CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that two oral presentations and two posters featuring data from its pipeline of lysosomal disorder gene therapy programs will be shared at the 24th virtual Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), May 11-14, 2021.
Accepted abstracts will include new data points from an investigator-sponsored Phase 1/2 trial1 in cystinosis and from AVROBIO’s preclinical program in Pompe disease. Encore data from AVROBIO’s clinical programs in Fabry disease and Gaucher disease type 1 will also be presented.
The presentations are listed below and the full preliminary program is available online at the ASGCT website.
LIVE ORAL PRESENTATIONS:
“Long-term hematopoietic stem cell lentiviral gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice”
Tuesday, May 11, 2021, 6:30-6:45 p.m. ET
Niek van Til, Ph.D., senior director, gene therapy, AVROBIO, will present new preclinical data for AVR-RD-03, a gene therapy for Pompe disease, and answer questions in a live Q&A.
“AVR-RD-01, an investigational lentiviral gene therapy for Fabry disease: Clinical data trends from Phase 1 and Phase 2 studies up to 3.5 years”
Friday, May 14, 2021, 12:30 p.m. - 12:45 p.m. ET
Mark Thomas, M.D., principal investigator of the AVROBIO-sponsored FAB-GT Phase 2 trial of AVR-RD-01, an investigational gene therapy for Fabry disease, nephrologist at the Department of Nephrology, Royal Perth Hospital and clinical professor at the University of Western Australia Medical School, will present encore data and answer questions in a live Q&A.
RECORDED POSTER PRESENTATIONS:
“Hematopoietic stem cell lentiviral gene therapy for cystinosis: Updated results from a Phase 1/2 clinical trial”
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 cystinosis, and associate professor of pediatrics at University of California, San Diego, School of Medicine, will present new data on the third patient dosed in the trial and recap existing data on the first two patients dosed.
“Preliminary results from the Phase 1/2 Guard1 trial: A first-in-human study of ex vivo lentiviral gene therapy (AVR-RD-02) in people with Gaucher disease type 1.”
Leslie Jacobsen, M.D., vice president, clinical development lead, AVROBIO, will present encore data from the first patient dosed in the Phase 1/2 trial of AVR-RD-02, an investigational therapy for Gaucher disease type 1.
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. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by our industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.
This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as “aims,” “anticipates,” “believes,” “could,” “designed to,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.
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1 Collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 is funded in part by grants to UCSD from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and National Institutes of Health (NIH).