Exploring the Genetic Foundations of Autism and Gene Therapy Treatments

Principal investigator Alex Pollen, PhD, with his co-investigators and team of researchers. As outlined in Proposition 14, one of the goals of the California Institute for Regenerative Medicine (CIRM) is to help accelerate discoveries that enhance our understanding of central nervous system (CNS) disorders. As part of this goal, CIRM launched the Research using Multidisciplinary, … Continue reading Exploring the Genetic Foundations of Autism and Gene Therapy Treatments

CIRM Funds new UCSD VERGE Center to Better Understand Schizophrenia and Autism

From top left to right: Anne Bang, PhD; Carrie Bearden, PhD; Eugene Yeo, PhD; Jonathan Sebat, PhD Bottom left to right: Alysson Moutri, PhD; Lilia Iakoucheva, PhD; Sébastien Jacquemont; Trey Ideker, PhD  This blog is part of a series highlighting our ReMIND program and the researchers it’s supporting. To explore all features in the series, … Continue reading CIRM Funds new UCSD VERGE Center to Better Understand Schizophrenia and Autism

World AIDS Day at the 3rd Annual Timothy Ray Brown Community Cure Symposium

Event recap written by guest writer Jacqueline Hantgan, CIRM Sr. Outreach Advisor for Outreach and Advocacy December is HIV/AIDS Awareness Month. To commemorate this important event, CIRM recently supported the 3rd Annual Timothy Ray Brown Community Cure Symposium which took place at the Palm Springs Art Museum on World AIDS Day−December 1, 2024. It was … Continue reading World AIDS Day at the 3rd Annual Timothy Ray Brown Community Cure Symposium

Gene Therapy Brings Hope to Families Affected by Rare Immune Disorder

Photo Courtesy of Andrea Fernandez de Soto Imagine a life where every time you step outside, everything in your environment poses a life-threatening risk. For children born with ADA-SCID (adenosine deaminase severe combined immunodeficiency), this is a reality. Their immune systems are so compromised that a common cold or a simple scrape on the knee … Continue reading Gene Therapy Brings Hope to Families Affected by Rare Immune Disorder

A Step Forward in the Fight Against Advanced Kidney Cancer

Photo Courtesy: Allogene Therapeutics Metastatic renal cell carcinoma (RCC) is an advanced form of kidney cancer with few treatment options and a five-year survival rate of less than 17%. With funding support from CIRM, Allogene Therapeutics is developing ALLO-316, an allogeneic CAR-T cell therapy which targets CD70—a protein highly expressed on clear cell renal cell … Continue reading A Step Forward in the Fight Against Advanced Kidney Cancer

Two CIRM-funded research projects receive RMAT designation from FDA

The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to two research projects funded by the California Institute for Regenerative Medicine (CIRM). RMAT is a fast-track designation that can help speed up the development, review, and potential approval of treatments for serious or life-threatening diseases. The first RMAT designation … Continue reading Two CIRM-funded research projects receive RMAT designation from FDA

Remembering CIRM board member and ALS patient advocate Fred Fisher

CIRM would like to honor the contributions of Fred Fisher, MSW, LCSW, a dedicated member of our Independent Citizens’ Oversight Committee (ICOC) governing board and an enthusiastic patient advocate. Fred passed away in September.   Fred joined the CIRM ICOC as the ALS patient advocate in June 2021 and held various roles during his tenure, including … Continue reading Remembering CIRM board member and ALS patient advocate Fred Fisher

UCLA researchers exploring ways to prevent heart failure after heart attacks

ENPP1 is expressed in the region of scarring after injury to the heart. Cardiac muscle shown in red and ENPP1 in green demonstrates expression of ENPP1 in the region of scar formation. | Credit: Arjun Deb Lab/UCLA At the California Institute for Regenerative Medicine (CIRM), we support groundbreaking cardiovascular research from Discovery to Clinical as … Continue reading UCLA researchers exploring ways to prevent heart failure after heart attacks

How California is advancing our understanding of Alzheimer’s disease using stem cells 

Over 14,000 people in Fresno County are living with Alzheimer's, and it’s estimated that more than 720,000 people are living with the brain disorder in California.   That’s why the California Institute for Regenerative Medicine (CIRM)—a state of California agency dedicated to funding regenerative medicine research—joined the Alzheimer's Association Walk to End Alzheimer’s in Fresno/Madera to … Continue reading How California is advancing our understanding of Alzheimer’s disease using stem cells 

Story of Hope: Elpida Therapeutics’ Pioneer Study on Rare Nerve Disease CMT4J

Image source: SickKids -Terry, Georgia, and Michael Pirovolakis CIRM awarded Elpida Therapeutics a $3.9 million grant in December 2023 to support a pioneering preclinical study on Charcot Marie Tooth disease type 4J (CMT4J). CMT4J is an ultra-rare, inherited condition that primarily affects the nerves outside the brain and spinal cord. It accounts for about 0.24% … Continue reading Story of Hope: Elpida Therapeutics’ Pioneer Study on Rare Nerve Disease CMT4J