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Benefits - Canadian Genetic Diseases Network - CGDN

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Bridging the gap from lab to bedside: Clinical trials for new cancer drug started with early support from CGDN
 

They call it the "Valley of Death" – that high-risk stage when a "discovery" moves from the laboratory, to the patent office, to clinical trials, and finally to patients. CGDN is providing critical support that is helping start-ups navigate this rocky road.

Aegera Therapeutics Inc. is a rising star on the global biotechnology scene. With $20 million in new funding, the Montreal start-up launched Phase 1 clinical trials in March 2004 of a new drug candidate that could prolong the lives of cancer patients with advanced tumours.

It's a promising new therapeutic that might never have left the lab without some critical funding and management support from the Canadian Genetic Diseases Network (CGDN).

Aegera is commercializing technology developed by Drs. Robert Korneluk and Alex MacKenzie of the Children's Hospital of Eastern Ontario. The two geneticists, with ongoing CGDN support, identified a family of genes that is responsible for programmed cell death, or apoptosis, in humans. This fundamental research is fuelling Aegera's efforts to develop therapeutics that kill cancer cells by inducing apoptosis, rescue neurons from cell death, and implant stem cells to restore function.

The initial discoveries were promising, but far too early in the development pipeline to attract serious interest from venture capitalists. Investors want to see much more than an innovative idea or even a discovery. They want protected intellectual property, a strong management team, and a clearly defined target market.

"Research funding usually dries up once a discovery is made," explains Dr. Ron Woznow, CEO of CGDN. "So what do you do? You need money to do the additional experiments to strengthen your intellectual property. This is a black hole that exists within the commercialization process."

CGDN moved aggressively to deal with this gap in 1994 with the launch of its Strategic Grant program, the first program of its kind in Canada. Drs. Korneluk and MacKenzie were among the beneficiaries, receiving $50,000 to do the additional research required for proof-of-principle and the protection of patent claims. CGDN also leveraged its contacts in the Canadian life sciences community to assemble a high-profile interim management team, help secure start-up capital, and launch a new company to study the therapeutic potential of apoptosis control.

In addition, CGDN made its national network of core research facilities available to Drs. Korneluk and MacKenzie. "If CGDN members are in a race to protect a patent, these researchers can get preferential treatment for the use of our core facilities across the country," says Dr. Woznow.

Apoptogen Inc. was launched in 1995 with $8.5 million in seed funding provided by CDP Capital-Technology Ventures, Canadian Medical Discoveries Fund, Neuroscience Partners Limited Partnership, and Working Ventures Canadian Fund. In 2000, Apoptogen merged with Exogen Neurosciences to become Aegera. CGDN continues to hold equity in the company.

CGDN has garnered an impressive track record for commercializing genetic discoveries. The $800,000 awarded through its Strategic Grant program has led to the creation of six new biotechnology companies, including Aegera, Xenon Pharmaceuticals Inc., SignalGene Inc., and NeuroVir Therapeutics (acquired by MediGene). These spinoffs in turn have raised more than $135 million in investments and generated more than 900 jobs.

"Now that Aegera is in clinical trials," Dr. Woznow adds, "it validates the CGDN Strategic Grant program goal of catalyzing the translation of genetic discoveries into new treatments and cures."

www.cgdn.ca

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