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Small molecules lead to big partnership
SAN FRANCISCO—The University of California, San Francisco's (UCSF) Small Molecule Discovery Center (SMDC) has signed a partnership agreement with Genentech Inc., a wholly owned member of the Roche Group, to discover and develop drug candidates as armor in the fight against neurodegenerative diseases.
Through the agreement, Genentech will provide funding and its research acumen in neuroscience and will collaborate with the SMDC to identify small molecule drugs. Those familiar with the agreement were unwilling to elaborate on the diseases the university would study through this partnership.
Small-molecule drugs—which target a specific protein or enzyme that modulates a biochemical or cellular process and have the potential to alter disease states—have traditionally been highly effective, but are difficult to identify through standard, high-throughput screening.
Genentech will support the work of several researchers at the SMDC, which is administered by the UCSF School of Pharmacy and located in the California Institute for Quantitative Biosciences on the UCSF Mission Bay campus. A research team at Genentech will work closely with the university researchers to develop a drug candidate based on prior academic research conducted at the SMDC and discoveries at Genentech.
Those closest to the agreement say it's a partnership of two unique but complementary organizations.
Dr. Michelle Arkin, associate director of the SMDC, says the match is in part the result of a long-standing relationship the SMDC's director, Dr. Jim Wells, has with Genentech.
"Jim was a scientific leader at Genentech for 16 years before he went on to found Sunesis Pharma," Arkin says. "He then became a chaired professor of pharmaceutical chemistry at UCSF. The SMDC is focused on the application of innovative technologies to challenging targets. We have expertise in lead discovery, but to translate our basic discoveries into new therapies, we need the resources and expertise of a pharmaceutical partner. They are a science-driven organization that is willing to put resources on novel targets towards important unmet medical needs."
Wells and the center's associate directors, Arkin and Dr. Adam Renslo, will lead the project in collaboration with the Genentech team. Preclinical work will be conducted both at Genentech and at UCSF under the direction of a joint steering committee, but Genentech is responsible for clinical development.
Genentech spokeswoman Caroline Pecquet agrees that the match was a perfect fit.
"Since Genentech first announced neuroscience as a research focus area in March of 2008, we have been building upon our in-house expertise to better understand the biology behind neuroscience and translate discoveries into innovative therapies for this area of tremendous unmet need," she says. "This collaboration builds upon the existing master agreement between Genentech and UCSF, which allows the two to collaborate in a streamlined manner. To date, we have entered into more than 15 research collaborations with UCSF, across several therapeutic areas. We believe that this latest agreement with SMDC enhances our relationship with the university and creates a new model for important industry-academic drug development partnership."
According to Pecquet, the master agreement, which was established in 2005, allows the two entities to collaborate in a streamlined manner.
"This relationship can significantly reduce the time it takes to negotiate agreements, thereby accelerating the pace of research efforts," she explains.
Genentech's Pecquet says that the specific terms of the agreement will be kept under wraps, but notes that it is accurate to say that Genentech's financial commitment will support the work of several researchers at the SMDC.
In the future, if certain development and commercial benchmarks are met, the university stands to gain further funding to the tune of at least $13 million, plus the potential for royalties.
Those working within the new partnership say that initial results are heartening.
"Finding targeted compounds is a major obstacle in the drug discovery process, in part because most academic researchers don't have access to this type of facility," says Wells. "This collaboration shows how a center like the SMDC can help support the path from new biology into therapeutic products to help improve patients' lives."
"In the three months we have been working together, we have been impressed by Genentech's commitment and talent," Arkin says. "There is a perfect synergy between their interests in the biology of neurodegenerative diseases and our lead discovery approaches."
The SMDC offers all UC biomedical researchers access to modern small-molecule discovery technologies, including high-throughput assays and follow-up medicinal chemistry, including a novel approach called "tethering," which Wells pioneered before founding the SMDC. The center performs biochemical and cell-based assays using liquid handling robots and a screening library of more than 180,000 compounds. The center acts as a central research facility for scientists whose research would benefit from such technology. Its ultimate goal is to help pave the way for the discovery of new small molecules for research and potential therapeutics.
It also houses a group of medicinal chemistry researchers who optimize screening hits into drug leads. Over the past four years, the center has grown to 18 biologists and chemists, many with pharmaceutical experience.
Genentech has been human genetic information to discover, develop, manufacture and commercialize medicines to treat patients with serious or life-threatening medical conditions for more than 30 years. Today, Genentech is considered among the world's leading biotech companies, with multiple products on the market and a promising development pipeline.