The Orphan Therapeutics Accelerator is a non-profit biotech company focused on efficiently advancing promising “shelved” treatments for ultra-rare conditions through late-stage clinical development and approvals and providing a viable commercial pathway to make them available to patients in need.

Our model utilizes tax and other incentives to dramatically reduce up-front costs to obtain and develop therapeutic assets, features unique opportunities for collaboration across the orphan disease landscape, and leverages philanthropic and impact investment to establish a regenerative source of funding to build and sustain a pipeline of ultra-orphan drugs.