Design Therapeutics has launched with $45 million in funding to advance its lead candidate for the treatment of Friedreich’s ataxia into clinical development, and support programs for other nucleotide repeat disorders such as fragile X syndrome, and myotonic dystrophy.
These genetic diseases are caused by excessive repeats of nucleotides — the building blocks of DNA — in certain genes.
Depending on where they are located, these repeats may significantly change a gene’s instructions to produce a specific protein. This may impair protein production or result in an abnormal, toxic product, contributing to the development of neurodegenerative and neuromuscular diseases.
In Friedreich’s ataxia, excessive repeats of three nucleotides (called GAA triplets — G for guanine and A for adenine) within the FXN gene significantly decrease the production of frataxin, an essential protein for the normal functioning of mitochondria, which are cells’ powerhouses. This leads to mitochondrial dysfunction and impaired energy production in several organs.
“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,” Pratik Shah, PhD, Design Therapeutics’ co-founder, said in a press release.
Aseem Ansari, PhD, Design’s other co-founder and also a member of the board of directors, said “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.”
Design in-licensed its technology from the Wisconsin Alumni Research Foundation (WARF) and intends to take a unique disease-modifying approach to treat nucleotide repeat disorders.
The company’s lead therapeutic candidate works by unblocking RNA generation from the FXN gene, which potentially could restore frataxin levels and prevent further damage in people with Friedreich’s ataxia.
With these funds, Design plans to conduct studies that enable the start of clinical trials in its Friedreich’s ataxia program.
“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 Simeon George, MD, Design’s lead investor and member of the board of directors, and CEO of SR One.
“We are excited to have the opportunity to work with Pratik and Aseem, two experienced [therapy] developers who have helped deliver novel therapies to patients. Their pipeline is compelling, with encouraging preclinical data in Friedreich’s ataxia,” George said. “I believe their unique therapeutic approach to restoring natural gene expression holds tremendous potential, and we are excited to support their future.”
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