New class of small molecules targets DNA damage repair and Huntington’s disease

Charles River researchers have developed a number of small molecules that target ATM kinase and have shown promise in preclinical studies for the treatment of Huntington’s disease

There is currently no cure for Huntington’s disease. But researchers like Philip Mitchell, a science director at Charles River who works with Diana Miszczuk, associate director in CNS Pharmacology at Charles River and oversees work on Huntington’s disease, are working hard to change that.

The Huntington’s disease research community is very focused on delaying the onset of the disease or slowing its progression by targeting pathological proteins using multiple approaches.

Philip Mitchell

Huntington’s disease therapeutic development has mainly focused on small molecules and antisense oligonucleotides (ASO). The latter are small strands of RNA or DNA that can be designed to target the HTT mRNA transcript and prevent the mutated protein from being made.

In collaboration with the Cure Huntington’s Disease Initiative (CHDI), a privately funded, nonprofit biomedical research organization devoted to developing therapeutics for Huntington’s disease, Charles River’s team recently published an article in the Journal of Medicinal Chemistry which the optimization of a new class of small molecule inhibitors that could ameliorate Huntington’s disease.

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