Therapies

We are focused on providing access to ultra-rare disease treatments that most companies aren’t able to commercialize due to high costs and very small numbers of treatable patients.

We do so through partnerships, practices and technology platforms that create fit-for-scale approaches that are repeatable across multiple therapies.

We are currently working in partnership with Fondazione Telethon to prepare for the US commercial launch of a gene therapy that has recently been approved by the US FDA. Please check back soon for more details.

If you’re interested in working with us to make therapies available to patients that would otherwise not be accessible, please contact us at info@orphantxl.com.