Neurodegenerative disorders affect over 50 million people globally, with prevalence rising due to increased life expectancy. These conditions lead to progressive cognitive and motor decline, often resulting in death, and primarily affect those over 65, though some, like Amyotrophic lateral sclerosis (ALS), can appear earlier.
Despite the high burden, effective disease-modifying therapies are scarce. Traditional research has focused on down-regulating a limited number of targets, neglecting many genetically validated targets due to challenges in controlled upregulation.
These targets, crucial for neurodegenerative mechanisms like lysosomal function and RNA metabolism, require precise and safe approaches to halt disease progression, which current gene therapy and editing technologies cannot adequately provide.
Jan Thirkettle, CEO of the UK preclinical biotech Harness Therapeutics, told In Vivo about the company’s approach, which aims to slow the somatic expansion process, offering a disease-modifying therapy.
Its Huntington’s disease (HD) program focuses on the controlled upregulation of FAN1, a nuclease shown to slow disease progression. The company is pioneering this approach, which has strong genetic and biochemical validation, and it may also benefit other triplet expansion disorders like Fragile X and Spinocerebellar ataxias. Recent research published in Cell and Nature Medicine demonstrate this link between somatic DNA-repeat expansion and disease progression.
Thirkettle discussed the applicability of the Harness approach to other indications within neurodegeneration such as Alzheimer’s disease and Parkinson’s disease, as well as its strategy to partner for future clinical development.
Time stamps:
1:20 The scientific roots of Harness Therapeutics
5:54 New investors and taking the next step
7:26 The Harness technology
10:47 New data validating the Harness approach to neurodegeneration
14:05 Differentiating from others in the field
16:00 Expected clinical timelines
17: 26 Applicability in Parkinson’s disease
