Programs

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.

Plasticity and Mechanisms of Cognitive Remediation in Older Adults

Plasticity and Mechanisms of Cognitive Remediation in Older Adults supports a grant for a multicenter clinical research trial on remediating age-related cognitive decline through mindfulness-based stress reduction and exercise.

Biomarkers Consortium - Workshop: Defining an Evidentiary Criteria Framework for Surrogate Endpoint Qualification

The FNIH Biomarkers Consortium and FDA hosted a workshop to provide a Framework for Defining the Evidentiary Criteria for Surrogate Endpoint Qualification on July 30-31, 2018. The workshop aimed to create alignment of the biomedical community and regulators on the levels of evidence required to qualify biomarkers for use in drug development, with an emphasis on surrogate endpoints and specific clinical outcome measures.