Every year California performs around 100 kidney transplants in children but, on average, around 50 of these patients will have their body reject the transplant. These children then have to undergo regular dialysis while waiting for a new organ. Even the successful transplants require a lifetime of immunosuppression medications. These medications can prevent rejection but … Continue reading Making transplants easier for kids, and charting a new approach to fighting solid tumors.
IND
CIRM-catalyzed spinout files for IPO to develop therapies for genetic diseases
Graphite Bio, a CIRM-catalyzed spinout from Stanford University that launched just 14 months ago has now filed the official SEC paperwork for an initial public offering (IPO). The company was formed by CIRM-funded researchers Matt Porteus, M.D., Ph.D. and Maria Grazia Roncarolo, M.D. Six years ago, Dr. Porteus and Dr. Roncarolo, in conjunction with Stanford … Continue reading CIRM-catalyzed spinout files for IPO to develop therapies for genetic diseases
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
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
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
CIRM Board Expands COVID-19 Efforts
Coronavirus particles, illustration. Courtesy KTSDesign/Science Photo Library This past Friday, the governing Board of the California Institute for Regenerative Medicine (CIRM) expanded the eligibility criteria for COVID-19 related projects to develop new treatments against the virus. Just two weeks ago, the Board approved $5 million in emergency funding for COVID-19 research. One major addition is allowing research related … Continue reading CIRM Board Expands COVID-19 Efforts
Turning the corner with the FDA and NIH; CIRM creates new collaborations to advance stem cell research
This blog is part of the Month of CIRM series on the Stem Cellar A lot can change in a couple of years. Just take our relationship with the US Food and Drug Administration (FDA). When we were putting together our Strategic Plan in 2015 we did a survey of key players and stakeholders at … Continue reading Turning the corner with the FDA and NIH; CIRM creates new collaborations to advance stem cell research
ViaCyte Advances Cell Replacement Therapy for High Risk Type 1 Diabetes
San Diego regenerative medicine company ViaCyte announced this week that the Food and Drug Administration (FDA) approved their Investigational New Drug (IND) Application for PEC-Direct, a cell-based therapy to treat patients at risk for severe complications caused by type 1 diabetes. In the US, IND approval is the final regulatory step required before a therapy … Continue reading ViaCyte Advances Cell Replacement Therapy for High Risk Type 1 Diabetes
A ‘Call to Action’ for change at the FDA
It’s bad enough to have to battle a debilitating and ultimately deadly disease like Huntington’s disease (HD). But it becomes doubly difficult and frustrating when you feel that the best efforts to develop a therapy for HD are running into a brick wall. That’s how patients and patient advocates working on HD feel as they … Continue reading A ‘Call to Action’ for change at the FDA
Creating a “Pitching Machine” to speed up our delivery of stem cell treatments to patients
When baseball players are trying to improve their hitting they’ll use a pitching machine to help them fine tune their stroke. Having a device that delivers a ball at a consistent speed can help a batter be more consistent and effective in their swing, and hopefully get more hits. That’s what we are hoping our … Continue reading Creating a “Pitching Machine” to speed up our delivery of stem cell treatments to patients
The Stem Cellar 