Foundation for the National Institutes of Health Inc

PROGRAM ONE - RESEARCH PROGRAMS - ACCELERATING MEDICINES PARTNERSHIP PROGRAM (AMP) The FNIH manages the Accelerating Medicines Partnership (AMP) program with the interests of the collective scientific and medical research communities in mind. Our mission is to improve understanding of disease pathways, facilitate better selection of targets for treatment, and identify platforms and processes to accelerate new and effective therapies to patients. All AMP projects operate under the broad principle of delivering pre-competitive advancements to the research and medical fields and enabling the broadest possible access and use of AMP research discoveries. A critical component of each public-private partnership in the AMP program is an agreement among partners to make data and analyses arising from the collaboration publicly accessible to benefit the broader biomedical community. Individual AMP projects frequently establish public portals to rapidly disseminate data from their research efforts, and AMP-funded publications are required to be made available publicly within specific timeframes. At the center of the AMP program is a common goal of accelerating new and effective therapies to patients. AMP projects work towards this goal by identifying clinically relevant disease targets, improving identification of patients most likely to respond to a particular treatment, and safely reducing the development timelines for life-saving therapies and improvements in patient outcomes. Biomarkers Consortium The Biomarkers Consortium convenes government, industry, patients and patient advocacy groups, and not-for-profit organizations to address one of the most pressing needs in the diagnosis and treatment of disease: the development and the seeking of regulatory approval for disease biomarkers and surrogates. The Biomarkers Consortium creates and leads cross-sector efforts that validate and qualify biomarkers and other drug development tools to accelerate better decision making for the development of new therapeutics and health technologies. The core operations of the Biomarkers Consortium are supported through contributing membership. Organizations representing private industry (including the pharmaceutical, biotechnology, diagnostics, and information technology industries) and not-for-profit organizations (including associations, advocacy groups, trade organizations, and philanthropic organizations) that wish to support biomarkers development are eligible to become contributing members. GeneConvene The GeneConvene Global Collaborative advances best practices and informed decision making for development of genetic biocontrol technologies to improve public health. GeneConvene offers technical information, advice, training, and coordination for research on gene drive and other genetic biocontrol technologies - technologies that use genetic variants of a target species, like a disease-transmitting mosquito, to control its undesirable impacts. Efforts to control the spread of mosquito-borne diseases save hundreds of thousands of lives every year, but the tools and resources available are not sufficient to protect everyone. Gene drive is recognized as a potentially powerful tool to help control malaria. Genetic biocontrol has been used on disease-transmitting mosquitoes for over 60 years, but scientific advances in the past 20 years have dramatically improved the ability of researchers to engineer new genetic biocontrol approaches, including gene drives. GeneConvene was created to support informed decision making on the open scientific, regulatory, and policy questions raised by these new technologies. In 2023, GeneConvene members published the following: - An article in a special collection volume (organized by GeneConvene Global Collaborative) of Transgenic Research focusing on the regulatory requirements for implementing gene drive technologies in national or regional public health programs. - An article in Frontiers in Bioengineering and Biotechnology considering the approval procedures and market entry strategies for implementing gene drive-modified mosquitoes. - An assessment by a neutral third party, CSIRO, of risks associated with the release of genetically modified mosquitoes by the Target Malaria project. - A multi-country study in Malaria Journal exploring the insights and recommendations of key stakeholders across Africa on the potential of gene drive-modified mosquitoes for malaria control and elimination. Members also: - Supported training activities on operationalization of biosafety oversight and genetically modified organism decision making processes at African institutions. - Advised the United Nations Convention on Biological Diversity on developing guidance for risk assessment of engineered gene drive-modified mosquitoes. RESEARCH PROGRAMS FOR ACCELERATING NEW THERAPIES NIMBLE Results Set Stage for Improved Diagnosis, Treatment of Liver Disease To diagnose metabolic dysfunction-associated steatohepatitis (MASH), a leading cause of liver-related mortality, patients must undergo a liver biopsy, a painful and expensive procedure that carries some risk of death for patients. The Biomarkers Consortium's Noninvasive Biomarkers of Metabolic Liver Disease (NIMBLE) project aims to identify noninvasive blood-based and imaging biomarkers to assess their performance in diagnosing MASH, compared to liver biopsy, and to evaluate their suitability for monitoring treatment response in clinical trials. By expanding diagnostic options, more healthcare professionals and facilities would be able to diagnose MASH with greater safety, and the use of noninvasive tests may assist enrollment in future studies. - Four noninvasive blood tests for MASH and advanced liver disease were identified for assessment in NIMBLE 2. Study results published inNature Medicine. - Six imaging biomarkers shown to have the potential to measure liver fat, fibrosis, and body composition in those at risk for MASH. Study results published in Radiology. Expanding Access to Gene Therapy for Sickle Cell Disease Worldwide The FDA recently approved two treatments that use gene-editing technologies to mitigate the inherited gene mutation that causes sickle cell disease (SCD). These therapies involve procuring a patient's own bone marrow cells to repair the defective gene outside the body (ex vivo) and then transplanting the modified cells back into the patient's blood. The FNIH has supported, and continues to support, the underlying gene-editing research at NIH's National Heart, Lung, and Blood Institute (NHLBI) that helped lay the foundation for this breakthrough. Still, access to these therapies remains limited for people in low- and middle-income countries that may not have a cell-processing center and related resources necessary for ex vivo gene therapy. This is where the FNIH is seeking to broaden access to critical gene editing therapies for patients with SCD. In partnership with the NHLBI, the FNIH launched a project to explore techniques for administering gene therapy strategies entirely inside a patient's body (in vivo), which would eliminate the need for an advanced healthcare infrastructure and make SCD treatment more broadly available. BGTC Launches Clinical Trial Portfolio There are more than 10,000 known rare genetic diseases, affecting more than 30 million Americans and their families, but there is typically little to no commercial interest in developing treatments for a disorder that may impact only a dozen or so patients, leaving many such patients with little or no hope for beneficial treatments. Gene therapies hold promise for treating such patients, but progress is slowed by uncertainties about manufacturing and a clear regulatory path. Part of the AMP program, the Bespoke Gene Therapy Consortium (BGTC) partnership, now in its second year, brings experts across the biosciences ecosystem together to define the best ways to safely advance these treatments and encourage increased investments in their development. BGTC announced in 2023 the eight rare diseases that will comprise the program's initial clinical trial portfolio, which will pioneer a novel approach to the development of adeno-associated virus (AAV) gene therapy for rare diseases. The goal is to demonstrate that manufacturing and testing standards can provide a streamlined approval pathway for first-in-human clinical trials.