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Novel Tocopherol and Tocopheryl Quinone Derivatives as Therapeutics for Lysosomal Storage Disorders

Novel tocopherol derivatives and tocopheryl quinone derivatives useful in the decrease of lysosomal substrate accumulation, the restoration of normal lysosomal size, and the treatment of lysosomal storage disorders (LSDs) are provided. The inventors have discovered that tocopherol and tocopheryl quinone derivatives with side chain modifications (such...

Published: 7/25/2024 | Inventor(s): Jingbo Xiao, Wei Zheng, John McKew, Juan Marugan

Keywords(s): ANALOGUES, Correctors, DISORDERS, IB6XXX, IBXXXX, IXXXXX, Lysosomal, Storage, Tocopherol

Category(s): Collaboration Sought > Collaboration, Application > Vaccines, Application > Therapeutics, TherapeuticArea > Reproductive Health, Application > Diagnostics, Application > Research Materials

Novel Small Molecule Inhibitors for the Treatment of Huntington’s Disease

This technology is a collection of small molecules screened for their ability to prevent or reduce the cytotoxic effects of the protein, Huntingtin. Huntington's disease is a neurodegenerative disorder due to a dominantly acting expansion of a CAG trinucleotide repeat in exon 1 of the Huntington (HTT) gene resulting in production of the altered (mutant)...

Published: 7/25/2024 | Inventor(s): Joshua McCoy, Samarjit Patnaik, Steven Titus, Wei Zheng, Noel Southall, Wenwei Huang, Juan Marugan

Keywords(s): ANALOGUES, Disease, HUNTINGTON'S, IB6XXX, IBXXXX, IXXXXX, NCTT, Novel, Thio-ether, treatment

Category(s): Collaboration Sought > Collaboration, Collaboration Sought > Licensing, Application > Therapeutics, Application > Vaccines, TherapeuticArea > Reproductive Health, Application > Research Materials, Application > Diagnostics