Harness Therapeutics Launches MISBA® Duo Platform And Partners With Ono Venture Investment For Research Collaboration

Harness Therapeutics, a biotechnology company focused on targeting previously undruggable mechanisms to transform treatments for neurodegenerative diseases, has announced the launch of the second generation of its MISBA® (microRNA site blocking ASO) platform, called MISBA Duo. The first application of this next-generation platform will support a new research and development programme in collaboration with Ono Venture Investment, Inc. (OVI), the corporate venture capital arm of Ono Pharmaceutical Co., Ltd.

The original MISBA platform is designed to enable controlled, target-specific upregulation of genes. MISBA Duo builds on this capability, introducing bimodal modulation for the first time, which allows simultaneous upregulation of one target and downregulation of another. This dual-target approach is expected to provide new opportunities for disease-modifying therapies in neurodegenerative diseases and other central nervous system disorders.

Dr Jan Thirkettle, CEO of Harness Therapeutics said in a statement, “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. We’re looking forward to working closely with OVI as we explore the potential of the MISBA Duo platform for bimodal protein modulation. Such high-quality partnerships are a key strand of our wider strategy as we seek to deliver novel, disease-modifying therapies across a range of devastating CNS disorders.”

Dr Shunichiro Matsumoto, President & CEO of Ono Venture Investment, stated, “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.”

Under this collaboration, Harness and OVI will leverage the MISBA Duo platform alongside expertise gained from Harness’ wholly owned FAN1 programme for Huntington’s Disease, currently in preclinical studies. The new programme will focus on developing disease-modifying therapies for an undisclosed rare triplet repeat disorder. Harness will be responsible for designing and evaluating MISBA Duo constructs to generate candidate molecules capable of selectively modulating two targets, with the goal of achieving therapeutic effects across all stages of the disease.

OVI will fund the initiative, building on its initial investment in Harness in January 2025 that supported the progress of the lead Huntington’s Disease programme. The venture arm will also secure the exclusive option to negotiate rights to any resulting candidates and associated intellectual property developed for this rare triplet repeat disorder. This collaboration highlights Harness Therapeutics’ commitment to advancing innovative molecular approaches for complex neurodegenerative diseases, offering potential for new therapeutic modalities where traditional strategies have been limited.