12 Results

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Small Molecules for Treating Cystic Fibrosis ApplicationSmall molecule compounds to potentiate most common type of CFTR mutation in cystic fibrosis patients.Key BenefitsPotentiates ΔF508-hCFTR mutation, addressing more than 60 percent of CF patients. Market SummaryCystic fibrosis transmembrane conductance regulator (CFTR) is a membrane protein and ion channel that malfunctions in the genetic... Published: 4/5/2023       Contributor(s): Nael McCarty, Guiying Cui, Hanoch Senderowitz, Netaly Khazanov

Whole-Animal Genetic Model to Mimic Aspects of the Patient Phenotype of Classic Galactosemia ­ Application An animal model for studying the underlying causes of galactosemia and screening for novel drugs to treat the condition. Key Benefits The first animal model for elucidating the underlying causes of galactosemia in infants. Animals with galactosemia causing genetic knockout exhibit similar disease phenotypes and complications... Published: 2/8/2023       Contributor(s): Judith Fridovich-Keil

Antisense Oligonucleotides Therapeutics Targeting Specific Tissues or Cells Application Antisense oligonucleotide (ASO) therapies for the treatment of rare diseases and cancer. Key Benefits Novel approach to ASO therapeutics that potentially improve tissue/cell targeting capabilities. Increases the efficacy of ASOs while decrease off-target toxicity. Potential to treat a variety of genetic diseases such as cancer, blood... Published: 5/8/2023       Contributor(s): Jiahui Zhang, Khalid Salaita, Hanjoong Jo

Predicting Treatment Response in Patients with Spinal Muscular Atrophy (SMA) Application Prognostic model predicts treatment response to nusinersen (Spinraza®) in patients with spinal muscular atrophy. Key Benefits A prediction model using baseline CHOP-INTEND, IL-8, fractalkine, and MCP-1 accurately predicts CHOP-INTEND outcomes in children treated with nusinersen The model can also be incorporated into future therapeutic... Published: 4/5/2023       Contributor(s): William Hu, Sumit Verma

3D Model for Analysis and Treatment Planning of Vascular Disease Application A patient-specific, 3D model of pulmonary atresia for surgical planning. Key Benefits Patient-specific model. Allows for accurate surgical planning. Enables new anastomosis procedure. Market Summary Pulmonary atresia (PA), ventricular septal defect (VSD) with major aortopulmonary collateral arteries (MAPCAs) is a rare congenital... Published: 12/6/2022       Contributor(s): Vahid Serpooshan, Holly Bauser-Heaton, Martin Tomov

CFTR Modulators for Treating Rare Forms of Cystic Fibrosis Application Small molecules to treat cystic fibrosis and other pulmonary diseases. Key Benefits Rescue rare variants of CFTR gene. Serve as PDE4 inhibitors with strong anti-inflammatory effects. Market Summary Cystic fibrosis (CF) is a progressive, genetic disorder that arises from mutations in the cystic fibrosis transmembrane conductance... Published: 5/4/2023       Contributor(s): Eric Sorscher, Jeong Hong, Haian Fu, Yuhong Du, Huw Davies, Andras Rab, Candela Manfredi, Xun Yang, Zhi Ren

Combination Therapy for Increasing Venetoclax Sensitivity in Multiple Myeloma Application Marker and co-therapy that inhibits succinate dehydrogenase to increase patient response to venetoclax. Key Benefits Co-therapy increases MM sensitivity to venetoclax treatment. Identifies patients who will respond to venetoclax therapy. Market Summary Multiple Myeloma (MM) is a cancer of plasma cells responsible for making antibody... Published: 4/5/2023       Contributor(s): Malathy (mala) Shanmugam, Richa Bajpai

Assay for Non-Nucleoside Inhibitors of the Measles and Nipah Viruses Application A cell-based assay to screen for inhibitors of the measles virus (MV). Highly active compounds have been identified and analogs have been synthesized and evaluated. Key Benefits Potent (sub-nM range), non-peptidic, non-nucleosidic compounds. Robust, cell-based, high-throughput screening assay. Technical Summary Vaccination serves... Published: 5/4/2023       Contributor(s): Richard Plemper, James Snyder, Aiming Sun

Combination Drug Treatment for Neoplasms Associated with Tuberous Sclerosis Application Combination of rapamycin and imatinib for the treatment of neoplasms associated with tuberous sclerosis (TS). Key Benefits Combined therapy shows 97% decrease in tumor volume in mouse model of TS compared to vehicle treatment. Dual therapy blocks two major signaling pathways implicated in TS. Market Summary Tuberous sclerosis (TS)... Published: 12/6/2022       Contributor(s): Jack Arbiser

Drosophila Model of Galactosemia Application Whole animal genetic model for Galactosemia. Fly model can be used to test efficacy of novel therapeutic interventions. Genetic system enables screening for suppressors and/or genetic modifiers of Galactosemia. Key Benefits First whole-animal genetic model to study GALT and GALE function. Only model that mimics acute... Published: 7/29/2022       Contributor(s): Kenneth (Ken) Moberg, Judith Fridovich-Keil, Rebecca Sanders, Jennifer Daenzer, Rebekah Kushner, Emily Ryan, Kerry Garza

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