Clinical In Vivo Gene Therapy



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Children bear a disproportionate burden of genetic disease. Gene-based therapies are rapidly evolving to provide disease altering and potentially curative therapeutic options. The Clinical In Vivo Gene Therapy group (CIGT) is dedicated to developing and conducting adeno-associated virus gene transfer and molecular therapy trials at Children’s Hospital of Philadelphia. Through industry partnerships and investigator-initiated trials, CIGT seeks to further the development of transformative human therapies and to build upon CHOP’s legacy as a leader in gene therapy research.

Led by experts in in vivo gene therapy research, with more than 10 years of clinical trial experience, the CIGT team has a wealth of knowledge to navigate the complex safety, regulatory, and clinical care considerations of gene therapy clinical research. Our group offers resources to support the startup and conduct of industry-sponsored trials, as well as regulatory and clinical operations support for CHOP investigator-initiated clinical trials.

For CHOP investigators wishing to translate their own research into the clinic, CIGT can help facilitate continuity among the various groups involved in the translational process (e.g., the Clinical Vector Core or Cell and Gene Therapy Laboratory).