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 - Inflammatory Markers for Early Detection and Subtyping of Neurodegenerative and Mood Disorders

This project will aim to standardize and validate measurement methods for inflammatory markers associated with Alzheimer’s Disease and/or Major Depressive Disorder to ultimately identify a unique biosignature of disease. The identified biosignature would greatly assist with medication development, patient diagnosing, and patient selection for clinical trials.

Biomarkers Consortium - Bone Quality Project

The Biomarkers Consortium’s Bone Quality Project aims to evaluate and to identify biomarkers of bone strength and quality changes by analyzing pooled imaging and biochemical data from multiple clinical studies to allow definition of better clinical endpoints.

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.