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Cell and gene therapies have offered hope to patients with genetic abnormalities (e.g., rare diseases) or long-term disorders such as cancer. Innovative approaches for correcting sequences via editing or replacement at the genome level have changed the ways that genetic payloads are delivered to target tissues or organs. For gene therapies to be safe and efficacious, they must be delivered to the right cell type, evade immune responses, and correct the disorder they are designed for. To date, the most successful delivery vehicles are based on adeno-associated viruses (AAVs).

In this cell and gene therapy webinar, our distinguished speakers, Dr. Dominik Fischer and Dr. Michelle Fraser, will discuss recent advances and novel strategies for genetic modulation and payload delivery systems. In his talk, Dr. Fischer will discuss the potential and limitations of adeno-associated virus-based delivery of genetic therapies in the eye. His talk will sketch out the ideal payload and delivery system for ocular therapies and contrast them to AAVs. In her presentation, Dr. Fraser will share current and future trends in the development and use of genetic payloads. She will discuss some of the exciting advances in gene editing platforms and how these enable more complex editing tasks while reducing the risk of unintended genomic rearrangements.

A live Q&A session followed the presentation, offering a chance to pose questions to our expert panelists.

Dominik Fischer
Dominik Fischer, MD, PhD
Ophthalmic Surgeon at the
Oxford Eye Hospital
Professor of Ophthalmology
University of Oxford
Michelle Fraser
Michelle Fraser, PhD
Head, Cell & Gene Therapy Business Unit