• Modified nucleic acid functional molecule for the treatment of rare genetic diseases

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Gene Editing, Rare Diseases

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Modified nucleic acid functional molecule for the treatment of rare genetic diseases

Drug Design & Delivery Systems | New Drugs & Therapies

Introduction

Non-coding long RNAs (IncRNAs), known as SINEUP, are a new class of RNA that play key roles in cell physiology. In particular, thanks to their ability to increase the efficiency of protein synthesis, they are used as a possible therapy for genetic diseases. So far, SINEUP has been administered via plasmid transfection. However, this method is ineffective because it requires the export of transcribed RNA from the nucleus to the cytoplasm for co-localization with the target mrna.

PT20055_IIT_Immagine 1

Technical Features

The functional nucleic acid molecule of the invention can be administered as bare or unpackaged RNA, or through a carrier. The composition may include standard carriers, such as liposomes, exosomes, lipid particles or nanoparticles, which help deliver RNA to the target cell, directly into the cytoplasm. It can also be administered as an oligoribonucleotide containing the modified molecule. The functional molecule consists of a complementary sequence of the target mrn to increase protein translation and a regulatory sequence with a SINE B2 element or active fragment. The molecule is chemically modified to improve stability and efficacy, both in in vitro transcribed RNA (IVT) and in transcribed RNA in the cell (ICT). Changes may include 2'-O-Methyl, Pseudouridine and others, applied co- or post-transcriptionally.

Possible Applications

  • Treatment of diseases associated with genetic defects (e.g. reduction of protein levels and/or loss of coding gene functions)
  • Treatment of diseases caused by a quantitative decrease in normal protein level

Advantages

  • Controlled expression thanks to stimulation of specific target mrna without altering the endogenous mrn
  • Improved stability
  • Small size: IVT SINEUP can be reduced to the minimum functional size, facilitating transport to target organs with minimal invasive effects.
  • Versatility in administration
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