Award Data

ABSTRACT This Phase STTR Phase II proposal aims to engineer and scale-up a synthetic metabolic pathway in a microbial host to produce UDCA and related compounds. Additionally, the UDCA produced from the engineered synthetic metabolic pathway will be used to synthesize derivatives for testing in our Alzheimer’s Disease cell and animal models. The proposed work has high intellectual merit for the ...

Project Summary MarPam Pharma aims to develop a one-time treatment for achieving durable remission of human immunodeficiency virus (HIV), after which patients will no longer need to take antiretroviral therapy. Our treatment is an autologous HIV-specific chimeric antigen receptor (CAR) immune cell therapy that employs the CXCR5 chemokine receptor as a homing device to direct either anti-HIV T cell ...

PROJECT SUMMARY/ABSTRACT The long-term goal of this proposal is to improve the care of patients suffering from Large Vessel Occlusion Acute Ischemic stroke. Currently, mechanical thrombectomy techniques can achieve revascularization in over 70% of cases, and physicians are rapidly adoption aspiration thrombectomy over stent-trievers. Aspiration maintains equivalent angiographic and clinical outcom ...

PROJECT SUMMARY The goal of this proposal is to advance a new therapeutic approach targeting myosin for polycystic kidney disease (PKD). PKD is a major life-threatening Mendelian disorder that affects 12,000,000 individuals, representing a market opportunity of ~$1 billion. In PKD, tiny tubules in the kidneys, liver, and other organs gradually expand into fluid-filled cysts, leading to organ failu ...

PROJECT SUMMARY Genomics has become an essential element of biomedical science, playing multiple roles in rapidly addressing the COVID-19 pandemic, understanding medical complications to develop more effective preventative care and treatments, and modeling diseases in laboratory specimens for study. Despite the increasing relevance of genomics to people's everyday lives, genomics literacy in the U ...

PROJECT SUMMARY/ ABSTRACT Currently in the US there are over 400,000 kidney patients on dialysis. The primary therapy mode is hemodialysis and is performed in a hospital or private clinic. Current dialysis therapy requires a large device that must be continuously connected to a reverse osmosis (RO) system. A single treatment will require over 150L of AAMI dialysate making portable device unattaina ...

PROJECT ABSTRACT Myriel, Inc., is a pharmaceutical start-up company founded in 2022 to find cures for tauopathies, including Alzheimer’s disease and related dementias (ADRD), which impair cognition in 32% of US adults 65 years and older. The goal of this Phase 1 STTR application is to provide Myriel with a brain-penetrant analogue of our lead compound, GPHR ‘739, the first potent, selective, r ...

Project Summary The goal of this Fast Track STTR project is to determine proof-of-principle and efficacy of a novel blood brain barrier (BBB) penetrating therapeutic nanoparticle for the potential treatment of otherwise intractable brain tumors like glioblastoma multiforme (GBM). This project seeks to demonstrate that novel, targetable nanoparticles can delivery therapeutic substances to human bra ...

Project Summary The goal of this project is to design and prepare diagnostic biomarker reference standards for the endogenous glycosaminoglycan (GAG) Mucopolysaccharidosis type I (MPS-I) and type II (MPS- II) biomarkers. MPS-I and MPS-II are lysosomal storage disorders (LSDs) which are listed on the Recommended Uniform Screening Panel (RUSP) in the United States and are included in global Newborn ...

PROJECT SUMMARY Duchenne muscular dystrophy (DMD) is the most common type of muscular dystrophy, caused due to mutations in the dystrophin gene. DMD is characterized by progressive muscle wasting and overall loss of muscle function. DMD patients become wheel chair dependant by 12 years of age and the associated cardiorespiratory complications result in their early death around 20 years of age. Dys ...