microRNA Platform Technology – RoadMap
At Regulus, our microRNA Development Platform provides a total, integrated solution for microRNA therapeutics from discovery all the way through commercialization. Core technologies in microRNA Chemistry and Delivery combine seamlessly to generate ground breaking therapies, while our commitment to Quality and investment in Global Supply Chain and manufacturing infrastructure ensure the highest quality and speed to market.
Regulus has established a robust platform for the design of anti-miRs that target and inhibit the action of microRNAs. Regulus relies upon a core set of chemical modifications to tailor the specificity, potency, and safety of the oligonucleotides in our pipeline. Regulus has benefited from over 20 years of innovation in RNA technology, including the use of the phosphorothioate backbone to improve metabolic stability, 2’-O-methoxyethyl (MOE) nucleosides to modulate safety, and high-affinity bicyclic modifications such as constrained ethyl (cEt) nucleosides to create more potent compounds. In addition to these core chemistries, Regulus has invested in building a team of chemists to profile and develop new chemical modifications that will further enhance the therapeutic potential of our oligonucleotides.
Regulus is also moving aggressively to develop microRNA “mimics” that will supplement the activity of under-expressed microRNAs. Extensive profiling of chemical modifications is being used to advance the design and development of miR-mimics for multiple therapeutic indications.
At Regulus, we embrace the use of diverse strategies for the delivery of therapeutic oligonucleotides to specific tissues and cell types. Regulus has access to a rich portfolio of delivery technologies, including lipid nanoparticle (LNP) formulations and cell-targeting technologies. Recent advances in the use of GalNAc-conjugation to enhance delivery to hepatocytes have been exploited in numerous programs, including our lead clinical program, RG-101. Regulus continues to explore the development of new delivery technologies both through internal efforts and via external partnerships and collaborations, and has invested heavily in efforts to identify strategies that might be used to enhance delivery to a variety of tissues and cell types.
Chemistry Manufacturing and Controls
Regulus has invested in numerous manufacturing partnerships in order to ensure a robust supply of key raw materials and API. Both internal and external expertise have been established in areas of QA, QC, CMC analytical and CMC manufacturing processes. Regulus is well-situated to manage and advance both current clinical activities and future commercialization.
As part of our corporate Quality by Design plan we are firmly committed to ensuring the quality of our drugs that will be used in patients. To achieve this goal we have established an experienced Quality Management team dedicated to API and drug product development, testing, and manufacturing, from early discovery through clinical trials and ultimately toward commercialization through global world-class manufacturing partnerships.
Global Supply Chain Management
Regulus has established and enabled global supply chain management to provide critical raw materials, API and Drug Product that support all phases of drug development, current world wide clinical trials and future commercialization.