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
Rare Diseases
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
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
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
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
CIRM awards $8 million to Support Groundbreaking Gene Therapy for AML and MDS
Image source: Canva Around 20,000 Americans are affected by acute myeloid leukemia (AML) each year. AML is a type of cancer that originates in the bone marrow and rapidly enters the bloodstream. Additionally, many individuals are impacted by myelodysplastic syndromes (MDS), which are blood disorders that result in the improper formation or functioning of blood … Continue reading CIRM awards $8 million to Support Groundbreaking Gene Therapy for AML and MDS
CIRM awards $26 million to boost clinical trials for cancer, heart, and rare diseases
The California Institute for Regenerative Medicine (CIRM) is proud to announce the allocation of $26 million to support clinical research targeting a range of cancers, heart conditions, and rare diseases. This significant funding underscores CIRM’s commitment to advancing innovative stem cell and gene therapy-based treatments through all stages of clinical trial development. This latest round … Continue reading CIRM awards $26 million to boost clinical trials for cancer, heart, and rare diseases
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
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 