🧬 Biotech Startup of the Week: Harness Therapeutics
What if instead of silencing genes, we turned the right ones back on?
Every week, I highlight one biotech startup that’s doing something quietly (or loudly) disruptive. This week, we're looking at a UK-based company tackling neurodegeneration from a completely different angle.
🔬 1. A New Oligo Paradigm
Most oligonucleotide therapies act by knocking down gene expression. Harness Therapeutics has taken the opposite path: they’re using antisense oligonucleotides (ASOs) to upregulate endogenous genes, effectively unlocking protective biology that was previously “undruggable” with traditional approaches.
ASOs are typically used to silence RNA; here, Harness uses them to enhance it.
Their technology targets the post-transcriptional regulation of protein synthesis—modulating mRNA to increase protein output in a controlled, physiological range.
🧬 2. Lead Programme: FAN1 for Huntington’s Disease
Harness’s premier project focuses on FAN1, a DNA repair nuclease. Genetic studies show that higher FAN1 levels correlate with delayed onset of Huntington’s disease, making it a compelling target.
In Huntington’s, somatic CAG repeat expansions drive disease progression.
By boosting FAN1 using ASOs, Harness aims to stabilise CAG repeat length in neurons—a mechanism upstream of downstream toxicity.
Preclinical data so far shows strong FAN1 upregulation and reduced somatic instability in patient-derived neuronal models. The company is preparing for CTA/IND-enabling studies in 2025, with Phase I/II trials expected in 2026.
🧪 3. Biomarkers & Preclinical Readiness
Harness is leveraging nanopore sequencing to measure CAG expansion in iPSC-derived neurons—an advanced biomarker approach presented at the CHDI HD conference in February 2025, crucial for demonstrating pharmacodynamic impact.
⚗️ 4. Advanced Platform & Expanding Pipeline
Beyond HD, Harness is broadening its horizon:
Second programme targeting nuclear import receptors for ALS and Alzheimer’s
Third programme for Parkinson’s disease set for early 2025
Their oligo platform can be adapted to diverse CNS targets, promising a modular approach to tackling complex neurobiology .
👥 5. Science Guided by Top Experts
Harness Therapeutics was founded by two academic leaders:
Dr Rickie Patani (UCL & Francis Crick Institute), expert in iPSC models of neurodegeneration
Dr James Nathan (University of Cambridge), specialist in stress signalling and proteostasis
Their scientific vision is supported by CEO Mike Romanos (ex-Crescendo Biologics, Kymab) and board chair Dr Heather Preston (Oxford Nanopore, Oxford Biomedica), alongside biotech executive Meenu Chhabra Karson.
In 2024, Harness established a Scientific Advisory Board featuring:
Prof Roger Barker (Cambridge)
Prof Jim Gusella (Harvard)
Dr Irina Antonijevic, Dr Tim Harris, and Dr Beth Borowsky
Together, this team brings deep expertise in RNA biology, Huntington’s disease, and translational neurotherapeutics—ensuring Harness’s scientific strategy is both ambitious and grounded.
💸 6. Funding Anchored to Scientific Milestones
Raised £4M in November 2023, bringing total to ~£17.6M, accelerating HD direction.
In January 2025, a new round led by Ono Ventures deepened funding commitments towards CTA/IND stage, corresponding to the appointment of Dr Heather Preston as Non-Executive Chair.
🎯 Why It Matters
Harness is targeting a fundamental problem in neurodegeneration—somatic repeat expansion—with a targeted ASO platform. They aren’t just producing a drug; they’re building a versatile platform capable of regulating endogenous proteins across myriad neuro diseases—an approach that could redefine “druggable” in CNS.
📘 At a Glance – The Science Summary
Focus AreaKey ProgressUpregulation PlatformASOs used to increase expressionLead ProgrammeFAN1 ASO for Huntington’s DiseaseBiomarker StrategyNanopore sequencing for CAGsPipelineALS/Alzheimer’s, Parkinson’sSAB GuidanceField-leading neuro expertsFunding Milestones£17.6M to date; active funding path
Harness Therapeutics isn’t just developing a drug—it’s launching a new therapeutic paradigm. For anyone tracking the next chapter in oligonucleotide innovation or neurodegenerative disease treatment, this one demands attention.
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