EdiGene to Present Data on its Lead RNA Base Editing Program to Treat Patients with the Most Severe Form of MPS I

EdiGene, Inc., which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, will present data on its lead therapeutic program for patients with the most severe form of mucopolysaccharidosis type I (MPS I), known as Hurler syndrome, at the 23rd American Society of Gene & Cell Therapy (ASGCT) Annual Meeting on Tuesday, May 12, 2020 at 10:15 a.m. EDT.

MPS I is a rare and potentially fatal lysosomal storage disorder that can affect organs and tissue throughout the body. It is a genetic condition that results in a deficiency of the enzyme ?-L-iduronidase (IDUA). As a result, metabolic waste accumulates in cells and cause progressive damage. There is no cure for MPS I. Patients today are either treated with a bone marrow transplant or enzyme replacement therapy.

Over 40% of the patients with Hurler syndrome, are caused by presence of W402X (c1205 G ->A point mutation) in IDUA. EdiGene's therapeutic program for these patients uses LEAPERtm (Leveraging endogenous ADAR for programmable editing of RNA), a novel RNA base editing technology. Oligonucleotides of various design, called arRNA, were developed to produce precise and sequence specific A-to-I conversion in the mRNA sequence for codon 402, leading to generation of the wild type IDUA mRNA and protein. Data showed significant IDUA enzymatic activity with long duration in MPS I patient-derived cells.

"Even with the current standard of care, unmet needs remain for patients with Hurler Syndrome," said Dong Wei, Ph.D., CEO of EdiGene, "The pre-clinical data suggest encouraging progress for our program, and we hope to further develop it into a potential treatment for these patients."

LEAPERtm is developed by Professor Wensheng Wei's lab at Peking University. Professor Wei, who is also the founder of EdiGene, commented: "LEAPERtm is a robust, precise and efficient editing technology with versatility across multiple delivery platforms. It is uniquely positioned for in vivo genome editing treatment for selected diseases and we will dedicate ourselves to turning this technology into therapies for patients in need."

Details of the presentation:

Title: A Novel Oligonucleotide-Based RNA Base Editing Therapeutic Approach for the Treatment of Hurler Syndrome

Date: 10:15 a.m., Tuesday, May 12, 2020, EDT

Session Name: Oligonucleotide Therapies for Genetic Diseases

About EdiGene, Inc.

EdiGene is a biotechnology company focused on leveraging the cutting-edge genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of genetic diseases and cancer. The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. Founded in 2015, EdiGene is headquartered in Beijing, with subsidiaries in Guangzhou, China and Cambridge, Massachusetts, USA. More information can be found at www.edigene.com.