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In Vivo Hematopoietic Stem Cell Engineering: Building the Bone Marrow as a Living Factory for One-Time Genetic Medicines

June 22, 2026
Type: Breakout Session
Focus Area: Cell and Gene Therapy and Genome Editing
In vivo hematopoietic stem cell (HSC) engineering represents the next inflection point in genetic medicine - moving beyond transient delivery or ex vivo manipulation to direct, permanent reprogramming of the body’s own stem cells. By targeting HSCs in the body’s native bone marrow, emerging in vivo platforms such as viral-like particles, base and epigenetic editing, lipid nanoparticles, lentivirus and fusogen-based systems are advancing the therapeutic paradigm toward one-time, off-the-shelf treatments. This panel will convene thought leaders from this emerging field to explore scientific breakthroughs enabling in vivo HSC targeting, therapeutic opportunities across disease areas, challenges of delivery and safety, and the regulatory and commercial models needed for success. Attendees will leave with a stronger understanding of why this is one of the most important and emerging areas in therapeutic innovation.
Speakers
David Barrett
VP, Cell Biology & Translational Medicine
Kite Pharma
Jim Burns
Chief Executive Officer
Ensoma
Hans-Peter Kiem, M.D., Ph.D.
Deputy Director & Professor, Translational Science and Therapeutics Division; Professor, Vaccine an
Fred Hutchinson Cancer Center
John Lo, Ph.D.
Chief Commercial Officer
Beam Therapeutics

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