Use of L-Enantiomeric Peptide Ligands of Natively Folded Human Superoxide Dismutase 1 (SOD1) for the Therapy of Amyotrophic Lateral Sclerosis (ALS)

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This invention relates to peptides, specifically L-enantiomeric peptide ligands with the amino acid sequence SEQ ID NO: 1 (and homologues/fragments), that selectively bind to the native (correctly folded) form of human superoxide dismutase 1 (SOD1). The peptide stabilizes native SOD1 and prevents its pathological aggregation. This approach is proposed for the treatment of amyotrophic lateral sclerosis (ALS), a currently untreatable neurodegenerative disease often associated with misfolded and aggregating SOD1.

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• Therapeutic agent for the treatment of amyotrophic lateral sclerosis (ALS) in patients with familial or sporadic forms of the disease.
• Potential use in stabilizing SOD1 protein to prevent or slow neurodegeneration caused by SOD1 aggregation in ALS.
• Research tool for studying protein misfolding diseases involving SOD1, and potentially identifying new targets for other protein aggregation-related neurodegenerative diseases.
• Development of drug candidates for other disorders involving protein aggregation, by adapting the peptide selection and stabilization method described.

BenefitsContent extracted from patent full text and abstract with AI.

• Addresses the root cause of ALS in cases related to SOD1 misfolding by stabilizing the native protein structure.
• Presents a novel potential therapy that could extend survival and slow progression in ALS patients, for whom effective treatments are currently lacking.
• Peptides can be highly specific and designed for high affinity, reducing the risk of off-target effects and toxicity compared to less specific interventions.
• The described method allows development of homologous or optimized peptides with improved pharmaceutical properties (e.g., better brain penetration, stability).
• May reduce formation of toxic SOD1 aggregates and thereby alleviate neurotoxicity in affected cells.

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Patent Abstract

The invention relates to a peptide containing the amino acid sequence according to SEQ ID NO: 1 and homologues, fragments and parts thereof, and to such a peptide for use in the treatment of amyotrophic lateral sclerosis (ALS).