Derek Jackson, BS, MA, and Kilian Guse, PhD, on High-Capacity Adenovirus Vectors for Gene Therapy

“I would say that gene therapy for monogenetic disease is validated now, and now I think we need to take gene therapy to the next level, which is developing them for prevalent nongenetic diseases. That's where the HCAd platform seems to be particularly suitable.”

Pacira Biosciences’ gene therapy product enekinragene inzadenovec (PCRX-201) is currently in clinical stage evaluation for the treatment of osteoarthritis (OA) of the knee. Notably, the gene therapy utilizes a high-capacity adenovirus (HCAd) vector, setting it apart from the many gene therapies currently on the market and in clinical trials that utilize adeno-associated virus (AAV) vectors. At the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2024, in New Orleans, LA, Pacira held a symposium entitled “High-capacity adenoviral vectors: advancing gene therapy beyond AAV to deliver cost-effective therapies for common diseases” focused on the rationale behind using HCAd and exploring how it may be a cost-effective platform for a range of gene therapy products aimed at treating prevalent conditions. The symposium featured speakers Brendan Lee, a professor of molecular and human genetics at Baylor College of Medicine; Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira; and Kilian Guse, PhD, the vice president of genetic medicine platforms at Pacira.

The day before the symposium, CGTLive® sat down with Jackson and Guse on the floor of ASGCT to learn more about HCAd vectors and the topics that would be covered at the symposium. Guse explained the background behind the development of HCad vectors and their use in PCRX-201 and beyond. Jackson noted that one of the advantages of using adenovirus is that it has excellent transduction efficiency, which could allow for the use of lower doses and reduced costs in commercial products. He highlighted the importance of this for PCRX-201 because it will be competing with a number of other treatments currently available for OA and thus will need to be priced in a range that is competitive for that market.

Click here to view more coverage of the 2025 ASGCT Annual Meeting.

REFERENCE
1. Pacira Biosciences to present new data on clinical immunogenicity of intra-articular PCRX-201 and its implications for dosing strategy in knee osteoarthritis. News release. Pacira BioSciences, Inc. May 2, 2025. Accessed July 9, 2025. https://investor.pacira.com/news-releases/news-release-details/pacira-biosciences-present-new-data-clinical-immunogenicity