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.

Multi-site Efficacy and Safety Trial of Intrapartum Azithromycin in Low- and Middle-Income Countries

This study will test whether an antibiotic taken during labor can prevent infections in mothers and newborns in seven low- and middle-income countries. It will be conducted by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) through their Global Network for Women’s and Children’s Health Research (Global Network) in partnership with the Bill & Melinda Gates Foundation.

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.