Children’s Hospital of Philadelphia announced on May 12 that its physicians will present new and updated data at the American Society of Gene and Cell Therapy annual conference in Boston, Massachusetts, taking place from May 11 to May 15. More than 30 presentations are scheduled, offering insight into the hospital’s approach to gene-based treatment strategies.
The event is significant as it highlights ongoing advancements in gene therapy research. Several CHOP researchers will also be recognized with awards for their contributions to the field.
Beverly Davidson, PhD, Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics and Chief Scientific Strategy Officer at CHOP, will receive the Outstanding Achievement Award on Thursday, May 14. The award recognizes an ASGCT member who has achieved pioneering research success or made significant contributions over a career. 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 will be presented on Tuesday, May 12, recognizing transformative work that accelerates cell and gene therapies from discovery to clinical application. This year’s recipients include Rebecca Ahrens-Nicklas, MD, PhD; Danaher Corporation; Kiran Musunuru, MD, PhD; and Fyodor Urnov, PhD. The award honors their collaboration leading to “the first personalized gene editing therapy.”
Highlighted presentations by CHOP experts during the conference include sessions led by William Peranteau on germline editing techniques; Denise Sabatino on mechanisms regulating AAV transgene expression; Lindsey George on understanding AAV toxicities; Benjamin Samelson-Jones co-chairing a session about recent toxicities in AAV gene therapies; Rebecca Ahrens-Nicklas and Kiran Musunuru discussing clinical translation of somatic genome editing as a therapeutic platform; Peter Kurre presenting positive clonal selection of curative HSCs; and Samelson-Jones sharing results from multiyear studies in hemophilia B dogs.
The participation underscores CHOP’s continued involvement in advancing genetic medicine through both research innovation and collaborative efforts.
