LoQus23 Therapeutics is focused on developing oral small molecule therapies aimed at addressing the underlying mechanisms of neurodegenerative diseases, particularly Huntington's disease. The company’s research highlights the critical role of somatic expansion of the CAG nucleotide sequence in the Huntingtin gene, which leads to neurodegeneration. Unlike the inherited mutation, it is the further expansion of this sequence during a person's lifetime that drives disease progression. This process is mediated by aberrant DNA mismatch repair, which has been identified as a primary disease driver. The toxic huntingtin protein produced as a result of exceeding a certain expansion threshold contributes to neuronal dysfunction and cell death, leading to the characteristic symptoms of Huntington's disease.

To combat this, LoQus23 is targeting the MutSβ heterodimer protein, a key player in the mismatch repair pathway, as a potential drug target to inhibit somatic expansion. The company employs a structure-based approach to discover allosteric inhibitors of MutSβ, aiming to halt DNA instability and slow the progression of Huntington's disease and other related conditions. By focusing on oral small molecule drugs, LoQus23 aims to provide a more convenient treatment option for patients, leveraging the established efficacy of such therapies in managing complex brain diseases.

United Kingdom

LoQus23 Therapeutics is a company that targets DNA mismatch repair pathways to slow neurodegeneration in Huntington's Disease and other triplet repeat disorders. Recognizing the urgent need to find a treatment for patients with triplet repeat expansion diseases, LoQus23 Therapeutics was co-founded in 2019 by David.

LoQus23 Therapeutics

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