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9873 documents

Orally available NOD2 agonist, HPX-612, for the treatment of visceral leishmaniasis

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Jean-Loup Romet-Lemonne · TRAVERSE BIOTECH II INC · 2024 · $298,938 — Project Summary We are developing the first orally available immunotherapy product, HPX-612, for the treatment of visceral leishmaniasis, a parasitic disease caused by Leishmania and transmitted by sandflies. Visceral leishmaniasis remains endemic in 65 countries worldwide, with over 4 million peopl…

Targeted delivery of nucleic acid formulations for in vivo CAR-Treg generation

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Chester Edward Markwalter · OPTIMEOS LIFE SCIENCES, INC. · 2024 · $300,000 — In this Phase I SBIR, Optimeos will develop an off-the-shelf, in situ CAR-Treg therapy through the nanoparticle-mediated delivery of a CAR-FoxP3 construct to CD4+ T cells as a treatment for transplant rejection. We envision an off-the-shelf therapy that reduces the healthcare costs and high burden o…

Creation of the Next Generation RNAi Therapeutics and an in vivo model for their study

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Prem Khovabutr Premsrirut · MIRIMUS, INC. · 2024 · $350,000 — Abstract RNA interference (RNAi) – a natural biological mechanism by which eukaryotic cells control gene expression – can be used to target and silence with high specificity virtually any expressed gene, making it a potentially powerful therapeutic approach. Importantly, RNAi mechanisms utilize DNA/…

PLK1 and EGFR targeted nanoconstruct as a monotherapy and a radiation sensitizer for lung cancer

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Worapol Ngamcherdtrakul · PDX PHARMACEUTICALS, INC. · 2024 · $914,249 — Lung cancer is the leading cause of cancer mortality, exceeding deaths from colon, breast, and prostate cancers combined. Resistance to chemo and targeted therapy is very common. Radiation therapy (RT) remains a key treatment in about 60% of lung cancer patients. We seek to develop a novel nanothera…

Role of chive-derived exosome-like nanoparticles in suppressing inflammation in obesity

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Jiujiu Yu · CASE WESTERN RESERVE UNIVERSITY · 2024 · $315,809 — Obesity is a chronic progressive disease that leads to the development of heart disease, stroke, and type 2 diabetes, which are among the top ten leading causes of death in the United States. The initiation and progression of obesity-related diseases is strongly associated with chronic low-grade inf…

Sphingolipid signaling in the endothelium during viral host defense

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Timothy Tun Hla · BOSTON CHILDREN'S HOSPITAL · 2024 · $712,226 — SUMMARY Endothelial cell (EC) dysfunction is a key factor that promotes poor host defense, pro-thrombotic cardiovascular complications and bleeding during respiratory viral infections. The applicant’s laboratory has made long-standing contributions in vascular effects of sphingosine 1-phosphate (S1…

Precision targeting of bladder cancer using CRISPR

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Mats Ljungman · UNIVERSITY OF MICHIGAN AT ANN ARBOR · 2024 · $566,310 — ABSTRACT Chromosome rearrangements (structural variants) are common in cancer and they drive tumor development by altering the expression or function of oncogenes and tumor suppressor genes by copy number alterations, formation of oncogenic fusions or by alterations of DNA elements responsible for r…

Clinically-useful vehicles for in vivo delivery of mRNA to stem and progenitor cells

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

John Ramunas · REJUVENATION TECHNOLOGIES, INC. · 2024 · $324,998 — Abstract Rejuvenation Technologies Inc. (RTI) aims to expand the utility of our telomere extension biologic, telomerase (TERT) mRNA, and our world-leading lipid nanoparticle (LNP) delivery vehicle by adding progenitor cell (PC) targeting capability. Telomeres are the protective DNA tips of chromosom…

A novel bioengineering approach to restoring permanent periodontal inflammatory bone loss

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Hae Lin Jang · BRIGHAM AND WOMEN'S HOSPITAL · 2024 · $798,032 — About 80% of Americans experience periodontitis in their lifetime. Alveolar bone loss leads to loosening or loss of teeth or dental implants that disrupts the most basic daily functions, such as eating and speaking. Various bone grafts are being used to restore alveolar bone loss, but poor prognosis…

Multifunctional Nanoparticle Platform to Prevent Alcohol-Associated HCC Development

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Jyothi Unnikrishna Menon · UNIVERSITY OF RHODE ISLAND · 2024 · $406,851 — ABSTRACT Hepatocellular carcinoma (HCC), the fastest rising cause of cancer-related deaths worldwide with a 5-year survival of <20%, affects more than 41,000 individuals in the United States every year. Heavy alcohol consumption leading to fatty liver, hepatitis and cirrhosis has been identified as …

