Polysialic acid and use for treatment of neurodegenerative and neuroinflammatory diseases

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This patent describes the use of low molecular weight polysialic acid (chains of 14–26 sialic acid units, especially the α(2→8)-linked form) as a medicinal compound that can prevent or treat neurodegenerative and neuroinflammatory diseases in the central nervous system (CNS) and retina. The invention reports that these specific polysialic acid fragments can inhibit pro-inflammatory cytokine production by microglia and tissue macrophages without toxic effects, and demonstrates therapeutic benefit in disease models of multiple sclerosis and retinal degeneration.

Use CasesContent extracted from patent full text and abstract with AI.

• Treatment of neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, and dementia with Lewy bodies.
• Prevention or treatment of neuroinflammatory diseases including multiple sclerosis, septic encephalopathy, and other CNS inflammatory conditions.
• Treatment or prophylaxis of degenerative or inflammatory diseases of the retina, such as age-related macular degeneration, diabetic retinopathy, uveitis, and inherited retinal diseases.
• Therapy to prevent or reduce microglia-mediated neurotoxicity associated with amyloid-beta in Alzheimer's disease.
• Intervention to reduce inflammation-induced progression of neurodegenerative diseases triggered by systemic infections or sepsis.

BenefitsContent extracted from patent full text and abstract with AI.

• Selectivity: The use of polysialic acid fragments with a specific chain length avoids toxicity and provides effective anti-inflammatory activity.
• Multi-disease application: Offers potential treatment across a spectrum of CNS and retinal degenerative and inflammatory disorders.
• Novel mechanism: Reduces pro-inflammatory cytokine and reactive oxygen species production by microglia/macrophages, a key driver of neuronal damage.
• Prophylactic potential: Can be used not just for therapy but also for prevention or delay of disease progression.
• Good compatibility: Linear α(2→8)-linked polysialic acid matches endogenous human forms, enhancing biological safety.
• Demonstrated efficacy in preclinical models: Shows reductions in disease symptoms and pathology in animal models of multiple sclerosis and macular degeneration.

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Inventors & Applicants

Patent Abstract

The present invention relates to a branched or unbranched free or glycosidically bound polysialic acid according to general formula (1) as given as follows (poly-±(2 †’ 8 or 2 †’ 9)Neu5Ac) n (1) wherein Neu5Ac is N-acetylneuraminic acid, and n is an integer in the range from 14 to 26 and/or pharmaceutically acceptable salts thereof, a polysaccharide composition comprising the polysialic acid (1), and the use as a medicament, particularly in the therapeutic and/or prophylactic treatment of degenerative, demyelinating and inflammatory diseases of the central nervous system, and degenerative or inflammatory retinal diseases.