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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: 11/20/2024
Contributor(s): Peng Jin, Chuan He, Zhongyu Zou
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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: 7/30/2024
Contributor(s): Nael McCarty, Guiying Cui, Hanoch Senderowitz, Netaly Khazanov
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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: 7/30/2024
Contributor(s): Judith Fridovich-Keil
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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: 11/20/2024
Contributor(s): Jiahui Zhang, Khalid Salaita, Hanjoong Jo
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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: 11/20/2024
Contributor(s): William Hu, Sumit Verma
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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: 10/28/2024
Contributor(s): Vahid Serpooshan, Holly Bauser-Heaton, Martin Tomov
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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: 11/20/2024
Contributor(s): Eric J. Sorscher, Jeong Hong, Haian Fu, Yuhong Du, Huw Davies, Andras Rab, Candela Manfredi, Xun Yang, Zhi Ren
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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: 10/28/2024
Contributor(s): Malathy (mala) Shanmugam, Richa Bajpai
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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: 9/26/2024
Contributor(s): Richard Plemper, James Snyder, Aiming Sun
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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: 7/30/2024
Contributor(s): Jack Arbiser
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