Children’s Hospital of Philadelphia (CHOP) today announced that CHOP physicians conducting genetic research will present new and updated data at the American Society of Gene and Cell Therapy (ASGCT) annual conference in Boston, Massachusetts, May 11–15, 2026. Data from more than 30 presentations, provide insight on CHOP’s approach to gene-based strategies for treatment.
Additionally, multiple gene editing innovators from CHOP will receive awards at ASGCT. Recipients include:
The Outstanding Achievement Award
Thursday, May 14, 2026
MCEC Exhibit Hall B1
2:30 p.m. EST
Beverly Davidson, PhD, Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Chief Scientific Strategy Officer at CHOP, and Professor of Pathology and Laboratory Medicine at Penn Medicine, will receive the society’s highest honor. The award recognizes an ASGCT member who has achieved a pioneering research success, a specific high-impact accomplishment, or a lifetime of significant contributions to the field of cell and gene therapy. Dr. Davidson will be honored for foundational discoveries into the molecular mechanisms of fatal inherited brain diseases and for leading translational efforts that have advanced Huntington’s disease (HD) research toward potentially disease‑modifying therapies.
The Catalyst Award
Tuesday, May 12, 2026
MCEC Exhibit Hall B1
2:30 p.m. EST
The award honors an ASGCT member or team whose transformative work accelerates the translation of cell and gene therapies from discovery to the clinic. This year’s award was shared among four distinguished recipients recognizing their collaboration and work leading to the first personalized gene editing therapy. They include:
- Rebecca Ahrens-Nicklas, MD, PhD, a pediatric geneticist and Director of CHOP’s Gene Therapy for Inherited Metabolic Disorders Program
- Danaher Corporation
- Kiran Musunuru, MD, PhD, MPH, ML, MRA, Co-Director of the Orphan Disease Center, a partnership between CHOP and Penn Medicine
- Fyodor Urnov, PhD, Professor of Molecular Therapeutics at UC Berkeley and Director of Therapeutic R&D at IGI
Highlighted CHOP presentations at ASGCT include:
May 12
William Peranteau, MD
Germline Editing: Technical Background Perinatal
10:15 - 10:35 a.m. EST
Denise Sabatino, PhD
Dissecting SUMOylation-mediated mechanisms that regulate AAV transgene expression
4 - 4:15 p.m. EST
May 13
Lindsey George, MD
Evolving understanding of AAV toxicities
10:15 - 10:41 a.m. EST
Benjamin Samelson-Jones, MD, PhD (Session Co-Chair)
Recent toxicities in AAV gene therapies: Translational lessons from preclinical and clinical studies
10:15 a.m. - noon EST
Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, MPH, ML, MRA
Clinical translation of somatic genome editing as a therapeutic platform
3:30 - 3:45 p.m. EST
Lindsey George, MD
AAV and systemic toxicities in clinic trials and clinical practice
3:30 - 3:52 p.m. EST
Top Abstract: Lindsey George, MD
Neuroepithelial Tumor Associated with AAV Integration After Intracisternal Magna Vector Delivery
May 15
Peter Kurre, MD
Positive clonal selection of curative HSCs - introductory component
10:15 - 10:41 a.m. EST
Benjamin Samelson-Jones, MD, PhD
Multiyear stable transgene factor IX levels after AAV hepatocyte-directed gene in neonatal and juvenile hemophilia B dogs
3:30 - 3:45 p.m. EST
Learn more here.
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Children’s Hospital of Philadelphia (CHOP) today announced that CHOP physicians conducting genetic research will present new and updated data at the American Society of Gene and Cell Therapy (ASGCT) annual conference in Boston, Massachusetts, May 11–15, 2026. Data from more than 30 presentations, provide insight on CHOP’s approach to gene-based strategies for treatment.
Additionally, multiple gene editing innovators from CHOP will receive awards at ASGCT. Recipients include:
The Outstanding Achievement Award
Thursday, May 14, 2026
MCEC Exhibit Hall B1
2:30 p.m. EST
Beverly Davidson, PhD, Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Chief Scientific Strategy Officer at CHOP, and Professor of Pathology and Laboratory Medicine at Penn Medicine, will receive the society’s highest honor. The award recognizes an ASGCT member who has achieved a pioneering research success, a specific high-impact accomplishment, or a lifetime of significant contributions to the field of cell and gene therapy. Dr. Davidson will be honored for foundational discoveries into the molecular mechanisms of fatal inherited brain diseases and for leading translational efforts that have advanced Huntington’s disease (HD) research toward potentially disease‑modifying therapies.
The Catalyst Award
Tuesday, May 12, 2026
MCEC Exhibit Hall B1
2:30 p.m. EST
The award honors an ASGCT member or team whose transformative work accelerates the translation of cell and gene therapies from discovery to the clinic. This year’s award was shared among four distinguished recipients recognizing their collaboration and work leading to the first personalized gene editing therapy. They include:
- Rebecca Ahrens-Nicklas, MD, PhD, a pediatric geneticist and Director of CHOP’s Gene Therapy for Inherited Metabolic Disorders Program
- Danaher Corporation
- Kiran Musunuru, MD, PhD, MPH, ML, MRA, Co-Director of the Orphan Disease Center, a partnership between CHOP and Penn Medicine
- Fyodor Urnov, PhD, Professor of Molecular Therapeutics at UC Berkeley and Director of Therapeutic R&D at IGI
Highlighted CHOP presentations at ASGCT include:
May 12
William Peranteau, MD
Germline Editing: Technical Background Perinatal
10:15 - 10:35 a.m. EST
Denise Sabatino, PhD
Dissecting SUMOylation-mediated mechanisms that regulate AAV transgene expression
4 - 4:15 p.m. EST
May 13
Lindsey George, MD
Evolving understanding of AAV toxicities
10:15 - 10:41 a.m. EST
Benjamin Samelson-Jones, MD, PhD (Session Co-Chair)
Recent toxicities in AAV gene therapies: Translational lessons from preclinical and clinical studies
10:15 a.m. - noon EST
Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, MPH, ML, MRA
Clinical translation of somatic genome editing as a therapeutic platform
3:30 - 3:45 p.m. EST
Lindsey George, MD
AAV and systemic toxicities in clinic trials and clinical practice
3:30 - 3:52 p.m. EST
Top Abstract: Lindsey George, MD
Neuroepithelial Tumor Associated with AAV Integration After Intracisternal Magna Vector Delivery
May 15
Peter Kurre, MD
Positive clonal selection of curative HSCs - introductory component
10:15 - 10:41 a.m. EST
Benjamin Samelson-Jones, MD, PhD
Multiyear stable transgene factor IX levels after AAV hepatocyte-directed gene in neonatal and juvenile hemophilia B dogs
3:30 - 3:45 p.m. EST
Learn more here.
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Ashley Moore
Gene Therapy for Inherited Metabolic Disorders Program