Design Therapeutics is pioneering novel small-molecule therapeutic candidates, called GeneTAC™ molecules (Gene Targeted Chimera), that are designed to be disease-modifying and target the underlying cause of inherited nucleotide repeat expansion diseases.
Our GeneTAC™ molecules are designed to selectively bind to genetic repeat sequences, modulate gene expression either by restoring or blocking transcription, and restore cellular health. As a platform, we believe that GeneTAC™ molecules have broad potential applicability across monogenic nucleotide repeat expansion diseases.
Board of Directors
Founders
Pratik Shah, Ph.D.
Chairperson and CEO
Pratik Shah, Ph.D., is co-founder, chairperson and chief executive officer of Design Therapeutics. In addition, Dr. Shah serves as chairperson of the board at ARS Pharmaceuticals. Until February of 2020, he served as chairperson of Synthorx, Inc., a biotechnology company developing innovative protein therapeutics for cancer and autoimmune disorders that was acquired by Sanofi for $2.5 billion. Before that, Dr. Shah was president and chief executive officer of Auspex Pharmaceuticals, a biopharmaceutical company focused on the development and commercialization of novel medicines for people with movement disorders, including AUSTEDO® (deutetrabenazine). He became involved in Auspex as an investor in 2007 and served as its chairperson until he took over as its chief executive officer in 2013, after which he took the company public and oversaw its growth until its acquisition by Teva Pharmaceuticals for $3.5 billion in 2015. Before that, Dr. Shah was a partner in the healthcare venture capital firm Thomas, McNerney & Partners for nearly a decade, and prior to that, he co-founded two biotechnology companies and was also a consultant at McKinsey & Company in San Francisco. Dr. Shah obtained his Ph.D. in biochemistry and molecular biology as well as his MBA in finance from the University of Chicago.
Sean Jeffries, Ph.D.
Chief Operating Officer
Sean Jeffries, Ph.D., is the chief operating officer at Design Therapeutics. Dr. Jeffries brings over twenty years of experience in biopharma with extensive experience in strategy and R&D. Prior to Design, Dr. Jeffries led projects to develop R&D and therapeutic strategies for biopharma at the Boston Consulting Group. He was instrumental in pioneering miniPCR, which performed the first PCR on the international space station. Prior to that, Dr. Jeffries established and led the informatics group at Kalypsys. Dr. Jeffries completed his Ph.D. at the University of Cambridge in epigenetics as an NIH-Cambridge scholar.