Research Investigator Julia Kaye, PhD, stands for a portrait in a lab at Gladstone Institutes. Photo courtesy of Gladstone Institutes. Before there can be any therapies for disease, scientists need to understand what goes wrong in the first place. Are cells lost that could be replaced? Is there too much or too little of a … Continue reading How Patient-Derived Cells Are Changing ALS Research
Month: March 2025
A CIRM Bridges student discovers a passion for engineering
CIRM Bridges student Esme Ruiz-Garcia Esmerelda “Esme” Ruiz-Garcia grew up in California in a Mexican-American family, where pursuing a career in healthcare is considered a family calling, with most of her relatives in the nursing and medical fields. “I’ve always loved science, but I was not that interested in the medical field itself,” said Esme. … Continue reading A CIRM Bridges student discovers a passion for engineering
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
Advancing Heart Health: CIRM Funds Tenaya Therapeutics’ Gene Therapy Trial for ARVC
Histology of human cardiac muscle under microscope. Credit: Shutterstock The California Institute for Regenerative Medicine (CIRM) recently announced funding a series of projects spanning the earliest stages of research through to clinical trials. One of those projects was an $8 million award to Matthew Pollman, MD, MS, Senior Vice President of Clinical Development at Tenaya … Continue reading Advancing Heart Health: CIRM Funds Tenaya Therapeutics’ Gene Therapy Trial for ARVC