Biotechnology company LoQus23 Therapeutics has nominated its drug candidate LQT-23 for development as a treatment for Huntington’s Disease (HD). 

LQT-23 is a first-in-class oral drug candidate which has shown potential to slow or stop the progression of HD. The candidate works as an allosteric small molecule inhibitor of MSH3/MutSβ, proving to be a unique mechanism of action.  

DNA mismatch repair has been shown as a key driver of HD and other triplet repeat diseases. Convergent evidence from human genetics, post-mortem studies, and mechanistic research identifies MSH3/MutSβ as the most promising target of the DNA mismatch repair pathway. However, the protein is a highly challenging target for small molecule therapeutic intervention. 

It is believed LQT-23 could be a groundbreaking advancement in MutSβ modulation for the treatment of HD, offering the most advanced and potent allosteric small molecule inhibitor of MutSβ to date. 

“The nomination of LQT-23 as our development candidate is a significant moment for LoQus23 and reaffirms our strategy of advancing our pipeline of MSH3/MutSβ inhibitors to transform the lives of patients,” said Dr David Reynolds, Chief Executive Officer of LoQus23 Therapeutics. 

“I want to salute the dedication of our team which has worked tirelessly over many years to identify this candidate for such a difficult target, and we look forward to advancing it to IND/CTA filing later this year.” 

“MSH3/MutSβ is the most promising and best validated target for HD, but has been difficult to drug,” added Dr Cyrus Mozayeni, Chair of the Board of Directors of LoQus23.  

“The team has made exceptional progress since the financing in 2024, and the nomination of LQT-23 as our lead development candidate underscores our deep domain expertise, while positioning LoQus23 at the forefront of emerging therapies for the treatment of HD.” 

Pre-clinical development of LQT-23 will continue in 2026. 

Bruno Quinney, Content Team, DDW