Revolutionizing Treatment: How Nanoparticle Platforms Are Unlocking the Potential of Genetic Medicine
Genetic medicine holds the potential to treat and potentially cure complex diseases. The molecular tools available to clinicians utilizing gene therapies have progressed significantly in the past two decades with the development of antisense oligonucleic acids (ASOs), and CRISPR based gene editing modalities.
However, progress in drug delivery technology has not kept pace with the advances in gene therapy cargos, largely due to a lack of throughput in screening new delivery vehicles. At Battelle, we’ve built and pioneered a gene delivery vehicle discovery platform called HIT SCANTM (High Throughput Synthesis, Characterization and Assessment of Nanoparticles), designed to do just that. The HIT SCAN platform enables drug delivery scientists to change the discovery paradigm from screening several candidates at a time to hundreds. The HIT SCAN platform is focused on screening delivery vehicles that draw on the compositional diversity of combinatorial polymer chemistry.
Traditional delivery methods like lipid nanoparticles (LNPs) and adeno-associated viruses (AAVs) are not always capable of navigating the biological complexities of the human body effectively. Polymeric nanoparticles (PNPs) have gained traction as a promising solution to the challenges faced in the delivery of genetic medicine.
Unlocking Genetic Medicine's Potential with PNPs
PNPs stand out for their ability to encapsulate and protect genetic material, ensuring it reaches target cells efficiently and intact. This capability is a significant improvement over LNPs, which, while valid, offer less versatility in terms of payload capacity and targeting specificity. PNPs, by contrast, can be engineered to meet the specific needs of different genetic materials and therapeutic targets, making them a more adaptable and effective choice.
Expanded Regulatory and Commercialization Pathways
The regulatory landscape is adapting to support the development of these innovative technologies that are redefining the delivery of genetic therapies.
The FDA’s Platform Technology Designation Program for Drug Development is one example of this shift. This program recognizes the potential of PNPs as more than just one-off delivery vehicles for drugs, but platforms, and facilitates their path to market by allowing developers to leverage existing data to streamline the approval process.
This approach allows for a "combination product" — a device and a drug — to be integrated, to streamline the regulatory pathway. This integration is crucial for PNPs as it allows for the combination of nanoparticle carriers with genetic payloads. The FDA's guidance on platform technologies corroborates the importance of viewing these carriers as platforms rather than mere components.
The benefits are clear: enhanced efficiency, reduced development costs, and faster patient access to crucial therapies.
At Battelle, we’re using a robust discovery platform to match these vital therapies to PNPs designed for their specific payload and destination in the body. HIT SCAN is enabling researchers to automate nanoparticle synthesis, rapidly testing and evaluating large amounts of PNPs to find the ideal delivery candidates.
The future of PNPs in genetic medicine is bright, with ongoing research aimed at expanding their capabilities and applications. Innovations in PNP technology continue to push boundaries to deliver more targeted, efficient, and safe genetic therapies.