Modulators of the unconventional secretory pathway for use in the treatment of Alexander disease

Simple SummaryContent extracted from patent full text and abstract with AI.

This patent introduces a new therapeutic approach for treating Alexander disease, a rare and fatal neurodegenerative disorder. The invention centers on modulators (including certain known drugs and genetic interventions) that regulate the 'unconventional secretory pathway' within cells. This pathway, when dysfunctional due to specific mutations in the GFAP protein, disrupts the secretion of important enzymes like insulin-degrading enzyme (IDE). The patent claims that using specific small-molecule drugs (such as statins or kinase inhibitors) or gene/cell therapies can restore this secretion process and potentially treat or alleviate Alexander disease symptoms.

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

• Treatment of Alexander disease in adults, children, and infants carrying GFAP mutations.
• Development of pharmaceutical compositions (oral, injectable, etc.) containing listed modulators for clinical use.
• Adjunct therapy to slow progression or improve symptoms in Alexander disease patients.
• Gene or cell-based therapies using engineered viruses or modified cells to correct secretory pathway dysfunctions in the central nervous system.
• Preclinical research platforms to test unconventional secretory pathway modulators for Alexander disease or related neurodegenerative disorders.

BenefitsContent extracted from patent full text and abstract with AI.

• Provides the first targeted therapeutic approach for Alexander disease, a disorder previously considered untreatable.
• Utilizes several classes of modulators, including repurposed drugs like statins, which may speed up clinical translation and regulatory approval.
• Offers both pharmacological and gene/cell-based treatment strategies, addressing different patient needs and disease severities.
• Has the potential to alleviate symptoms, slow progression, and improve quality of life for Alexander disease patients.
• Restores normal secretion of insulin-degrading enzyme (IDE), potentially impacting not only Alexander disease but also other neurodegenerative diseases involving the unconventional secretory pathway.

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Inventors & Applicants

Patent Abstract

The present invention relates to modulators of the unconventional secretory pathway for use in the treatment of Alexander disease.