Introduction

The present invention refers to the development and validation of a method to select recombinant antibody domains able to selectively bind and silence intracellular post-translationally modified (PTM) targets. Post-translational modifications (PTMs) are a wide class of therapeutic targets, very relevant for cancer, inflammation and neurodegenerative diseases.

Technical features

This invention is based on the Post-translational Intracellular Silencing Antibody Platform (PISA). The antibodies generated by means of this technology can be used for both therapeutic/ diagnostic applications and research. PTM proteins are a wide class of relevant therapeutic targets. Nevertheless, PTM-associated diseases are still untreatable because individual PTMs cannot be selectively blocked and validated as pharmacological targets even with powerful genetic methods. As a matter of fact, none of the drugs so far approved by the Food and Drug Administration (USA) is directed against a specific PTM of the corresponding target protein. The patented technology overcomes this shortcoming, allowing us to select specific antibodies able to recognise PTM proteins (in particular intracellular ones) by binding and silencing an individual PTM responsible for a given disease.

Possible Applications

• New generation therapeutic treatments;
• Target validation;
• Cellular screening;
• New generation diagnostic methods.

Advantages

• Access to undruggable targets;
• Molecular precision;
• Submolecular precision;
• Products administered as genes.