LoQus23 Therapeutics raises £35m to fight Huntington’s disease

Based at Babraham Research Campus, LoQus23 is a private company investigating small molecule drugs that could stop DNA instability and slow neurodegeneration in Huntington’s, myotonic dystrophy type 1 and similar triplet repeat expansion diseases.

The Series A round was led by Dutch VC Forbion alongside existing investors SV Health Investors’ Dementia Discovery Fund (DDF) and Novartis Venture Fund (NVF). Forbion General Partner Rogier Rooswinkel will join LoQus23’s Board.

LoQus23 was founded in 2019 by Entrepreneurs in Residence at DDF Dr David Reynolds, Dr Caroline Benn, and Dr Ruth McKernan. It received additional seed funding from NVF in 2021.

The company has since established a platform of assays and a small molecule series of MutSα and MutSβ inhibitors which are therapeutically relevant in up to 30 triplet repeat diseases, including Huntington’s.

The Mismatch Repair branches of the DNA Damage Repair system repair DNA insertions, deletions and misincorporation errors during transcription and/or cellular replication, with two main pathways dependent upon the size of mismatch – MutSα for small mismatches and MutSβ for larger insertion/deletion loops.

Huntington’s is an autosomal dominant neurodegenerative disorder for which there is currently no disease modifying treatment available and which has 30,000 patients in the US alone.

By targeting somatic expansion, LoQus23 is hoping to slow or even halt the onset and progression of Huntington’s disease – a concept supported by genetic studies and recently strengthened by pre-clinical models.

The proceeds of the Series A will primarily be used to support the pre-clinical development and initial clinical studies of LoQus23’s lead programme, an allosteric small molecule MutSβ inhibitor. The company is preparing its lead programme to enter the clinic in 2026.

LoQus23 CEO Dr David Reynolds, said: “This financing will enable us to develop key clinical data to support the development of our exciting lead programme. The ever-increasing body of data pointing to somatic expansion, caused by aberrant DNA mismatch repair, as being the primary culprit in Huntington’s disease provides great support that our approach of developing oral small molecule therapies will be transformative for patients with this dreadful inherited disease.

“We welcome Forbion as the lead investor of this round, alongside our existing high calibre investors, and look forward to benefiting from their support and expertise.”

Rogier Rooswinkel added: “Somatic expansion is a key driver of triplet repeat diseases like Huntington’s. In somatic expansion, MutSβ seems the most promising and best validated target, with the potential to bring disease progression to a halt.

“LoQus23 is leading the charge in developing allosteric small molecules against MutSβ, and we look forward to working with them to progress their molecules towards the clinic and ultimately to benefit patients.”