LNPs are designed to stay in the circulation long enough to accumulate at disease sites, such as the liver or cancerous tumors. Through a process called endocytosis, cells take up the LNPs which allows them to migrate into the cell. The LNPs then undergo an interaction within the cell and the RNAi trigger molecules are released, mediating RNAi.
To realize the tremendous therapeutic potential of RNAi-based drugs, effective delivery is critical. Our proprietary delivery technology:
Additionally, multi-dosing with LNP has proven to be well-tolerated with treatments out to one year.
Our manufacturing process is rapid, scalable, and highly reproducible – enabling the commercialization of LNP-based products. Through our various collaboration arrangements, including the U.S. Department of Defense (DoD) on our TKM-Ebola program, Gritstone Oncology and Alexion Pharmaceuticals, we developed a number of innovations for our LNP delivery technology, such as a “commercial-scale” manufacturing processes and ability to lyophilize (freeze-dry) LNP.
On August 11, 2018, Arbutus LNP Licensee, Alnylam Pharmaceuticals, announced the approval of ONPATTRO™ (patisiran), for the treatment of ATTR - Amyloidosis. This approval represents unprecedented clinical validation of Arbutus’ LNP technology.
In addition to our LNP delivery platform, we have developed a proprietary N-Acetylgalactosamine (GalNAc) conjugate technology to enable subcutaneous delivery of an RNAi therapeutic targeting HBsAg and/or other HBV targets.
In April 2018, we launched Genevant Sciences, a company jointly owned by Arbutus and Roivant Sciences. Arbutus contributed a broad license to the LNP and ligand conjugate delivery platforms to Genevant for all applications outside of HBV. Genevant aims to advance multiple product candidates into the clinic across RNAi, mRNA, and gene editing modalities using the Arbutus LNP and ligand conjugate delivery platforms.