In this month’s ACGT Research News Roundup, we’re reading new publications in cell and gene therapy research, new developments in the clinic and biotech, and catching up on research from our ACGT Scientific Advisory Council (SAC) and Research Fellows.
Cancer Cell and Gene Therapy Industry News
Kernal Bio awarded up to $48 million from ARPA-H
The biotech company Kernal Bio was awarded a grant of $48 million from the Advanced Research Projects Agency for Health, with the funds meant to advance Kernal Bio’s in vivo CAR T-cell therapy program and expand the capabilities of its mRNA 2.0 platform.
The program aims to improve cancer care by increasing the accessibility of advanced CAR T-cell therapies at a lower cost to patients.
Cytiva receives ARPA-H award with ambition to help transform CAR-T cell therapy for solid tumors.
ARPA-H also issued funding to Cytiva, which will lead a new effort aimed at developing a transformative in vivo CAR-T cell therapy platform for gastrointestinal (GI) cancers with significantly reduced manufacturing costs.
The research is anticipated to include contributions from the Innovative Genomics Institute (IGI); University of California, San Francisco (UCSF); University of California, Berkeley; Stanford Medicine; and The Ohio State University. Fellow Danaher subsidiaries Integrated DNA Technologies (IDT) and Aldevron will contribute clinic-enabling solutions.
BlueWhale Bio doses first patient with new Synecta platform
The Philadelphia-based biotechnology startup BlueWhale Bio announced that it has dosed its first patientusing a first-in-line product from its cell-derived Synecta platform, using it to streamline the manufacturing process for huCART19-IL18 cells in the treatment of blood cancers.
According to BlueWhale Bio, the trial showed “complete and durable” responses in patients with refractory lymphoma. ACGT Scientific Advisory Council member and Research Fellow, Carl H. June, MD (University of Pennsylvania), serves as scientific co-founder of BlueWhale Bio.
Kite, a Gilead Company, to acquire Interius BioTherapeutics, a privately held biotechnology company developing in vivo CAR therapeutics, for $350 million.
This acquisition complements Kite’s expertise in cell therapy by incorporating Interius’s in vivo platform. This approach enables the generation of CAR T cells directly within the patient’s body and may offer a more durable and long-lasting therapeutic effect by inserting DNA into the patient’s genome. Unlike traditional CAR T therapies that require cell harvesting, engineering and reinfusion, Interius’s innovative, off-the-shelf yet personalized approach is designed to be delivered via a single intravenous infusion, eliminating the need for preconditioning chemotherapy and complex cell processing.
Dispatch Bio launches, targeting new approaches to solid tumors.
A new biotechnology company, Dispatch Bio, announced its launch this summer with a mission to deliver a universal treatment for solid tumors using a novel immunotherapy approach. The company’s first-in-class Flare platform is designed to address targeting and resistance mechanisms in solid tumors to expand the curative potential of immunotherapies for cancer patients.
The board of the new company has important ACGT representation. ACGT SAC member and Research Fellow Carl H. June, MD (University of Pennsylvania), is a co-founder of Dispatch Bio and also serves on the company’s Scientific Advisory Board, along with Christine Brown, PhD (City of Hope), also an ACGT SAC member.
June, Sadelain receive 2025 Broermann Medical Innovation Award.
ACGT SAC members Carl H. June, MD (University of Pennsylvania) and Michel Sadelain, MD, PhD (Columbia University) will be honored with the inaugural Broermann Medical Innovation Award, recognizing their groundbreaking contributions to the development of CAR T-cell therapy for cancer.
Drs. June and Sadelain played leading roles in the early conception and research into CAR T-cell therapy, helping to usher in a new age of cancer treatment. The prize, which carries with it a one million Euro endowment, will be presented at a ceremony in Wiesbaden, Germany, by Hessian Prime Minister Boris Rhein.
Lifileucel gets Canadian approval for advanced melanoma.
Continuing its commercial rollout, Iovance’s Amtagvi® (lifileucel) received Health Canada approval for advanced melanoma, becoming the first T cell therapy for a solid tumor cancer and the first treatment option approved in Canada for advanced melanoma after anti-PD-1 and targeted therapy.
Amtagvi received FDA approval in Feb. 2024 for advanced melanoma that had stopped responding to a PD-1 blocking drug either by itself or in combination, and in the case of BRAF mutation-positive cancer, a BRAF-inhibitor drug with or without a MEK inhibitor drug that had also stopped working.
Cancer Cell and Gene Therapy Research Highlights from ACGT’s SAC and Research Fellows
Cell Reports Medicine: Chlorotoxin-directed CAR T cell therapy for recurrent glioblastoma: Interim clinical experience demonstrating feasibility and safety.
