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
clinical trials
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
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
Gene Therapy Brings Hope to Families Affected by Rare Immune Disorder
Photo Courtesy of Andrea Fernandez de Soto Imagine a life where every time you step outside, everything in your environment poses a life-threatening risk. For children born with ADA-SCID (adenosine deaminase severe combined immunodeficiency), this is a reality. Their immune systems are so compromised that a common cold or a simple scrape on the knee … Continue reading Gene Therapy Brings Hope to Families Affected by Rare Immune Disorder
A Step Forward in the Fight Against Advanced Kidney Cancer
Photo Courtesy: Allogene Therapeutics Metastatic renal cell carcinoma (RCC) is an advanced form of kidney cancer with few treatment options and a five-year survival rate of less than 17%. With funding support from CIRM, Allogene Therapeutics is developing ALLO-316, an allogeneic CAR-T cell therapy which targets CD70—a protein highly expressed on clear cell renal cell … Continue reading A Step Forward in the Fight Against Advanced Kidney Cancer
Two CIRM-funded research projects receive RMAT designation from FDA
The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to two research projects funded by the California Institute for Regenerative Medicine (CIRM). RMAT is a fast-track designation that can help speed up the development, review, and potential approval of treatments for serious or life-threatening diseases. The first RMAT designation … Continue reading Two CIRM-funded research projects receive RMAT designation from FDA
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
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
Where Are They Now: Jake Javier
The California Institute for Regenerative Medicine (CIRM) in partnership with the University of Southern California (USC) recently hosted the 2024 CIRM Trainee Network Conference, which provided trainees from CIRM’s education and training programs an opportunity to engage with patient advocates and other professionals in the field. On the first day, Jake Javier took the stage … Continue reading Where Are They Now: Jake Javier
A Conversation with CEO Cory Nicholas on Neurona Therapeutics’ Recent RMAT Designation
At the California Institute for Regenerative Medicine (CIRM), we enjoy highlighting achievements that come out of the research we support. We recently sat down with Cory Nicholas, PhD, CEO and Co-Founder of Neurona Therapeutics, to discuss an important milestone for the clinical-stage company. Neurona recently received the FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation for … Continue reading A Conversation with CEO Cory Nicholas on Neurona Therapeutics’ Recent RMAT Designation