How Patient-Derived Cells Are Changing ALS Research

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

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

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