New Perspectives On Accelerating Genetic Nanomedicines Through The Regulatory Pathway

By Tony Duong, Ph.D., Nanoscientist, Lead Drug Delivery Scientist

Gene therapies and nanomedicines have the potential to revolutionize medicine, but their success depends on navigating regulatory pathways effectively. Collaboration, transparency, and patient safety are crucial in accelerating the development and use of these therapies. Gene therapies combined with nanocarrier technology provide precise delivery vehicles for targeted treatments. The FDA classifies gene therapies and nanocarriers as drugs, devices, or biological products, and combination products can be created by combining drugs and devices. Regulatory bodies are adopting a risk-based approach to address the unique challenges of genetic nanomedicines, and stakeholder engagement is essential.

The FDA approval process for genetic nanomedicines using lipid nanoparticles involves considering them as novel excipients or as components of the final biologic product. A platform approach with a family of nanocarriers can de-risk clinical trials and accelerate the development of new biological drug products. Battelle has developed the HIT SCAN platform, which combines automated polymer synthesis, high-throughput screening, and machine learning to create a diverse library of nanoparticles for gene therapy delivery. This platform allows for tracking nanoparticle biodistribution and behavior, and artificial neural networks are used to predict their performance based on chemical makeup.

The goal is to use nanoparticles as a platform for delivering various biotherapeutics, rather than just for specific payloads. The development of a library of well-characterized nanoparticles with data on their composition, molecular weight, biodistribution, toxicity, and delivery efficiency is seen as a valuable tool. Battelle is confident that the HIT SCAN platform has the potential to revolutionize gene therapy and treat a wide range of diseases. With the potential to transform medicine, gene therapies and nanomedicines are paving the way for innovative treatments and improved patient outcomes.