Taking Combination Products To The Next Level: Combining Gene Therapies, Carrier Systems, And Medical Devices

By Stefanie Johns, PhD, Director of Regulatory Affairs, Kymanox Corporation

Drug delivery methods present considerable challenges to the development of gene therapies, including difficulties in accessing the therapeutic target, prevention of degradation, and facilitation of drug activity to achieve its intended purpose. Even one of the most popular delivery methods, adeno-associated virus (AAV)-based technology, poses serious risk to patient safety through severe adverse side effects.

Combination products incorporating a small molecule drug or biologic have already demonstrated success by improving accessibility for patients and expanding therapeutics indications. Novel drug delivery carrier systems that combine novel medical devices to improve delivery of gene therapies hold significant potential to expand the utility of gene therapy by improving access to gene targets while reducing toxicity. While recent successes in delivery carrier systems methods - delivering mRNA using lipid nanoparticles and macromolecule ligand tags - have led to more specific localization and processing of RNA-based drugs, the new frontier for developing gene therapies lies in combining these drug carrier systems and the medical device used to deliver the drug into the patient effectively and safely. Examine the opportunities for success in combining novel medical devices to improve delivery of gene therapies.