Preventing the progression of Huntington’s Disease

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Unlocking New Targets to Transform the Treatment of Neurodegenerative Diseases

At Harness Therapeutics we have one mission – physiological upregulation of targets that have the promise to provide disease-modifying therapeutics for neurodegenerative diseases. We use a range of mRNA-targeted oligonucleotide-based methodologies to alter the post-transcriptional regulation of protein synthesis and so increase the target protein in a controlled manner.

Our focus on neurodegenerative disease is driven by our passion to develop truly disease-modifying therapies for diseases that affect so many people, yet for which there are few if any therapies that can slow or halt the progression of the disease.

The ability to effect controlled increases of protein levels is essential in being able to safely drug targets with narrow therapeutic windows, something that existing platforms cannot achieve. This is critical for neurodegenerative disease as there are now a number of targets that have strong genetic validation linked to disease progression but require controlled increases rather than knock-down or over-expression.

Our goal is to open up the target space for neurodegenerative disease and be able to advance effective treatment solutions for the tens of thousands of patients and their families who are facing the certainty of irreversible disease progression, loss of quality of life, and ultimately death.

Neurodegenerative disorders are chronic conditions that destroy parts of the nervous system over time, especially the brain. They result in progressive loss of cognitive and motor function and eventually, death. The most common neurodegenerative diseases include Alzheimer’s disease, Parkinson’s disease, Amyotrophic lateral sclerosis (ALS), Motor neuron disease (MND), Huntington’s disease (HD), Spinal muscular atrophy (SMA), and Spinocerebellar ataxia (SCA).

It is estimated that more than 50 million people worldwide are affected by neurodegenerative diseases and this number is increasing as overall life expectancy increases. Most of these conditions are much more likely to happen in people over 65, but some conditions like Huntington’s disease and ALS often appear much earlier.

To learn more please send us an email or complete our enquiry form.

Our vision

Building the leading platform for physiological upregulation of neurodegenerative disease targets to enable truly disease-modifying therapies.

Our approach

Deep expertise & unique capabilities in RNA biology & neurodegeneration

High value targets

Ready to scale

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Preventing the progression of Huntington’s Disease

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