There is no treatment for inherited degenerative disorders like Friedreich's ataxia, so care focuses on relieving symptoms. With $45 million and technology out of the University of Wisconsin, Design Therapeutics is trying to change that.
The company uncloaked Friday with $45 million in series A funding to push a treatment for Friedreich's ataxia into the clinic. It will also advance its discovery-stage programs in other degenerative disorders such as Fragile X syndrome and myotonic dystrophy.
San Diego-based Design Therapeutics is taking aim at nucleotide repeat disorders, where a mutation increases the number of times a DNA sequence is repeated. These mutations cause disease in multiple ways—the affected gene may produce an abnormal protein, or it may not produce enough of a required protein, as is the case with Friedreich's ataxia.
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Most patients with Friedreich's ataxia have an expanded GAA repeat in their FXN gene that blocks transcription, resulting in limited production of the frataxin protein. Design’s lead program aims to unblock transcription. The series A funding, from SR One, Cormorant Asset Management, Quan Capital and West River Group, will bankroll IND-enabling studies for the program.
“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 Pratik Shah, Ph.D., Design co-founder and executive chairman, in a statement. “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.”
Triplet Therapeutics, which launched in December, is taking a different tack. It’s looking at a specific subset of repeat expansion disorders called triplet repeat disorders, so called because they result from repeats of three nucleotide bases, or letters. Rather than developing drugs on a disease-by-disease basis, it is doing so on a tissue-by-tissue basis, so it could potentially treat multiple diseases with the same drug.