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Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment March 11, 2025
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 impacts over 2,000 people worldwide and around 500-600 people in […]
Advancing Heart Health: CIRM Funds Tenaya Therapeutics’ Gene Therapy Trial for ARVC March 7, 2025
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 Therapeutics, to support a clinical trial focused on treating […]
Honoring Dr. John Zaia: A Legacy of Science, Leadership, and Kindness February 27, 2025
The scientific community lost a true pioneer with the passing of John A. Zaia, MD, a visionary virologist, gene therapy trailblazer, and esteemed leader at City of Hope. Dr. Zaia dedicated over four decades to advancing regenerative medicine, leaving a profound impact on HIV research, pediatric diseases, stem cell transplantation, and cell and gene therapy. […]