Search Results - c.+bennett

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Combinations of ASOs for the Modulation of SMN Expression in SMA Patients

Unmet NeedSpinal muscular atrophy (SMA) is a devastating neuromuscular disease that predominantly affects children and is the most common cause of hereditary infant mortality. The disease is caused by low levels of survival motor neuron (SMN) protein, due to recessive mutations in the SMN1 gene. Importantly, the copy number variation of SMN2, a homologous...

Published: 3/13/2026 | Inventor(s): Charlotte Sumner, Constantin d'Ydewalle, Frank Rigo, C. Bennett

Keywords(s): CNS and Neurological Disorders, Combination, Disease Indication, Drug Delivery Vehicle, Muscle Atrophy, Muscular Disorders, Neurodegenerative Diseases, Peptides/Prodrugs, Single, Targeted Therapy, Therapeutic Matter, Therapeutics, Therapy Type

Category(s): Clinical and Disease Specializations > Neurology > Motor Neuron Diseases, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Technology Classifications > Therapeutic Modalities > Proteins

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: 3/12/2026 | 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