Event preview of the 6th iPSC Drug Development Summit

Clinical translation of iPSC therapies

As iPSC-derived cell therapies progress through clinical development, sessions at the summit will examine first-in-human experiences and advancing data across Parkinson's disease and ocular indications, with a focus on safety, engraftment, durability, and functional outcomes. Ron Cohen (Chief Executive Officer, Oryon Cell Therapies) will offer an inside look at how Oryon is advancing autologous iPSC-derived dopaminergic neurons in Parkinson's disease. The session will address the real challenges of demonstrating safety, durability, and manufacturability while aligning CMC and regulatory strategy to support clinical progression, offering particular value for teams navigating complex comparability and scale considerations in patient-specific iPSC programs.

The summit will also provide insight into how autologous and allogeneic development paths are converging as programs approach pivotal readiness, reflecting the field's broadening clinical ambition and the strategic choices developers face when selecting between patient-specific and universal approaches.

Manufacturability, CMC, and GMP readiness

Scaling iPSC platforms to meet GMP standards remains one of the field's most pressing challenges. Sessions in this track will share real-world lessons on building scalable, GMP-ready iPSC workflows, including donor and cell line selection, comparability strategies, and the control of CQAs.

Rachel Knopp (Lead, iPSC Characterization Team, Astellas Pharma) will address what 'fit for purpose' characterization looks like in large-scale iPSC programs, sharing lessons from big pharma on building integrated analytical and QC frameworks that confirm correct genetic editing, functional phenotypes, and consistency early enough to prevent costly downstream failures. The session is designed for teams serious about GMP readiness and regulatory confidence.

A dedicated roundtable, 'Implementing functional QC frameworks to predict potency and reduce line-to-line variability early in development', will bring together Yanzheng Liu (Senior Director, Quality Control, Aspen Neuroscience) and Eric Law (Head of CMC Regulatory Affairs, Century Therapeutics) to discuss how functional assays can assess differentiation, proliferative potential, and therapeutic relevance early in development. The discussion will also address the industry-wide gap in functional characterization and how QC systems can de-risk variability across clones, donors, and maturation states, ensuring controlled, predictable behavior before products enter GMP workflows.

Genome editing and next-generation iPSC platforms

Genome editing is increasingly central to the design of next-generation iPSC-derived therapies, including immune-evasive platforms and CAR-NK and microglia-based approaches. Sessions in this track will explore how editing decisions made in preclinical development affect long-term safety, durability, and regulatory confidence.

Mahendra Rao (Chief Scientific Officer, Vita Therapeutics) will present on 'Implementing GMP-compatible gene editing strategies to enable scalable iPSC-derived cell manufacturing'. The session will cover how to translate complex immune-evasion gene editing strategies into manufacturable iPSC platforms without committing to multiple costly master cell lines, and how to design flexible, staged CRISPR editing and evaluation workflows that balance product customization, speed, and control in GMP manufacturing. Real-world constraints, including CRISPR selection, IP and licensing considerations, and CDMO readiness, will also be addressed.

A fireside chat, 'Balancing immune evasion with safety controls to prevent oncogenic outgrowth and improve preclinical risk predictability', will feature William Hendriks (Director, PSC Biology, BlueRock Therapeutics) and John Steel (Director, Voyager Therapeutics). The discussion will examine how iPSC-derived cells may evade or escape immune surveillance and the implications for uncontrolled proliferation in vivo, how kill-switches, regulated elimination systems, and early oncogenicity assays can counter immune-escape-driven safety concerns, and how to design preclinical studies that distinguish acceptable immune evasion for persistence from high-risk profiles that could trigger cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), or tumor-like expansion.

Investment, de-risking, and commercial readiness

The closing session of the summit will address the commercial and investment landscape for iPSC programs, bringing together pharma, venture capital, and funding stakeholders to examine how early strategic alignment can reduce development risk in a capital-intensive field.

A panel discussion, 'De-risking iPSC therapies by aligning investors, pharma, and stakeholders to unlock commercial success', will feature Aaron Goldman (Director, Drug Resistance Group and Faculty, Instructor in Medicine / Operating Partner, Harvard Medical School / BroadOak Capital), Daniel Friedman (Managing Director, BroadOak Capital), and Steven Kattman (External Diligence and Competitive Intelligence Advisor, Astellas Pharma). The discussion will explore how to engage pharma business development teams, VCs, and payers early to address the high cost of goods and long development timelines that characterize iPSC programs, what investors look for in scalable, robust manufacturing strategies before committing capital, and how to coordinate government funding, venture investment, and pharma partnerships to support sustainable clinical and commercial success. 

Across two focused days, the 6th iPSC Drug Development Summit will provide an in-depth forum for examining how iPSC platforms are advancing from preclinical promise to clinical and commercial reality. With sessions spanning translational data, GMP manufacturing, genome editing strategy, and regulatory alignment, the summit offers a comprehensive view of the decisions and capabilities shaping the next generation of iPSC-derived therapies. Bringing together leaders from organizations including Oryon Cell Therapies, BlueRock Therapeutics, BroadOak Capital, Astellas Pharma, Aspen Neuroscience, Century Therapeutics, and Vita Therapeutics, the meeting represents a timely and focused opportunity to engage with the science, strategy, and infrastructure driving this rapidly maturing field. 

You can register for the 6th iPSC Drug Development Summit here.

Additionally, to find out what other cell and gene therapy events are upcoming, you can find our online Events Calendar here.