Serpin Inhibitors for the Treatment of Prion and Prion-Like Diseases

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

This invention relates to new small molecule inhibitors of the human SERPINA3 protein for the prevention and treatment of prion and prion-like diseases, including various neurodegenerative disorders. The compounds inhibit the activity of SERPINA3, which is upregulated in prion diseases and contributes to harmful protein misfolding and aggregation in neural cells. By blocking SERPINA3, these inhibitors reduce the accumulation of misfolded, disease-causing proteins, thereby slowing or preventing disease onset and progression in both humans and animals. The invention also covers pharmaceutical compositions containing these inhibitors for various modes of administration.

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

• Treatment of human prion diseases such as Creutzfeldt-Jakob Disease (CJD), fatal familial insomnia (FFI), Gerstmann-Straussler-Scheinker syndrome (GSS), and variant CJD.
• Therapy or prevention for neurodegenerative prion-like diseases including Alzheimer’s, Parkinson’s, Huntington’s disease, Amyotrophic Lateral Sclerosis (ALS), and Frontotemporal Dementia (FTD).
• Treatment or prophylaxis of animal prion diseases such as Bovine Spongiform Encephalopathy (BSE), scrapie in sheep, chronic wasting disease in deer, and others.
• Development of pharmaceutical products (tablets, injectables, nasal sprays, etc.) targeting SERPINA3 for veterinary or human medicine.
• Early intervention in individuals at risk of prion or prion-like disease before clinical symptoms appear.

BenefitsContent extracted from patent full text and abstract with AI.

• Provides a novel therapeutic approach for diseases with few or no effective treatments, especially prion diseases and prion-like neurodegenerative disorders.
• Targets an upstream molecular mechanism common to many neurodegenerative conditions, potentially offering broad-spectrum neuroprotection.
• Demonstrates significant reduction of misfolded prion proteins in cellular models, indicating potential to slow or stop disease progression.
• May be effective for both treatment and prevention, including administration before disease onset.
• Potential for use in both human and veterinary medicine, addressing public health and agricultural concerns.
• Small molecule inhibitors can be formulated for various routes of administration and could be easier to manufacture and distribute compared to biologics.
• By not interfering with normal prion protein, may avoid side effects linked to important natural functions of the target proteins.

Technical Classifications (CPCs)

Inventors & Applicants

Patent Abstract

The present invention is in the field of therapeutic treatment of prion and prion-like diseases. There is described a SERINA3 inhibitor compound and a pharmaceutical composition comprising the same. The compound of the invention is effective against the onset and/or progression of the neuropathological processes associated with prion and prion-like disorders.