Adverum has invested $80 million into a North Carolina viral vector facility to support gene therapy candidate ADVM-022.
The GMP adeno-associated virus (AAV) manufacturing facility will be in Durham, North Carolina. Once operational in 2023, the 174,000 square-foot facility will boast 4,000 L of bioreactor capacity and create 200 jobs.
The inhouse facility will support large-scale production of Adverum’s ADVM-022, which is a potential gene therapy using “a single intravitreal injection targeting two mass markets – wet AMD and diabetic macular edema,” according to CEO Laurent Fischer, who added that if successful it has potential to be the “first mass marketed gene therapy.”
Image: iStock/3D_generator
The investment “sets the foundation of our overall manufacturing strategy and will provide dedicated commercial supply, which is a critical success factor for gene therapy,” Fischer told Bioprocess Insider.
Adverum’s manufacturing process is built on the Baculovirus Expression Vector System. “This approach is well suited for the production of large quantities of AAVs, as it takes advantage of the efficiency of viral infection coupled with the high density and scalability of insect cells grown in serum-free suspension cultures,” the firm says.
Inhouse vs outsource
Fischer told us Adverum chose to invest inhouse to control the manufacturing process for ADVM-022.
“In gene therapy, it’s critical to invest in your own manufacturing facility to maintain quality and timelines of commercial product supply. Having a dedicated site will enable us to deliver ADVM-022 to patients globally with a flexible and strongly controlled supply chain strategy.”
He added: “There is a scarcity of gene therapy capacity, especially at large scale and therefore it is essential that we control the manufacturing.”
Shortage of viral vector capacity has been well-documented and has led to increased inhouse investment by some gene therapy developers.
Clinical biotech Regenxbio, for example, recently said “finding gene therapy contractors has become more difficult” adding “the COVID-19 pandemic exacerbated the issue.” Meanwhile, firms including Pfizer and Astellas have cited the competitive advantage of having inhouse capabilities.
However, inhouse investment does not come without an element of risk. For example, monoclonal antibody developer Genmab has said it chooses to outsource all its biomanufacturing after buying what was described as a “very, very costly, and very inflexible” manufacturing plant.
Despite the $80 million investment inhouse, Adverum’s Fischer told us it will also use contract development and manufacturing organizations (CDMOs) for “flexible clinical and additional commercial supply,” stating “this multi-source strategy capitalizes on our internal AAV manufacturing expertise and puts us in a solid position.”