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news.AVROBIO, a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases and cancer, has raised $25m in a Series A financing co-led by Atlas Venture, Clarus and SV Life Sciences.
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AVROBIO will use the proceeds from this financing to accelerate development of its clinical stage programs in Fabry disease and acute myeloid leukemia (AML), and to expand its pipeline in rare diseases and solid and liquid cancers.
“AVROBIO’s highly innovative therapies offer potentially life-altering impact for patients following a single infusion of genetically-modified cells,” said Geoff MacKay, AVROBIO’s President and Chief Executive Officer.
“We are grateful for the funding and support we have received from our investors, as we continue to focus on displacing the standard of care for patients with Fabry disease or AML through the development of these disruptive gene therapies.”
Gene therapies represent a new paradigm in human health, with the potential to deliver dramatic disease-modifying effects with long-lasting, durable impact. Underlying these advances are a deeper understanding of cell biology, immunology and a newer generation of vector designs enabling safe and effective delivery of therapeutic genes targeted to specific cells.
AVROBIO’s initial two programs are leveraging the established safety and effectiveness of ex-vivo gene therapy to provide Fabry and AML patients with new therapies that have the potential to significantly improve both their quality of life and lifespan.
AVROBIO’s Phase 1 gene therapy to treat Fabry disease seeks to deliver lasting and meaningful benefits for Fabry patients. The patient’s stem cells are extracted and genetically modified by adding a new, functional copy of the faulty gene. The modified cells are then infused back into the patient via a one-time procedure.
A durable elevation of endogenous enzyme is expected, with the potential to significantly improve patient outcomes and eliminate costly lifetime biweekly intravenous infusions of enzyme replacement therapy.
AVROBIO’s Phase 1 gene therapy to treat AML seeks to recruit the patient’s own immune system to better detect and eradicate cancer cells. After initial chemotherapy, some of the patient’s residual cancer cells are extracted and genetically modified to express IL-12, a powerful immune system activator.
The modified cells are then infused back into the patient via a one-time procedure, with a goal to elicit a potent and durable anti-cancer immune response against remaining AML blasts in the patient’s body.
In conjunction with AVROBIO’s Series A financing, the company announced that Bruce Booth, DPhil, Partner at Atlas Venture, will serve as Chairman of the Board of Directors. Also joining the company’s Board are Scott Requadt, Managing Director at Clarus, and Joshua Resnick, MD, Partner at SV Life Sciences.
“Scott and Josh bring a wealth of experience in the growth and development of early-stage biotechnology companies,” said Bruce Booth.
“Their expertise, together with AVROBIO’s deeply experienced and highly talented management team, creates a dynamic leadership team capable of accelerating the development of these truly important therapies for patients in immense need.”