guide Cas9 protease to effectively cut DNA double strands, and modify genes through repair or recombination after DNA double strand breaks.

    CTX001 is an autologous in vitro CRISPR/Cas9 gene editing therapy under development, currently in clinical trials for the treatment of severe patients with TDT or SCD.

    The patient's hematopoietic stem cells are edited in vitro to increase the level of fetal hemoglobin (HbF) in the red blood cells.

    HbF is a kind of hemoglobin that is naturally present in the human body when it is born, and it is converted into adult hemoglobin as it grows up.

    Increasing HbF levels through CTX001 may alleviate the need for blood transfusions in TDT patients and reduce pain and sickle cell crisis in SCD patients.

    CTX001 has been granted the Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of TDT and SCD by the US FDA, as well as fast track and orphan drug designation.

    It has also been granted priority medicine (PRIME) qualification by the European Medicines Agency (EMA) for the treatment of SCD.

    Image source: 123RF According to the revised terms of the agreement, Vertex will lead the global development, production and promotion of CTX001 with the support of CRISPR Therapeutics to maximize the potential of CTX001 to change the lives of tens of thousands of patients.

    Vertex will be responsible for 60% of the cost of the CTX001 project and will obtain 60% of the profit of CTX001's future global sales.

    CRISPR Therapeutics will continue to support the development of CTX001, and invest in further innovation, bear 40% of the cost and get 40% of the profit.

    In addition, CRISPR Therapeutics will receive an upfront payment of US$900 million from Vertex, and may receive a milestone payment of US$200 million after CTX001 obtains the first regulatory approval.

    "Cell and gene therapies are the key to our strategy to develop transformative therapies for serious diseases.

    " said Dr.
    Jeffrey Leiden, Executive Chairman of Vertex.
    "Our increased investment in cooperation with CRISPR is based on CTX001's impressive clinical performance, showing that it has It may provide a long-lasting cure for patients with SCD and TDT.

    We believe that by making full use of Vertex's extensive capabilities, CTX001 can serve more patients around the world faster.

    "Through cooperation with Vertex, we are on CTX001 therapy.
    Great progress has been made.
    This is the first clinically proven CRISPR/Cas9-based therapy.
    Together, we have opened up a new field in the treatment of genetic diseases.

    We have now injected CTX001 into more than 30 patients, The longest follow-up time is more than two years.
    This year we will complete the registration of patients for these two clinical trials.

    " Dr.
    Samarth Kulkarni, CEO of CRISPR Therapeutics said, "We are adopting a new operating model to enable this drug as soon as possible.
    Benefit all patients who can benefit from it. "Reference: [1] Vertex Pharmaceuticals and CRISPR Therapeutics Amend Collaboration for Development, Manufacturing and Commercialization of CTX001™ in Sickle Cell Disease and Beta Thalassemia.
    Retrieved April 20, 2021, from release/2021/04/20/2213128/0/en/Vertex-Pharmaceuticals-and-CRISPR-Therapeutics-Amend-Collaboration-for-Development-Manufacturing-and-Commercialization-of-CTX001-in-Sickle-Cell-Disease- and-Beta-Thalassemia.
    html Note: This article aims to introduce the progress of medical and health research, not a treatment plan recommendation.

    If you need treatment plan guidance, please go to a regular hospital.

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