28TWENTYFOURSEVENBIOPHARMA Issue 1 / March 2026 LONZA Scaling GMP-compliant manufacturing for exosome-based therapies presents unique challenges compared to traditional biologics. Could you elaborate on the key hurdles Lonza encounters in this process, particularly regarding standardization and reproducibility? Furthermore, what are the most critical regulatory considerations that developers should prioritize when navigating the approval pathway for exosome therapeutics globally? Exosomes are heterogeneous nanoparticles that carry a plethora of biomolecules, such as proteins, small RNAs, DNA, and lipids. Their unique ability to cross the blood-brain barrier makes them attractive vectors for delivering therapeutic payloads, such as proteins and nucleic acids, to target specific tissues. If these capabilities are fully realized, the potential applications of exosomes could be vast, spanning disease treatment, immune modulation, and tissue regeneration. Despite their potential, manufacturing standards for exosome-based therapies are still in the early stages. One key hurdle manufacturers may face in building a Good Manufacturing Practices (GMP)-compliant process for exosome production is the limited understanding of how exosome characteristics influence their biological functions. This knowledge gap can hinder the development of scalable methods for isolating and purifying specific exosome populations from other extracellular vesicles and cellular debris. Additionally, during manufacturing, specific exosome subpopulations may be selected or excluded, affecting the critical quality attributes of the drug substance. Processes that rely on primary cells (e.g., mesenchymal stem cells [MSCs]) may be difficult to standardize as the exosome cargo may vary from one cell batch to the other. Furthermore, the lack of standardized analytics for exosome therapeutics hinders process development and product approval. Establishing GMP-compliant processes is an essential part of regulatory strategy and should be addressed in the earliest stages of development. When developing these processes, it is important to focus on defining a product’s critical quality attributes (CQAs), supported by qualified analytics, to enable the manufacture of a product that is safe and, secondly, effective.
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