Grant awarded through the Parkinson's Disease Therapeutics Pipeline Program to support advancement of Harness' new programme targeting GCase and LIMP2 for Parkinson's disease
Two-year programme expands Harness' neurodegenerative disease pipeline, leveraging proprietary MISBA® platform
Cambridge, UK, 9 June 2026: Harness Therapeutics (‘Harness’), a biotechnology company unlocking previously undruggable targets to transform the treatment of neurodegenerative diseases, today announces it has been awarded a grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to advance its Parkinson's disease (PD) programme targeting glucocerebrosidase (GCase) and lysosomal integral membrane protein-2 (LIMP2) in GBA1-PD.
Mutations of the GBA1 gene, encoding the lysosomal enzyme GCase, represent the most common genetic risk factor for PD, disrupting glycosphingolipid homeostasis and contributing to earlier onset and accelerated disease progression.
Harness will leverage its proprietary MISBA® (microRNA steric blocking ASO) platform technology to simultaneously upregulate GCase and its lysosomal transport protein, LIMP2. This unique approach aims to increase GCase abundance while ensuring its correct localisation in the cell, representing a compelling potential strategy to address the underlying biology of the disease and to potentially deliver disease modification in patients with GBA1-PD and other synucleinopathies.
The two-year programme will focus on demonstrating proof-of-concept for the controlled upregulation of GCase and LIMP2, with the goal of identifying potential therapeutic candidates for further development.
This work expands Harness' growing neurodegenerative disease pipeline, including its lead candidate HRN001 for Huntington's disease, now advancing towards clinical trials, alongside programmes in ALS and Alzheimer's disease.
Dr Jan Thirkettle, CEO of Harness Therapeutics, commented: "We are delighted to receive this grant from The Michael J. Fox Foundation, to address these compelling, genetically validated targets in Parkinson's disease through our proprietary MISBA platform. This programme represents an important strategic expansion of our neurodegenerative pipeline and demonstrates the versatility of our platform across multiple disease areas. We look forward to advancing this work and building on the momentum across our broader pipeline as we work to deliver transformative therapies for patients living with devastating neurodegenerative diseases."
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About Harness Therapeutics
Harness Therapeutics is a biotechnology company focused on unlocking previously undruggable targets to transform the treatment of neurodegenerative diseases. Its MISBA® platform technology enables precise upregulation of target protein levels by modulating mechanisms controlling protein synthesis. Harness’ recently announced MISBA® Duo technology enables the design of single constructs which simultaneously upregulate one target whilst downregulating a second. Using its deep understanding of post-transcriptional regulation and sophisticated neuron-based models, Harness’ approach allows drugging of potentially disease-modifying targets, which would not be addressable using gene therapy or other modalities.
Its lead candidate in Huntington’s Disease, HRN001, targets FAN1 nuclease, a key protective protein, shown to slow disease progression. Its pipeline also includes programmes addressing compelling first in class targets for ALS, Alzheimer's Disease, and Parkinson’s Disease.
Harness has assembled a world-class team, supported by leading experts in neurodegeneration and RNA biology, and is based in Cambridge, UK. Its leading life science investors include the foundational investors Takeda Ventures and SV Health Investors’ Dementia Discovery Fund, alongside Epidarex Capital and Ono Ventures Investment.
For more information, please visit https://www.harnesstx.com/ and follow us on LinkedIn.
For further information, please contact:
ICR Healthcare
Amber Fennell, Stephanie Cuthbert,
Tel: +44 20 3709 5700
Email: harnesstx@icrinc.com
