Meet Ja’Ceon Golden. He was among the first patients treated at UC San Francisco, in partnership with St. Jude Children’s Research Hospital, in a CIRM-funded study for Severe Combined Immunodeficiency. Ja’Ceon was born without a functioning immune system, so even a simple cold could have been fatal. At UCSF, Dr. Mort Cowan’s team collected Ja’Ceon’s … Continue reading Celebrating Ja’Ceon Golden
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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). CIRM-funded researchers Matt Porteus, M.D., Ph.D., and Maria Grazia Roncarolo, M.D., formed the company. Six years ago, Dr. Porteus and Dr. Roncarolo, in conjunction with Stanford University, received … Continue reading CIRM-catalyzed spinout files for IPO to develop therapies for genetic diseases
Paving the Way in Treating Myelodysplastic Syndromes
CIRM just helped reach a major milestone in treating myelodysplastic syndromes (MDS), a rare disorder that affects red blood cell production. Positive early results Jasper Therapeutics announced new data from their Phase 1 clinical trial treating people with MDS. This group of disorders involves abnormal, immature blood‑forming cells in the bone marrow, leading to low … Continue reading Paving the Way in Treating Myelodysplastic Syndromes
New Study Shows CIRM-Supported Therapy Cures More than 95% of Children Born with a Fatal Immune Disorder
Dr. Donald B. Kohn; Photo courtesy UCLA A study published in the New England Journal of Medicine shows that an experimental form of stem cell and gene therapy has cured 48 of 50 children born with a deadly condition called ADA-SCID. Children with ADA-SCID, (severe combined immunodeficiency due to adenosine deaminase deficiency) lack a key … Continue reading New Study Shows CIRM-Supported Therapy Cures More than 95% of Children Born with a Fatal Immune Disorder
Biotechnology companies join forces in developing treatment for X-SCID
Jasper Therapeutics, Inc., a biotechnology company focused on blood stem cell therapies, and Graphite Bio, Inc., a biotechnology company focused on gene editing therapies to treat or cure serious diseases, announced a research and clinical collaboration for a treatment for X-SCID. X-SCID, which stands for X-linked severe combined immunodeficiency, is a genetic disorder that interferes … Continue reading Biotechnology companies join forces in developing treatment for X-SCID
Stem cell therapy for deadly childhood immune disorder goes four for four
The gold standard for any new therapy in the U.S. is approval by the Food and Drug Administration (FDA). This approval clears the therapy for sale and often also means it will be covered by insurance. But along the way there are other designations that can mean a lot to a company developing a new … Continue reading Stem cell therapy for deadly childhood immune disorder goes four for four
First patient in CIRM funded X-CGD trial gives back by working in patient care
Brenden Whittaker Brenden Whittaker was born with a rare genetic disorder called X-linked chronic granulomatous disease (X-CGD). This condition affects the immune system's ability to fight off common germs, specifically bacteria and fungi, and can result in infections that would only be mild for healthy people. Unfortunately for Brenden, he has suffered life-threatening infections that … Continue reading First patient in CIRM funded X-CGD trial gives back by working in patient care
CIRM-funded kidney transplant procedure progresses
Kidney transplant surgery. Medeor Therapeutics, which is running a CIRM-funded clinical trial to help people getting kidney transplants, just got some really good news. The US Food and Drug Administration (FDA) has just granted their product Regenerative Medicine Advanced Therapy (RMAT) designation. That’s a big deal because it may allow them to apply for faster … Continue reading CIRM-funded kidney transplant procedure progresses
Charting a new course for stem cell research
What are the latest advances in stem cell research targeting cancer? Can stem cells help people battling COVID-19 or even help develop a vaccine to stop the virus? What are researchers and the scientific community doing to help address the unmet medical needs of underserved communities? Those are just a few of the topics being … Continue reading Charting a new course for stem cell research
The treatment that saved Evie Vaccaro
Evie Vaccaro You can see in this photo that Evie Vaccaro is full of energy and life. It sometimes feels as if I have known her forever. In a way, I have. I feel lucky for that, and this photo is powerful because her life nearly ended before it began. Evie was born with severe … Continue reading The treatment that saved Evie Vaccaro