Precision Genome Engineering

Gene editing is revolutionizing biology and therapeutics. An ever-expanding toolbox of editing enzymes and delivery solutions enables the precise manipulation of the genome and it is fueling a diverse pipeline of clinical candidates. Emerging clinical data from gene-edited cell therapies and preclinical data from in vivo delivered gene-editing drug candidates are demonstrating the feasibility, versatility, and potential for efficacy and safety in humans. Furthermore, there is great interest and investment in gene editing for crops and livestock. The Symposium will cover most aspects of the genome-editing field: from fundamental biochemistry to tool development, to applications in biology, agriculture, and medicine. The meeting will bring together scientists from academia and biopharma, including regulators and ethicists. It will also uniquely bring together scientists who have studied natural genome editing systems and natural DNA damage repair with scientists trying to develop novel genome editing systems. Significance: Genome editing is a powerful and precise tool to change the sequence of cellular DNA for both a better understanding of human disease and the treatment of human disease. The basic understanding of how genome editing works, the development of new tools in genome editing, and the translation to the clinic are changing on a rapid basis. There is broad interest in genome editing from basic scientists to foundations to industry.

The specific aims of this meeting are:

• Provide an update on the state of understanding of the biochemistry of genome editing
• Relate genome editing to natural genome editing and DNA damage repair processes
• Provide an update on the latest tools in translational genome editing
• Provide an update on pre-clinical work using genome editing to provide better treatments for patients.
• Provide an update on the clinical trial results using genome editing in humans for both ex vivo and in vivo uses.
• Have an open discussion on the ethics of human genome editing. The outcome of this meeting is increased communication between different experts to enhance the safety and efficacy of genome editing for therapeutic use. The meeting will bring together expertise that is different than many similar meetings.

Sponsored by Ultragenyx Pharmaceutical Inc.

Organizers: Laura Sepp-Lorenzino and Matthew Porteus