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AVROBIO to Present Clinical and Preclinical Data at the ASGCT 24th Annual Meeting
AVROBIO to Present Clinical and Preclinical Data at the ASGCT 24th Annual Meeting
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”
“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”
RECORDED POSTER PRESENTATIONS:
“Hematopoietic stem cell lentiviral gene therapy for cystinosis: Updated results from a Phase 1/2 clinical trial”
“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.”
About
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.
Forward-Looking Statements
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.
Any forward-looking statements in this press release are based on AVROBIO’s current expectations, estimates and projections about our industry as well as management’s current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIO’s product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of
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1 Collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 is funded in part by grants to
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chris.brinzey@westwicke.com
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