Introduction

To date, despite numerous efforts, there are no drugs available for the cure of neurodegenerative diseases collectively referred to as prion and prion-like disorders. Through this breakthrough invention, we are presenting a new class of small molecules acting as SERPINA3 inhibitors, able to reduce PrPSc accumulation in prion-infected cell lines;  representing a new therapeutic treatment against prion and prion-like diseases.

Technical features

Current treatment regimens for prion-related and prion-like neurodegenerative disorders target only the symptoms and not the cause of the disorder. Symptomatic treatments include the administration of antipsychotics, such as quetiapine and clonazepam, for the treatment of myoclonus, and selective serotonin re-uptake inhibitors (SSRIs) for the treatment of depression. In light of this, a new therapeutic treatment is proposed, which overcomes the limits of symptomatic treatments and acts directly on the cause of the disease. A new class of small molecules is now available that act as SERPIN3 inhibitors, capable of reducing the accumulation of PrPSc in prion-infected cell lines. The development of specific SERPINA3 inhibitors with activity in the nanomolar range is expected. Characterization of structural patterns of small molecule binding to the protein should allow for the identification of novel inhibitors with greater potency.

TRL Level: 4 (Technology Validated in Lab)

Possible Applications

• Prion diseases treatment;
• Use in pharmaceutical compositions as serpina3 inhibitors;

Advantages

• Potentially effective therapy for the treatment of prion diseases;
• Valid alternative to the administration of antipsychotics;
• It allows the identification of new inhibitors for neurodegenerative diseases;
• Approved reduction in the concentration of infectious agents;