Promising phase 1 results for Legend’s in vivo CAR T
Legend Biotech’s in vivo CAR-T cell therapy has reduced or eliminated signs of lymphoma in all recipients, according to phase 1 data due to be presented at EHA 2026.
Of the six patients who received the higher dose, five had a complete response to LB2501, a CD19/CD20 dual‑targeting in vivo CAR T therapy for B‑cell malignancies. Eight of the participants experienced cytokine release syndrome, but only one needed an intervention beyond basic symptomatic treatments for fever, pain or nausea.
Legend’s shares rose 30 percent following the news. Source
Kelonia reveals promising phase 1 in vivo CAR T data
Kelonia Therapeutics’ in vivo BCMA CAR T therapy has eliminated signs of disease in all 18 patients with relapsed or refractory multiple myeloma one month after treatment.
Patients assessed six months after treatment still had no cancer, and the patient with the longest follow-up – beyond 10 months – also had no minimal residual disease.
Eli Lillu acquired Kelonia – alongside in vivo developer Orna Therapeutics – earlier this year. Source
iPSC therapy beefs up failing hearts
Induced pluripotent stem cell-derived engineered heart muscle can remuscularize failing myocardium and improve function in patients with heart failure and a reduced left ventricular ejection fraction, according to a phase 1-2 study.
A total of 20 patients were treated with biologic ventricular assist tissue (BioVAT), which is formulated from engineered heart muscle composed of cardiomyocytes and stromal cells derived from allogeneic iPSCs.
BioVAT transplantation resulted in increases in the target heart-wall thickness, left ventricular ejection fraction, and quality of life. “Longer-term follow-up and further clinical investigation are warranted,” the researchers, based in Germany, concluded. Source
Dual CAR T opens doors for kidney patients
CAR-T treatment has enabled two patients with end-stage kidney disease to receive previously improbable kidney transplants.
The researchers at the University of Pennsylvania, NYU Langone, and Mass General used dual CAR-T cell therapy to reduce the level of harmful immune antibodies in the two highly sensitized patients. The approach combines CD19‑targeted CAR T-cells, which eliminate memory B cells, and BCMA‑targeted CAR T cells, which deplete antibody‑producing plasma cells.
“This is the first demonstration that CAR T cells can be used not only to treat cancer, but also to help patients who previously had no opportunity to receive a compatible donor kidney,” said Ali Naji, the Jonathan E. Rhoads Professor of Surgery and principal investigator of the study. “For patients who have spent years on the kidney transplant waiting list, this approach could be transformative.” Source
Restoring CAR T’s navigation system
Researchers have developed a strategy to help CAR T cells migrate into lymph nodes – a significant obstacle for treating B-cell malignancies with lymph node involvement – and kill lymphoma cells in mice.
“Many lymphomas grow primarily in lymph nodes,” said lead author Maria Zschummel. “If therapeutic immune cells cannot reach these sites efficiently, even powerful therapies may fail.”
The approach involved engineering the CAR T cells to express the chemokine receptor CCR7, crucial for nodal homing, which had become downregulated during manufacturing.
In mouse models, these cells also eliminated lymphoma cells more effectively than conventional CAR T cells. “This was a surprise,” says Zschummel. “We expected improved migration, but CCR7 also boosted the cells’ killing efficiency.” Source
Irradiation keeps CAR T cells in the fight against solid tumors
Irradiation can help CAR T cells survive longer and work more effectively inside solid tumors, according to a preclinical study.
In mouse models of advanced lung cancer and melanoma, irradiation promoted dendritic cells to capture intact tumor surface proteins and display them on their own membranes, a process called "antigen dressing." These antigen-dressed dendritic cells then engaged the chimeric receptor on the CAR T cells, keeping them alive and multiplying within the tumor over several weeks.
The result was durable control of advanced lung tumors that CAR T cells alone could not eliminate.
"We found that dendritic cells can dress themselves in tumor proteins and use them to directly expand CAR T cells through the engineered receptor,” said lead author Jalal Ahmed from Mount Sinai, USA. “This was completely unexpected – dendritic cells normally engage T cells through an entirely different mechanism.” Source
A SMArTer – and safer – CRISPR?
Researchers have developed a new strategy to improve the precision and safety of CRISPR-Cas9 gene editing in human blood stem cells.
The study introduces SMArT (“Selection by Means of Artificial Transactivators”), which transiently implements AND reporter gates to achieve templated integration of a functional cassette at the target site.
The researchers used SMArT to enrich edited hematopoietic stem and progenitor cells (HSPCs) to 80-100 percent purity, while removing cells carrying unintended and potentially harmful genomic alterations generated during editing.
“Unintended outcomes, such as large deletions of DNA sequences, have emerged as one of the most important limitations to the broader application of gene editing, especially in stem cells intended for transplantation,” said Luigi Naldini from the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), who led the study. “With SMArT, we aimed to create an intelligent selection system capable of identifying and enriching only those cells that achieved the desired genetic correction while excluding cells carrying potentially dangerous alterations.” Source
FDA proposes using existing knowledge to accelerate gene therapy development
The US FDA has issued draft guidance that proposes allowing cell and gene therapy developers to make greater use of existing scientific and regulatory knowledge.
The draft guidance outlines how sponsors can use publicly available information and established platform knowledge, including chemistry, manufacturing and controls (CMC) data, nonclinical study results and clinical information, to streamline regulatory submissions for human gene therapy products that use genome editing in human somatic cells.
“By outlining how sponsors can intelligently build upon existing nonclinical, clinical, and manufacturing knowledge, we can meaningfully streamline development programs and lower the cost barriers that have historically slowed access to these potentially life-changing treatments,” said Vijay Kumar Acting Director of the Office of Therapeutic Products in CBER. Source
T-CURX acquires Pantherna to accelerate in vivo CAR T development
German cell therapy developer T-CURX has agreed to acquire Pantherna Therapeutics, a German biotechnology company specializing in mRNA engineering and lipid nanoparticle delivery technologies.
“We are taking a decisive step forward to accelerate the clinical development of differentiated, next-generation in vivo CAR-T therapies,” said Ulf Grawunder, CEO and co-founder of T-CURX. Source
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