Date: 25-Mar-2020

Design Therapeutics Launches With $45 Million To Develop A New Class Of Disease-Modifying Therapies For Serious Degenerative Disorders

Design Therapeutics announced today that it is launching to create and develop a new class of therapies for patients with serious degenerative disorders caused by nucleotide repeat expansions. The company has closed a $45 million Series A financing led by SR One, with participation from Cormorant Asset Management, Quan Capital and WestRiver Group, to advance its lead therapeutic candidate into clinical development for the treatment of Friedreich’s ataxia, and support advancement of its discovery programs for multiple other degenerative diseases, including fragile X syndrome and myotonic dystrophy.

Nucleotide repeat disorders are a set of genetic disorders caused by repeat expansion in certain genes. Depending on where it is located, the repeat may cause a change in the regulation of gene expression or produce a toxic gene product, all of which are recognized as significant contributors to degenerative diseases. Design Therapeutics in-licensed its technology from the Wisconsin Alumni Research Foundation (WARF), and aims to take a unique approach to treatment and provide hope for patients with degenerative disorders.

Design Therapeutics was co-founded by Pratik Shah, Ph.D., and Aseem Ansari, Ph.D. Dr. Shah serves as chairman of the board at ARS Pharmaceuticals, and prior to that, until its acquisition by Sanofi, was chairman of Synthorx. Earlier, Dr. Shah was president and chief executive officer of Auspex Pharmaceuticals until its acquisition by Teva in 2015. Dr. Ansari currently serves as chair of the Department of Chemical Biology and Therapeutics at St. Jude Children’s Research Hospital. Previously, Dr. Ansari was a faculty member at the University of Wisconsin, where during his tenure, he helped execute the license agreement with WARF. Design Therapeutics’ leadership team also includes Sean Jeffries, Ph.D., chief business officer, formerly with the Boston Consulting Group.

“Significant industry advancements have led to the understanding of root causes of multiple nucleotide repeat disorders, however, there remain few to no therapeutic options that slow the progression or reverse the course of disease,” said Dr. Shah. “Our company was founded with a goal of designing a new class of small molecule therapies that address the core etiology of diseases to deliver a biological effect typically only seen with complex molecules. The collective insights of our team, along with our financial strength, position us to transition into a clinical-stage company to explore the corrective benefit of our programs