Harness Therapeutics announced the launch of its second-generation MISBA Duo platform, marking a significant advancement in targeting previously undruggable proteins for neurodegenerative diseases. The Cambridge-based biotechnology company has simultaneously established a research collaboration with Ono Venture Investment (OVI) to develop disease-modifying therapies for an undisclosed rare triplet repeat disorder.
Revolutionary Dual-Target Modulation Technology
The MISBA Duo platform represents the first technology capable of simultaneous, controlled upregulation and downregulation of two targets. Building on Harness's original MISBA (microRNA site blocking ASO) platform designed for target-specific upregulation, MISBA Duo delivers bimodal modulation that could pave the way for advanced disease-modifying approaches to neurodegenerative diseases and other central nervous system disorders.
"Today's announcement is a natural evolution of our technology and testament to the insights we have gained from our lead programme in Huntington's Disease," said Dr Jan Thirkettle, CEO of Harness Therapeutics. "We're looking forward to working closely with OVI as we explore the potential of the MISBA Duo platform for bimodal protein modulation."
Strategic Partnership and Funding Structure
The collaboration leverages expertise established through Harness's wholly owned FAN1 programme for Huntington's Disease, currently in pre-clinical studies. OVI will fund the research initiative, following its initial investment in Harness in January 2025 to support the company's lead HD programme. Under the agreement, OVI will secure the exclusive option to negotiate exclusivity on any resulting candidates and associated intellectual property for the rare triplet repeat disorder.
Dr Shunichiro Matsumoto, President & CEO of Ono Venture Investment, commented: "We are pleased to further collaborate with Harness and look forward to working closely together to advance this programme in the months ahead. We remain committed to supporting the development of the Company's MISBA platform and pipeline of disease modifying therapies for neurodegenerative diseases."
Expanding Pipeline for CNS Disorders
Harness's technology drives controlled and precise upregulation of target protein levels by modulating mechanisms controlling protein synthesis. The company's approach allows targeting of potentially disease-modifying proteins that would not be addressable using gene therapy or other modalities. Beyond the new collaboration, Harness's pipeline includes programmes targeting nuclear import receptors for ALS and Alzheimer's Disease, with plans to initiate a third programme for Parkinson's Disease.
The company's lead programme targets FAN1 nuclease, identified as a key protective protein in slowing Huntington's Disease progression. Harness has assembled a team supported by a scientific advisory board of leading experts in HD and RNA biology, with backing from investors including Takeda Ventures, SV Health Investors' Dementia Discovery Fund, Epidarex Capital, and Ono Ventures Investment.
