Harness raises £4M with a focus on advancing Huntington’s treatment
New funding to further 'great strides' in early work in neurodegeneration
Harness Therapeutics has secured an additional £4 million (about $5 million) in funding, which the U.K. biotech company plans to use to advance development of an experimental treatment for Huntington’s disease.
“This further investment puts us in a strong position to address multiple neurodegenerative disease pathways, with an initial focus on progressing our lead FAN1 nuclease programme into clinical development for Huntington’s Disease,” Jan Thirkettle, PhD, Harness’ CEO, said in a company press release.
Harness initially focused on developing a new class of therapeutic RNA molecules, called SINEUPs, that can increase the production of virtually any target protein by binding to its messenger RNA (mRNA) molecule. mRNA is the intermediate molecule derived from DNA that serves as a template in protein production.
By boosting this translation of target mRNA molecules into proteins, this type of approach can increase or restore the levels of relevant proteins in conditions such as Huntington’s.
The insights gained with this initial investment allowed the Cambridge-based company to expand its RNA-based toolset for protein increase or upregulation, creating a promising RNA platform.
“We have made great strides in our early work on [mRNA-based] regulation of protein [production], and I am eager to bring the unique capabilities and target insights we have acquired into action with our refined focus on neurodegeneration,” Thirkettle said. “There is a pressing need for new approaches in neurodegeneration where compelling targets could be unlocked by controlled upregulation.”
Boosting FAN1 protein levels seen as viable Huntington’s treatment strategy
Harness’ lead program aims to use this approach to increase levels of a protein called FAN1, which is involved in DNA repair. Previous studies have shown that FAN1 helps prevent further expansion of disease-causing CAG repeats, and that lower levels of this protein are associated with an earlier disease onset in Huntington’s patients.
As such, boosting FAN1 levels might be a viable strategy as a treatment for Huntington’s.
“I strongly believe that by pursuing physiological protein upregulation of targets with strong biology and genetic validation we can deliver new medicines that currently cannot be discovered through other methods,” Thirkettle said.
Harness was launched in 2020 under the name Transine Therapeutics, with lead investors Takeda Ventures and The Dementia Discovery Fund (DDF). Epidarex Capital joined as an investor in 2022. With the new funding, which was led by these prior investors, the total invested in Harness is £17.6 million (about $22 million).
We believe that the Harness team has developed an exceptional RNA biology platform that is insightfully designed to overcome the limitations of other platforms, enabling novel approaches to address the underlying drivers of neurodegenerative diseases.
“We are very excited to extend further investment in support of Harness’ commitment to pursuing differentiated therapies for an area with considerable unmet need,” said Miles Gerson, executive investment director and partner at Takeda Ventures.
“We believe that the Harness team has developed an exceptional RNA biology platform that is insightfully designed to overcome the limitations of other platforms, enabling novel approaches to address the underlying drivers of neurodegenerative diseases,” Gerson said.
Christian Jung, a partner at SV Health Investors, investing out of DDF, said Harness’ technology “holds the potential to bring forward important new medicines that can change the lives of neurodegenerative disease patients who currently have few or no effective treatment options available to them.”
“We are delighted to have been an early investor in Harness and are excited to see its unique target-focused approach now being focused on neurodegenerative diseases,” added Jung, also the chairman of Harness’ board.