The study will expand into the U.S. in the second half of 2019 and includes five study periods: Screening, Baseline,Pre-transplant, Transplant, and Post-transplantFollow-up at 48 weeks. Patients will undergo a conditioning regimen administration with busulfan intravenously for four days to achieve myeloablation prior to the transplant. After study completion, consenting subjects will continue periodic safety and efficacy assessments for approximately 14 years, for a total of 15 years post-transplantfollow-up.
About the plato™ platform
The plato platform, AVROBIO’s commercial-scale platform for anticipated future worldwide commercialization and pipeline expansion activities, consists of a proprietarystate-of-the-art four-plasmid vector system, automation of a closed cell manufacturing process and a conditioning regimen that utilizes therapeutic drug monitoring (TDM). The plato platform is designed to enhance the potency, safety, efficacy, and long-term durability of AVROBIO’s gene therapies, and may additionally provide the capability to address central nervous system (CNS) manifestations that accompany many lysosomal storage diseases. The platform is additionally being incorporated in the planned Phase 1/2 clinical trial ofAVR-RD-02(GAU-201) in Canada and Australia. That trial is expected to begin in the second half of 2019.
About AVROBIO, Inc.
AVROBIO, Inc., is a Phase 2 clinical-stage gene therapy company developing gene therapies to potentially cure rare diseases with a single dose. AVROBIO’s lentiviral-based gene therapies employ hematopoietic stem cells that are collected from the patient and then modified with a lentiviral vector to insert a functional copy of the gene that is defective in the target disease. AVROBIO is focused on the development of its gene therapy,AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders includingGaucher disease,Cystinosis andPompe disease. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit www.avrobio.com.
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 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 business strategy, prospective products and goals, the therapeutic potential of our product candidates, anticipated benefits of our gene therapy platform including potential impact on our commercialization and pipeline expansion activities, the design, commencement, enrollment and timing of ongoing or planned clinical trials, and the timing, scope and likelihood of regulatory filings and approvals. 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.
