AVROBIO, a clinical-stage biotechnology company developing transformative cell and gene therapies targeting cancer and rare diseases, has announced its launch plans. The company's priority is to accelerate development of two novel cell and gene therapies pioneered within the labs of Dr. Christopher Paige and Dr. Jeffrey Medin (now at the Medical College of Wisconsin) at the University Health Network (UHN) in Toronto. Phase I programs will be in the clinic by early to mid-2016 in both acute myeloid leukemia (AML) and Fabry disease. The company will simultaneously work to expand its proprietary cell and gene therapy platform to treat additional indications.

"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 very proud to carry forward the groundbreaking work of our founding scientists with investment from Atlas Venture and partnership with the Center for Commercialization of Regenerative Medicine (CCRM). We have built a team capable of accelerating the development of truly important therapies for patients in immense need."

Joining MacKay on the leadership team are Dr. Kim Warren, head of Operations; Dr. Chris Mason, chief science officer; and Deanna Petersen, chief business officer. All are recognized veterans of the cell and gene therapy field and bring proven leadership in rare diseases.

"Following extensive research and pre-clinical development, we are happy to have our technology reach clinical development and are excited to work with the AVROBIO team to continue translating our science into proven medicine. Lentiviral gene therapy approaches have been demonstrated in clinical trials to be a safe and effective vehicle for the delivery of therapeutic genes into targeted cells. A broader goal is to leverage our proprietary ex-vivo gene therapy backbone across a number of serious diseases thereby accelerating the development of potential breakthrough therapies," said Dr. Jeffrey Medin.

Cell and 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 AML and Fabry patients with new therapies that will potentially significantly improve both their quality of life and lifespan.

AVR-01 is designed to be a potent anti-cancer immunotherapy which triggers the immune system to first detect, and then eradicate, tumor cells. The patient's cancer cells are genetically modified to express one of the most powerful immune signaling agents, the cytokine IL-12. The modified cells are then infused back into the patient via a one-time procedure, where they quickly activate cytotoxic CD4+ T cells which specifically target tumor cells and thus eliminate the cancer. A long-lasting anti-cancer immune response is maintained via both CD4+ and CD8+ cytotoxic T cells.

AVR-02 is designed to deliver lasting benefits for Fabry disease patients. The company's approach is to genetically modify a patient's own cells by adding a functional copy of the faulty gene. CD34+ hematopoetic stem cells are genetically modified to express the enzyme alpha-galactosidase A. The modified cells are then infused back into the patient via a one-time procedure. The objective is to deliver long-lasting or permanent, continuous elevation of endogenous enzyme thereby significantly improving patient outcomes and eliminating onerous lifetime biweekly intra-venous infusions of enzyme replacement therapy.