New Michael J Fox Foundation grant backs a two-pronged approach targeting one of Parkinson’s strongest genetic risk factors.
Parkinson’s disease remains one of medicine’s most stubborn challenges. More than six million people worldwide are living with the condition, and despite decades of research, treatments today largely focus on managing symptoms rather than slowing the disease itself.
Now, UK biotech Harness Therapeutics has secured backing from The Michael J Fox Foundation for Parkinson’s Research (MJFF) to advance a program that aims to tackle the disease closer to its roots.
The Cambridge-based company announced it has been awarded a grant through MJFF’s Parkinson’s Disease Therapeutics Pipeline Program to support a two-year research effort focused on a subgroup of Parkinson’s patients carrying mutations in the GBA1 gene, the most common known genetic risk factor for the disease [1].
A different way to approach Parkinson’s
Much of Parkinson’s drug development has centered on alleviating tremors, stiffness and movement problems. Harness is pursuing something more ambitious: a potential disease-modifying strategy designed to address biological processes believed to contribute to disease progression.
The company’s approach focuses on a protein called glucocerebrosidase, or GCase [2]. Researchers have long known that reduced GCase activity is linked to Parkinson’s disease, particularly among people with GBA1 mutations. These mutations are found in an estimated 5–15% of patients and are associated with earlier disease onset and faster progression. Rather than simply increasing GCase levels, Harness is attempting to solve a second problem that often receives less attention: getting the protein to the right place inside cells.
The company’s program simultaneously targets GCase and another protein known as LIMP2, which acts as a transport partner. In simple terms, one protein needs to be produced, while the other helps deliver it to where it can do its job.
Harness believes that increasing both together could prove more effective than approaches focused on GCase alone.
The grant highlights growing interest in therapies that target the biology underlying neurodegenerative diseases rather than their symptoms. For years, researchers have increasingly recognized the importance of cellular waste-disposal systems in disorders such as Parkinson’s and Alzheimer’s disease. When these systems become impaired, harmful proteins can accumulate and damage neurons.
Harness’ strategy is built around supporting this cellular housekeeping machinery. If successful, the approach could potentially benefit not only patients with GBA1-associated Parkinson’s disease but also broader groups of patients affected by related neurodegenerative disorders.
The company will use its proprietary MISBA platform to advance the program. Over the next two years, the goal is to generate proof-of-concept data and identify therapeutic candidates suitable for further development.
Expanding a growing neurodegeneration pipeline
The Parkinson’s initiative also marks a strategic expansion for Harness as it builds a portfolio focused on diseases of aging. The company’s lead candidate, HRN001 for Huntington’s disease, is progressing toward clinical trials, while additional programs are underway in ALS and Alzheimer’s disease.
Dr Jan Thirkettle, CEO of Harness Therapeutics, expressed great pleasure in receiving the grant from The Michael J Fox Foundation. He noted that the funding would allow the company to address compelling, genetically validated targets in Parkinson’s disease utilizing their proprietary MISBA platform.
“This program represents an important strategic expansion of our neurodegenerative pipeline and demonstrates the versatility of our platform across multiple disease areas,” he added. “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.”
While Parkinson’s disease is often discussed as a neurological condition, it is also fundamentally an aging-related disease. As populations live longer, the number of people affected is expected to continue rising, creating an urgent need for treatments that can preserve function and independence later in life. That is why programs like this matter beyond a single disease area.
Harness’ newly funded program remains at an early stage, and significant work lies ahead before any therapy reaches patients. Yet the grant signals confidence in an approach that seeks not merely to manage Parkinson’s disease but potentially to alter its course.
[1] https://www.harnesstx.com/news/harness-therapeutics-awarded-grant-from-the-michael-j-fox-foundation-for-parkinsons-research-to-advance-parkinsons-disease/
[2] https://www.harnesstx.com/our-programmes/parkinsons-disease/