ACGT SAC member Christine Brown, PhD (City of Hope), reported interim clinical experience of a phase 1 trial evaluating intracavity/intratumoral (ICT) delivery of CLTX-CAR T cells in four patients with MMP-2-expressing recurrent GBM (NCT04214392), with the primary objectives of feasibility and safety.
Cell Reports Medicine: CAR-T cells in solid tumors: Challenges and breakthroughs.
In this review, ACGT Research Fellow Marcela Maus, MD, PhD, and co-authors from Massachusetts General Hospital, discuss insights from recent successful clinical trials in advanced solid tumors and highlight groundbreaking strategies integrating synthetic biology and gene engineering to enhance CAR-T cell fitness, potency, and persistence, activate host immunity, reprogram the TME, and enable multi-antigen targeting.
Neuro-Oncology Advances: Translational advancements in tumor vaccine therapies for glioblastomas.
A team from Brigham and Women’s Hospital published a review of recent advancements and clinical trials aimed at creating a tumor vaccine for the brain cancer glioblastoma, along with preclinical studies focused on boosting the immune response.
The study, co-written by ACGT SAC member and Research Fellow E. Antonio Chiocca, MD, PhD, found that despite some encouraging progress, a significant amount of research is needed to continue into personalized vaccines and combination therapies in order to make major headway.
Beyond Molecular Therapy: From bench to bloodstream: Lipid nanoparticles drive on-demand CAR-T cells.
ACGT Scientific Advisory Board member and Research Fellow Carl H. June, MD (University of Pennsylvania), published an article in Beyond Molecular Therapy examining the advantages of in vivo CAR engineering to overcome traditional hurdles, such as autologous apheresis, vein-to-vein times, and costly manufacturing, among others.
Dr. June suggests that in vivo CAR-T engineering provides a “clean break from that paradigm,” using targeted lipid nanoparticles along with other delivery methods to transform cell therapy into an off-the-shelf process that can be applied more widely.
Cancer Cell: Genome-wide CRISPR screens identify critical targets to enhance CAR-NK cell antitumor potency.
MD Anderson Cancer Center published findings from a study into the use of a novel CRISPR screening tool to maximize the efficacy of natural killer (NK) cells across multiple cancer types, potentially enhancing CAR NK cell therapies.
The study, led by ACGT SAC member Katy Rezvani, MD, PhD, identified and edited multiple targets and pathways that control NK cells within the tumor microenvironment, thereby strengthening them and mitigating the effects of pressures that suppress immune activity.
Cancer Cell and Gene Therapy Research Publications
- Journal for ImmunoTherapy of Cancer: Tandem CAR-T cells targeting mesothelin and MUC16 overcome tumor heterogeneity by targeting one antigen at a time. Marcela Maus, MD, PhD (Massachusetts General Hospital) (ACGT Research Fellow).
- Nature: In vivo CRISPR screens identify modifiers of CAR T cell function in myeloma. Marcela Maus, MD, PhD (Massachusetts General Hospital) (ACGT Research Fellow).
- Cell Reports Medicine: Clinical and molecular dissection of CAR T cell resistance in pancreatic cancer.Joseph Fraietta, PhD, Carl June, MD (University of Pennsylvania) (ACGT Research Fellow).
- JCO Cancer Oncology: Next-Generation Sequencing of Intraoperatively Acquired Cerebrospinal Fluid and Matched Tumor Tissue in Patients Undergoing Surgical Resection for Glioblastoma. Nduka Amankulor, MD (University of Pennsylvania) (ACGT SAC member).
- Immunity: Oxidative-stress-induced telomere instability drives T cell dysfunction in cancer. Greg Delgoffe, PhD (University of Pittsburgh) (ACGT Research Fellow).
- Neuro-Oncology Advances: TRLS-02 Cerebrospinal Fluid-Derived Tumor Reactive T Cells (CSF-TRT Cells) for the Treatment of Melanoma Leptomeningeal Disease. Shari Pilon-Thomas, PhD (Moffitt Cancer Center) (ACGT SAC member).
- National Library of Medicine: Second signals for cancer immunotherapy. Michael T. Lotze, MD (University of Pittsburgh) (ACGT SAC member).
- Neuro–Oncology: Single-cell profiling of peripheral and local immune compartments reveal unique genotype-independent prognostic immune signatures across IDH-stratified glioma. Nduka Amankulor, MD (University of Pennsylvania) (ACGT SAC member).
- Therapeutics: Oncolytic viruses as anticancer agents: clinical progress and remaining challenges. E. Antonio Chiocca, MD, PhD (Brigham and Women’s Hospital) (ACGT SAC member).
- Cancer Research: Targeting Chronic Inflammation in Ovarian Cancer Through Immune Engineering. Michel Sadelain, MD, PhD (Columbia University) (ACGT SAC member).
