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.
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.
The National Institute of Nursing Research hosted a two-day Summit to gather a variety of stakeholder perspectives on the spectrum of caregiving issues and research for conditions and illnesses that may occur across the lifespan.
The International Summit in Human Genetics and Genomics is a five-year initiative (2016-2020) designed to help developing nations build and expand their knowledge base, infrastructure, systems and technologies in genetics and genomics. Each fall, researchers from abroad travel to the National Institutes of Health in Bethesda, Maryland for one month of in-person training at the National Human Genome Research Institute (NHGRI). The Summit helps them to understand the prevalence and basis of genetic diseases in their nations and to address these public health challenges. The 2020 Summit will be held on August 31 - October 1, 2020.
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.
The Sayer Vision Research Lecture and Award funds an annual presentation delivered by an investigator conducting outstanding research in the area of vision research or related fields. It also provides a grant to support the research of a promising independent investigator in the early stage of his or her career in the Division of Intramural Research whose work is closely related to aspects of vision research.
The Age-Related Eye Disease Study 2 (AREDS2) examined the effects of vitamin and mineral supplementation on the progression of age-related macular degeneration (AMD), which is one of the leading causes of blindness in the United States. Funds raised by the FNIH support the development of a genetic repository for the study, facilitating deeper analyses of the results.
The Baby Connectome Project (BCP) is a four-year study of children from birth through five years of age, intended to provide a better understanding of how the brain develops from infancy through early childhood and the factors that contribute to healthy brain development.
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.
This workshop aimed at creating alignment among scientific stakeholders including the FDA, the NIH, the biopharmaceutical industry, academic researchers and patient groups regarding a proposed framework for determining the levels of evidence required to qualify biomarkers for use in drug development.
whorton@fnih.orgThe Biomarkers Consortium’s PET Radioligand Project, completed in December 2012, developed improved, more sensitive PET radioligands with higher binding to the peripheral benzodiazepine receptor. Findings from this study suggest that the [11C]PBR38 ligand, in particular, may be useful in detecting progression from mild cognitive impairment or treatment response in Alzheimer’s Disease.