Award Data

Abstract: The objective of this project is to perform the required (and previously approved) IND-enabling experimental work for E1v1.11, a novel drug candidate for Spinal Muscular Atrophy (SMA). SMA is an autosomal recessive disorder that is the leading genetic cause of infantile death worldwide, occurring in ~1:10,000 live births. The gene responsible for SMA is called survival motor neuron-1 (SM ...

OTHER PROJECT INFORMATION – Unit 7 – Project Summary/Abstract This project addresses the growing need for medications for cannabis use disorder (CUD) which, in the absence of approved medications, is a major focus of NIDA’s mission. Currently available cannabinergic-based treatments for CUD include the directly acting CB1 agonists, e.g., Δ9-THC or nabilone, which have been reported to reduc ...

Project Summary Human exposure to toxic heavy metals existing in drinking water, food, and the environment is an area of increasing national and international concern. Heavy metals are significant environmental pollutants because they tend to persist, bioaccumulate and can result in serious adverse acute or chronic health effects after ingested or inhaled. Traditionally, trace metals are measured ...

PROJECT SUMMARY: Herpes Simplex Virus (HSV) infections are common, infecting more than half of the world’s population. While most cases of genital herpes are caused by HSV type 2 (HSV-2), it is estimated that more than 150 million people aged 15-49 have genital HSV type 1 (HSV-1) infections. Although several vaccines have been evaluated for preventing/treating HSV infections, none have received ...

Project Summary Crohn’s disease is a debilitating and chronic inflammatory disorder of the gastrointestinal tract characterized by aberrant healing and intestinal stricturing. In the US, rt500,000 patients suffer from Crohn’s disease and over 30,000 patients are newly diagnosed each year. No curative treatments exist. Instead, current treatments focus on managing symptoms and reducing the occu ...

Abstract Insomnia is a highly prevalent condition that significantly degrades the quality of life and health of millions of patients. By diagnostic definition, insomnia is a disease characterized by self-reported subjective criteria, thus therapeutic developers targeting insomnia typically evaluate their treatment’s efficacy based on patient reported outcomes in the form of sleep diaries. A crit ...

Project Summary The value of computational chemistry to commercial drug discovery is now well-established. Virtual screening (including molecular docking) now jumpstarts most discovery efforts. Tools such as molecular dynamics and free energy perturbation are increasingly used to inform the later stages of lead refinement. The growing importance of computational structure-based methods has influen ...

ABSTRACT The United States is experiencing a major mental health crisis. Per recent reports in the New York Times and The Washington Post, the gap between need and access to behavioral health services is rapidly increasing. There is an urgent need to solve both the access and quality problems with behavioral healthcare. With COVID and other stressors, 76% of surveyed employees are reporting at lea ...

PROJECT SUMMARY/ABSTRACT The mission of Antiger Therapeutics Inc. is to develop novel immunotherapies for transplant recipients to improve long-term graft outcomes and minimize adverse effects. Antibody-mediated rejection (AMR) is a leading cause of graft loss, with over 3000 new cases per year in the United States and estimated market size of $90 million. However, this estimate is likely to under ...

ABSTRACT Atrial fibrillation (AF) remains the most commonly occurring cardiac arrhythmia. It is associated with a lower quality of life and a higher rate of morbidity and mortality primarily due to poor hemodynamic performance and often stroke. One of the primary options to treat AF is cardiac ablation where the physician applies radiofrequency energy via percutaneous catheters to form a series of ...