To tackle the human health challenges that face the world today, the FNIH develops collaborations with top experts from government, industry, academia and the not-for-profit sector and provides a neutral environment where we can work productively toward a common goal.

Bespoke Gene Therapy Consortium (BGTC)

The Bespoke Gene Therapy Consortium (BGTC) is a program in development. Building on the successful Accelerating Medicines Partnership model, the BGTC represents a public-private partnership dedicated to making gene therapy a reality for people with rare genetic diseases affecting populations too small to be viable from the current commercial perspective. By developing a operational playbook that invokes the use of streamlined templates, master regulatory files, and uniform production processes, it is anticipated that following a pilot phase, a pathway toward the commercial viability of these therapies will be found. This may ultimately have a tremendously positive impact on the larger field of gene therapy it moves more broadly into the era of genome editing.

The NIH Undiagnosed Diseases Program

The Undiagnosed Diseases Program (UDP) at the National Institutes of Health assists with diagnosing patients who have long been unable to find any diagnosis for their symptoms.

Clinical Center Drug Donations

The Clinical Center In-Kind Drug Donation Program provides pharmaceuticals that are donated through the public-private partnership coordinated by the FNIH to the NIH Clinical Center.