Kits for advanced polymer-lipid nanocarriers for targeted delivery of RNAs to cardiac and skeletal muscle cells

Introduction

Currently, RNA therapies are available in clinics to treat liver diseases and as COVID-19 vaccines. Hence, their therapeutic potential still remains largely unexploited for the treatment of other disorders, such as age-related, rare genetic, and infectious diseases.

Problem Statement

One key problem for effective preclinical research on new RNA therapies is the lack of safe, efficient, and cell-specific delivery vehicles. Gold-standard cationic lipid transfection reagents suffer from:

• Dose-dependent cytotoxicity
• Inability for cell-targeted delivery
• Poor stability

Clinically approved lipid nanoparticles efficiently target liver cells, while attempts to target other cell types have achieved limited success.

Project Overview

In POLIRNA, I will develop and validate a new safe, efficient, and versatile platform for RNA release to target multiple cell types. This will be based on ligand-functionalized polymer-lipid nanocarriers, by exploiting a new patented method from my ERC-funded project BIORECAR (772168).

Research Kits

POLIRNA consists of “kits for research use,” allowing user-friendly preparation of nanocarriers able to effectively deliver non-coding RNAs (such as siRNAs and microRNAs) to target:

1. Cardiomyocytes
2. Skeletal muscle cells

This will facilitate preclinical research studies on cardiac regenerative medicine and muscle-related diseases (e.g., neuromuscular diseases).

Collaboration and Validation

Technology development and validation will benefit from collaboration with major experts in the fields and companies supporting the innovation route from lab to market.

Experience and Goals

My previous experience as coordinator of research and innovation projects, as well as being a co-founding member of an academic spin-off company, will drive the process of technology validation. The current involvement of my team in business plan competitions aims to:

• Strengthen technology value proposition versus current gold-standard solutions
• Found a start-up company
• Attract investments