Yesterday, the governing Board of the California Institute for Regenerative Medicine (CIRM) awarded $8.39 million to UC San Francisco to launch a clinical trial for sickle cell disease.

CIRM also awarded $51.08 million to fifteen California community colleges and universities to support undergraduate and master’s programs that train future stem cell researchers.

Sickle Cell Trial

Sickle cell disease (SCD) is an inherited blood disorder caused by a single mutation in the beta‑globin gene. This change produces defective hemoglobin that polymerizes and damages red blood cells, giving them their characteristic sickle shape. These damaged cells can block blood vessels, leading to organ damage, reduced quality of life, and shorter life expectancy.

Mark Walters, M.D., and his team at UCSF Benioff Children’s Hospital Oakland will run a clinical trial using CRISPR‑Cas9 gene editing to correct the mutation in patients’ blood stem cells. After editing, the team will return the corrected stem cells to patients to restore normal hemoglobin and healthy red blood cell production.

This clinical trial is eligible for co‑funding under the agreement between CIRM and the National Heart, Lung, and Blood Institute (NHLBI) as part of the NHLBI’s “Cure Sickle Cell” initiative. The partnership aims to speed the development of cell and gene therapies for SCD. CIRM previously supported this therapy’s preclinical and IND‑enabling studies through Translational and Late Stage Preclinical awards, in collaboration with NHLBI.

The CIRM Bridges to Stem Cell Research and Therapy program offers undergraduate and master’s students stem cell coursework and hands‑on laboratory experience in academic or biotechnology settings. Fifteen institutions received $51.08 million to continue training the next generation of scientists.

Awards summary

Application	Title	Institution	Award Amount
EDUC2-12607	Bridges to Stem Cell Research and Therapy at Pasadena City College	Pasadena City College	$3,605,500
EDUC2-12611	CIRM Bridges to Stem Cell Research and Therapy Training Grant	CSU San Marcos	$3,606,500
EDUC2-12617	Bridges to Stem Cell Research Internship Program	San Diego State University	$3,606,500
EDUC2-12620	CIRM Bridges 3.0	Humboldt State	$3,605,495
EDUC2-12638	CIRM Regenerative Medicine and Stem Cell Research Biotechnology Training Program	CSU Long Beach	$3,276,500
EDUC2-12677	Stem Cell Internships in Laboratory-based Learning (SCILL) continue to expand the scientific workforce for stem cells research and therapies.	San Jose State University	$3,606,500
EDUC2-12691	Strengthening the Pipeline of Master’s-level Scientific and Laboratory Personnel in Stem Cell Research	CSU Sacramento	$2,946,500
EDUC2-12693	CIRM Bridges Science Master’s Program	San Francisco State University	$3,606,500
EDUC2-12695	CIRM Graduate Student Training in Stem Cell Sciences in the Stem Cell Technology and Lab Management Emphasis of the MS Biotechnology Program	CSU Channel Islands	$3,606,500
EDUC2-12718	CSUN CIRM Bridges 3.0 Stem Cell Research & Therapy Training Program	CSU Northridge	$3,606,500
EDUC2-12720	Stem Cell Scholars: a workforce development pipeline, educating, training and engaging students from basic research to clinical translation.	CSU San Bernardino	$3,606,500
EDUC2-12726	Training Master’s Students to Advance the Regenerative Medicine Field	Cal Poly San Luis Obispo	$3,276,500
EDUC2-12730	Building Career Pathways into Stem Cell Research and Therapy Development	City College of San Francisco	$2,706,200
EDUC2-12734	Bridges to Stem Cell Research and Therapy: A Talent Development Program for Training Diverse Undergraduates for Careers in Regenerative Medicine	CSU Fullerton	$3,606,500
EDUC2-12738	CIRM Bridges to Stem Cell Research and Therapy	Berkeley City College	$2,806,896

“We are pleased to fund a promising trial for sickle cell disease that uses the Nobel Prize winning gene editing technology CRISPR-Cas9,” says Maria T. Millan, M.D., President and CEO of CIRM.  “This clinical trial is a testament to how the CIRM model supports promising early-stage research, accelerates it through translational development, and advances it into the clinics. As the field advances, we must also meet the demand for promising young scientists.  The CIRM Bridges programs across the state of California will provide students with the tools and resources to begin their careers in regenerative medicine.”