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Antisense oligonucleotide therapy and biomarker assay for C9ORF72 hexanucleotide expansion disorders

Therapy to Treat and Assay to Monitor Neurodegenerative Disease JHU REF: [C12195]Invention novelty: This technology is the use of antisense oligonucleotides (ASO) as a therapeutic for specific neurodegenerative diseases. The technology could also be used in development of a biomarker assay to monitor the efficacy of the ASO therapy. Value Proposition: Current...

Published: 3/13/2025 | Inventor(s): Christopher Donnelly, C. Bennett, Susan Freier, Rita Sattler, Jeffrey Rothstein

Keywords(s): Amyotrophic Lateral Sclerosis, Assay, Biologics, Clinical Diagnostics, CNS and Neurological Disorders, Dementia, Disease Indication, In Vitro Diagnostics, Neurodegeneration, Nucleic Acid, Therapeutic Matter, Therapeutic Substance, Therapeutics

Category(s): Clinical and Disease Specializations, Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Diagnostics > In Vitro Diagnostics, Technology Classifications > Research Tools > Assays, Technology Classifications > Diagnostics, Clinical and Disease Specializations > Neurology, Technology Classifications > Research Tools, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > Dementia, Clinical and Disease Specializations > Neurology > Neurodegeneration