Gene Therapy for Heart Failure: A Conversation with Eric Adler, MD

Image Credit: The Adler Lab at the University of California, San Diego In the fall of 2024, the California Institute for Regenerative Medicine (CIRM) helped sponsor the Gene Therapy Initiative symposium in La Jolla, CA. While there, CIRM staff caught up with a few CIRM grantees who are working on gene therapy approaches to treating … Continue reading Gene Therapy for Heart Failure: A Conversation with Eric Adler, MD

Gene Therapy for Osteoarthritis Shows Positive Results in Early Trial

Image Credit: Shutterstock Here’s a shocking statistic: in 2020, 7.6% of the world’s population was living with the most common form of arthritis – osteoarthritis (OA). That amounts to 595 million people globally and 32.5 million adults in the U.S. That’s a lot of people with painful knees, hips, and other joints. Although pain medication … Continue reading Gene Therapy for Osteoarthritis Shows Positive Results in Early Trial

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 

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

CIRM Showcases Strategic Vision and New Initiatives at 7th Annual Alpha Clinics Network Symposium 

Leveraging CIRM Infrastructure to Improve Accessibility Panel - From left to right: Noah Federman, MD (moderator), Catriona Jamieson, MD, PhD, Mehrdad Abedi, MD, Daniela Bota, MD, PhD, Thomas Buchanan, MD, Brian Fury, MS, Dhruv Sareen, PhD, and Taby Ahsan, PhD The California Institute for Regenerative Medicine (CIRM) recently sponsored the 7th Annual CIRM Alpha Clinics … Continue reading CIRM Showcases Strategic Vision and New Initiatives at 7th Annual Alpha Clinics Network Symposium 

Story of Hope: Elpida Therapeutics’ Pioneer Study on Rare Nerve Disease CMT4J

Image source: SickKids -Terry, Georgia, and Michael Pirovolakis CIRM awarded Elpida Therapeutics a $3.9 million grant in December 2023 to support a pioneering preclinical study on Charcot Marie Tooth disease type 4J (CMT4J). CMT4J is an ultra-rare, inherited condition that primarily affects the nerves outside the brain and spinal cord. It accounts for about 0.24% … Continue reading Story of Hope: Elpida Therapeutics’ Pioneer Study on Rare Nerve Disease CMT4J

Inaugural UC San Diego Gene Therapy Initiative Symposium is fueling progress in the field 

There was no shortage of enthusiasm at the inaugural UC San Diego Gene Therapy Initiative Symposium hosted at the Roth Auditorium at the prestigious Sanford Consortium, where researchers, clinicians, academia, industry, and patient advocates shared discoveries in gene therapy, a modern-day treatment that’s revolutionizing medicine.CIRM was a platinum sponsor of the event.The gene therapy field … Continue reading Inaugural UC San Diego Gene Therapy Initiative Symposium is fueling progress in the field 

Gene therapy trial tests cancer-killing virus in brain tumors 

Dr. Noriyuki Kasahara from UCSF has been awarded $11.8 million by the California Institute for Regenerative Medicine (CIRM) to target newly diagnosed high-grade gliomas. This groundbreaking clinical trial aims to treat high-grade gliomas, one of the most challenging brain cancers to manage.   These aggressive cancers develop in the brain or spinal cord and are notoriously … Continue reading Gene therapy trial tests cancer-killing virus in brain tumors 

Equipping California nurses with skills to support cell and gene therapy patients 

Regenerative medicine is a rapidly evolving field. As more therapies reach commercialization and clinical trial stages, there remains an immediate need to equip direct patient care nurses with skills to support patients on complex clinical trials as well as novel standard-of-care therapies.  To address this need and continue efforts to build a regenerative medicine workforce … Continue reading Equipping California nurses with skills to support cell and gene therapy patients 

Update on SCID patient enrolled in CIRM-funded gene therapy trial

Photo credit: UCSF Hataalii Tiisyatonii Begay (HT) is paving the road for newborns with SCID. When HT was born in 2018 in a remote part of the Navajo nation, he was quickly diagnosed with a rare and -usually fatal- condition. Today, thanks to a therapy developed at UCSF and funded by CIRM, he’s a healthy … Continue reading Update on SCID patient enrolled in CIRM-funded gene therapy trial