GENE EDITING

Series supported by Intellia Therapeutics

Intellia’s Modular CRISPR/Cas9 Gene Editing Platform for the Treatment of Disease

Laura Sepp-Lorenzino, Ph.D., Executive Vice President, Chief Scientific Officer, Intellia Therapeutics

About Laura Sepp-Lorenzino

Laura Sepp-Lorenzino, Ph.D. oversees all platform and pipeline research activities at Intellia Therapeutics in her role as Chief Scientific Officer. Intellia’s leading CRISPR/Cas9-based platform supports a full-spectrum strategy, which deploys differentiated, modular solutions across in vivo and ex vivo therapeutic applications. Before joining Intellia, Laura was VP, Head of Nucleic Acid Therapies and member of the External Innovation team at Vertex Pharmaceuticals, with previous positions as VP and entrepreneur-in-residence at Alnylam Pharmaceuticals, Inc., and Executive Director RNA Therapeutics at Merck & Co., Inc. In addition to her extensive experience in nucleic acid therapies, Laura has expertise in oncology drug discovery and development acquired earlier in her career by leading the Cancer Research Department at Merck West Point and working as an assistant attending molecular biologist at Memorial Sloan-Kettering Cancer Center. She received her professional degree in Biochemistry from the University of Buenos Aires, Argentina and both her M.S. and Ph.D. in Biochemistry from New York University. Laura is a member of Taysha Gene Therapies’ board of directors and sits on the scientific advisory board of Thermo Fisher Scientific, Lodo Therapeutics, the U.K. Nucleic Acid Therapy Accelerator.

Safety considerations in therapeutic genome editing

Toni Cathomen, University of Freiburg

About Toni Cathomen

Toni Cathomen is Director of the Institute for Transfusion Medicine and Gene Therapy at the Medical Center of the University of Freiburg, Germany. His research activities focus on developing cell therapies based on induced pluripotent stem cells (iPSCs), improving the effectiveness and safety of designer nucleases (CRISPR-Cas and TALEN), and developing and manufacturing genome edited stem cell and immune cell preparations for the treatment of HIV infection, primary immunodeficiencies and cancer.

Repurposing of bacterial CRISPR-Cas immunity for genome editing

Virgis Siksnys, Vilnius University, Institute of Biotechnology

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About Virgis Siksnys

Virginijus Siksnys studied chemistry at Vilnius University and obtained his Ph.D from Moscow State University before returning to Vilnius where he moved through different research ranks at the Institute of Applied Enzymology/Institute of Biotechnology. Since 2002 he holds the position of Professor of Vilnius University and serves as Chief scientist at the Institute of Biotechnology. Dr. Siksnys has made a major and sustained contribution to the understanding of the structure and function of restriction enzymes. His research on the CRISPR-Cas has had a major impact on the field. His studies of the Cas9 protein paved the way for development of novel tools for genome editing applications. He is a member of Academia Europaea, EMBO, Lithuania Academy of Sciences and Norwegian Academy of Sciences and Letters. His work has been recently recognized with several awards and prizes.