Design Therapeutics Snags $45M in Series A Round of Financing

Design Therapeutics obtained $45 million from Series A round of financing

Fremont, CA: Design Therapeutics raised $45 million in series A round of financing to push a treatment for Friedreich's ataxia into the clinic. The round was led by SR One, with the participation from Cormorant Asset Management. The firm will also use the proceeds of the funding to advance its discovery-stage programs in other degenerative disorders such as Fragile X syndrome and myotonic dystrophy.

"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 the disease," said Dr Shah. "Our company was founded to design 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."

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Joining Dr Shah and Dr. Ansari on the Board of Directors, are lead investor Simeon George, M.D., chief executive officer of SR One, and co-lead investor Stella Xu, Ph.D., managing director at Quan Capital, as well as Rodney Lappe, Ph.D., a seasoned R&D leader and the former chairman of Mirati Therapeutics. 

"Design Therapeutics has an opportunity to truly transform the way severe degenerative diseases are managed with their proprietary technology and disease-modifying approach to treatment," said Dr George. "We are excited to have the opportunity to work with Pratik and Aseem, two experienced drug developers who have helped deliver novel therapies to patients. Their pipeline is compelling, with encouraging preclinical data in Friedreich's ataxia. I believe their unique therapeutic approach to restoring natural gene expression holds tremendous potential, and we are excited to support their future."

The company's pipeline is extended by a novel program for Friedreich's ataxia, the most commonly occurring among hereditary in the U.S. It affects 1 in every 50,000 people. Friedreich's ataxia affects the nervous system resulting in progressive and life-altering movement problems and decreased life expectancy. Patients with Friedreich's ataxia have an expanded GAA repeat in the first intron of the FXN gene, which withholds transcription and limits production of the frataxin protein. Design Therapeutics has developed a new program that unblocks transcription, thereby restoring the natural production and function of frataxin. Design Therapeutic aims to conduct IND-enabling studies and initiate clinical development for its program to treat Friedreich's ataxia using the proceeds of this aforementioned investment round. 

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