Story of Hope: Elpida Therapeutics’ Pioneer Study on Rare Nerve Disease CMT4J

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).

CIRM Supports Innovative Gene Therapy for Rare Heart Disorder

Image Source: American Heart Association Journals  The California Institute for Regenerative Medicine (CIRM) awarded $4 million to support a gene therapy for rare life-threatening genetic disorder. A rare heart disorder Known as desmoplakin gene variant arrhythmogenic cardiomyopathy (DSP ACM), the genetic disorder is a rare heart disorder.   This preclinical award went to Deborah D. Ascheim, … Continue reading CIRM Supports Innovative Gene Therapy for Rare Heart Disorder