Cure Rare Disease’s mission is to develop advanced therapeutics to treat individuals with rare and ultra-rare genetic diseases including Duchenne muscular dystrophy (Duchenne). 

On Sunday, October 29, 2023, our team joined the Tckachenko family and community of Scottsdale, Arizona for the inaugural community fundraiser organized by Heal DMD - a nonprofit organization committed to promoting healing and well-being for individuals with Duchenne Muscular Dystrophy (DMD) and their families....

This past week, Cure Rare Disease’s first therapeutic 一developed using CRISPR technology to treat Duchenne muscular dystrophy一began its final stage of testing leading up to dosing this fall. This CRISPR therapeutic will be one of the first of its kind to be delivered systemically to the human body, an accomplishment built on decades of science and research. As we approach this huge milestone in CRD’s mission, we wanted to reflect on the path that has gotten us here today.

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....