A new home for stem cell research? Have you ever been at a party where someone says “hey, I’ve got a good idea” and then before you know it everyone in the room is adding to it with ideas and suggestions of their own and suddenly you find yourself with 27 pages of notes, all … Continue reading Charting a course for the future
FDA
Month of CIRM: Making sure stem cell therapies don’t get lost in Translation
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 blog written by … Continue reading Month of CIRM: Making sure stem cell therapies don’t get lost in Translation
Progress in the fight against Sickle Cell Disease
Marissa Cors, sickle cell disease patient advocate Last November Marissa Cors, a patient advocate in the fight against Sickle Cell Disease (SCD), told the Stem Cellar “A stem cell cure will end generations of guilt, suffering, pain and early death. It will give SCD families relief from the financial, emotional and spiritual burden of caring … Continue reading Progress in the fight against Sickle Cell Disease
A Month of CIRM: Where we’ve been, where we’re going
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. We kick off this event with a … Continue reading A Month of CIRM: Where we’ve been, where we’re going
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
Making a good thing better
Thomas Edison Legend has it that Thomas Edison “failed” 1,000 times before he managed to create the incandescent lightbulb. Edison says he didn’t get discouraged, instead he looked at each unsuccessful experiment as being one step closer to finding the method that really worked. That’s a lesson in optimism and persistence for all of us. … Continue reading Making a good thing better
You can’t take it if you don’t make it
Biomedical specialist Mamadou Dialio at work in the Cedars-Sinai Biomanufacturing Center. Photo by Cedars-Sinai. Following the race to develop a vaccine for COVID-19 has been a crash course in learning how complicated creating a new therapy is. It’s not just the science involved, but the logistics. Coming up with a vaccine that is both safe … Continue reading You can’t take it if you don’t make it
CIRM-funded therapy to ease the impact of chemotherapy
Treatments for cancer have advanced a lot in recent years, but many still rely on the use of chemotherapy to either shrink tumors before surgery or help remove cancerous cells the surgery missed. The chemo can be very effective, but it’s also very toxic. Angiocrine Bioscience Inc. is developing a way to reduce those toxic … Continue reading CIRM-funded therapy to ease the impact of chemotherapy
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
CIRM-funded treatment gets orphan drug and rare pediatric disease designations from FDA
From left to right: Brian Lookofsky , Taylor Lookofsky, and Rosa Bacchetta, M.D. Picture taken October 2019 Last year, CIRM awarded $5.53 million to Rosa Bacchetta, M.D. at Stanford University to complete the work necessary to conduct a clinical trial for IPEX syndrome. This is a rare disease caused by mutations in the FOXP3 gene, which leaves people … Continue reading CIRM-funded treatment gets orphan drug and rare pediatric disease designations from FDA