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
Clinical Trials
Hitting our Goals: Scoring a half century
Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves … Continue reading Hitting our Goals: Scoring a half century
Going the extra mile to save a patient’s life
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
Hitting our goals: regulatory reform
Way, way back in 2015 – seems like a lifetime ago doesn’t it – the team at CIRM sat down and planned out our Big 6 goals for the next five years. The end result was a Strategic Plan that was bold, ambitious and set us on course to do great things or kill ourselves … Continue reading Hitting our goals: regulatory reform
A Match Made in Heaven, if heaven were in Oakland!
The Matchmaker - by Gerrit van Honthorst Throughout history, matchmakers have played an important role in bringing together couples for arranged marriages. Fast forward to today and CIRM is now playing a similar role. We’re not looking to get anyone hitched, what we are trying to do is create partnerships between people we are funding … Continue reading A Match Made in Heaven, if heaven were in Oakland!
A little history in the making by helping the tiniest patients
Dr. Diana Farmer stands with Dr. Aijun Wang and their UC Davis research team. It’s appropriate that at the start of Women’s History Month, UC Davis’ Dr. Diana Farmer is making a little history of her own. She launched the world’s first clinical trial using stem cells to treat spina bifida before the child is … Continue reading A little history in the making by helping the tiniest patients
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
Charting a course for the future
A new home for stem cell research? Have you ever been at a party where someone says “hey, I’ve got a good idea” and then before you know it everyone in the room is adding to it with ideas and suggestions of their own and suddenly you find yourself with 27 pages of notes, all … Continue reading Charting a course for the future
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
Surviving with Joy
Dr. Tippi MacKenzie (left) of UCSF Benioff Children’s Hospital San Francisco, visits with newborn Elianna and parents Nichelle Obar and Chris Constantino. Photo by Noah Berger Alpha thalassemia major is, by any stretch of the imagination, a dreadful, heart breaker of a disease. It's caused by four missing or mutated genes and it almost always … Continue reading Surviving with Joy
The Stem Cellar 