UCSF, Genentech partner to discover and develop drug candidates for neurodegenerative diseases

The University of California, San Francisco has signed a partnership agreement with Genentech, Inc., a wholly owned member of the Roche Group, to discover and develop drug candidates for neurodegenerative diseases.

Through the agreement, Genentech will provide funding and its research acumen in neuroscience and will collaborate with UCSF to identify small molecules.

Genentech will support the work of several researchers at the UCSF Small Molecule Discovery Center (SMDC), which is administered by the UCSF School of Pharmacy and located in the California Institute for Quantitative Biosciences (QB3) on the UCSF Mission Bay campus. A research team at Genentech will work closely with UCSF to develop a drug candidate based on prior academic research conducted at the SMDC and discoveries at Genentech.

In addition to receiving financial support from Genentech for its research function, UCSF has the potential for further funding in excess of $13 million, if certain development and commercial milestones are met, plus royalties.

This is the first major collaboration the SMDC has formed with an industry partner, according to Jim Wells, PhD, who founded the center in 2005 and serves as its director. Wells and the center's associate directors - Adam Renslo, PhD, and Michelle Arkin, PhD - will lead the project in collaboration with the Genentech team.

"What is transformative about this agreement from the University's perspective is that it is a true collaboration between UCSF and Genentech scientists with the intent to generate drug candidates. This is different from a standard out-license or simple research collaboration," said Wells, who is also chair of the Department of Pharmaceutical Chemistry in the UCSF School of Pharmacy and a faculty affiliate of QB3.

"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," Wells said. "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."

This collaboration builds upon the existing master agreement between Genentech and UCSF, which allows the two to collaborate in a streamlined manner.

"We are very pleased to enter this agreement and are hopeful that together we can develop new treatments to help people with neurodegenerative diseases," said Marc Tessier-Lavigne, PhD, executive vice president, research and chief scientific officer of Genentech. "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 partnerships."

The partnership also coincides with the UCSF School of Pharmacy's goal of shaping the future of pharmacy science by working in fresh, collaborative ways, according to Mary Anne Koda-Kimble, PharmD, dean of the UCSF School of Pharmacy.

"This is a tremendous new partnership and one that fits perfectly with one of the school's major strategic goals," Koda-Kimble said. "Our work with QB3 is a great example of how we are doing this. We believe the new UCSF partnership with Genentech takes this to a whole new level."

The SMDC offers all UC biomedical researchers access to modern small-molecule discovery technologies, including high-throughput screening and follow-up medicinal chemistry. The center performs biochemical and cell-based assays using liquid handling robots and a screening library of more than 180,000 compounds. 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.

The center is based on high-throughput screening, medicinal chemistry, and fragment-based screening, including a novel approach called "Tethering," which Wells pioneered in his previous role as founder, president, and chief scientific officer at Sunesis Pharmaceuticals, Inc.

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.

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.