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A GALT-null Rat Model for Studies of Classic Galactosemia
Application
Pre-clinical rodent model to study classic galactosemia disease.
Key Benefits
Identify striking differences in GALT-independent galactose metabolism among tissues and across post-natal time points.
Study the relationship between galactose metabolite levels in blood and other tissues.
Study the impact of rigorous galactose restriction...
Published: 2/13/2025
Contributor(s): Judith Fridovich-Keil
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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: 2/13/2025
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: 2/13/2025
Contributor(s): Nael McCarty, Guiying Cui, Hanoch Senderowitz, Netaly Khazanov
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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: 2/13/2025
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: 2/13/2025
Contributor(s): William Hu, Sumit Verma
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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: 2/13/2025
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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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: 2/13/2025
Contributor(s): Richard Plemper, James Snyder, Aiming Sun
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