Experiment in Genome Editing Inside a Living Person Occurred on Monday, November 13, 2017

44-year-old Brian Madeux, who has MPS II (Hunter syndrome), was the first patient to undergo an experiment in genome editing inside a living person on Nov. 13, 2017 in Oakland, California using Sangamo Therapeutics, Inc.‘s zinc finger nuclease (ZFN) genome editing technology. The procedure was performed at UCSF Benioff Children’s Hospital Oakland. If successful, this genome editing will not repair Brian Madeux’s existing damage from MPS II, but he hopes it will end his need for weekly enzyme replacement therapy infusions.

This is a Phase 1/2 clinical trial (“the CHAMPIONS study”) evaluating SB-913, an investigational in vivo genome editing therapy. The CHAMPIONS study is an open-label clinical study designed to assess the safety, tolerability and preliminary efficacy of the SB-913 investigational genome editing therapy in up to nine adult males with MPS II. Other CHAMPIONS study sites include Minneapolis, Chapel Hill, Chicago and Philadelphia. Currently at these study sites, prospective study participants are being screened. In Minneapolis at the University of Minnesota, the Lysosomal Disease Network’s principal investigator Dr. Chester B. Whitley is conducting the CHAMPIONS study.

Two additional clinical trials are now underway in the United States to evaluate Sangamo’s in vivo genome editing therapeutics for hemophilia B and MPS I (Hurler or Hurler-Scheie syndrome). The Lysosomal Disease Network’s principal investigator Dr. Chester B. Whitley is also conducting the MPS I genome editing study at the University of Minnesota. Other sites in this MPS I study include Oakland, New York City and Cincinnati. All three trials use ZFNs designed to edit liver cells at the same location in the albumin gene, but they differ in delivering the corrective gene relevant to the respective disease. All three of Sangamo’s in vivo genome editing product candidates have received Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration. Additionally, SB-318 for MPS I and SB-913 for MPS II have received Rare Pediatric Disease designations from the FDA.

Consensus Conference to Convene for Cognitive Endpoints in MPS I, MPS II and MPS III

Elsa Shapiro, PhD, neuropsychologist and expert in rare pediatric diseases; Mark Dant, president and CEO of the National MPS Society (USA); and Christine Lavery, executive director of the Society for Mucopolysaccharide Diseases in the UK, have planned a program to bring experts together from around the world to establish a consensus for cognitive endpoints in MPS I, MPS II and MPS III. Such endpoints are absolutely essential for conducting clinical trials of potential treatments. The consensus conference will be held December 1-3, 2016 in London.

With support from 18 pharmaceutical companies, more than 80 doctors and psychologists from countries around the world will attend, including China, Egypt, Australia and Japan. The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are sending representatives who will present. The consensus proceedings and the selected consensus panel comprised of experts in MPS, statistics and psychological assessments will be facilitated by J.H. van der Lee, MD, PhD, of the Academic Medical Center, Amsterdam, Netherlands. Participants also include the Lysosomal Disease Network’s Dr. Chester Whitley, Dr. Igor Nestrasil and Dr. Julie Eisengart, all of the University of Minnesota. A paper will be published documenting the outcome.