Image source: SickKids -Terry, Georgia, and Michael Pirovolakis CIRM awarded Elpida Therapeutics a $3.9 million grant in December 2023 to support a pioneering preclinical study on Charcot Marie Tooth disease type 4J (CMT4J). CMT4J is an ultra-rare, inherited condition that primarily affects the nerves outside the brain and spinal cord. It accounts for about 0.24% … Continue reading Story of Hope: Elpida Therapeutics’ Pioneer Study on Rare Nerve Disease CMT4J
clinical trial
Neurona Therapeutics Raises $120M Financing, Cell Therapy Shows Positive Outcomes in CIRM-Funded Trial
Photo Courtesy of Neurona Therapeutics/Curtis Myers Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapy candidates for the treatment of neurological disorders, recently announced the successful completion of a $120 million financing. Neurona’s financial success is an encouraging sign for the stem cell and gene therapy sector, highlighting partnerships with investors and public organizations … Continue reading Neurona Therapeutics Raises $120M Financing, Cell Therapy Shows Positive Outcomes in CIRM-Funded Trial
Tratando malformaciones congénitas antes del nacimiento
El bebé, Tobi recibió un tratamiento de células madre, financiado por el CIRM, mientras aún estaba en el útero. To read this blog in English, click here. Michelle y Jeff se llenaron de felicidad cuando se enteraron de que iban a tener un bebé. Luego, un examen de ultrasonido a las 20 semanas del embarazo … Continue reading Tratando malformaciones congénitas antes del nacimiento
Pioneering a new approach to HIV/AIDS
Dr. Steven Deeks. Photo courtesy UCSF I’ve always been impressed by the willingness of individuals to step forward and volunteer for a clinical trial. Even more so when they are the first person ever to test a first-in-human therapy. They really are pioneers in helping advance a whole new approach to treating disease. That’s certainly … Continue reading Pioneering a new approach to HIV/AIDS
The power of the patient advocate: how a quick visit led to an $11M grant to fund a clinical trial
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Members of NFOSD visiting UC Davis in 2013 At the California Institute for Regenerative Medicine (CIRM) we are fortunate in having enough money to fund the most promising research to be tested in a clinical trial. Those are expensive projects, often costing tens of millions of … Continue reading The power of the patient advocate: how a quick visit led to an $11M grant to fund a clinical trial
Breaking down barriers: Expanding patient access and accelerating research
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST 10 years ago I was presented with an incredibly unique opportunity- to become the fifth patient with spinal cord injuries to participate in the world's first clinical trial testing a treatment made from human embryonic stem cells. It was not only a risky and potentially life-changing decision, … Continue reading Breaking down barriers: Expanding patient access and accelerating research
Stem cell therapy may help mend a broken heart
Blausen.com staff (2014). "Medical gallery of Blausen Medical 2014 Dilated cardiomyopathy (DCM), a condition where the muscles of the heart are weak and can lead to heart failure, is considered rare in children. However, because the symptoms are not always easy to recognize the condition can go unnoticed for many years, and in severe cases … Continue reading Stem cell therapy may help mend a broken heart
Paving the way for a treatment for dementia
What happens in a stroke When someone has a stroke, the blood flow to the brain is blocked. This kills some nerve cells and injures others. The damaged nerve cells are unable to communicate with other cells, which often results in people having impaired speech or movement. While ischemic and hemorrhagic strokes affect large blood … Continue reading Paving the way for a treatment for dementia
Stem cell therapy for diabetic foot ulcers shows promise in new study
For individuals with diabetes, the body's inability to properly control blood sugar levels can lead to a wide range of other problems as time passes. One major issue is a diabetic foot ulcer (DFU), an open sore or wound that is commonly located on the bottom of the foot and caused by poor blood circulation … Continue reading Stem cell therapy for diabetic foot ulcers shows promise in new study
CIRM-Funded Project Targeting Sickle Cell Disease Gets Green Light for Clinical Trial
Dr. Matthew Porteus The US Food and Drug Administration (FDA) has granted Investigational New Drug (IND) permission enabling Graphite Bio to test the investigational, potentially revolutionary gene editing therapy GPH101 developed under the supervision of Matthew Porteus, MD, PhD, in a clinical trial for people with sickle cell disease (SCD). The California Institute for Regenerative … Continue reading CIRM-Funded Project Targeting Sickle Cell Disease Gets Green Light for Clinical Trial