UCLA Health Sciences Secures $13.9 Million CIRM Grant to Map Genetic Drug Targets Using Advanced Stem Cell Models

UCLA researchers use stem cell "cell villages" and CRISPR to identify drug targets for autism and schizophrenia under a new $13.9 million CIRM grant.

By: AXL Media

Published: Mar 27, 2026, 9:17 AM EDT

Source: Information for this report was sourced from University of California - Los Angeles Health Sciences

Strategic Investment in Neurodevelopmental Drug Discovery

The recent allocation of $13.9 million by the California Institute for Regenerative Medicine to UCLA represents a significant pivot toward biology-based drug discovery for neuropsychiatric conditions. Currently, pharmacological interventions for autism and schizophrenia are limited because the precise molecular differences between neurotypical and affected brain cells remain unmapped. Dr. Daniel Geschwind, a professor of human genetics at UCLA, emphasizes that this research is designed to create a functional roadmap where none currently exists. By studying a vast array of genetic variants in parallel, the team intends to provide the pharmaceutical industry with concrete biological targets that could eventually facilitate the move from laboratory findings to clinical applications.

Precision Modeling Through CRISPR and Organoid Technology

The first phase of the research strategy involves a high-precision approach to known genetic risks using CRISPR gene editing. Researchers will introduce 36 variants linked to autism and 32 variants linked to schizophrenia into human stem cells to observe their developmental trajectories. These edited cells will be grown into 3D organoids—miniature tissue clusters that mimic the architecture of the developing human brain—and assembloids, which are formed by fusing multiple organoids to study inter-regional communication. This methodology allows the team to pinpoint how specific mutations alter the structural and electrical properties of the brain during its most critical prenatal stages.

The Cell Village Platform and Technical Noise Reduction

To address the majority of cases where no single genetic cause is identified, the team is deploying a "cell village" platform pioneered by co-investigator Dr. Michael F. Wells. This technique involves growing stem cell-derived neurons from over 300 diverse donors within a single shared culture dish. Traditionally, subtle biological variations are often obscured by "technical noise" such as slight fluctuations in temperature or oxygen across different wells. By co-culturing cells, these variables are neutralized, and because each donor’s DNA serves as a natural barcode, researchers can accurately trace phenotypic differences back to their specific genetic origins. This approach not only increases the sensitivity of the data but...