Optimizing lipid nanoparticles for retinal gene editing in the NHP

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Renee Christine Ryals · OREGON HEALTH & SCIENCE UNIVERSITY · 2024 · $100,786 — PROJECT SUMMARY Inherited retinal dystrophies (IRDs) are a group of genetically and clinically heterogenous diseases, inherited in an autosomal dominant, recessive, or X-linked pattern. With an estimated incidence of 1:2000-1:3000, IRDs are the leading cause of vision loss in persons aged 15 to 45. …

A Novel Hybrid Viral Vector for Therapeutic CRISPR Insertion of Multiple Transgenes

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Logan Thrasher Collins · WASHINGTON UNIVERSITY · 2024 · $48,974 — PROJECT SUMMARY Existing delivery systems for CRISPR-Cas gene editing machinery have very little capacity for making multigenic insertions. As such, the scope of existing gene therapies has been limited primarily to monogenic diseases. Yet multigenic insertions could facilitate treatment of a much b…

Immunotherapy via engineered therapeutic programs in tumors using RNA

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Yizhou Dong · MASSACHUSETTS INSTITUTE OF TECHNOLOGY · 2024 · $607,159 — Immunotherapy treatments such as checkpoint blockade and chimeric antigen receptor T cell therapy have demonstrated the power of the immune system to eradicate metastatic cancer, but the efficacy of immunotherapies in solid tumors remains confined to a minority of patients. A series of interlinked e…

Harnessing Platelet-Endothelial Interactions for Exosome Delivery

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Ke Cheng · COLUMBIA UNIV NEW YORK MORNINGSIDE · 2024 · $735,942 — PROJECT SUMMARY Studies have demonstrated that adult stem cells such as mesenchymal stem cells (MSCs) repair myocardial infarction (MI) or ischemia/reperfusion (I/R) injury by indirect paracrine mechanisms rather than by differentiation and tissue replacement. In the past decade, exosomes have emer…

Controlled release of RNA-targeting therapy to promote healing of diabetic ulcers

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Adam G Berger · HARVARD MEDICAL SCHOOL · 2024 · $53,974 — Project Summary Non-healing ulcers are a common complication of diabetes, resulting in decreased quality of life, elevated rates of amputation, increased risk of mortality, and high healthcare costs. Unfortunately, current treatments remain outdated and inadequate. In diabetes, neuropathy and microv…

The essential roles of primary cilia in heterotopic ossification

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Haitao Wang · MAYO CLINIC ROCHESTER · 2024 · $410,535 — PROJECT SUMMARY/ABSTRACT Heterotopic ossification (HO), a diverse pathologic process of formation of extraskeletal bone in muscle and soft tissues, can be classified into hereditary and nonhereditary (acquired) HO types. A rare and devastating form of hereditary HO is fibrodysplasia ossificans progr…

Nanotechnology-based platform for the development of next-generation vaccines against opioid use disorder (OUD)

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Fatima Alamin Awad Alkareem Hamid · UNIVERSITY OF MINNESOTA · 2024 · $31,294 — Project Abstract: The highly complex OUD and overdose epidemic poses a huge public health and economic burden. Current FDA-approved pharmacotherapies against OUD and overdose use opioid receptor agonists and antagonists. These therapies show overall limited efficacy, due to their side effects, subop…

Structure and Function of Serum Amyloid A in Health and Disease

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Olga Gursky · BOSTON UNIVERSITY MEDICAL CAMPUS · 2024 · $106,758 — Abstract Amyloid diseases afflict >10 million patients worldwide and involve pathologic deposition of ~40 human proteins as fibrils. This project is focused on AA amyloidosis, a life-threatening complication of chronic inflammation wherein deposition of a small plasma protein, serum amyloid A (SAA),…

Therapeutic targets and novel anticancer agents for endocrine cancers

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Naris Nilubol · DIVISION OF BASIC SCIENCES - NCI · 2024 · $802,871 — Clinical trial development of MELK and CDK1-2 inhibitor is pending as we need to find a substitute CDK1/2 inhibitor because Merck did not want to provide their inhibitor. OncoTherapy Science has agreed to provide OTS167. We currently have three manuscripts in preparation. We are also working on the …

Core C- BIDMC Core

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Ioannis Vlachos · BETH ISRAEL DEACONESS MEDICAL CENTER · 2024 · $303,065 — SUMMARY The proposed multi-disciplinary Program Project Grant (PPG) will utilize interdisciplinary expertise, key established resources, cutting-edge technologies, and extensive in vivo and human studies to bypass substantial roadblocks in our understanding, management, and treatment of lupus nephri…