Dr. Matthew Porteus The US Food and Drug Administration (FDA) has granted Investigational New Drug (IND) permission enabling Graphite Bio to test the investigational, potentially revolutionary gene editing therapy GPH101 developed under the supervision of Matthew Porteus, MD, PhD, in a clinical trial for people with sickle cell disease (SCD). The California Institute for Regenerative … Continue reading CIRM-Funded Project Targeting Sickle Cell Disease Gets Green Light for Clinical Trial
Stem cells
CIRM-Funded Clinical Trial for Sickle Cell Gives Hope to People Battling the Disease
Marissa Cors (right) with her mother Adrienne Shapiro Marissa Cors has lived with Sickle Cell Disease (SCD) for more than 40 years. The co-founder of The Sickle Cell Experience Live, an online platform designed to bring more awareness to Sickle Cell Disease around the world, says it’s hard, knowing that at any moment you may … Continue reading CIRM-Funded Clinical Trial for Sickle Cell Gives Hope to People Battling the Disease
Thank you
Bob Klein These last few days have been interesting on so many levels. First the presidential race has kept the nation on tenterhooks. Closer to home the vote count for Proposition 14, to refunded CIRM, has been painstakingly slow (by the way, painstakingly means “with great care and thoroughness” for which we thank all the … Continue reading Thank you
CIRM-funded development of stem cell therapy for Canavan disease shows promising results
Yanhong Shi, Ph.D., City of Hope Canavan disease is a fatal neurological disorder, the most prevalent form of which begins in infancy. It is caused by mutation of the ASPA gene, resulting in the deterioration of white matter (myelin) in the brain and preventing the proper transmission of nerve signals. The mutated ASPA gene causes … Continue reading CIRM-funded development of stem cell therapy for Canavan disease shows promising results
A look back at 15 years of CIRM funding at UCLA
Researchers in the lab of CIRM grantee Donald Kohn, M.D. Image Credit: UCLA Broad Stem Cell Center Since the first grant was issued in April 2006, CIRM has funded a wide range of research conducted by top scientists at UCLA for a wide range of diseases. To give a retrospective look at all the research, … Continue reading A look back at 15 years of CIRM funding at UCLA
Cures, clinical trials and unmet medical needs
When you have a great story to tell there’s no shame in repeating it as often as you can. After all, not everyone gets to hear first time around. Or second or third time. So that’s why we wanted to give you another opportunity to tune into some of the great presentations and discussions at … Continue reading Cures, clinical trials and unmet medical needs
Partners in health
From left to right: Heather Dahlenburg, staff research associate; Jan Nolta, director of the Stem Cell Program; Jeannine Logan White, advanced cell therapy project manager; Sheng Yang, graduate student, Bridges Program, Humboldt State University, October 18, 2019. (AJ Cheline/UC Davis) At CIRM we are modest enough to know that we can't do everything by ourselves. … Continue reading Partners in health
CIRM-funded kidney transplant procedure eyeing faster approval
Kidney transplant surgery. Medeor Therapeutics, which is running a CIRM-funded clinical trial to help people getting kidney transplants, just got some really good news. The US Food and Drug Administration (FDA) has just granted their product Regenerative Medicine Advanced Therapy (RMAT) designation. That’s a big deal because it means they may be able to apply … Continue reading CIRM-funded kidney transplant procedure eyeing faster approval
Meet the people who are changing the future
Kristin MacDonald Every so often you hear a story and your first reaction is “oh, I have to share this with someone, anyone, everyone.” That’s what happened to me the other day. I was talking with Kristin MacDonald, an amazing woman, a fierce patient advocate and someone who took part in a CIRM-funded clinical trial … Continue reading Meet the people who are changing the future
Perseverance: from theory to therapy. Our story over the last year – and a half
Some of the stars of our Annual Report It’s been a long time coming. Eighteen months to be precise. Which is a peculiarly long time for an Annual Report. The world is certainly a very different place today than when we started, and yet our core mission hasn’t changed at all, except to spring into … Continue reading Perseverance: from theory to therapy. Our story over the last year – and a half