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 developing 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. Building on the successful Accelerating Medicines Partnership model, this program will focus on developing an operational playbook that invokes the use of streamlined templates, master regulatory files, and uniform production processes. It is anticipated that following a pilot phase of 4-6 test cases, 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 if it moves more broadly into the era of genome editing.

Biomarkers Consortium - The Autism Biomarkers Consortium for Clinical Trials (ABC-CT)

The Consortium will establish a technical and data infrastructure for reliably measuring social function, allowing the collaborating sites to work together as a single unit. The goal is to create a set of measures that can be used in clinical trials to determine which treatments are best for which patients and who will benefit from a particular treatment. The ultimate goal is to further develop and validate a set of measures that can be used as stratification biomarkers and/or sensitive and reliable objective measures of social impairment in autism spectrum disorders that could serve as indicative markers of long term clinical outcome.