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.

Accelerating Medicines Partnership - Parkinson's Disease

In 2016, the AMP Executive Committee approved the planning of an AMP effort to confront the challenges presented by Parkinson’s disease (PD). This complements current efforts in the areas of Alzheimer’s disease, type 2 diabetes and the autoimmune disorders of rheumatoid arthritis and systemic lupus erythematosus (lupus). A critical component of this partnership is that all members have agreed to make the AMP Parkinson’s disease (AMP PD) data and analyses publicly available to the broad biomedical community.

Accelerating Medicines Partnership - Alzheimer's Disease

The Accelerating Medicines Partnership Alzheimer’s Disease Project (AMP-AD) is a precompetitive partnership among government, industry, and nonprofit organizations that focuses on discovering novel, clinically relevant therapeutic targets and on developing biomarkers to help validate existing therapeutic targets. 

Biomarkers Consortium - Treatments Against RA and Effect on FDG PET-CT (TARGET Biomarker Study)

The Biomarkers Consortium’s TARGET Biomarker Study seeks to utilize validated proteomic biomarkers of rheumatoid arthritis (RA) disease activity and inflammation to categorize baseline and disease-modifying antirheumatic drug (DMARD)-associated changes in vascular inflammation in RA patients.

Biomarkers Consortium - Longitudinal Proteomic Changes in CSF from ADNI: Towards Better Defining the Trajectory of Prodromal and Early Alzheimer’s Disease

The Biomarkers Consortium’s Longitudinal CSF Proteomics Project, completed in Q4Y20, was the third stage of a multi-phased effort utilizing samples from the Alzheimer’s Disease Neuroimaging Initiative (ADNI) expanding on the identification of promising proteins in a previous Biomarkers Consortium project to provide early validation for candidate AD biomarkers. Concentrations of the candidate biomarkers in CSF were measured using a state-of-the-art targeted stable isotope-based quantitative mass spectrometry assay developed and implemented during the first two program stages.