Harness Therapeutics appoints Leading World Experts in Neurodegenerative Diseases to its Scientific Advisory Board (SAB)

in Portfolio News

Newly established SAB of prominent geneticists, biologists and Huntington’s disease specialists’ marks pivotal step in accelerating progress of lead programme HRN001

Cambridge, UK, May 30, 2024: Harness Therapeutics, a biotechnology company focused on protein upregulation to develop next generation therapeutics for neurodegenerative diseases, today announces the formation of its new SAB, consisting of renowned leaders in the genetics, biology, and clinical development of Huntington’s disease (HD) and other important neurodegenerative disorders:

  • Dr. Irina Antonijevic – CMO EveryONE Medicines
  • Prof. Roger Barker – University of Cambridge
  • Prof. Jim Gusella – MGH, Harvard
  • Prof. Jeff Long – University of Iowa
  • Dr. Eric Marcusson – Independent Consultant
  • Dr. Tom Massey – University of Cardiff
  • Dr. Ralf Reilmann – George Huntington Institute
  • Dr. Jennifer Sims – Independent Consultant

The SAB members will support Harness Therapeutics’ research and development activities, offering invaluable scientific and clinical guidance as the Company progresses its lead HD programme HRN001 through CTA-enabling studies, scheduled to begin in 2025, and beyond this towards clinical development.

HRN001 is designed to increase levels of FAN1 nuclease, the strongest genetic modifier of HD onset. Increases in FAN1 have been shown to slow down the expansion of CAG ‘triplets’ (somatic instability), which is now understood to be the key driver of HD progression. Harness is leading efforts to exploit this important target through its deep understanding of the relevant translational biology and by utilising its platform for physiological upregulation, based on optimising protein translation from endogenous mRNA. By means of this controlled increase in target protein expression, Harness hopes to develop HRN001 as the first truly disease-modifying therapy for HD.

With input from the SAB, Harness will also continue to build its pipeline of neurodegenerative disease assets. The Company has now introduced a second programme aiming to target a specific process to reduce the formation of TPD-43 aggregates which are believed to drive some of the most common neurodegenerative diseases including Alzheimer’s disease and amyotrophic lateral sclerosis (ALS).

Dr Jan Thirkettle, CEO of Harness Therapeutics, commented “I am thrilled to announce the establishment of Harness’ Scientific Advisory Board. These highly accomplished scientists and clinicians bring a wealth of knowledge and experience across all aspects of Huntington's disease research and treatment as well as the broader neurodegenerative disease landscape. Their expertise will not only help us accelerate the development of a potentially transformative treatment for HD patients but will also bolster our efforts to explore promising avenues of research for other neurodegenerative disorders that may benefit from our approach to controlled physiological upregulation, including Alzheimer’s disease, Parkinson’s disease and ALS”.

Dr. Irina Antonijevic, chair of the SAB added,“Recent advancements in the Huntington’s disease field have put a spotlight on the importance of FAN1 as a target for the development of novel therapies for this disease which, if successfully exploited, could provide significant benefits to patients. Harness is leading this charge with HRN001, which is designed to selectively increase FAN1 nuclease levels in a controlled manner to reduce the triplet expansion that drives Huntington’s disease progression. This is a highly promising approach and I and the SAB look forward to helping the company progress this programme for the many people with HD in need as well as advancing the platform to enable development of disease-modifying therapies for other neurodegenerative disorders”


About Harness Therapeutics

Harness Therapeutics is a biotechnology company focused on the development of next-generation therapeutics for the treatment of neurodegenerative diseases, with a lead programme in Huntington’s disease targeting FAN1 to limit somatic expansion. Focussing on neurodegeneration targets with strong biology and genetic validation, Harness Therapeutics utilises its deep understanding of RNA biology and post-transcriptional regulation to enable drugging of targets with oligonucleotide-based drugs to achieve controlled increases in protein expression.

Harness Therapeutics has assembled a world-class team focused on the mission of building the most efficient platform for controlled upregulation of CNS targets that underpin neurodegenerative disease. This includes extensive RNA biology and bioinformatics expertise, specialist analytics capabilities and unique neuronal cell models. Harness Therapeutics’ unique platform generates multiple orthogonal upregulation strategies that are tested in highly translatable in vitro cell models to identify constructs capable of increasing endogenous protein translation. This specific and controlled approach to increasing target proteins provides a mechanism to drug targets that are not addressable using gene therapy or other modalities.

Harness Therapeutics is based in Cambridge, UK, and was originally established by leading life science investors Takeda Ventures and SV Health Investors’ Dementia Discovery Fund in 2020. Harness Therapeutic’s current investor base now also includes Epidarex Capital.

For more information, please visit www.harnesstx.com/ and follow us on LinkedIn.


Harness Therapeutics
Dr Jan Thirkettle, CEO



Sylvie Berrebi, Frazer Hall


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