AVROBIO Announces First Patient Dosed Using plato™ Platform
Patient is the fourth dosed in Phase 2 trial of AVR-RD-01, an investigational gene therapy for Fabry disease
plato is AVROBIO’s platform for worldwide gene therapy commercialization
The plato platform includes a state-of-the-art lentiviral vector (LV2) designed to optimize vector copy number, transduction efficiency and resulting enzyme activity; a personalized conditioning regimen with precision dosing via therapeutic drug monitoring intended to enhance safety and engraftment; advanced cryopreservation to extend shelf life and enable flexible scheduling for patients; and an automated, closed-manufacturing process intended to improve consistency and predictability of the drug product. The Company expects that plato’s self-contained manufacturing pods will facilitate global manufacturing site expansion.
The first patient dosed using plato is enrolled in FAB-201, the Company’s Phase 2 trial of AVR-RD-01, an investigational gene therapy for Fabry disease. That trial is evaluating the safety and efficacy of AVR-RD-01 in 8 to 12 treatment-naïve patients, with ongoing recruitment in the U.S.,
“Our plato platform sets
plato has received regulatory clearance for clinical use and will be used for all patients going forward in FAB-201, AVROBIO’s Phase 2 Fabry disease trial, and in GAU-201, AVROBIO’s Phase 1/2 clinical trial of AVR-RD-02, an investigational gene therapy for Gaucher disease.
AVROBIO’s mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease progression throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we are also advancing a program in Pompe disease.
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 such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our plato platform, including the expected benefits and results of our implementation of the plato platform in our FAB-201 trial and other programs, our business strategy, prospective products and goals, the therapeutic potential of our product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, and the anticipated benefits of our gene therapy platform including potential impact on our commercialization activities. 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
Christopher F. Brinzey
Westwicke, an ICR Company
Ten Bridge Communications