As the field of advanced therapeutics continues to evolve, one area drawing increasing attention is the use of extracellular vesicles (EVs), naturally occurring nanoparticles involved in intercellular communication, as potential drug delivery vehicles. Their ability to traverse biological barriers, their immune-silent nature, and their intrinsic capacity to target specific tissues make EVs uniquely suited for therapeutic applications.

In a recent webinar, Vijetha Bhat, a technology expert on Lonza’s licensing team, introduced Lonza’s Xcite^® EV Platform, a novel technology aimed at engineering exosomes for therapeutic use. This platform builds on foundational work developed by Codiak Biosciences and offers a scalable, flexible approach to designing exosome-based delivery systems.

EVs: Nature’s Nanocarriers

EVs are increasingly seen as a promising modality in therapeutic delivery due to several favorable properties. Unlike synthetic nanoparticles or viral vectors, EVs are naturally produced by cells and are well tolerated by the immune system. They can be loaded with a variety of cargo including proteins, peptides, and nucleic acids, and they exhibit selective tropism for certain tissue types. These features position them as highly versatile delivery vehicles.

What makes the Xcite^® EV Platform notable is its systematic approach to exosome engineering. Through the use of specialized scaffold proteins PTGFRN and BASP1, therapeutic molecules can be either displayed on the EV surface or encapsulated within the vesicle. This dual capacity significantly broadens the range of therapeutic payloads that can be effectively delivered.

Inside the Xcite^® EV Platform

At the core of the platform is a plasmid-based system that allows developers to fuse therapeutic targets to the scaffold proteins. Once transfected into a target cell line, these engineered cells produce exosomes that are pre-loaded with the desired cargo. This approach enables precision design of EVs tailored to specific applications from oncology to regenerative medicine.

Beyond protein-based payloads, the platform also accommodates other modalities such as oligonucleotides and small molecules using post-purification loading techniques. Lonza offers supporting protocols and intellectual property protections to assist partners in navigating these technical pathways.

From Concept to Clinic: Real-World Examples

Bhat presented several examples of how the Xcite^® technology is already being leveraged in preclinical and clinical contexts. A particularly compelling case study focused on exoIL-12™, an engineered EV expressing interleukin-12 on its surface. In early-phase clinical trials for cutaneous T-cell lymphoma, patients treated with exoIL-12™ showed strong tumor retention of the therapeutic and encouraging response rates without systemic inflammatory side effects, a common challenge with cytokine-based therapies.

Lonza also highlighted the potential of exoVACC^®, a vaccine development platform that uses exosomes to co-deliver antigens and adjuvants to the same antigen-presenting cells. Early data suggests this strategy may support more selective immune targeting and longer-lasting responses, with additional benefits in manufacturability and stability.

Challenges Ahead and a Path Forward

Despite the promise, the development of EV-based therapies is not without challenges. Understanding EV biology, establishing robust analytical methods, and ensuring scalability for GMP production are all critical hurdles. Bhat emphasized that Lonza’s experience in biologics, cell, and gene therapy manufacturing uniquely positions the company to support partners in overcoming these obstacles.

To accelerate innovation in this space, Lonza has made the Xcite^® EV Platform available through its “Lonza In Your Lab^®” licensing model, which provides research teams with access to the same tools used internally by Lonza’s CDMO operations. This includes plasmids, transposase systems, and know-how around host cells and culture media, all designed to help innovators generate proof-of-concept data more efficiently.

A Platform Poised to Shape the Future

As interest in EV-based therapeutics continues to grow, platforms like Xcite^® could play a key role in translating promise into clinical reality. By offering a modular, well-characterized approach to exosome engineering, Lonza is contributing to the maturation of this emerging modality and supporting developers looking to explore its full therapeutic potential.

For those interested in learning more about the Xcite^® platform and how it may fit into their development roadmap, the full webinar is available to watch on demand above.

About the Presenter

Vijetha Bhat is a technology expert working within Licensing in Lonza’s Specialized Modalities Business Platform. She has 15 years of experience in the biopharma industry. Vijetha started her scientific career at Lonza in 2017 and has since contributed to a variety of projects focusing on developing production platforms and analytics for the cell and gene therapy space. She currently supports with providing in-depth technical direction to users working with Lonza’s expression platforms.