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 ACGT’s Scientific Advisory Council and Research Fellows.
Cancer Cell and Gene Therapy News
UCLA announces development of CAR-NKT breast cancer therapy
Scientists at UCLA announced that they have developed what they call a “one product fits all” CAR-NKT cell therapy that has been shown to be more effective than current immunotherapies at fighting cancer in tumor samples from late-stage triple-negative breast cancer patients.
The team that developed the treatment hopes that the same cell product could also be used to treat other types of cancer that manifest in solid tumors and express the protein mesothelin, including ovarian, pancreatic, and lung cancers.
Replimune announces FDA acceptance of BLA resubmission of RP1 for advanced melanoma
The Massachusetts-based biotechnology company Replimune announced that the FDA has accepted its resubmission of the novel oncolytic immunotherapy RP1 (vusolimogene oderparepvec) in combination with nivolumab for the treatment of advanced melanoma in patients who progress on an anti-PD-1-containing regimen.
RP1 is designed to treat cancer by using a modified herpes simplex virus to kill tumor cells and trigger an immune response. The FDA set a PDUFA date of April 10, 2026.
Roswell Park researchers identify key genetic features in glioblastoma
In what could be a major development in the clinical understanding of glioblastoma, researchers from Roswell Park Comprehensive Cancer Center have identified molecular features that could be used to select patients most likely to respond to the peptide-conjugate vaccine SurVaxM.
Glioblastoma is the most common primary brain cancer and is one of the most aggressive and difficult-to-treat cancers in existence with a five-year survival rate of less than 7 percent. The vaccine SurVaxM is currently in a Phase IIa clinical trial and is being advanced by MimiVax, a clinical-stage biotech company focused on developing novel immunotherapies for the treatment of brain tumors.
Chen awarded Excellence in Genetics Research Award
ACGT Research Fellow Sidi Chen, PhD (Yale School of Medicine) was awarded the Excellence in Genetics Research Award by the Association of Chinese Geneticists in America, which recognized Dr. Chen’s work creating CRISPR-based in vivo genomics platforms to identify genetic factors key to the progression of cancer.
Chen was previously awarded a grant by ACGT in 2020, which helped fund his development of a program he called MAEGI – Multiplexed Activation of Endogenous Genes as an Immunotherapy – in the treatment of pancreatic cancer, specifically pancreatic ductal adenocarcinoma. Read more about Chen’s ACGT-funded research here.
Maus receives ACGT grant for pancreatic cancer research
ACGT announced that it has issued a $500,000 grant to fund a team of Massachusetts General Hospital scientists, led by Marcela Maus, MD, PhD, who are probing the use of in vivo CRISPR screening to improve CAR T cell therapy in pancreatic cancer.
The grant will support Dr. Maus’s research into using a CRISPR tool to create a screening process that can help identify necessary changes to CAR T cells to make them more effective in solid tumors. Dr. Maus is building on prior research that identified ways to enhance the effectiveness of CAR T cells against pancreatic cancer.
Cancer Cell and Gene Therapy Research Highlights from ACGT’s SAC and Research Fellows
Science Translational Medicine: Serial multiomics uncovers anti-glioblastoma responses not evident by routine clinical analyses.
ACGT Scientific Advisory Council member and Research Fellow, E. Antonio Chiocca, MD, PhD (Brigham and Women’s Hospital), published the results of longitudinal tumor biopsy collection and multi-omic readouts for two patients participating in a clinical trial of the oncolytic HSV CAN-3110. Samples from both study participants provided evidence for reshaping of the GBM tumor microenvironment in response to CAN-3110, including expansion of T cell responses, features that would not be apparent by traditional MRI. These data both suggest a potential therapeutic benefit for CAN-3110 and demonstrate the feasibility of implementing longitudinal tumor sampling in GBM. CAN-3110 is being developed by Candel Therapeutics, a clinical-stage biopharmaceutical company focused on developing multimodal biological immunotherapies.
