LNP Delivery Platform

Arbutus’ Lipid Nanoparticle (LNP) technology represents the most widely adopted RNAi delivery technology to date. Administered intravenously, our LNP delivery technology allows RNAi drugs to be encapsulated in tiny particles made of lipids (fats or oils). These tiny particles – LNP – travel through the bloodstream to target tissues.

 

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.

The Benefits of LNP Technology

To realize the tremendous therapeutic potential of RNAi-based drugs, effective delivery is critical. Our proprietary delivery technology:

  • Protects the potent trigger in a LNP;
  • Distributes it rapidly and efficiently;
  • Releases it inside the cell; and,
  • Minimizes immunotoxicity and other undesired side effects.

 

Additionally, multi-dosing with LNP has proven to be well-tolerated with treatments out to one year.

Development of LNP-Based Products

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. 

Ligand Conjugate Delivery 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.

 

Genevant Sciences

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.

 

Read more about RNAi Therapeutics in our R&D Portfolio.