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
Rare Pediatric Disease Designation
Good news on two CIRM-funded research on blood disorders
Dr. Rosa Bacchetta on the right with Brian Lookofsky (left) and Taylor Lookofsky after CIRM funded Dr. Bacchetta's work in October 2019. Taylor has IPEX syndrome It’s always lovely to end the week on a bright note and that’s certainly the case this week, thanks to some encouraging news about two CIRM-funded research on blood … Continue reading Good news on two CIRM-funded research on blood disorders
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
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
Progress to a cure for Bubble Baby Disease
Evie, a former "bubble baby" enjoying life by playing inside a giant plastic bubble 'Bubble baby disease' will one day be a thing of the past. That’s a bold statement, but I say it with confidence because of the recent advancements in stem cell gene therapies that are curing infants of this life-threatening immune disease. … Continue reading Progress to a cure for Bubble Baby Disease
CIRM-funded life-saving stem cell therapy gets nod of approval from FDA
If you have read our 2016 Annual Report (and if you haven’t you should, it’s brilliant) or just seen the cover you’ll know that it features very prominently a young girl named Evie Padilla Vaccaro. Evie was born with Severe Combined Immunodeficiency or SCID – also known as “bubble baby disease”; we’ve written about it … Continue reading CIRM-funded life-saving stem cell therapy gets nod of approval from FDA