You can tell an awful lot about a company by the people it hires and the ability it gives them to do their job in an ethical, principled way. By that measure Rocket Pharma is a pretty darn cool company. Rocket Pharma is running a CIRM-funded clinical trial for Leukocyte Adhesion Deficiency-I (LAD-I), a rare … Continue reading Going the extra mile to save a patient’s life
Immune Disease
A word from our Chair, several in fact
In 2005, the New Oxford American Dictionary named “podcast" its word of the year. At the time a podcast was something many had heard of but not that many actually tuned in to. My how times have changed. Now there are some two million podcasts to chose from, at least according to the New York … Continue reading A word from our Chair, several in fact
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
Cures, clinical trials and unmet medical needs
When you have a great story to tell there’s no shame in repeating it as often as you can. After all, not everyone gets to hear first time around. Or second or third time. So that’s why we wanted to give you another opportunity to tune into some of the great presentations and discussions at … Continue reading Cures, clinical trials and unmet medical needs
It’s all about the patients
Ronnie, born with a fatal immune disorder now leading a normal life thanks to a CIRM-funded stem cell/gene therapy: Photo courtesy of his mum Upasana Whenever you are designing something new you always have to keep in mind who the end user is. You can make something that works perfectly fine for you, but if … Continue reading It’s all about the patients
CIRM-funded kidney transplant procedure eyeing faster approval
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 means they may be able to apply … Continue reading CIRM-funded kidney transplant procedure eyeing faster approval
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
Building a progressive pipeline
Dr. Kelly Shepard By Dr. Kelly Shepard One of our favorite things to do at CIRM is deliver exciting news about CIRM projects. This usually entails discussion of recent discoveries that made headlines, or announcing the launch of a new CIRM-funded clinical trial …. tangible signs of progress towards addressing unmet medical needs through advances … Continue reading Building a progressive pipeline
Meet the people who are changing the future
Kristin MacDonald Every so often you hear a story and your first reaction is “oh, I have to share this with someone, anyone, everyone.” That’s what happened to me the other day. I was talking with Kristin MacDonald, an amazing woman, a fierce patient advocate and someone who took part in a CIRM-funded clinical trial … Continue reading Meet the people who are changing the future
Stem Cell All-Stars, All For You
Dr. Larry Goldstein, UC San Diego It’s not often you get a chance to hear some of the brightest minds around talk about their stem cell research and what it could mean for you, me and everyone else. That’s why we’re delighted to be bringing some of the sharpest tools in the stem cell shed … Continue reading Stem Cell All-Stars, All For You
The Stem Cellar 