checkAd

     105  0 Kommentare AVROBIO to Present New Clinical and Preclinical Data at the ASGCT 25th Annual Meeting

    AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced that new data from its ongoing, collaborator-sponsored1 Phase 1/2 clinical trial in cystinosis will be presented on May 16, 2022, at the 25th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), held in Washington, D.C. Additionally, new preclinical data from AVROBIO’s Pompe disease program will be presented during the four-day conference.

    The planned presentations are listed below and the full preliminary program is available online at the ASGCT website.

    ORAL PRESENTATION:

    Hematopoietic stem cell gene therapy for cystinosis: updated results from a Phase 1/2 clinical trial
    Monday, May 16, 2022, 1:30-3:15 p.m. ET
    Oral Abstract Session on “Gene and Cell Therapy Trials in Progress”

    Stephanie Cherqui, Ph.D., principal investigator of the Phase 1/2 clinical trial of AVR-RD-04 (CTNS-RD-04), an investigational gene therapy for infantile nephropathic cystinosis, and associate professor of pediatrics at the University of California, San Diego, School of Medicine, will present new clinical data from patients dosed in the ongoing trial.

    POSTER PRESENTATION:

    Long-term efficacy and safety in preclinical hematopoietic stem cell gene therapy in Pompe Mice
    Wednesday, May 18, 5:30-7 p.m. ET
    Poster session on “Musculo-skeletal diseases”

    Niek van Til, Ph.D., consultant to AVROBIO, will share new preclinical data for AVR-RD-03, a gene therapy for Pompe disease.

    About AVROBIO
    Our vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle and bone. AVROBIO’s pipeline is powered by our industry-leading plato gene therapy platform, our foundation designed to deliver gene therapy worldwide. It includes clinical programs in cystinosis and Gaucher disease type 1, as well as preclinical programs in Gaucher disease type 3, Hunter syndrome and Pompe disease. We are headquartered in Cambridge, Mass. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

    Seite 1 von 3


    Business Wire (engl.)
    0 Follower
    Autor folgen

    AVROBIO to Present New Clinical and Preclinical Data at the ASGCT 25th Annual Meeting AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced that new data from its ongoing, collaborator-sponsored1 Phase 1/2 clinical trial in …