By targeting a dysfunctional DNA repair system, inhibitors could slow or halt the progression of Huntington’s disease. Image: Shutterstock/ Andrii Yalanskyi.

LoQus23 closed a round of Series A financing to advance development of its lead candidate for Huntington’s Disease.

The Cambridge, UK-based biotechnology company announced in a 2 October release it had raised £35m ($43m) in this latest round, led by venture capital firm Forbion. The proceeds will advance preclinical and clinical research of LoQus23’s foremost asset, its small molecule MutSβ inhibitor.

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Along with Forbion were prior investors Novartis Venture Fund and SV Health Investors’ Dementia Discovery Fund. In addition, Forbion General Partner Rogier Rooswinkel will join the LoQus23 board of directors.

LoQus23’s MutSβ inhibitor is set to enter clinical development in 2026. The allosteric small molecule targets MutSβ, a protein component of the mismatch repair system (MMR) strongly implicated as the central cause of Huntington’s Disease.

The MMR system, which when functioning properly resolves errors in DNA structure, is known to drive disease pathophysiology by increasing CAG nucleotide repeats within the Huntingtin gene. Once these repeats reach a critical threshold, patients’ symptoms of neuronal dysfunction progress and ultimately lead to death. LoQus23 aims to slow or halt this progression by inhibiting the MMR system.

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The company has established a series of small molecule inhibitors targeting MutSβ and MutSα, another MMR component. The proteins are thought to be relevant in up to 30 triplet-repeat diseases. Aside from Huntington’s Disease, these inhibitors are being investigated to treat myotonic dystrophy and broader genetic disorders.

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In 2019, LoQus23 raised $5.9m in seed capital to advance its candidates, then a further $9.4m in 2021. In 2022, the company entered into a research collaboration with the Chinese biotech HitGen to screen for small molecule candidates using its DNA-encoded library platform. HitGen has also partnered with Pfizer, Boehringer Ingelheim, and Cancer Research UK among others to leverage its high throughput screening capabilities.

Huntington’s Disease affects roughly 30,000 patients in the US alone, for whom there is not yet any disease-modifying treatment available. Rogier Rooswinkel, the newly appointed LoQus23 board member, said that for these patients, “MutSβ seems the most promising and best validated target, with the potential to bring disease progression to a halt.”

Dr David Reynolds, LoQus23 CEO, said, “This financing will enable us to develop key clinical data to support the development of our exciting lead programme.” He also emphasised the company’s focus on developing an oral formulation for the therapy to increase patient adherence and convenience.