August 2019 | Research & Development Update
Cure Rare Disease has finished developing the infrastructure necessary to enable continuous development of precision medicine therapeutics for genetic diseases
- We have onboarded two critical partners to enable continuous development: Charles River Labs and Nationwide Children’s Hospital.
- Charles River will lead regulatory engagement and GLP toxicology studies both of which are critical to reaching patient dosing.
- Nationwide Children’s Hospital will produce research and clinical grade AAV (viral vector) for systemically delivering the therapeutic to patients.
Our first patient is expected to be dosed in summer 2020 while we have recently enrolled (2) additional DMD patients into the precision medicine process.
- We have engaged FDA to begin regulatory conversations ultimately leading into clinical trial.