The Molecular Therapeutics Initiative:
Harnessing Campus-wide Research for New Drug Discovery
By Kirsten Mickelwait
Last summer, MCB launched a new division, Molecular Therapeutics (MTx), for those seeking to translate fundamental discoveries from cell biology, biochemistry, and genetics into next-generation therapeutics to improve human health. The program pursues research in biochemical and cellular mechanisms in disease that can be used to develop new therapeutic modalities.
On January 1, MTx launched a new enterprise: the Molecular Therapeutics Initiative (MTI), which will accelerate drug discovery at the intersection of academia and the biotech industry. Catalyzed by a generous $10 million gift from a private donor, the initiative will harness foundational biological and chemical discoveries within molecular therapeutics research across campus to grow those projects into drug-discovery programs.
The program's infrastructure will be formed by an interdisciplinary community of scientists from UC Berkeley labs, external academic institutes, biopharma companies, and venture capital partners. Their initial research will focus on the interaction between metabolites and proteins to reveal new biological pathways that can modulate “undruggable” proteins.
Over 90 percent of all human proteins are undruggable— they don't possess well-defined binding pockets for drug targeting. The challenge is to develop small molecules, biologics, or any other therapeutic modality to develop drugs against these targets. MTI’s collaborative research will help to create next-generation, induced-proximity modalities in the greatest areas of need such as cancer, rare diseases, and neurodegeneration.
“In academia, we’re able to address complex biological challenges and deliver transformative innovations in areas where pharma may not have the bandwidth, but we often lack the resources to push those innovations into the clinic,” says Daniel Nomura, MTI co-director and professor of chemical biology in the Departments of Chemistry, Molecular and Cell Biology, and Nutritional Sciences and Toxicology. “We envision the MTI as the catalytic spark in a pipeline engine, enabling us to more quickly advance promising early-stage research into development programs—whether as independent biotech start-ups or through pharma collaborations.”
He cites transformational discoveries that set the precedent for the initiative, like Jennifer Doudna’s CRISPR-Cas9 and the Innovative Genomics Institute (IGI) development, which is harnessing gene-editing technologies for patient impact. The academic nature of the MTI will allow its researchers to focus on diseases that don’t afflict many patients—such as early childhood diseases—and are often neglected by large pharmaceutical companies. The initiative is anticipated to be a parallel effort to the IGI, where it can develop therapeutic technologies or harness already developed therapeutic technologies using a variety of approaches.
“The MTI comes at the right time and place,” says MTI co-director Roberto Zoncu, associate professor of biochemistry, biophysics, and structural biology. “It builds on MCB’s unique strengths—in particular, its long history of fundamental discoveries that have changed our understanding of gene regulation, gene editing, cellular architecture, signal transduction, and protein quality control. We’re not only advancing R&D but also democratizing it by building industry connections for emerging researchers interested in biotech careers.”
To provide training opportunities for Berkeley students and to accelerate the translation of big biology breakthroughs into new drugs, some of the work at the MTI will be performed in collaboration with pharmaceutical companies, as well as new biotech start-ups to emerge from MTI efforts. “Everyone on our advisory board—which includes Berkeley faculty like Michael Rapé, Robert Tjian, and Jennifer Doudna as well as industry veterans—have started biotech companies and know what it takes to develop new drugs,” says Nomura. “We’ll leverage all that experience to translate our basic science more quickly.”
“The MTI will integrate labs and invest in shared technologies and instrumentation,” says Michael Rapé, MTx head and investigator of the Howard Hughes Medical Institute in the MCB Department. “Students will be at the forefront of translating their discoveries in biological mechanisms into new approaches to therapy. By uniting diverse expertise in this unique way, we’ll be innovating education and making significant advancements in small-molecule and biologic-based therapeutics.”
Banner photo credit: Keegan Houser
Back to Main Spring 2024 Newsletter Page
| Connect With Us! | ||||
 MCB X |
 LinkedIn Postdocs, PhDs, or Undergrads |
 Cal Alumni Network |
 Give to MCB |
|
