February 19, 2016
By Mark Terry, BioSpace.com Breaking News Staff
No financial details were disclosed. The company is founded on two novel cell and gene therapies that came out of the laboratories of Christopher Paige and Jeffrey Medin at the University Health Network (UHN) in Toronto, Ontario. Both researchers are currently at the Medical College of Wisconsin.
The two therapeutics are expected to begin Phase I trials in the clinic in mid-2016 in acute myeloid leukemia (AML) and in Fabry disease. It will also work on expanding its cell and gene therapy platform to other 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, in a statement. “We are very proud to carry forward the groundbreaking work of our founding scientists with investment from Atlas Venture and partnership with the Center or Commercialization of Regenerative Medicine (CCRM).”
CCRM is a Canadian nonprofit organization. It is funded by the Government of Canada’s Networks of Centres of Excellence program, the Province of Ontario, and several academic and industry partners. It focuses on the development of regenerative medicines and related technologies, specifically focusing on cell and gene therapy.
The company’s two lead products are AVR-01 and AVR-02. AVR-01 is an anti-cancer immunotherapy. The patient’s cancer cells are drawn from the body, genetically modified to express cytokine IL-12, an immune signaling agent, then infused back into the patient. The modified cells activate cytotoxic CD4+ T cells, which then attack the tumor cells.
AVR-02 utilizes a similar approach, but instead genetically modifies the patient’s own cells by adding a functional copy of the gene that is causing the disease. In this specific case, CD34+ hematopoietic stem cells are genetically modified to express an enzyme, alpha-galactosidase A. Once infused back into the patient, it is hoped they will deliver long-lasting or even permanent increase of the enzyme. Traditional treatment for Fabry is long-term, permanent biweekly intravenous enzyme replacement therapy.
The company also announced its executive team. Geoff MacKay is chief executive officer. Kim Warren will act as Head of Operations. Chris Mason will be chief science officer, and Deanna Petersen will be chief business officer.
MacKay is the former chief executive officer of Organogenesis Inc., and a board member and interim chief executive officer of eGenesis, a company focused on CRISPR Cas-9 gene editing. Formerly MacKay spent 11 years with Novartis in various senior positions of the Global Transplantation & Immunology division.
Warren spent the last 10 years at Lonza in support of GMP manufacturing of cellular therapeutics, including tissue acquisition, bioassays and other services. She also headed research and development at Cambrex, founded and ran Poietic Technologies, which was acquired by Cambrex in 1999, and was a research scientist at Otsuka and Cetus.
Mason is a Full Professor of Regenerative Medicine Bioprocessing at University College London. He is co-founder of the London Regenerative Medicine Network, a trustee of the UK Stem Cell Foundation, and services on the UK-Israel Science Council.
Peterson was previously head of business development for Shire (SHPGY)’s rare disease business. Prior to Shire, Petersen was vice president of business development at Agenus Inc. (AGEN), and at Coley Pharmaceutical Group. She serves on the board of directors of American DG Energy, and for the Massachusetts Biotechnology Association.
“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,” said Jeffrey Medin, in a statement. “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.”