There are 9,000+ “ultra-rare” diseases. Many affect children and have deadly, devastating, costly impacts.

Hundreds of promising treatments in clinical trials for ultra-rare conditions have been “shelved” in recent years due to shifting market and policy dynamics.

The Orphan Therapeutics Accelerator (OTXL) utilizes our non-profit status and related incentives to obtain, fund and complete development of these shelved therapies at low cost through a network of affiliated partners (Orphan ClinDevNet).

We then work with partners to ensure commercial access to approved treatments through several alternative approaches. A portion of all revenue generated is returned to OTXL to fund additional portfolios of ultra-rare disease treatments.