Last week two new papers came out echoing each other about the dangers of bogus “therapies” being offered by predatory stem cell clinics and the risks they pose to patients. The first was from the Pew Charitable Trusts entitled: ‘Harms Linked to Unapproved Stem Cell Interventions Highlight Need for Greater FDA Enforcement’ with a subtitle: … Continue reading Two voices, one message, watch out for predatory stem cell clinics
Food and Drug Administration
Hitting our Goals: Accelerating to the finish line
Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves … Continue reading Hitting our Goals: Accelerating to the finish line
Regulated, Reputable and Reliable: FDA’s Taking Additional Steps to Advance Safe and Effective Regenerative Medicine Products
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research In February 2020, CIRM presented a series of benchmarks for the responsible delivery of stem cell and regenerative medicine products. These benchmarks are outlined in the publication Regulated, reliable and reputable: Protect patients with uniform standards for stem cell treatments. In a nutshell, CIRM … Continue reading Regulated, Reputable and Reliable: FDA’s Taking Additional Steps to Advance Safe and Effective Regenerative Medicine Products
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
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
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
CIRM-funded treatment for cancer granted FDA breakthrough therapy designation
Mark Chao, M.D., Ph.D., cofounder of Forty Seven, Inc. and current VP of oncology clinical research at Gilead Sciences An antibody therapeutic, magrolimab, being tested for myelodysplastic syndrome (MDS), a group of cancers in which the bone marrow does not produce enough healthy blood cells , was granted breakthrough therapy designation with the Food and Drug Administration … Continue reading CIRM-funded treatment for cancer granted FDA breakthrough therapy designation
Graphite Bio launches and will prepare for clinical trial based on CIRM-funded research
Josh Lehrer, M.D., CEO of Graphite Bio This week saw the launch of the 45th startup company enabled by CIRM funding of translational research at California academic institutions. Graphite Bio officially launched with the help of $45M in funding led by bay area venture firms Versant Ventures and Samsara BioCapital to spinout a novel CRISPR … Continue reading Graphite Bio launches and will prepare for clinical trial based on CIRM-funded research
Unproven “stem cell” therapy injuries are more common than we realized
Jaime Imitola, senior author of the paper and director of the Comprehensive Multiple Sclerosis Center at UConn Health Here at CIRM we only fund clinical trials that meet the rigorous standards outlined by the Food and Drug Administration (FDA). These requirements are not only necessary to properly evaluate how effective a potential treatment may be, … Continue reading Unproven “stem cell” therapy injuries are more common than we realized
The Stem Cellar 