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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, including through our commercialization subsidiary Orphan Therapies. A portion of all revenue generated is returned to OTXL to fund additional portfolios of ultra-rare disease treatments.
Partner With Us
Fund or Invest – We are accepting grants and philanthropic donations, as well as investment from venture philanthropists, Donor Advised Funds (DAF), family offices and impact investors
Contribute Therapeutic Assets – If you have an ‘orphaned’ drug program with promising data and think our model may be the best path forward, please reach out
Become a Network Affiliate – If you are interested in becoming part of a network of dedicated CDMO and other affiliates, to support and be rewarded for efficient commercialization of Accelerator programs, please reach out