Realizing the Promise of CRISPR Therapeutics

ABSTRACT

Intellia Therapeutics is developing novel, potentially curative therapeutics leveraging CRISPR-based technologies. Our in vivo programs use IV-administered CRISPR as the therapy, in which proprietary lipid nanoparticle delivery technology enables precise editing of disease-causing genes directly within specific target tissues. Intellia’s ex vivo programs use CRISPR to engineer human T cells to treat cancer and autoimmune diseases. Preclinical and clinical data will be discussed.

BIOGRAPHY

Laura Sepp-Lorenzino, Ph.D. is Chief Scientific Officer and Executive Vice President at Intellia Therapeutics, a leading clinical-stage genome editing company developing novel, potentially curative medicines leveraging CRISPR-based technologies. 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 the board of directors of Taysha Gene Therapies and the Alliance for Regenerative Medicine, and sits on the scientific advisory boards of Thermo Fisher Scientific, the U.K. Nucleic Acid Therapy Accelerator, and Arsenal Capital Partners. Laura is a member of the Editorial Board of Nucleic Acid Therapeutics and Molecular Therapy Nucleic Acids.

The Stivala Lectures in Chemistry invites an outstanding scientist for a day of lecture and discussion on timely topics in chemistry. Dr. James Cooper established this lecture series in memory of his father Charles Cooper, who was a close friend of Prof. Salvatore Stivala. Prof. Stivala served the Stevens community as a faculty member from 1953 to 1994.