Cure Rare Disease

Cure Rare Disease envisions a future without rare diseases. To accomplish this goal, Cure Rare Disease collaborates with academic and clinical institutions across the Northern Hemisphere to engineer and test life-saving therapeutics for patients impacted by rare, neuromuscular diseases. Cure Rare Disease focuses on developing therapeutics for diseases for which there is no treatment or cure and for which there are too few patients to attract commercial drug development interests.

To realize our mission, Cure Rare Disease continues to develop research collaborations with institutions and industry members including Yale, Hospital for Sick Kids, Ohio State University, Leiden University Medical Center and other institutions around the globe. We have developed a novel framework by which therapeutics for rare and ultra-rare diseases are created. In partnership with our collaborators and patients, we are successfully advancing life-saving therapeutics.

Cure Rare Disease’s organizational capabilities include a staff members to support and engage with patients and patient families. Moreover, our research capabilities include 7+ research collaborations and 5+ partnerships with industry to advance our therapeutics. Moreover, we are supported by regulatory, clinical and manufacturing advisors and consultants to help guide the programs.

We have 19 active therapeutic development programs broken down into 5 phases. Our first therapeutic has entered the clinic while 3 other therapeutics are in the 4th stage of development. An additional 5 therapeutics are in the 3rd phase of development and the remaining therapeutics are split evenly between the first and second stages of development. In aggregate, over 200,000 people would be impacted if successful. Beyond drug development, we have conducted neutralizing antibody analyses on over 30 patients samples and have also conducted whole genome sequencing on over 15 patient samples.