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
rare disease
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
CIRM awards $2.8 million to develop a gene therapy for rare neuromuscular disorders
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
Bringing a community together at the 2nd Annual ALSP Conference
In addition to funding innovative programs and research that accelerate the development of stem cell and gene therapy treatments, the California Institute for Regenerative Medicine (CIRM) provides funding to support valuable mission-specific scientific conferences where people can meet to exchange scientific information to meet goals relevant to CIRM’s mission. Recently, CIRM supported the 2nd Annual … Continue reading Bringing a community together at the 2nd Annual ALSP Conference
Join the movement to fight rare diseases
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
Sweet 16 and counting for stem cell clinical trial
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
Celebrating academic success and overcoming obstacles
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
Creating a ‘bespoke’ approach to rare diseases
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Up until recently the word “bespoke” meant just one thing to me, a hand-made suit, customized and fitted to you. There’s a street in London, Saville Row, that specializes in these suits. They’re gorgeous. They’re also very expensive and so I thought I’d never have a … Continue reading Creating a ‘bespoke’ approach to rare diseases
The bootcamp helping in the fight against 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
Rare Disease: An Uphill Battle for Diagnosis and Treatment
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST From left to right: Baby Dalia pre-diagnosis, Dalia on her way to the kindergarten, and Dalia today. When Dalia was 5 years old, she was finally diagnosed with MERRF syndrome- an extremely rare form of mitochondrial disease. By then, her parents had been searching for an … Continue reading Rare Disease: An Uphill Battle for Diagnosis and Treatment