RARE Daily

Design Therapeutics Launches with $45 Million to Develop Treatments for Nucleotide Repeat Disorders

March 20, 2020

Rare Daily Staff

Design Therapeutics has launched with a $45 million series A financing to create and develop a new class of therapies for patients with serious degenerative disorders caused by nucleotide repeat expansions.

SR One led the round, which included investment from Cormorant Asset Management, Quan Capital and WestRiver Group.

The funding will be used to advance Design’s 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 was co-founded by Pratik Shah and Aseem Ansari. Shah serves as chairman of the board at ARS Pharmaceuticals, and prior to that, until its acquisition by Sanofi, was chairman of Synthorx. Ansari currently serves as chair of the Department of Chemical Biology and Therapeutics at St. Jude Children’s Research Hospital. Previously, Ansari was a faculty member at the University of Wisconsin, where during his tenure, he helped execute the license agreement with the Wisconsin Alumni Research Foundation.

Design Therapeutics in-licensed its technology from WARF, and aims to take a unique approach to treatment and provide hope for patients with degenerative disorders. Its pipeline is led by a novel program for Friedreich’s ataxia, the most common form of hereditary ataxia in the United States, affecting about 1 in every 50,000 people. Friedreich’s ataxia, affects the nervous system resulting in progressive and life-altering movement problems and shortened life expectancy. Patients with Friedreich’s ataxia have an expanded GAA repeat in the first intron of the FXN gene, which blocks transcription and limits production of the frataxin protein.

Design Therapeutics has developed a novel program that unblocks transcription, thereby restoring the natural production and function of frataxin. With the use of proceeds from the financing, the company intends to conduct IND-enabling studies and initiate clinical development for its program for Friedreich’s ataxia.

“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 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.”


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