SERPIN inhibitors for the treatment of prion and prion like diseases

Through this invention, we are presenting a new class of small molecules acting as SERPINA3 inhibitors, which are able to reduce PrPSc accumulation in prion-infected cell lines, thereby representing a new therapeutic treatment against prion and prion-like diseases.

Ref. Nr. TA-PT 1.2816G
Institute for Advanced Simulation

Development Status: Trial. The project is being developed at Scuola Internazionale Superiore di Studi Avanzati (SISSA). Thus far, the research team has characterized the system in vitro and partially in vivo. Further developments are expected in the coming months also depending on the identification of appropriate partnership.

We are continuously seeking for cooperation partners and/or Licensees in this and adjacent areas of research and application.

Potential Applications: Compounds for use in the therapeutic treatment of a prion or prion-like disease and to pharmaceutical compositions comprising these compounds.

Background

To date, despite numerous active efforts, there are no drugs available for the cure of the neurodegenerative diseases collectively referred as prion and prion-like disorders. Symptomatic treatment is the only available option, including the administration to patients of antipsychotics, such as quetiapine and clonazepam, to treat myoclonus, and of selective serotonin re-uptake inhibitors (SSRIs) to treat depression.

Problem addressed

So far, there are no effective therapies for prion diseases. The proposed technology aims at developing novel molecules with improved efficacy on these devastating diseases.

Solution

Through this invention, we are presenting a new class of small molecules acting as SERPINA3 inhibitors, which are able to reduce PrPSc accumulation in prion-infected cell lines, thereby representing a new therapeutic treatment against prion and prion-like diseases.

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Letzte Änderung: 06.02.2024