SUMMARY New compounds that inhibit an enzyme to reduce hydrogen sulfide in the carotid body, helping to normalize breathing and prevent sleep apnea by targeting the underlying cause of abnormal breathing during sleep The Unmet Need: Targeted, mechanism-based treatments that can restore… Read More
SUMMARY A non-hormonal therapeutic strategy for uterine fibroids targeting the upregulated m6A regulator METTL3 employing small molecule inhibitors thereby reducing fibroid cell proliferation and increasing apoptosis. The Unmet Need: Novel therapies that target fibroid growth without relying on hormonal manipulation Uterine… Read More
SUMMARY First-in-class small molecules to change how the PRNP gene is spliced, reducing harmful prion levels and offering a new treatment approach for prion diseases The Unmet Need: Safe, effective, and deliverable therapies to reduce prion protein levels in the central… Read More
SUMMARY An opioid painkiller combined with a peripherally restricted κ-opioid receptor agonist stimulating the carotid body to prevent opioid-induced respiratory depression, enhancing breathing without affecting pain relief or causing central side effects The Unmet Need: Effective approaches to reducing opioid-induced respiratory depression… Read More
SUMMARY Small molecule drugs developed to induce trained immunity in macrophage cells resulting in a heightened pro-inflammatory response without triggering a primary immune response The Unmet Need: Trained immunity induction that does not rely on pathogen-derived agents or endogenous molecules and without… Read More
SUMMARY According to the National Osteoporosis Foundation (NOF) approximately 10.2 million adults in the United States have osteoporosis, with an additional 43.4 million having low bone mass. One in eight women will be diagnosed with breast cancer in their lifetime. Because estrogen receptor-α (ERα) is expressed in ~70% of… Read More
SUMMARY Expanding CAR T cells with venetoclax enhances their ability to target and kill CD19+ tumor cells, increases their effectiveness, and reduces exhaustion, potentially leading to better treatment outcomes for diffuse large B cell lymphoma. The Unmet Need: Methods to increase CAR T efficacy… Read More
SUMMARY Novel machine learning-enable platform to guide in vitro experimental screening and discovery of small molecule immunomodulators to improve immune responses The unmet need: Platform to resolve unregulated immune potentiator enhancement and suppression of inflammatory and stimulatory responses The innate immune response… Read More
SUMMARY Imidazoquinolinone derivatives that activate Toll-like receptor (TLR) 7/8 are small molecule immune potentiators (SMIPs) that have potent activity as vaccine adjuvants and as anti-tumor agents. However, these molecules have high bioavailability that results in unacceptable levels of systemic inflammation due to adjuvant toxicity greatly limiting their use. The faculty… Read More
SUMMARY DNA hypomethylating agents have been widely used to treat various hematologic and non-hematologic malignancies. However, accumulating evidence has demonstrated that DNA methylation status does not correlate with clinical response to hypomethylating agents. Two RNA cytosine methyltransferases (RCMTs) NSUN1 and NSUN2 are responsible for catalyzing the majority of 5-methylcytosine in… Read More
SUMMARY There have been significant research efforts to develop effective vaccines that elicit a strong and durable T cell response against intracellular pathogens and cancer. A popular strategy has been to enhance vaccination efficacy by targeting dendritic cells (DCs). For DCs, functional heterogeneity is critical in the complex process of… Read More
SUMMARY TB bacterium, Mycobacterium tuberculosis (M. tuberculosis), is extremely slow growing compared to other bacteria and is naturally resistant to many of the powerful antibiotics that are effective against other bacterial infections. In recent years, multi-drug-resistant TB strains (MDR-TB) have arisen that are resistant to standard treatment and require extensive… Read More