Image Credit: The Adler Lab at the University of California, San Diego In the fall of 2024, the California Institute for Regenerative Medicine (CIRM) helped sponsor the Gene Therapy Initiative symposium in La Jolla, CA. While there, CIRM staff caught up with a few CIRM grantees who are working on gene therapy approaches to treating … Continue reading Gene Therapy for Heart Failure: A Conversation with Eric Adler, MD
Research & Science
CIRM funds science research from the discovery stage through all phases of clinical trials. We highlight new findings in gene and cell therapies, as well as technology and related science news.
Gene therapies for Friedreich’s Ataxia: A conversation with Liz Soragni, PhD
Liz Soragni, PhD, Director of Research at the Friedreich’s Ataxia Research Alliance In the fall of 2024, the California Institute for Regenerative Medicine (CIRM) helped sponsor the Gene Therapy Initiative symposium in La Jolla, CA. While there, CIRM staff caught up with a few CIRM grantees who are working on gene therapy approaches to treating … Continue reading Gene therapies for Friedreich’s Ataxia: A conversation with Liz Soragni, PhD
20 years of CIRM: After spurring new research, CIRM’s iPSC repository closes
In celebration of CIRM’s 20th anniversary, we have been reflecting on some of the early projects we supported and their outcomes. This blog is a little different, because it also marks the end of a project. Stem cell lines In 2013 CIRM announced a $32 million project to create a repository of stem cell lines … Continue reading 20 years of CIRM: After spurring new research, CIRM’s iPSC repository closes
A patient’s journey with an epilepsy stem cell therapy
Justin Graves diving by the Grand Cayman Islands In 2007, Justin Graves was working at a SCUBA shop in Louisville, Kentucky, when he had his first epileptic seizure. "SCUBA diving has just been something I've loved my whole life," he said. "My whole family loves it." Medication not always effective Justin had to give up … Continue reading A patient’s journey with an epilepsy stem cell therapy
Filling gaps in federal research funding with a focus on neurological diseases
Over the past 20 years, the California Institute for Regenerative Medicine (CIRM) has supercharged research in California in ways that have often filled gaps in federal research funding and most recently by focusing on gaps in neurological disease. A few different factors are limiting biomedical research funding across the United States. In addition to recent … Continue reading Filling gaps in federal research funding with a focus on neurological diseases
How Patient-Derived Cells Are Changing ALS Research
Research Investigator Julia Kaye, PhD, stands for a portrait in a lab at Gladstone Institutes. Photo courtesy of Gladstone Institutes. Before there can be any therapies for disease, scientists need to understand what goes wrong in the first place. Are cells lost that could be replaced? Is there too much or too little of a … Continue reading How Patient-Derived Cells Are Changing ALS Research
Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment
A 12 year-old Kurt Gillenberg. Photo courtesy of Kurt Gillenberg. When Kurt Gillenberg was 10 months old, his parents knew something wasn’t right. But it wasn’t until he reached 18 months that they found a doctor at the University of California, San Diego, who diagnosed Kurt with cystinosis – a rare genetic, metabolic defect that … Continue reading Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment
Honoring Dr. John Zaia: A Legacy of Science, Leadership, and Kindness
Photo Courtesy: City of Hope The scientific community lost a true pioneer with the passing of John A. Zaia, MD. Dr. Zaia was a visionary virologist, gene therapy trailblazer, and esteemed leader at City of Hope. He dedicated over four decades to advancing regenerative medicine. He left behind a profound impact on HIV research, pediatric … Continue reading Honoring Dr. John Zaia: A Legacy of Science, Leadership, and Kindness
Luxa Biotechnology’s CIRM-funded Dry AMD Therapy Milestone
Credit: Shutterstock The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to a promising new treatment for dry age-related macular degeneration (AMD)—a leading cause of vision loss. The therapy is being developed by Luxa Biotechnology and supported in part by the California Institute for Regenerative Medicine (CIRM). What RMAT Designation Means RMAT designation … Continue reading Luxa Biotechnology’s CIRM-funded Dry AMD Therapy Milestone
The Link Between Air Pollution and Autism
The California Institute for Regenerative Medicine (CIRM) is supporting innovative research into neuro diseases, including a project examining how air pollution may contribute to autism. Through the Research using Multidisciplinary, Innovative approaches in Neuro Diseases (ReMIND) program, Stuart Lipton, MD, PhD, and co-investigators Tomo Nakamura, PhD, John Yates, PhD, Marisa Roberto, PhD, and Nik Schork, … Continue reading The Link Between Air Pollution and Autism