30TWENTYFOURSEVENBIOPHARMA Issue 1 / March 2026 exosome production with evolving global standards. In this area, CDMOs can help developers navigate process validation, critical quality attributes, and compliance with GMP standards. This helps facilitate a smoother path to clinical and commercial success, especially for emerging modalities like exosomes. Exosome therapies are increasingly being explored as a means to enhance cell and gene therapies. In your view, how are exosomes revolutionizing these fields, and what are the most promising applications you foresee in the near future? The exosome therapy field today holds the kind of promise that antibody and protein-based therapeutics did years ago. Exosomes promise to offset, at least partially, some of the shortcomings observed with other cell and gene delivery platforms such as lipid nanoparticles (LNPs) and viral vectors. Specifically, initial clinical studies suggest that exosomes don’t present certain safety risks, including no reports of liver toxicity or adverse inflammatory events, enabling drug redosing and facilitating tissuespecific delivery and target engagement. Beyond cell and gene indications, exosomes may become a promising platform for vaccine development and immunotherapy approaches, including for cancer, where their drug-loading capacity and tissue-specific targeting can help minimize off-target effects and improve patient outcomes. Additionally, exosomes are currently explored as natural carriers of regenerative molecules (e.g., growth factors), enabling applications in wound healing and tissue regeneration. Earlier this year, Lonza and Exogenus Therapeutics announced an agreement to develop Exo-101, Exogenus’ exosome-based lead candidate. Could you provide insights into the specific aspects of this collaboration that highlight Lonza’s expertise in exosome development and manufacturing? What are the key milestones you are targeting in this collaboration? Lonza is leveraging its process and manufacturing expertise, specifically at our site in Siena, Italy, to develop a scalable process for the GMP manufacturing of Exo-101, a complex secretome product derived from cultured umbilical cord blood (UCB) cells. Our team of scientists and state-of-the art facilities draw on extensive experience with cell and gene therapies, providing a key advantage for technology providers, such as Exogenus, who require a bespoke process from upstream to downstream. Additionally, Lonza´s analytical characterization toolbox will enable Exogenus to identify and select key features of the product that will lead to the definition of its Critical Quality Attributes (CQAs) in GMP production. The manufacturing process has been fully assessed and a gap analysis performed to establish GMP manufacturability and scale. Following the initial feasibility and optimization studies, Lonza will perform confirmation runs with the optimized process to ensure preservation of Exo-101 CQAs and bioefficacy leading to GMP manufacturing. Lonza is very excited to undertake this project with Exogenus Therapeutics and to bring Exo-101 to clinical supply as efficiently as possible. Looking ahead, what are the critical advancements in exosome manufacturing technologies and regulatory frameworks that you believe will be essential to unlock the full potential of exosome-based therapeutics? Exosome manufacturers and regulatory agencies must work together, starting in the earliest stages of process development, to ensure alignment on expected critical quality attributes linked to product safety and potency. On the manufacturing side, we need continued innovation in processes, such as scalable, high-yield production systems that can meet commercial demand while maintaining product integrity. Automated purification workflows and engineered exosome platforms will be crucial for optimizing therapeutic delivery. From a regulatory perspective, clarity is essential. The industry needs standardized guidelines for exosome characterization, potency, and safety testing. Right now, different regulatory agencies have slightly different expectations, so harmonizing these frameworks will be essential to accelerating approval timelines and making exosome therapies widely available. LONZA DAVIDE ZOCCO Commercial Development Head for Exosomes and mRNA Technologies, Specialized Modalities Lonza
RkJQdWJsaXNoZXIy MjY2OTA4MA==