RNA trans-splicing molecule (RTM) for use in the treatment of cancer

US9655979B2 · kind B2 · utility

Assignees

• Johann Bauer · AT
• Christina Gruber · AT
• Ulrich Koller · AT

Inventors

• Johann Bauer · Ismaning, DE
• Christina Gruber · Bad Vigaun, AT
• Ulrich Koller · Neumarkt am Wallersee, AT

Key dates

• Filing date: Oct 31, 2013
• Grant date: May 23, 2017
• Expiry date: Oct 31, 2033

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2320/33
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present invention pertains to a novel pre-mRNA trans-splicing molecule (RTM) comprising a binding region which is complementary to a pre mRNA of a tumor associated gene, and a coding domain which encodes for a suicide gene. Specific embodiments of the present invention relate to RTMs which mediate trans-splicing of a suicide gene, such as thymidine kinase from herpes simplex virus (HSV-tk), with the pre mRNA of the squamous cell carcinoma associated gene solute carrier organic anion transporter family member 1B3 (SLCO1B3). The invention provides RTMs which selectively kill cells expressing SLCO1B3 and which are thus useful in the treatment of cancer, specifically squamous cell carcinoma associated with epidermolysis bullosa. Also provided are methods, kits and pharmaceutical compositions relating to the RTMs in accordance to the invention.