As previously announced on October 29, 2018, the companies entered into a global licensing and research collaboration focused on the discovery, development and commercialisation of potential new medicines in the areas of cardio-metabolic disease, neurodegeneration and pain.
The companies will utilise Dicerna's proprietary GalXC RNAi technology platform to progress new drug targets toward clinical development and commercialization.
In addition, the partners will collaborate to move beyond the current technical paradigm in order to generate next-generation oligonucleotide therapeutic agents.
Under the terms of the agreement, Dicerna will receive an upfront payment of USD 100m, as well as an equity investment of USD 100m at a premium.
Dicerna is also eligible to receive up to approximately USD 350m per target in development and commercialisation milestones, as well as tiered royalties ranging from the mid-single to low-double digits on product sales.
The two companies anticipate collaborating on more than 10 targets.
Dicerna's proprietary RNAi technology platform, called GalXC, aims to advance the development of next-generation RNAi-based therapies designed to silence disease-driving genes in the liver.
GalXC-based therapies are processed by the Dicer enzyme, which is the natural initiation point for RNAi within the human cell.
Using GalXC, Dicerna attaches N-acetylgalactosamine sugars directly to the extended region of the proprietary Dicer substrate short-interfering RNA (DsiRNA) molecules, yielding multiple conjugate delivery configurations that allow flexible and efficient conjugation to the targeting ligands while stabilising the RNAi duplex.
Dicerna believes this stabilisation will enable subcutaneous delivery of RNAi therapies to hepatocytes in the liver, where they are designed to specifically bind to receptors on target cells, potentially leading to internalization and access to the RNAi machinery within the cells.
By using the Dicer enzyme as the entry point into RNAi, the GalXC approach seeks to optimize the activity of the RNAi pathway so that it operates in the most specific and potent fashion.
Compounds produced via GalXC are intended to be broadly applicable across multiple therapeutic areas, including rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases.
Dicerna Pharmaceuticals is a biopharmaceutical company focused on the discovery and development of innovative, subcutaneously delivered RNAi-based therapeutics for the treatment of diseases involving the liver, including rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases.
The company is leveraging its proprietary GalXC RNAi technology platform to build a broad pipeline in these core therapeutic areas, focusing on target genes where connections between target gene and diseases are well understood and documented.
Dicerna plans to discover, develop and commercialize novel therapeutics either on its own or in collaboration with pharmaceutical partners. Dicerna has strategic collaborations with Boehringer Ingelheim, Eli Lilly and Company, and Alexion Pharmaceuticals.
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