Orphan Therapies is a subsidiary of Orphan Therapeutics Accelerator (OTXL), a non-profit biotech company focused on advancing shelved ultra-rare disease treatments through clinical trials.

Orphan Therapies is led and advised by the executive team and board of OTXL, with decades of experience in development and commercialization of advanced therapies for rare diseases. We are building out a dedicated team of experienced professionals and partners with expertise in patient services, medical affairs, market access and related fields.

Orphan Therapies is now partnering with Fondazione Telethon on US distribution and commercial access to a gene therapy that was recently approved by the US FDA.

If you are passionate about enabling rare disease treatments to reach patients and want to join our team or collaborate on commercial activities, please contact us at info@orphantxl.com.