Jordan Janz knows how lucky he is. It's been almost six years since Jordan volunteered to be patient number one in a clinical trial at the University of California, San Diego (UCSD) to test a new treatment for cystinosis. People with this rare genetic disease accumulate the amino acid cystine in their organs. The toxic … Continue reading Treatment for one rare disease but plans for many more
Recently, the California Institute for Regenerative Medicine (CIRM) announced a new approach for how its funding can advance treatments for people living with rare diseases. Individually, few people have any single rare disease, but in aggregate, about 30 million people in the U.S. have one of the roughly 10,000 rare diseases, and 95% have no … Continue reading New program to help patients with rare disease
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
Credit: Shutterstock The California Institute for Regenerative Medicine (CIRM) announced in January $100 million in funding for a broad range of projects, including four to support clinical programs. One of these was a $2.8 million award to Patricio Sepulveda, PhD, MBA, CEO of Amplo Biotechnology, Inc. That grant will support the development of a gene … Continue reading CIRM awards $2.8 million to develop a gene therapy for rare neuromuscular disorders
In addition to funding programs that advance stem cell and gene therapy development, CIRM supports mission‑focused scientific conferences that foster information exchange aligned with its goals. Second annual ALSP Conference Recently, CIRM supported the 2nd Annual ALSP Community Conference: Insights and Potential of Cell‑Based Therapies for Leukodystrophies. The conference brought together patients, advocates, care partners, … Continue reading Bringing a community together at the 2nd Annual ALSP Conference
Tomorrow, February 28th, is Rare Disease Day. It’s a day to remind ourselves of the millions of people, and their families, struggling with these diseases. These conditions are also called orphan diseases because, in many cases, drug companies were not interested in adopting them to develop treatments. Here at the California Institute for Regenerative Medicine (CIRM), we … Continue reading Join the movement to fight rare diseases
Dr. Judy Shizuru: Photo courtesy Jasper Therapeutics Over the years the California Institute for Regenerative Medicine (CIRM) has invested a lot in helping children born with severe combined immunodeficiency (SCID), a fatal immune disorder. And we have seen great results with some researchers reporting a 95 percent success rate in curing these children. Now there’s … Continue reading Sweet 16 and counting for stem cell clinical trial
Congratulations to Yasmine Arafa (she/hers), a CIRM Bridges Student Intern at UC Davis Institute for Regenerative Cures! She recently graduated from California State University-Sacramento, officially concluding her Master’s degree and Fulbright Association journey. She conducted research with the aim of developing new therapeutic approaches for rare diseases. Yasmine says, "I have finally passed my thesis defense and am … Continue reading Celebrating academic success and overcoming obstacles
Until recently, “bespoke” meant one thing to me: a custom suit from London’s Savile Row. They’re beautiful—and expensive—so I assumed I’d never own anything bespoke. A tailored approach I was wrong. CIRM is now part of a bespoke effort far more important than clothing. This group aims to create tailor‑made gene therapies for rare diseases. … Continue reading Bespoke approach to rare diseases
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Dr. Emil Kakkis at the Rare Entrepreneur Bootcamp Imagine you or someone you love is diagnosed with a rare disease and then told, “There is no cure, there are no treatments and because it’s so rare no one is even doing any research into developing a … Continue reading The bootcamp helping in the fight against rare diseases