ISCT 2026: Exosomes Scientific Signature Series Event

This session will examine GMP-qualified MSC-EV production strategies, emphasizing meaningful definitions of scale, dose-relevant yield assessment, and analytical approaches for evaluating process and product performance. Speakers will address practical manufacturing challenges and emerging technologies that enhance reliability, consistency, and cost control.

Key Learning Objectives:

1. Understand how to define manufacturing scale using therapeutic dose equivalents, and recognize the limitations of particle counts and protein yield as standalone indicators of production efficiency.

2. Evaluate GMP-ready production platforms, including bioreactor innovations (e.g., microturbulence-based systems) and their impact on yield, consistency, and cost.

3. Identify analytical tools and CMC parameters that support process qualification and ensure product comparability across manufacturing runs.

This session will focus on the mechanistic foundations required to design regulatory-compliant potency assays for MSC-EV therapies. Experts will discuss the gap between commonly described “modes of action” and the mechanistic specificity needed to define potency-linked CQAs.

Key Learning Objectives:

1. Differentiate between mode of action and mechanism of action, and understand why regulatory potency assays require mechanistic, not descriptive, linkage.

2. Identify strategies for defining mechanistically relevant potency CQAs, including approaches informed by clinical and preclinical data.

3. Recognize the regulatory expectations for potency assay development, and how mechanistic definition improves assay relevance, reliability, and harmonization across jurisdictions.

This session will explore challenges and pathways for financing, regulatory progression, and clinical adoption of high-cost MSC-EV therapies. Speakers will draw on real-world experiences—including organizational, clinical, and regulatory lessons—to outline viable models for sustaining development through late-stage trials and into market access.

Key Learning Objectives:

1. Understand the financing barriers and investment dynamics affecting advanced MSC-EV programs, including lessons from companies facing resource constraints.

2. Evaluate regulatory and clinical translation pathways, informed by existing clinical programs and cross-regional regulatory considerations.

3. Identify market access and reimbursement challenges for high-cost cell-free therapies and assess strategies to support sustainable adoption.