Unlocking Access: Cell & Gene Therapy at Scale
November 9 - 10, 2026
San Raffaele Hospital
Milan, Italy
About the Event
Marking 30 years of pioneering research, the San Raffaele Telethon Institute for Gene Therapy and Danaher Corporation will convene a scientific symposium at San Raffaele Hospital in Milan. Over 1.5 days, researchers, clinicians, and scientific partners will come together to work across the translational gaps in hematopoietic stem cell (HSC) gene therapy, examining how scientific progress becomes durable, resulting in repeatable outcomes for patients in real-world settings. The program is structured around the full continuum, from discovery through clinical application and long-term follow-up, with a focus on what it takes to move from promising results to consistent delivery at scale. This includes engaging directly with the scientific, operational, and system-level challenges that continue to limit reproducibility and patient access. Grounded in patient impact, the discussion will bring together diverse perspectives to build a more complete view of the system—where progress is being made, where friction remains, and what responsible advancement requires from across the field. By convening leaders across disciplines, this symposium aims to strengthen shared understanding and foster the kind of collaboration needed to translate innovation into reliable outcomes for more patients.
THE AGENDA
Monday November 9
8:30 - 13:00
DAY 1 · MORNING
Monday, November 9 | 08:30–13:00 | Open to Public
Recognizing 30 Years of SR-TIGET
08:30 | Arrivals
08:45 | Welcome and Institutional Opening Remarks
Chiara Bonini, OSR Director
Ilaria Villa, Fondazione Telethon General Manager
09:00 | 30 Years of SR-TIGET
Luigi Naldini, Director, SR-TIGET
Maria Grazia Roncarolo, Former Director, SR-TIGET; Stanford University; Tr1X Inc.
09:30 | Wiskott–Aldrich Syndrome Gene Therapy: From Discovery to First-in-Human Trials and Approval
Alessandro Aiuti, Deputy Director, SR-TIGET
Jacob Thomas, Patient testimonial
10:15 | Coffee Break
Keynote Lectures
10:45 | Genetic Engineering of Hematopoiesis: From Gene Addition to Genome Editing
Luigi Naldini, SR-TIGET, Italy
11:15 | Mutation-Specific, Mutation-Agnostic, and Disease-Agnostic Therapeutic Genome Editing
David Liu, Broad Institute of MIT and Harvard, US
12:00 | Panel Discussion: Partnership as the Engine of Translation
Moderator
Jose-Carlos Gutierrez-Ramos, Danaher, US
Panelists
Matthew Porteus, Stanford University, US
Maria Grazia Roncarolo, Stanford University and Tr1X Inc., US
Alessandro Aiuti, SR-TIGET, Milan
Priya Stephen, WAS Foundation, US
Monday, November 9
14:30 - 18:30
DAY 1 · AFTERNOON
Monday, November 9 | 14:30–18:30 | Registered Attendees Only
HSC Gene Therapy: State of the Art, Achievements, and Remaining Challenges
14:30 | How Hospital Innovation Can Reach Patients When There Is No Market: The Case of SCID-Artemis Within the ATMP@AP-HP Platform
Marina Cavazzana, Hôpital Necker-Enfants Malades, France
15:00 | Toward Sustainable HSC Gene Therapy for Patients with Rare Diseases: Lessons from the Spanish Network on Advanced Therapies
Juan A. Bueren, CIEMAT, Spain
15:30 | 25 Years of HSC Gene Therapy at GOSH
Adrian J. Thrasher, Great Ormond Street Hospital, UK
16:00 | Gene Editing in Thalassemia: From Seminal Studies to Real-World Application
Franco Locatelli, Bambino Gesù Children's Hospital, Italy
16:30 | Coffee Break
17:00 | HSC Gene Therapy in Lysosomal Storage Disorders: Therapeutic Benefit vs. Allo-HSCT, Long-Term Outcomes, and Ongoing Developments
Maria Ester Bernardo, SR-TIGET, Italy
Safety and Clonal Dynamics in HSC Gene Therapy
17:30 | Viral DNA Integration: Mechanisms and Consequences
Frederic D. Bushman, University of Pennsylvania, US
18:00 | Clonal Tracking in HSC Gene Therapy: Insights into Human Hematopoiesis and Long-Term Safety
Eugenio Montini, SR-TIGET, Italy
Tuesday, November 10
8:45 - 13:30
DAY 2 · MORNING
Tuesday, November 10 | 08:45–13:30 | Registered Attendees Only
Manufacturing for Advanced Therapies
08:45 | Designing Advanced Therapies That Survive Scale: From Early Decisions to Commercial Readiness
Daria Donati, Cytiva, US
Novel Editing Technologies
9:15 | The Next Generation of Genome Editing for Sickle Cell Disease: Toward Safer, More Accessible Curative Therapies
John Tisdale, NIH, US
09:45 | Generalist Editing Approaches for Bone Marrow Failure
Daniel Bauer, Dana-Farber/Boston Children’s Hospital, US
10:15 | Asymmetric Collaborations to Unlock Genetically Engineered Stem Cells as Curative Drugs
Matthew Porteus, Stanford University, US
10:45 | Advancing Personalized Gene Editing Strategies for Fanconi Anemia
Paula Rio, CIEMAT, Spain
11:15 | Coffee Break
11:45 | Engineering Protected Hematopoiesis to Unlock Safer and More Accessible Cell and Gene Therapies
Pietro Genovese, Harvard Medical School, US
Strategy for Microglia Reconstitution by Engineered Hematopoiesis
12:15 | Targeting Neurometabolic and Neurodegenerative Diseases via Hematopoietic Stem Cell Gene Therapy
Alessandra Biffi, Padua University Hospital, Italy
Closing Keynote Lecture
12:45 | Advanced Therapies: From Breakthrough Science to Sustainable Access
Claire Booth, Great Ormond Street Hospital, UK
13:15 | Closing Remarks
Tom Foti, Danaher, US
Luigi Naldini, SR-TIGET, Italy
13:30 | Lunch & Departures
A scientific symposia held in collaboration by
