In this month’s ACGT Research News Roundup, we’re covering new publications in cell and gene therapy research, exciting developments in the clinic and biotech, and highlighting research from our ACGT Scientific Advisory Council (SAC) and Research Fellows.
Cancer Cell and Gene Therapy Industry News
Aucatzyl receives UK approval for leukemia
The National Institute for Health and Care Excellence (NICE) in the United Kingdom announced that the T-cell therapy Aucatzyl (obecabtagene autoleucel) has been recommended as a treatment option for patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia, and the treatment will be available through the National Health Service.
Aucatzyl was granted conditional marketing authorization by the UK Medicines and Healthcare products Regulatory Agency in April, months after the FELIX study results were published in the New England Journal of Medicine.
Breyanzi approved for mantle cell lymphoma in the EU
On Nov. 24, the European Commission approved the CAR T-cell therapy Breyanzi (lisocabtagene maraleucel) for patients with relapsed or refractory mantle cell lymphoma after at least two lines of systemic therapy that include a Bruton’s tyrosine kinase (BTK) inhibitor.
Breyanzi had previously been approved in the European Union for the treatment of three other forms of relapsed and refractory lymphoma, and received this expanded approval after results from the TRANSCEND NHL 001 trial, which showed a complete response rate of 82.7% among patients in a third-line-plus setting.
Xenetic extends collaboration with Scripps Research Institute
The biotechnology firm Xenetic Biosciences announced that it has reached a four-month extension on its research collaboration with the Scripps Research Institute, aimed at continuing to advance research into the combination of CAR T-cell therapies and systemic DNase I.
Xenetic is currently in preclinical development of a systemic DNase I candidate, XBIO-015, which is being developed for both hematologic and solid tumors.
Mellman awarded Richard V. Smalley Memorial Award and Lectureship
ACGT SAC member Ira Mellman, PhD (Parker Institute for Cancer Immunotherapy, Medici Therapeutics) received the prestigious Richard V. Smalley, MD Memorial Award and Lectureship, recognizing his pioneering work in cancer immunology, at this year’s Society for Immunotherapy of Cancer (SITC) 2025 Annual Meeting in National Harbor, Maryland.
The award is the highest offered by SITC. Dr. Mellman previously headed Genentech’s early cancer vaccine programs against patient-specific mutant neo-antigens using mRNA and DNA, and has since made contributions such as the elucidation of how dendritic cells initiate immunity or maintain immune tolerance, and how T cell signaling is regulated by immune checkpoints, among other achievements.
ACGT hosts Lunch Symposium at SITC 2025
The ACGT had a strong presence at SITC 2025, hosting a Lunch Symposium that brought together leading experts in cancer cell and gene therapy to connect and share insights.
The ACGT Lunch Symposium included a fireside chat with cell and gene therapy trailblazer and ACGT SAC member Carl H. June, MD (University of Pennsylvania), moderated by ACGT Board of Directors member Marc Engelsgjerd, MD (Royalty Pharma), in which Dr. June shared his perspective on his groundbreaking career, the current state of the field and future directions for cell and gene therapy.
Cancer Cell and Gene Therapy Research Highlights from ACGT’s SAC and Research Fellows:
Journal for Immunotherapy of Cancer: Chimeric antigen receptor-engineered TIL (CAR-TIL) show potent tumor recognition and greater tumor infiltration than conventional CAR-T cells.
A team of researchers, including ACGT SAC member Shari Pilon-Thomas, PhD (Moffitt Cancer Center), published a study demonstrating the “feasibility of CAR-TIL manufacture” and illustrating their superiority in recognizing and infiltrating solid tumors over more conventional CAR-T cells.
Journal for Immunotherapy of Cancer: The science of tumor-infiltrating lymphocytes (TIL): perspectives from the SITC Surgery Committee.
Members of the SITC Surgery Committee, including ACGT Research Fellow George Coukos, MD, PhD (University of Lausanne), published an article on the science, production, and use of tumor-infiltrating lymphocytes (TILs) in cancer treatment, with a focus on early TIL studies in non-melanoma epithelial neoplasms.
Nature Communications: On-target off-tumor toxicity of claudin18.2-directed CAR-T cells in preclinical models.
A team of researchers, including ACGT Research Fellow Marcela Maus, MD (Massachusetts General Hospital), from a Phase I study on toxicity in Claudin 18.2-directed CAR-T, finding that toxicity is independent of the CAR construct’s design or the tumor model.
Cancer Cell and Gene Therapy Research Publications
Blood: Long-term Follow-up of Gastrointestinal CAR T-cell Lymphoma: Homing, Clonal Expansion, and Response to Cyclosporine. Crystal Mackall, MD (Stanford University).
New England Journal of Medicine: FDA’s New Plausible Mechanism Pathway.
Blood Immunology and Cancer Therapy: Viral vector-free generation of orthogonal IL2-responsive CAR T cells through gene editing of IL2 and its receptor. Carl June, MD (University of Pennsylvania).
Journal for Immunotherapy of Cancer: A mutant KRAS peptide vaccine (mKRAS-VAX) combined with immune checkpoint blockade recruits vaccine-induced T-cells into metastatic MMRp/MSS colorectal tumors.
Journal for Immunotherapy of Cancer: Single-cell characterization of novel chlorotoxin-directed CAR T cells for the treatment of patients with MMP2+ glioblastoma. Christine Brown, PhD (City of Hope).
Journal for Immunotherapy of Cancer: All-in-one CAR T cell platform for safe and effective targeting of metastatic castration-resistant prostate cancer. Carl June, MD (University of Pennsylvania).
Journal for Immunotherapy of Cancer: Evaluation of huCART19-IL18 manufactured using Synecta™ CDNP technology to improve CAR-T yield and consistency in cohort D of an ongoing phase I study. Carl June, MD (University of Pennsylvania).
