Salute e Benessere
LoQus23 Therapeutics announces £35 million (c.$43 million) Series A financing to advance its small molecule somatic expansion inhibition therapy for Huntington's disease
The Mismatch Repair (MMR) branches of the DNA Damage Repair (DDR) 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 disease 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 financing will be primarily 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.
" MutSβ MutSβ "
The DDR system is a complex set of pathways involving more than 400 genes/proteins, which is required for long-term maintenance of an organism's DNA to promote cellular health throughout a lifetime of damaging events.
MutSα and MutSβ are part of the MMR system, which is heavily implicated by human genetic and mechanistic studies to be the root cause of Huntington's disease and other related triplet repeat diseases. It is now clear that the initial driver of disease pathophysiology is MMR-mediated CAG repeat expansion in the Huntingtin gene, a process known as somatic expansion. Once a critical threshold number of CAG repeats is reached, then the second phase of neuronal dysfunction and ultimately death follows, which causes the slowly progressing symptoms in patients.
LoQus23 is a biotech company based in Cambridge, UK , developing small molecule somatic expansion inhibitors for the treatment of Huntington's Disease and other triplet repeat disorders. Huntington's disease is an autosomal dominant neurodegenerative disorder for which there is currently no disease modifying treatment available and which currently has 30,000 patients in the US alone.
LoQus23's approach has the potential to stop DNA instability and therefore slow neurodegeneration in these diseases. LoQus23 is focused on using a structure-based approach to design small molecule drugs, which can offer more convenient administration than other approaches. Oral small molecule drugs have a strong track record in treating complex brain diseases and provide greater convenience for patients compared with other advanced treatment modalities.
LoQus23 has a highly experienced leadership team, built on world-class science. It was originally established in 2019 by Dr David Reynolds , Dr Caroline Benn , and Dr Ruth McKernan CBE , FMedSci, Entrepreneurs in Residence at SV Health Investors' Dementia Discovery Fund, which also acted as the initial seed investor.
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Forbion is a dedicated life sciences venture capital firm with offices in The Netherlands , Boston and Germany . Forbion invests in life sciences companies that are active in the (bio-) pharmaceutical space. Forbion manages €3.2 billion across multiple fund strategies that cover all stages of (bio-) pharmaceutical drug development. Forbion's current team consists of over 30 life sciences investment professionals that have built an impressive performance track record since the late nineties with investments in over 110 companies across nine funds. Forbion's record of sourcing, building and guiding life sciences companies has resulted in many approved breakthrough therapies and valuable exits. Besides financial objectives, Forbion selects investments that will positively affect the health and well-being of patients. The firm is a signatory to the United Nations Principles for Responsible Investment. Forbion operates a joint venture with BGV, the manager of seed and early-stage funds, especially focused on Benelux and Germany .
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