Derek Jackson, BS, MA, on Evaluating Gene Therapy PCRX-201 for Osteoarthritis of the Knee

“...In our study, which was a large study for phase 1 (72 patients), 50% of the population had baseline neutralizing antibodies for adenovirus serotype 5, which is our capsid. In that study, we saw no impact of neutralizing antibody on efficacy or safety. This is encouraging for us because it does potentially pave the path for us exploring the ability to redose our therapy, which could be very meaningful for its market presentation.”

Pacira Biosciences is currently evaluating enekinragene inzadenovec (PCRX-201), an investigational high-capacity adenovirus (HCAd) vector gene therapy intended to treat osteoarthritis (OA) of the knee through local administration, in a phase 1 clinical trial. At the the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2024, in New Orleans, LA, Mi Jeong Kim, PhD, the senior director of translational sciences at Pacira BioSciences, gave a presentation on the data from the study entitled “Understanding the clinical immunogenicity of locally injected HCAd vector provides insight into optimizing dosing strategy”.

At the conference, CGTLive® sat down with Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira, to learn more about the findings that Kim presented. Jackson discussed the key data, which related to effect of preexisting neutralizing antibodies to the HCAd vector used in PCRX-201, highlighting that no impact of these antibodies on safety or efficacy was observed in patients in the study. He emphasized that this may allow the therapy to be dosed multiple times.

Jackson also discussed the potential role of PCRX-201 in treating knee OA more generally and spoke about future plans for the therapy’s development. In particular, he went over plans for the phase 2 randomized, double-blind ASCEND clinical trial (NCT06884865), which was recently initiated and will enable a more detailed analysis of the therapy’s efficacy compared to the phase 1 trial, which was primarily focused on safety.

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 8, 2025. https://investor.pacira.com/news-releases/news-release-details/pacira-biosciences-present-new-data-clinical-immunogenicity