Cure Rare Disease, in partnership with Charles River Laboratories, has successfully created 3 mouse models that harbor the humanized form of the dystrophin gene. Each model has a mutation that causes Duchenne muscular dystrophy that CRD is currently working to treat, meaning they can be utilized for critical in vivo studies to test the safety and efficacy of mutation-specific gene editing therapeutics. The success of this project paves the way for future therapeutics to be developed to treat rare and ultra-rare genetic disorders.

​​We are proud to announce the creation of our Scientific Advisory Board (SAB)! Joining the SAB are leading experts in gene therapy and neuromuscular disorders who will help advance our preclinical and clinical development of gene therapy and antisense oligonucleotide programs.

Unlike traditional methods for developing therapeutics for common and (relatively) rare diseases, ultra-rare disease drug development requires a different approach and mechanism in light of a limited ability to commercialize and even reach BLA/NDA approval once clinical trials are complete....