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

Honoring Dr. John Zaia: A Legacy of Science, Leadership, and Kindness 

Photo Courtesy: City of Hope 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, … Continue reading Honoring Dr. John Zaia: A Legacy of Science, Leadership, and Kindness 

FDA Grants RMAT Designation to Luxa Biotechnology’s CIRM-funded Dry AMD Therapy 

Credit: Shutterstock The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation for a project focused on vision loss that was funded in part by the California Institute for Regenerative Medicine (CIRM). This designation is given to highly promising regenerative medicine therapies that treat serious or life-threatening diseases with the goal of accelerating the path … Continue reading FDA Grants RMAT Designation to Luxa Biotechnology’s CIRM-funded Dry AMD Therapy 

Bridging the Gap: CIRM Tackles Cell and Gene Therapy Manufacturing Challenges

CIRM Manufacturing Network Symposium attendees. The California Institute for Regenerative Medicine (CIRM) is driving conversations and collaboration to address the manufacturing challenges of cell and gene therapies (CGT). CIRM hosted its inaugural Manufacturing Network Symposium in South San Francisco, bringing together more than 100 experts and stakeholders from the regenerative medicine field, including scientific leaders … Continue reading Bridging the Gap: CIRM Tackles Cell and Gene Therapy Manufacturing Challenges

Discovery Research into the Link Between Air Pollution and Autism

Infographic, provided by Dr. Liption and team, showcasing the link between air pollution and autism spectrum disorder As outlined in Proposition 14, one of the goals of the California Institute for Regenerative Medicine (CIRM) is to help accelerate discoveries that enhance our understanding of central nervous system (CNS) disorders. As part of this goal, CIRM … Continue reading Discovery Research into the Link Between Air Pollution and Autism

Using Genetic Sequencing and AI to Identify and Treat Schizophrenia

Top left to right: Tomasz Nowakowski, PhD, and Nevan Krogan, PhD Bottom left to right: Aparna Bhaduri, PhD; Matt State, PhD; Alice Ting, PhD; Trey Ideker, PhD; Willow Coyote-Maestas, PhD As outlined in Proposition 14, one of the goals of the California Institute for Regenerative Medicine (CIRM) is to help accelerate discoveries that enhance our … Continue reading Using Genetic Sequencing and AI to Identify and Treat Schizophrenia

How Discovery Research is Unlocking the DNA Behind Psychiatric Disorders

From top left to right: Xin Jin, PhD; Joseph Ecker, PhD; Rusty Gage, PhD Bottom left to right: Eran Mukamel, PhD and Maria Carol Marchetto, PhD As outlined in Proposition 14, one of the goals of the California Institute for Regenerative Medicine (CIRM) is to help accelerate discoveries that enhance our understanding of central nervous … Continue reading How Discovery Research is Unlocking the DNA Behind Psychiatric Disorders

Exploring the Genetic Foundations of Autism and Gene Therapy Treatments

Principal investigator Alex Pollen, PhD, with his co-investigators and team of researchers. As outlined in Proposition 14, one of the goals of the California Institute for Regenerative Medicine (CIRM) is to help accelerate discoveries that enhance our understanding of central nervous system (CNS) disorders. As part of this goal, CIRM launched the Research using Multidisciplinary, … Continue reading Exploring the Genetic Foundations of Autism and Gene Therapy Treatments

CIRM Funds new UCSD VERGE Center to Better Understand Schizophrenia and Autism

From top left to right: Anne Bang, PhD; Carrie Bearden, PhD; Eugene Yeo, PhD; Jonathan Sebat, PhD Bottom left to right: Alysson Moutri, PhD; Lilia Iakoucheva, PhD; Sébastien Jacquemont; Trey Ideker, PhD  This blog is part of a series highlighting our ReMIND program and the researchers it’s supporting. To explore all features in the series, … Continue reading CIRM Funds new UCSD VERGE Center to Better Understand Schizophrenia and Autism