Harness Therapeutics awarded grant from The Michael J. Fox Foundation for Parkinson’s Research
Harness Therapeutics, a biotechnology company unlocking previously undruggable targets to transform the treatment of neurodegenerative diseases, 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.
