Written by Holly Alyssa MacCormick Image Credit: Audrey Davidow－Calvin outside Wanting the best for your child is a universal experience, and Audrey Davidow wanted her baby to have a bright future full of possibility. But in March of 2012, her 13-month-old son Calvin was diagnosed with a rare disease called Pitt Hopkins Syndrome, and she … Continue reading Early-stage research could—one day—benefit many people living with genetic diseases
Patient Advocacy
Winner of Beast Games becomes champion for his son’s rare disease
Written by Holly Alyssa MacCormick You can do a lot with $10 million. Yet, when Jeffrey Randall Allen won the largest prize in game show history on Amazon Prime Video’s Beast Games there was only one thing he wanted to spend his $10 million on—research that might help find a cure for his son’s rare … Continue reading Winner of Beast Games becomes champion for his son’s 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
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
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
ICYMI – From Undergratuate to Hero: Solano Student’s Life-Saving Bone Marrow Match Begins with One Swab
Image Source: Jose Solorio At the California Institute for Regenerative Medicine (CIRM), we believe stem cells have the potential to change lives. Jose Solorio, a Solano Community College student and participant in CIRM’s COMPASS training program, has firsthand experience in witnessing the power of stem cells as a donor. As part of CIRM's COMPASS program, … Continue reading ICYMI – From Undergratuate to Hero: Solano Student’s Life-Saving Bone Marrow Match Begins with One Swab
Turning Hope into Reality: How a Genetic Therapy Helped Connor
Photo credit: n-Lorem Connor Dalby is a 15-year-old teenager who loves music and the beach, but his journey has been far from ordinary. Diagnosed with a rare mutation in the SCN2A gene, his life has been filled with challenges. From the time he was just a few weeks old, he had trouble sleeping and struggled … Continue reading Turning Hope into Reality: How a Genetic Therapy Helped Connor
Where Are They Now: Jake Javier
The California Institute for Regenerative Medicine (CIRM) in partnership with the University of Southern California (USC) recently hosted the 2024 CIRM Trainee Network Conference, which provided trainees from CIRM’s education and training programs an opportunity to engage with patient advocates and other professionals in the field. On the first day, Jake Javier took the stage … Continue reading Where Are They Now: Jake Javier
Exploring Advances in Eye Disease Webinar
The California Institute for Regenerative Medicine (CIRM) has been at the forefront of advancing cellular and gene therapies for various forms of vision loss—including retinitis pigmentosa (RP)—making significant progress in this critical field. Ongoing research and clinical trials are vital to further these advancements. We invite you to join our webinar, Exploring Advances in Eye Disease … Continue reading Exploring Advances in Eye Disease Webinar
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
The Stem Cellar 