Genetic Engineering and Biotechnology News

Adeno-associated virus
Credit: Thom Leach / Science Photo Library / Getty Images

Translating the Promise of AAVs: From Discovery to Delivery

Credit: Thom Leach / Science Photo Library / Getty Images

Panelists:

Image of Lindsey A. George, MD

Lindsey A. George, MD

Assistant Professor of Pediatrics,
The Perelman School of Medicine
University of Pennsylvania
Director, Clinical In Vivo Gene Therapy
Children’s Hospital of Philadelphia

Panelist

Image of Lindsey A. George, MD

Lindsey A. George, MD

Dr. George is a physician-scientist whose clinical expertise is in disorders of hemostasis and thrombosis with a particular interest in hemophilia and hemophilia gene therapy. Her basic science laboratory studies the molecular basis of coagulation that in diminished or excess functional states leads to disorders of hemostasis and thrombosis, respectively. The current focus of the George lab is to merge mechanistic studies aimed at understanding the regulation of factor VIII cofactor function with translational efforts in hemophilia A gene therapy. Her group is additionally interested in understanding the mechanistic basis of questions that have emerged from current hemophilia gene therapy clinical trials as well as general studies of adeno-associated viral vectors (AAV). Dr. George was previously the lead clinical principal investigator of multiple early phase hemophilia A and B adeno-associated virus-mediated gene addition trials. In addition to her clinical practice and laboratory, she directs the Clinical In Vivo Gene Therapy at the Children’s Hospital of Philadelphia that long-term aims to safely and efficiently advance translational and clinical research for in vivo gene therapy for children with genetic disorders.
Image of Steven Gray, PhD

Steven Gray, PhD

Professor, Department of Pediatrics
Co-Director, Gene Therapy Program
Director, Viral Vector Facility
University of Texas Southwestern Medical Center

Panelist

Image of Steven Gray, PhD

Steven Gray, PhD

Dr. Steven Gray is a Professor of Pediatrics at the University of Texas Southwestern Medical Center, where he co-directs the Gene Therapy Program and leads the Viral Vector Facility. An expert in AAV gene therapy vector engineering and nervous system gene delivery, his research has helped advance gene therapies for neurological disorders including Rett syndrome, Tay-Sachs disease, Batten disease, and Giant Axonal Neuropathy. Dr. Gray has authored more than 90 peer-reviewed publications, holds over 20 patents, and has contributed to multiple approved and ongoing clinical trials. His work has earned numerous honors, including the American Society of Gene and Cell Therapy’s Outstanding Young Investigator Award.

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Adeno-associated viruses (AAVs) have emerged as one of the most promising platforms for in vivo gene delivery. Ongoing innovation in vector engineering, delivery, and clinical translation is expanding the therapeutic potential of AAV-based approaches across a range of genetic disorders.

This episode of GEN Live will explore the rapidly evolving field of AAV gene therapy. Leaders from clinical and translational research will discuss current advances and challenges in AAV vector development, delivery, safety, and long-term therapeutic efficacy.

The session will provide a broad overview of the current state of the field and foster discussion to define the next era of gene therapy. We will also take questions from the audience, so please bring your questions on AAVs for our panelists as well.

Produced with support from:

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GEN LiveMultimediaAdeno-associated virusGene therapy (Therapeutics)Genetic diseases