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

A look back at 15 years of CIRM funding at UCLA

Researchers in the lab of CIRM grantee Donald Kohn, M.D. Image Credit: UCLA Broad Stem Cell Center Since the first grant was issued in April 2006, CIRM has funded a wide range of research conducted by top scientists at UCLA for a wide range of diseases. To give a retrospective look at all the research, … Continue reading A look back at 15 years of CIRM funding at UCLA

Want to help us solve a mystery?

Patient that has recovered from Covid-19 donating blood plasma. Photo courtesy Science Photo Convalescent plasma has been in the news a lot lately as a potential treatment for people infected with the coronavirus. In August the US Food and Drug Administration (FDA) granted emergency use authorization (EUA) to use these products based on preliminary data … Continue reading Want to help us solve a mystery?

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

‘Mini lung’ model shows scientists early stages of new coronavirus infection

Representative image of three-dimensional human lung alveolar organoid showing alveolar stem cell marker, HTII-280 (red) and SARS-CoV-2 entry protein, ACE2 (green) Image Credit: Jeonghwan Youk, Taewoo Kim, and Seon Pyo Hong The development of organoid modeling has significantly expanded our understanding of human organs and the diseases that can affect them. For those unfamiliar with … Continue reading ‘Mini lung’ model shows scientists early stages of new coronavirus infection

How stem cells are helping her win the fight of her life

We have all read about people who smoke a pack of cigarettes and drink a bottle of whiskey a day and somehow manage to live a long, healthy life. Then there are people like Sandra Dillon. She lived as healthy a life as you can imagine; she exercised a lot, ate a healthy diet and … Continue reading How stem cells are helping her win the fight of her life

Delivering a protein to testes could help treat male infertility

Delivering a protein (red fluorescence) to mice testes with a fibroin nanoparticle-encapsulated cationic lipid complex (green) restored male fertility. Credit: Adapted from ACS Nano 2020, DOI: 10.1021/acsnano.0c04936 For many couples that are ready to start a family, infertility, which is the inability to conceive children, can be a devastating setback. In fact, according to the Mayo Clinic, … Continue reading Delivering a protein to testes could help treat male infertility

Repairing damaged muscles

Close-up of the arm of a 70-year-old male patient with a torn biceps muscle as a result of a bowling injury; Photo courtesy Science Photo Library In the time of coronavirus an awful lot of people are not just working from home they’re also working out at home. That’s a good thing; exercise is a … Continue reading Repairing damaged muscles

CIRM funded trial for LAD-I announces positive results

Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease caused by a mutation in a specific gene that causes low levels of a protein called CD18. Due to low levels of CD18, the adhesion of immune cells is affected, which negatively impacts the body’s ability to combat infections. Rocket Pharmaceuticals has announced positive results from … Continue reading CIRM funded trial for LAD-I announces positive results

A model for success

Dr. Maria Millan, CIRM's President & CEO Funding models are rarely talked about in excited tones.  It’s normally relegated to the dry tomes of academia. But in CIRM’s case, the funding model we have created is not just fundamental to our success in advancing regenerative medicine in California, it’s also proving to be a model … Continue reading A model for success