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    Home > Active Ingredient News > Blood System > Obtained FDA Fast Track designation to expand the benefits of the treatment of acute myeloid leukemia with engineered hematopoietic stem cells lacking CD33, Yi Mai Meng broke the news

    Obtained FDA Fast Track designation to expand the benefits of the treatment of acute myeloid leukemia with engineered hematopoietic stem cells lacking CD33, Yi Mai Meng broke the news

    • Last Update: 2021-10-02
    • Source: Internet
    • Author: User
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    Recent popular reports from Yimaike ★NoticeShanghai Minhang Biopharmaceutical Industry Development Conference and 2021 Life and Health Industry Summit Forum is about to open in Shanghai ★September·ShanghaiOncolytic Virus Drug Development Forum is about to open Click on the picture and sign up for the conference September 11, 2021 Japan/MedClub News/--September 9, 2021, Vor Biopharma, a clinical-stage cell and genome engineering company, announced its main engineered hematopoietic stem cell (eHSC) drug candidate for the treatment of acute myeloid leukemia (AML) VOR33 has obtained the fast track designation granted by the FDA
    .

    VOR33 are hematopoietic stem cells engineered through CRISPR gene editing.
    These cells have been designed to lack CD33 expression
    .

    Infusion of VOR33 into the patient's body, VOR33 will differentiate into blood cells that do not express CD33 in the patient's body, participating in the normal operation of the human blood system; at the same time, reducing healthy blood cells expressing CD33, aiming to protect the patient's healthy cells from anti-CD33 therapy Influence
    .

    If the patient is subsequently treated with CD33-targeted therapy, then CD33-targeted therapy molecules will not attack blood cells differentiated from VOR33, but will only attack target cells with CD33
    .

    That is to "expose tumor cells, hide healthy cells"
    .

    This avoids the killing of relevant healthy cells by CD33 targeted therapy, and expands the treatment range and effectiveness of CD33 targeted therapy, which may expand the clinical benefit of acute myeloid leukemia
    .

    Robert Pietrusko, Ph.
    D.
    , Chief Regulatory and Quality Officer of Vor, said: "Achieving Fast Track designation is an important milestone for Vor.
    This marks the FDA's recognition of the serious and life-threatening medical condition of patients facing acute myeloid leukemia, and the resolution of VOR33.
    The potential of this unmet medical need
    .

    We will continue to work closely with the FDA to accelerate the development of VOR33.
    The company is currently actively participating in Phase 1/2a clinical trials for patients with AML who currently have limited treatment options
    .

    We will continue as planned.
    initial clinical data in the first half 2022 report VOR33 in
    .

    "About Vor BiopharmaVor Biopharma is a clinical-stage cell and genome engineering company, aims to change the cancer patient's life by creating works of hematopoietic stem cells (eHSC) treatment platform release The potential of targeted therapy
    .

    Vor's eHSC first collects HSCs from matched healthy donors, and then the HSCs are genetically engineered to knock out genes that express the target protein, and then infused into patients
    .

    ▲ Schematic diagram of Vor's candidate products (source: official website) In February 2019, Vor Biopharma completed a US$42 million Series A financing
    .

    In July 2020, the company announced the completion of a US$110 million Series B financing to advance VOR33's entry into clinical trials, deepen its product portfolio, and accelerate the verification of other targets on its scientific platform
    .

    Recommended reading: New pretreatment methods for blood tumor targeted therapy? Xinrui Biopharmaceuticals received 110 million U.
    S.
    dollars to advance hematopoietic stem cells into clinicsYi Mai Meng broke the news.
    On January 15, 2021, according to Nasdaq, Vor Biopharma submitted an S-1 file application to the U.
    S.
    Securities and Exchange Commission to raise up to 150 million Initial public offering of U.
    S.
    dollars
    .

    Recommended reading: Pharmaceutical financingRuizheng Gene has completed tens of millions of dollars in seed round financing to build a gene editing and delivery industrialization platform.
    For more details, scan the QR code to read the complete reference: 1.
    https:// .
    com/article/releases/vor33-granted-us-fda-fast-track-designation-for-aml/
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