Ono & Reborna Partner for CNS RNA-Targeting Drug Discovery
Ono Pharmaceutical Co., Ltd. (Headquartered in Osaka, Japan; President and COO: Toichi Takino; hereafter referred to as “Ono”) has announced a strategic collaboration agreement with Reborna Biosciences, Inc. (Headquartered in Kanagawa, Japan; Representative Director: Koji Fuji; hereafter referred to as “Reborna”). This collaboration aims to develop novel small-molecule compounds targeting ribonucleic acid (RNA) in the field of central nervous system (CNS) disorders. The agreement represents a significant step in leveraging cutting-edge RNA-targeting technologies to address rare neurological diseases that currently lack effective treatments.
Overview of the Agreement
The partnership between Ono and Reborna focuses on utilizing Reborna’s proprietary RNA-targeting drug discovery platform to conduct joint research and development efforts. Through this collaboration, the companies intend to identify and optimize small-molecule compounds that have the potential to become viable drug candidates for treating specific rare neurological disorders selected by both parties.
Under the terms of the agreement, Ono has secured an exclusive option right to further develop, manufacture, and commercialize the small-molecule compounds discovered through this partnership on a global scale. As part of the collaboration, Reborna will receive an upfront payment from Ono, along with research funding to support the collaborative efforts. Additionally, Reborna will be entitled to milestone payments based on the research and development (R&D) progress of the compounds, as well as sales milestones. Furthermore, the company will receive tiered royalties based on the net sales of the successfully developed drugs.
Significance of RNA-Targeting Drug Discovery in CNS Disorders
The advent of RNA-targeting drug discovery presents new possibilities in treating neurological disorders that have remained difficult to address with conventional drug development methods. Many rare neurological diseases are caused by genetic mutations that lead to dysfunctional RNA processing, impairing protein synthesis and cellular function. Traditional small-molecule drugs primarily target proteins, but this approach is often ineffective for diseases stemming from genetic mutations that affect RNA function.
Reborna’s RNA-targeting drug discovery platform has been designed to regulate disease-related RNA molecules, restoring normal cellular function. This innovative approach holds promise in addressing diseases for which there are currently limited or no treatment options. The partnership with Ono allows Reborna to apply its platform to real-world drug development projects with the potential to transform treatment paradigms for patients suffering from rare neurological disorders.
Statements from Key Executives
Seishi Katsumata, Corporate Officer / Executive Director of Discovery & Research at Ono, emphasized the importance of Reborna’s innovative technology in tackling previously undruggable targets.
“We highly value Reborna’s technology, which enables the regulation of targets that were previously difficult to address with conventional drug discovery methods through the normalization of RNA function. We will work with Reborna to advance new drug discovery projects targeting difficult-to-treat rare neurological disorders, striving to deliver innovative new drugs to patients as soon as possible,” said Katsumata.
Koji Fuji, Representative Director of Reborna, expressed enthusiasm for the collaboration, highlighting the alignment between the two companies’ missions.
“We are pleased to begin this research collaboration with Ono, which has a drug discovery policy of ‘creating original and innovative new drugs.’ This partnership marks a significant step forward in the discovery of drugs for genetic rare diseases. We are committed to maximizing our drug discovery research and development capabilities and working with Ono to create a new drug with high unmet medical needs,” said Fuji.
Addressing Unmet Medical Needs in Neurology
Rare neurological diseases are a significant area of unmet medical need. Many of these conditions are genetic and progressive, severely impacting patients’ quality of life and, in some cases, leading to early mortality. Due to their rarity, developing treatments for these conditions poses unique challenges, including limited patient populations for clinical trials, high research and development costs, and the need for specialized expertise in neurological drug discovery.
Ono has a history of developing and commercializing innovative therapies across various therapeutic areas, including oncology, immunology, and neurology. This collaboration aligns with Ono’s broader strategy to expand its pipeline in neurology by leveraging emerging scientific approaches such as RNA-targeting small molecules. By partnering with Reborna, which specializes in RNA biology and drug discovery, Ono aims to pioneer new treatment modalities for rare CNS disorders.
Future Implications of the Collaboration
The success of this collaboration could have significant implications not only for the treatment of rare neurological diseases but also for the broader field of RNA-targeted therapeutics. Over the past decade, there has been increasing interest in RNA-based drug discovery, with notable advancements in antisense oligonucleotides, RNA interference (RNAi), and messenger RNA (mRNA) technologies. However, the development of small molecules capable of modulating RNA function represents a relatively novel approach with untapped potential.
If the partnership leads to the discovery of viable drug candidates, it could pave the way for further investments in RNA-targeting therapeutics, expanding the treatment landscape for various genetic disorders beyond neurology. Furthermore, successful commercialization of an RNA-targeting small molecule drug could establish a new precedent in drug development, demonstrating that small molecules can be effectively designed to influence RNA biology in a therapeutic context.
The collaboration between Ono Pharmaceutical and Reborna Biosciences represents a promising convergence of expertise in drug discovery and RNA-targeting technologies. By working together, the two companies hope to unlock new possibilities in treating rare neurological diseases that have long remained beyond the reach of traditional drug development approaches. With an exclusive global commercialization option in place, Ono is well-positioned to advance these potential therapies from research to market, ensuring that patients with unmet medical needs may benefit from novel, life-changing treatments in the near future.
As the field of RNA-targeted drug discovery continues to evolve, this collaboration stands as a testament to the growing recognition of RNA as a crucial therapeutic target. If successful, the partnership could mark a turning point in the fight against rare neurological diseases, bringing much-needed hope to patients and their families worldwide
