Lipid nanoparticles for lung-targeted mRNA delivery

Background:

Therapeutics that deliver mRNA to a patient’s lungs offer significant potential for treating a broad spectrum of lung diseases. However, specifically and efficiently delivering mRNA to lung tissue remains a challenge. At present, clinical trials of lung-targeted mRNA therapeutics are based on local delivery via inhalation. This can be a problem in emergency cases, or when the patient is unable to inhale the medication effectively. Systemic delivery of mRNA to the lungs may be more effective, but may present additional challenges. Recently, a cationic lipid-based mRNA delivery system has been developed for lung targeting. However, further research found this system may result in severe adverse effects.

Technology Overview:

This technology is based on new lipid nanoparticles that offer great potential to enable new mRNA therapeutics for treatment of lung-associated diseases and conditions. These materials have very different chemical structures and physicochemical properties compared to previously reported lipid materials. Mouse animal models indicate that after a single intravenous dose of these new lipid nanoparticles complexed with mRNA, the mRNA can be delivered specifically to the lungs and promote protein production. The mouse models demonstrate that these lung-targeting lipid nanoparticles loaded with gene engineering mRNA can induce genome recombination in the lung cells. The new lipid nanoparticle platform outperformed the current benchmark lipid nanoparticles in terms of efficacy and specificity for lung targeting. In addition, the new lipid nanoparticles demonstrated a favorable safety profile, with no observed local or systemic inflammation and toxicity. Beyond mRNA, this versatile lipid nanoparticle platform can potentially deliver small molecule drugs, siRNA, DNA, and other therapeutic agents to the lungs.

Advantages:

•    Delivers mRNA specifically to the lungs, promoting protein production.
•    Induces genome editing in lung cells. 
•    Safe (no local and systemic inflammation and toxicity induced).
•    Well-defined molecular and supramolecular structure for reproducibility.

Applications:

The primary application for this technology is developing mRNA-based treatments for lung diseases.

Intellectual Property Summary:

Patent application filed

Stage of Development:

TRL 3 – Experimental proof of concept

Licensing Status:

This technology is available for licensing.