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
Research News
Bubble baby treatment cleared to restart clinical trial
Evie Vaccaro: Photo courtesy Nancy Ramos Three families battling a life-threatening immune disorder got some great news last week. A clinical trial that could save the life of their child has once again been given the go-ahead by the US Food and Drug Administration (FDA). The clinical trial is the work of UCLA’s Dr. Don … Continue reading Bubble baby treatment cleared to restart clinical trial
Sweet 16 and counting for stem cell clinical trial
Dr. Judy Shizuru: Photo courtesy Jasper Therapeutics Over the years the California Institute for Regenerative Medicine (CIRM) has invested a lot in helping children born with severe combined immunodeficiency (SCID), a fatal immune disorder. And we have seen great results with some researchers reporting a 95 percent success rate in curing these children. Now there’s … Continue reading Sweet 16 and counting for stem cell clinical trial
Pioneering a new approach to HIV/AIDS
Dr. Steven Deeks. Photo courtesy UCSF I’ve always been impressed by the willingness of individuals to step forward and volunteer for a clinical trial. Even more so when they are the first person ever to test a first-in-human therapy. They really are pioneers in helping advance a whole new approach to treating disease. That’s certainly … Continue reading Pioneering a new approach to HIV/AIDS
So far, some encouraging news for stem cell clinical trial treating epilepsy
Neurona Therapeutics is testing a new therapy for a drug-resistant form of epilepsy and has just released some encouraging early findings. The first patient treated went from having more than 30 seizures a month to just four seizures over a three-month period. This clinical trial, funded by the California Institute for Regenerative Medicine (CIRM), is … Continue reading So far, some encouraging news for stem cell clinical trial treating epilepsy
Funding a Clinical Trial for a Functional Cure for HIV
The use of antiretroviral drugs has turned HIV/AIDS from a fatal disease to one that can, in many cases in the US, be controlled. But these drugs are not a cure. That's why the governing Board of the California Institute for Regenerative Medicine (CIRM) voted to approve investing $6.85 million in a therapy that aims … Continue reading Funding a Clinical Trial for a Functional Cure for HIV
Fast Track Designation for a therapy making transplants safer for children with a fatal immune disorder
Bone marrow transplant For children born with severe combined immunodeficiency (SCID) life can be very challenging. SCID means they have no functioning immune system, so even a simple infection can prove life threatening. Left untreated, children with SCID often die in the first few years of life. There are stem cell/gene therapies funded by the … Continue reading Fast Track Designation for a therapy making transplants safer for children with a fatal immune disorder
The race to cure sickle cell disease
September is National Sickle Cell Awareness Month, a time to refocus our efforts to find new treatments, even a cure, for people with sickle cell disease. Until we get those, CIRM remains committed to doing everything we can to reduce the stigma and bias that surrounds it. Sickle cell disease (SCD) is a rare, inherited … Continue reading The race to cure sickle cell disease
Stem Cell Agency Expands Industry Alliance Program to Accelerate Therapies
An ever-growing array of academic and industry resources are required to rapidly translate scientific discoveries and emerging technologies toward safe and effective regenerative medicine therapies for patients. To help, the California Institute for Regenerative Medicine (CIRM) is creating a network of Industry Resource Partners (IRP) that will make its unique resources available to help accelerate … Continue reading Stem Cell Agency Expands Industry Alliance Program to Accelerate Therapies
The present and future of regenerative medicine
One of the great pleasures of my job is getting to meet the high school students who take part in our SPARK or Summer Internship to Accelerate Regenerative Medicine Knowledge program. It's a summer internship for high school students where they get to spend a couple of months working in a world class stem cell … Continue reading The present and future of regenerative medicine