Lonza launches AAV cell line platform for gene therapy scale-up

Lonza, a Switzerland-headquartered contract development and manufacturing organization (CDMO), announced the launch of its Xcite adeno-associated virus (AAV) stable producer cell line platform designed to help gene therapy developers improve scalability, process robustness and cost efficiency as programs move toward commercialization.

The company said the platform uses a manufacturing approach in which all AAV-producing components are stably integrated into a cell line, reducing variability and operational complexity commonly associated with transient transfection methods. During beta testing with a clinically relevant gene of interest and engineered capsid, the platform demonstrated a reported 10- to 15-fold increase in titer compared with transient transfection approaches.

Lonza said the platform is intended to support manufacturing for indications requiring larger patient populations or higher dosing levels, including oncology and neurodegenerative diseases. The company added that the technology could reduce cost of goods by 80% or more through raw material efficiencies, scalable manufacturing and productivity gains.

According to the company, customers can access the platform through Lonza’s process development laboratories to evaluate manufacturability during early-stage development and support go/no-go decisions. The company also said developers can in-license the platform for internal product development and commercial manufacturing efforts.

“Xcite AAV PCL platform represents the culmination of years of Lonza innovation and hands-on experience in AAV manufacturing,” said Senthil Ramaswamy, vice president of research and development for specialized modalities at Lonza, in a statement. “The technology has the potential to easily scale up processes to reduce COGS by 80% or more through raw material efficiencies, scalable manufacturing, and productivity gains.”

The launch expands Lonza’s recent investments in advanced biologics and cell and gene therapy manufacturing. Earlier this month, the company announced an exclusive licensing agreement with Bristol Myers Squibb that provides access to Lonza’s SYNtecan linker-payload platform for development of an antibody-drug conjugate targeting oncology applications. The agreement also included access to the company’s GlycoConnect and HydraSpace ADC technologies.