About

Treatment approaches that target genetic causes of rare diseases can offer significant, potentially curative, benefits to patients.

However, diseases with very small numbers of patients have limited sales potential, and these medicines are also costly to develop, manufacture and distribute. Recent market and policy shifts have made commercialization of ultra-rare disease treatments even more difficult, causing many companies to abandon promising therapies.

Orphan Therapies is a subsidiary of the Orphan Therapeutics Accelerator (OTXL), a non-profit organization that obtains shelved treatments that have advanced to clinical trials and works with partners to complete their development at low cost. Once approved, these treatments may be commercialized through Orphan Therapies or licensing agreements with biotech or other organizations.

Our streamlined, non-profit model enables us to operate with a primary focus on sustaining and scaling access to treatments, while generating sufficient revenues to support ongoing efforts to deliver more treatments to patients.

Diagram showing transition from a nonprofit biotech company, Orphan Therapeutics Accelerator, to a subsidiary, Orphan Therapies. Text explains the transition from developing ultra-rare disease treatments to moving to a marketing arm.

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