Rgenta Therapeutics Announces Formation of Repeat Expansion Diseases Focused Scientific Advisory Board

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Jun 21, 2023, 08:30 ET

CAMBRIDGE, Mass., June 21, 2023 /PRNewswire/ -- Rgenta Therapeutics today announced the formation of a Repeat Expansion Diseases (REDs) Focused Scientific Advisory Board (REDs SAB). REDs SAB is an esteemed group of preclinical and clinical experts who contribute to basic and translational research on the mechanisms and treatments for repeat expansion diseases.

Repeat expansion diseases are a large class of diseases caused by the occurrence of expanded repeats in genes and include many devastating neurological and neuromuscular diseases such as Huntington's disease, fragile X syndrome, Friedreich's ataxia, myotonic dystrophy, amyotrophic lateral sclerosis that currently do not have effective disease modifying treatments. Leveraging an integrated RNA-targeting platform, Rgenta is dedicated to developing novel small molecule-based therapies that target the mismatch repair pathway underlying the expansion of nucleotide repeats in REDs.

"We are proud and honored to introduce our REDs focused SAB," said Dr Zhiping Weng, Rgenta SAB chair. "We can't thank these outstanding advisors enough for committing their time and expertise to advance the science with us."

"We are excited and privileged to have the opportunity to work with this group of leading researchers and clinicians in the repeat expansion disease fields," said Dr. Travis Wager, co-founder, President & CSO Rgenta. "Their knowledge and insights to the repeat expansion diseases will help advance the preclinical and clinical development of our small molecule programs."

The REDs SAB is composed of the following members:

Dr. Sarah Tabrizi is an award-winning scientist who has published over 300 peer-reviewed publications, been elected a fellow of the UK Academy of Medical Sciences, co-founded the UCL Huntington's Disease Centre, and helped set up the UK All-Party Parliamentary Group for Huntington's disease. Her complementary research strategies - basic science focusing on cellular mechanisms of neurodegeneration, and a program to translate those findings into treatments and cures - have led to the clinical development of a novel 'gene silencing' treatment for Huntington's disease. Sarah was presented with the Yahr Award, Alexander Morison Medal in 2019, and MRC Millennium Medal and Osler Medal in 2022 for her contributions to research.

Dr. Darren Monckton is currently Professor of Human Genetics and leads an active research team investigating the basis and consequences of genetic instability in human disease. He is internationally recognized for his work in defining the role of DNA repair and somatic expansion of the CTG•CAG as a major driver of pathology in myotonic dystrophy and Huntington's disease and as a novel therapeutic target. He is a scientific advisor to the Myotonic Dystrophy Support Group (UK), the European Dystrophia Myotonica Association, and the Myotonic Dystrophy Foundation (USA), and was the Muscular Dystrophy UK 2017 Scientist of the Year and received the 2019 Huntington Disease Society of America Research Award.

Dr. Nicholas Johnson is an associate professor and vice chair of research in the Department of Neurology at Virginia Commonwealth University with a focus on inherited neuromuscular disorders. His laboratory is focused on identifying the pathogenesis of myotonic dystrophy, the limb girdle muscular dystrophies, and facioscapulohumeral muscular dystrophy and identifying appropriate clinical endpoints for these conditions. Johnson conducts therapeutic trials in many other inherited nerve and muscle disorders.

Dr. Richard H. Myers is Emeritus Professor of Neurology at the Chobanian and Avedisian School of Medicine at Boston University. Dr. Myers has had a long-standing interest in Huntington's disease and has participated in a wide range of research investigations into this disease. He has been a member of the New England Huntington's Disease "Center Without Walls" since its inception in 1980. His HD studies are best characterized as 'Neurobiological Studies' in that they include studies into the mechanisms of disease expression, including complex genetic modifier studies and a series of neuropathological studies of effects of disease expression in the brain. Dr. Myers has published more than 300 peer-reviewed articles, 38 individual NIH-funded grants.

About Rgenta Therapeutics
Rgenta Therapeutics is developing a pipeline of oral, small-molecule RNA-targeting medicines with an initial focus on oncology and neurological disorders. Our proprietary platform mines the massive genomics data to identify targetable RNA processing events and design small-molecule glues to modulate the interactions among the spliceosome, regulatory proteins, and RNAs. Our lead programs and unique approach are unlocking the therapeutic potential of historically undruggable targets in human diseases. Learn more at http://www.rgentatx.com

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