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Novel Therapeutic Target for Fragile X Syndrome and Neurodevelopmental Disorders
Application Salvianolic acid C for treating Fragile X Syndrome (FXS). Key Benefits New therapeutic drug candidate for the treatment of FXS. Preliminary data in the laboratory in organoids treated with the drug showed rescued cell phenotypes known to be associated with the disease in humans. Potential to treat other neurodevelopmental disorders. Market...
Published: 3/1/2024       Contributor(s): Peng Jin, Chuan He, Zhongyu Zou
Cell and Gene Therapies for Hemophagocytic Lymphohistiocytosis
Application Gene/cell therapy to treat hemophagocytic lymphohistiocytosis. Key Benefits Gene therapy approach for treating patients with UNC13D mutated hemophagocytic lymphohistiocytosis. Preliminary in vitro and in vivo data show the ability of the gene therapy to increase UNC13D protein levels and decrease T cell activation. It is administered...
Published: 1/17/2024       Contributor(s): H. (Harold) Trent Spencer, Christopher Doering, Shanmuganathan Chandrakasan, Sarah Takushi
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: 7/28/2023       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: 7/28/2023       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: 4/8/2024       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: 7/28/2023       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: 12/7/2023       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: 4/8/2024       Contributor(s): Eric J. 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: 12/1/2023       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: 11/6/2023       Contributor(s): Richard Plemper, James Snyder, Aiming Sun
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