Sickle shaped red blood cells The University of California, San Francisco (UCSF), in collaboration with UC Berkeley (UCB) and UC Los Angeles (UCLA), have been given permission by the US Food and Drug Administration (FDA) to launch a first-in-human clinical trial using CRISPR technology as a gene-editing technique to cure Sickle Cell Disease. This research … Continue reading Three UC’s Join Forces to Launch CRISPR Clinical Trial Targeting Sickle Cell Disease
bioengineering
Prime Time for Rocket
Rocket Pharmaceuticals, a company that specializes in developing genetic therapies for rare childhood disorders, just got a big boost from the European Medicines Agency (EMA). They were given a Priority Medicines (PRIME) designation for their therapy for Leukocyte Adhesion Deficiency-1 (LAD-1). CIRM is funding ($6.56 million) Rocket’s clinical trial for LAD-I, an immune disorder that … Continue reading Prime Time for Rocket
A Match Made in Heaven, if heaven were in Oakland!
The Matchmaker - by Gerrit van Honthorst Throughout history, matchmakers have played an important role in bringing together couples for arranged marriages. Fast forward to today and CIRM is now playing a similar role. We’re not looking to get anyone hitched, what we are trying to do is create partnerships between people we are funding … Continue reading A Match Made in Heaven, if heaven were in Oakland!
A little history in the making by helping the tiniest patients
Dr. Diana Farmer stands with Dr. Aijun Wang and their UC Davis research team. It’s appropriate that at the start of Women’s History Month, UC Davis’ Dr. Diana Farmer is making a little history of her own. She launched the world’s first clinical trial using stem cells to treat spina bifida before the child is … Continue reading A little history in the making by helping the tiniest patients
A word from our Chair, several in fact
In 2005, the New Oxford American Dictionary named “podcast" its word of the year. At the time a podcast was something many had heard of but not that many actually tuned in to. My how times have changed. Now there are some two million podcasts to chose from, at least according to the New York … Continue reading A word from our Chair, several in fact
Tipping our hat to the good guys (& gals)
A search on Google using the term “stem cell blogs” quickly produces a host of sites offering treatments for everything from ankle, hip and knee problems, to Parkinson’s disease and asthma. Amazingly the therapies for those very different conditions all use the same kind of cells produced in the same way. It’s like magic. Sadly, … Continue reading Tipping our hat to the good guys (& gals)
Everything you wanted to know about COVID vaccines but never got a chance to ask
All this month we are using our blog and social media to highlight a new chapter in CIRM’s life, thanks to the voters approving Proposition 14. We are looking back at what we have done since we were created in 2004, and also looking forward to the future. Today we feature a rare treat, an … Continue reading Everything you wanted to know about COVID vaccines but never got a chance to ask
How a CIRM scholar helped create a life-saving COVID vaccine
Dr. Derrick Rossi might be the most famous man whose name you don’t recognize. Dr. Rossi is the co-founder of Moderna. Yes, that Moderna. The COVID-19 vaccine Moderna. The vaccine that in clinical trials proved to be around 95 percent effective against the coronavirus. Dr. Rossi also has another claim to fame. He is a … Continue reading How a CIRM scholar helped create a life-saving COVID vaccine
Persistence pays off in search for clue to heart defects
A team of scientists led by Benoit Bruneau (left), including Irfan Kathiriya (center) and Kavitha Rao (right), make inroads into understanding what genes are improperly deployed in some cases of congenital heart disease. Photo courtesy Gladstone Institute For more than 20 years Dr. Benoit Bruneau has been trying to identify the causes of congenital heart … Continue reading Persistence pays off in search for clue to heart defects
CIRM-Funded Project Targeting Sickle Cell Disease Gets Green Light for Clinical Trial
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
The Stem Cellar 