Canadian Biotech Startup Tackles Blood Cancer and Rare Disease

A new Toronto-based biotech company has announced two novel ex vivo treatments for blood cancer and a rare disease, using gene therapy. AvroBio is a joint venture by Dr. Christopher Paige and Dr. Jeffrey Medin – both researchers at the University of Toronto.

The company is now collaborating with the University Health Network (UHN), and is backed by support from Atlas Venture, and the Center for Commercialization of Regenerative Medicine (CCRM). The founders – along with the newly-appointed members of the corporate leadership team – say their first Phase I clinical trial will begin by mid-2016, with a focus on treating acute myeloid leukemia (AVR-01) and Fabry disease (AVR-02).

The company’s experimental treatment AVR-01, is an immunotherapy-based treatment that promotes a patient’s immune system to target and destroy cancer cells. The therapy is on-trend with the recent shift in oncology therapies that aim to use the patient’s immune system to eradicate a tumor.

AvroBio’s immunotherapy AVR-01 works by genetically modifying patient-derived tumor cells to express cytokine IL-12 – a molecule that elicits a strong immune response. Once the cells have been modified, they are reintroduced into the patient where they attract cytotoxic CD4-positive T cells that are able to identify and destroy the cancerous cells.

“AvroBio’s highly innovative therapies offer potentially life-altering impact for patients following a single infusion of genetically-modified cells,” said Geoff Mackay, CEO of AvroBio. “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.”

The effectiveness of this one-time procedure is maintained by the stimulation of CD4-positive as well as CD8-positive cytotoxic T cells. This technique has also been used by other oncology drug developers to target various types of cancer.

Ziopharm Oncology – a Boston-based biotech – is currently in preliminary stages of testing their immunotherapy, Ad-RTS-IL-12, for melanoma. A similar approach is used by Bristol-Myers Squibb’s skin cancer drug Yervoy, which is administered via four infusions over the course of a year.

Acute myeloid leukemia has the lowest survival rate among all adult forms of the disease, and accounts for approximately 25 percent of cases of leukemia. Novartis is the most recent company to get an FDA breakthrough drug designation for their acute myeloid leukemia drug, PKC412.

AvroBio’s AVR-02 is designed as a functional therapy for patients with Fabry disease. Like AVR-01, this approach uses genetic engineering to introduce a functional copy of the faulty gene into the cells.

Fabry disease is an inherited condition, with only 11,000 people in the US carrying the mutation. Despite its status as a rare disease, Sanofi Genzyme’s Fabrazyme has been a market leading treatment, at $200,000 per patient. Fabrazyme is an enzyme replacement therapy that requires patients to undergo biweekly infusions for the rest of their lives.

Genzyme is currently testing an oral version of their enzyme replacement therapy, which would negate the need for patients to receive regular IV infusions. Amicus Therapeutics has already developed an oral form of the therapy for Fabry disease, which the New Jersey-based company expects will gain regulatory approval later this year.

Both oral therapies developed by Genzyme and Amicus would still need to be taken regularly, whereas AvroBio’s modified cell infusion would only need to be done once. If approved, this treatment could have an advantage over competitors as it could deliver a permanent solution to patients with the disease.