Dry Powder Formulation of RNA Nanoparticles for Inhalation and Improved Storage and Transport Conditions

Introduction

RNhale aims to establish technical and commercial proof-of-concept for our existing and protected protocols to be transformed into a platform technology with the long-term goal of licensing agreements with large pharmaceutical companies which are equipped for continuous manufacturing of large scales of dry powder RNA formulations.

Commercial Positioning

RNhale will assess the commercial positioning of a platform technology for spray-drying RNA nanoparticles (NPs). While RNA medicines have revolutionized how we can address a pandemic, RNA therapeutics have not yet broadly changed patient outcomes but are specifically used for very limited indications due to two main reasons:

1. The high immunogenicity and poor stability of RNA.
2. The accumulation of nano-encapsulated RNA in the liver and resulting poor targetability of other organs with RNA-loaded NPs.

Addressing Shortcomings

These shortcomings have been addressed in my current ERC project Novel Asthma Therapy by spray-drying polymer-based RNA NPs.

Refinement of Protocols

RNhale will refine existing protocols for spray-drying RNA NPs made with diverse RNA NPs to overcome current challenges by:

1. Improving storage and transport conditions of spray-dried RNA medicines in general.
2. Gaining access to delivery beyond the liver via inhalation delivery to the lung.