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
Orphan Drug Designation
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
CIRM-supported therapy for blood cancers gets FDA fast track
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST People often complain about how long it can take to turn a scientific discovery into an approved therapy for patients. And they’re right. It can take years, decades even. But for Immune-Onc Therapeutics the path to FDA approval may just have been shortened. Back in April … Continue reading CIRM-supported therapy for blood cancers gets FDA fast track
Looking back and looking forward: good news for two CIRM-supported studies
https://open.spotify.com/episode/4IimCtQ8STpFikwZRXqSAB 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 CIRM-funded research targeting blood … Continue reading Looking back and looking forward: good news for two CIRM-supported studies
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
CIRM-funded treatment for Cystinosis receives orphan drug designation
Dr. Stephanie Cherqui, UC San Diego Orphan drug designation is a special status given by the Food and Drug Administration (FDA) for potential treatments of rare diseases that affect fewer than 200,000 in the U.S. This type of status can significantly help advance treatments for rare diseases by providing financial incentives in the form of … Continue reading CIRM-funded treatment for Cystinosis receives orphan drug designation
How developing a treatment for a rare disease could lead to therapies for other, not-so-rare conditions
Logan Lacy, a child with AADC Deficiency: Photo courtesy Chambersburg Public Opinion Tomorrow, the last day in February, is Rare Disease Day. It’s a day dedicated to raising awareness about rare diseases and the impact they have on patients and their families. But the truth is rare diseases are not so rare. There are around … Continue reading How developing a treatment for a rare disease could lead to therapies for other, not-so-rare conditions
Stories that caught our eye: FDA grants orphan drug status to CIRM-funded therapy; stunning discovery upends ideas of cell formation; and how tadpoles grow new tails
Gut busting discovery Intestinal stem cells: Photo courtesy Klaus Kaestner, Penn Institute for Regenerative Medicine It’s not often you read the word “sensational” in a news release about stem cells. But this week researchers at the University of Copenhagen released findings that are overturning long-held ideas about the development of cells in our stomachs. So … Continue reading Stories that caught our eye: FDA grants orphan drug status to CIRM-funded therapy; stunning discovery upends ideas of cell formation; and how tadpoles grow new tails
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
The Stem Cellar 