Laura Sepp-Lorenzino, Ph.D.
Executive Vice President, Chief Scientific Officer
Laura Sepp-Lorenzino oversees all platform and pipeline research activities across in vivoMeaning “within the living”, this type of therapy is administered directly into the patient, targeting the cells and editing the genome from inside the body. and ex vivoAlso referred to as a cell therapy. In an ex vivo therapy, cells are removed from the body for modification. Modification is done by administering therapy directly to the cells before they are returned to the body. In the case of ex vivo CRISPR/Cas9 therapies, CRISPR/Cas9 is used to modify the extracted cells to repair them back to their proper function or add desired functions. The engineered cells are then administered to the patient so they can treat a particular disease. (engineered cell therapyType of therapy where engineered cells are transferred into a patient’s body to grow, replace or repair damaged tissue, or perform another desired function. Cells used in these therapies may originate from the patient (autologous cells) or from a donor (allogeneic cells). A common type of cell therapy is blood transfusions, where red blood cells, white blood cells and platelets from one or more donors are transferred into the body of a patient.) areas as Intellia’s Chief Scientific Officer. Before joining Intellia, she was vice president, Head of Nucleic Acid Therapies, Research, and member of the External Innovation team at Vertex Pharmaceuticals, Inc. She also served as vice president, entrepreneur-in-residence at Alnylam Pharmaceuticals, Inc., a leader in the development of RNAi Therapeutics. At Alnylam, she was responsible for the Hepatic Infectious Disease Strategic Therapeutic Area, championed extra hepatic siRNA delivery, and was active in licensing and partnering.
Laura spent 14 years at Merck & Co., most recently having served as executive director and department head, RNA Therapeutics Discovery Biology. In this role, she was responsible for identification and optimization of siRNAs and delivery vehicles, advancement of preclinical candidates, and development of an siRNA-conjugate platform to expand the repertoire of tissues accessible to in vivo siRNA delivery. Laura also 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 lab member and 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 holds professional affiliations with key scientific organizations, including the Oligonucleotide Therapeutics Society, the American Society for Gene and Cell Therapy, the European Society of Gene and Cell Therapy, and the New York Academy of Sciences, as well as a number of oncology societies. She also sits on the scientific advisory board of Thermo Fisher Scientific, Lodo Therapeutics, the U.K. Nucleic Acid Therapy Accelerator and is a member of Taysha Gene Therapies’ board of directors.