checkAd

     197  0 Kommentare AVROBIO Announces Positive Data from Phase 1/2 Clinical Trial of Investigational Gene Therapy for Cystinosis at the ASGCT 26th Annual Meeting

    AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced follow-up data demonstrating a durable treatment effect across key measures out to 36 months from a collaborator-sponsored Phase 1/2 clinical trial1 evaluating an investigational gene therapy for the treatment of cystinosis. These data are being presented at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles, California, on May 18, 2023.

    Cystinosis is a rare, progressive disease with a high treatment burden and unmet need. The fully enrolled Phase 1/2 clinical trial is monitoring long-term safety and efficacy in six adult patients affected by the most severe and common form of cystinosis who previously had been treated with the standard of care (SOC), cysteamine. The patients’ own hematopoietic stem cells (HSCs) were genetically modified to express a functional version of cystinosin, the protein that is deficient in people living with cystinosis. Preliminary data suggest that post-gene therapy, functional cystinosin is produced throughout the body as evidenced by clinical measures in multiple tissues, including the eyes, skin and gastrointestinal mucosa, as well as by neurocognitive tests suggestive of activity in the central nervous system. No adverse events (AEs) related to the drug product or serious adverse events have been reported to date.

    “These data show that genetically modifying a patient’s own HSCs has the potential to restore functional cystinosin and systemically reduce the accumulation of cystine, laying the foundation for a registration-enabling clinical trial,” said AVROBIO Chief Medical Officer Essra Ridha, M.D., MRCP, FFPM. “We are excited about moving this investigational gene therapy closer to patients.”

    In addition to the data presented, the Company also announced positive and productive meetings with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and U.S. Food and Drug Administration (FDA) designed to align on regulatory paths and obtain feedback on this program.

    Data show investigational HSC gene therapy durably and systemically impacts neurocognitive measures and reduces cystine levels in the blood, and crystal accumulation in the skin and gastrointestinal mucosa

    Follow-up data suggest that after receiving HSC gene therapy, patients can produce functional cystinosin protein throughout the body. As a result, leukocyte cystine levels in the blood have decreased below baseline in all six patients, and stabilized up to 36 months out from treatment. Skin and gastrointestinal mucosa biopsies reveal a decline in tissue cystine crystals below baseline in the first four patients, who have been observed for at least 12 months, with two patients observed up to 24 months.

    Seite 1 von 5




    Business Wire (engl.)
    0 Follower
    Autor folgen

    AVROBIO Announces Positive Data from Phase 1/2 Clinical Trial of Investigational Gene Therapy for Cystinosis at the ASGCT 26th Annual Meeting AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced follow-up data demonstrating a durable treatment effect across key measures out to 36 months from …

    Auch bei Lesern beliebt

    Schreibe Deinen Kommentar

    Disclaimer