By Debbie Strickland

Four months into a collaborative agreement with Genetics Institute Inc. (GI), a small, privately held developmental biology company has identified a potential therapeutic protein.

Ontogeny Inc., of Cambridge, Mass., has purchased an exclusive, nine-month licensing option for an undisclosed protein from GI's DiscoverEase library of novel secreted human proteins.

"While we realize that at such an early stage any one protein or gene has a low probability of becoming a product, this early success validates both companies' initiatives," said Doros Platika, president and CEO of Ontogeny.

Platika said his company has screened more than 100 DiscoverEase proteins and will continue to participate through the library's development even as the company works on further characterization of the optioned molecule.

DiscoverEase is the product of GI's methodical program of identifying and filing patents on all novel secreted human proteins. The proteins are shipped, 94 at a time, to collaborative partners. The package includes a database of genetic information, which also tells users in which tissues the proteins are secreted and whether they are extracellular or membrane associated. The company is filing composition-of-matter patent applications for each protein upon discovery.

GI won't say how many proteins it has identified, but estimates the completed library will be finished in two years and will comprise at least 5,000 entries.

Ontogeny is a three-year-old company with no products yet approved, but GI's other DiscoverEase partners are all established corporations with sales in the $1 billion range: Chiron Corp., of Emeryville, Calif., Genentech Inc., of South San Francisco, and Kirin Brewery Ltd., of Tokyo. Each partner has equal access, said GI spokesman Marc Blaustein.

"Small companies and large companies can benefit from the program," said Blaustein. "Ontogeny is a small company with very good science and it was the first one to take this step [of optioning a protein]. It shows that it's not just a question of the resources you can put toward the program, it's a question of whether you have good science in a specific area."

Ontogeny picked the protein using its OntoScreen assay system, which includes traditional and proprietary assays for determining expression patterns of genes during development and in adult organs.

The company focuses on identifying control molecules that cause cell and tissue differentiation. Disease targets are Parkinson's disease and Alzheimer's disease; diabetes; bone and cartilage disorders, including osteoporosis and fractures; fertility problems and cancer.

If Ontogeny's DiscoverEase protein pans out, the company may exercise its option for a license covering the protein, corresponding cDNA and related technology. In addition to license fees, GI may either receive milestone and royalty payments, or codevelop and co-commercialize resulting therapeutics. Specific financial terms have not been disclosed.

GI also is pursuing big pharma partners interested in small-molecule approaches to disease, according to Blaustein.

"Our program was founded as a protein therapeutics program," he said, "but we've expanded the terms to cover small-molecule drugs, and with that change, larger pharmaceutical companies are now more interested."

If a useful small-molecule interaction is noted and the partnering company opts to go forward with drug development, it will pay GI benchmarks and royalties, but GI will have no codevelopment rights. *