Nature Biomechanical Engineering: CCR5-targeted allogeneic gamma–delta CD19 chimeric antigen receptor T cells for HIV-associated B cell-malignancy immunotherapy.
A team of researchers, including ACGT Scientific Advisory Council members Michael T. Lotze, MD (University of Pittsburgh) and Carl June, MD (University of Pennsylvania) and ACGT Research Fellow Joseph A. Fraietta (University of Pennsylvania) published a study demonstrating a method for in vitro activation and expansion of T cells to help facilitate more efficient CAR integration in HIV-associated B-cell lymphoma and leukemia.
Nature Reviews Immunology: Microenvironmental regulation of solid tumor resistance to CAR T cell therapy.
ACGT Scientific Advisory Council member Carl June, MD (University of Pennsylvania) was a co-author on a review covering the various barriers that CAR T-cell therapy faces in attaining efficacy in the highly immunosuppressive solid tumor microenvironment, while discussing potential strategies to overcome these challenges.
Cytotherapy: Outcomes following CD22 CAR T-cells in B-ALL: a tale of two manufacturing strategies.
ACGT Scientific Advisory Council member Crystal Mackall, MD (Stanford University), was a co-author on a study comparing the outcomes of two different manufacturing systems of CD22 CAR T cells used in patients with B-cell acute lymphoblastic leukemia – the two methods examined were bag-culture and Prodigy CAR T cells.
Nature Reviews Urology: The opportunities and barriers for developing tumor-infiltrating lymphocyte therapy for patients with advanced genitourinary cancers.
ACGT Scientific Advisory Council member Shari Pilon-Thomas, PhD (Moffitt Cancer Center), was a co-author of a publication highlighting recent advances in TIL generation, manipulation techniques, preparative regimens, and combination with immune checkpoint inhibitors, offering new hope for re-examining optimized TIL therapy for genitourinary cancers.
Trends in Cancer: Overcoming solid-tumor barriers: armored CAR-T cell therapy.
ACGT Research Fellow, Yvonne Chen, PhD (University of California, Los Angeles), published a paper discussing the “armoring” of CAR-T cells to express chemokine receptors, enzymes that degrade extracellular matrix components, proinflammatory cytokines, or factors that modulate immunosuppressive signals to empower CAR-T cells to overcome barriers associated with solid tumors. Translating promising preclinical results into reliable clinical benefit for patients with solid tumors remains challenging. This review critically examines emerging CAR-T cell armoring approaches and highlights key translational hurdles, as well as the need for innovations in human-relevant disease models, safety designs, and treatment strategies to enable effective translation.
Cancer Cell and Gene Therapy Research Publications
- Arruda, L. C. M., Karbach, J., Kiselicki, D., Altmannsberger, H. M., Sinelnikov, E., Gustavus, D., … Jäger, E. (2025). Tumor-infiltrating lymphocytes-derived CD8+ clonotypes infiltrate the tumor tissue and mediate tumor regression in glioblastoma. OncoImmunology, 14(1). https://doi.org/10.1080/2162402X.2025.2559784.
- Walton CM, Bell M, O’Neil R, Sahin O, Choi BD, Fecci PE, Strickland BA. Chimeric antigen receptor (CAR) T-cell therapy for glioblastoma (GBM): current clinical insights, challenges, and future directions. J Immunother Cancer. 2025 Oct 31;13(10):e012308. doi: 10.1136/jitc-2025-012308. PMID: 41176315; PMCID: PMC12581071.
- Nierengarten MB. CAR T-cell therapy shows impressive outcomes for patients with refractory multiple myeloma: One third of patients did not need maintenance or subsequent treatment and presented without disease progression at 5 years and beyond. Cancer. 2025 Oct 15;131(20):e70057. doi: 10.1002/cncr.70057. PMID: 41104638.
- Oncolytic viruses as anticancer agents: clinical progress and remaining challenges. Appleton, Elizabeth et al. The Lancet, Volume 406, Issue 10509, 1295 – 1312.
