Gene Therapy for Heart Failure: A Conversation with Eric Adler, MD

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

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

Inaugural UC San Diego Gene Therapy Initiative Symposium is fueling progress in the field 

There was no shortage of enthusiasm at the inaugural UC San Diego Gene Therapy Initiative Symposium hosted at the Roth Auditorium at the prestigious Sanford Consortium, where researchers, clinicians, academia, industry, and patient advocates shared discoveries in gene therapy, a modern-day treatment that’s revolutionizing medicine.CIRM was a platinum sponsor of the event.The gene therapy field … Continue reading Inaugural UC San Diego Gene Therapy Initiative Symposium is fueling progress in the field 

CIRM awards $25 million for discovery and clinical research, infrastructure programs

Stock image of a researcher The California Institute for Regenerative Medicine (CIRM) has recently approved awarding $25 million to fund multiple projects across discovery research, clinical research, and infrastructure programs.This funding will support 11 projects under CIRM's Foundation Awards Program, which aims to drive rigorous studies addressing critical basic knowledge gaps in the biology of … Continue reading CIRM awards $25 million for discovery and clinical research, infrastructure programs

Novartis acquisition validates CIRM’s impact on cystinosis treatment

Dr. Stephanie Cherqui The future looks brighter for cystinosis patients and their families.   As one of 7,000 rare diseases, cystinosis causes an abnormal build-up of the amino acid cystine that can lead to organ failure and premature death. With little to no treatment options, children and young adults primarily affected by the condition often have … Continue reading Novartis acquisition validates CIRM’s impact on cystinosis treatment

Making transplants easier for kids, and charting a new approach to fighting solid tumors.

Every year California performs around 100 kidney transplants in children but, on average, around 50 of these patients will have their body reject the transplant. These children then have to undergo regular dialysis while waiting for a new organ. Even the successful transplants require a lifetime of immunosuppression medications. These medications can prevent rejection but … Continue reading Making transplants easier for kids, and charting a new approach to fighting solid tumors.

Reasons to be thankful this Thanksgiving: creative nerds

We at the California Institute for Regenerative Medicine have a lot to be thankful for this Thanksgiving. We get to work with some extraordinary colleagues, we get to know some remarkable patient advocates who are pioneers in volunteering for stem cell and gene therapies, and we have a front row seat in a movement that … Continue reading Reasons to be thankful this Thanksgiving: creative nerds

CIRM Board Approves Funding for New Clinical Trial Targeting Brain Tumors

The governing Board of the California Institute for Regenerative Medicine (CIRM) has awarded almost $12 million to carry out a clinical trial targeting brain tumors. This brings the total number of CIRM funded clinical trials to 83.   $11,999,984 was awarded to Dr. Jana Portnow at the Beckman Research Institute of City of Hope. They are using Neural stem … Continue reading CIRM Board Approves Funding for New Clinical Trial Targeting Brain Tumors

How the Tooth Fairy is helping unlock the secrets of autism

Our 2021-22 Annual Report is now online. It’s filled with information about the work we have done over the last year (we are on a fiscal calendar year from July 1 – June 30), the people who have helped us do that work, and some of the people who have benefited from that work. One … Continue reading How the Tooth Fairy is helping unlock the secrets of autism

From our house to the White House. Kinda

Jackie Ward, PhD. Photo courtesy National Institute of Neurological Diseases and Stroke It's always fun to meet someone early in their career and see how they grow and evolve and take on new challenges. I first met Jackie Ward when she received a training grant from CIRM while she studied for her PhD at the … Continue reading From our house to the White House. Kinda