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AlveoGene Receives Rare Pediatric Disease Designation (RPDD) from FDA for AVG-002, Its Novel, Inhaled Gene Therapy for Lethal Neonatal Surfactant Protein B (SP-B) Deficiency

November 15, 2024

[AlveoGene] AlveoGene announced that it has been granted a RPDD by the US FDA for AVG-002, its novel inhaled gene therapy for lethal neonatal SP-B deficiency.

Intravenous and Intracranial GD2-CAR T Cells for H3K27M+ Diffuse Midline Gliomas

November 13, 2024

[Nature] Arm A of Phase I trial no. NCT04196413 administered one intravenous dose of autologous GD2-CART to patients with H3K27M-mutant pontine or spinal DMG at two dose levels following lymphodepleting chemotherapy.

Maturation and Persistence of CAR T Cells Derived from Human Pluripotent Stem Cells via Chemical Inhibition of G9a/GLP

November 6, 2024

[Cell Stem Cell] Using a stroma-free human iPSC-T cell differentiation platform, researchers screened for epigenetic modulators that influenced T cell specification and identified the H3K9-directed histone methyltransferases G9a/GLP as repressors of T cell fate.

PD-1+CD8+ T Cell-Mediated Hepatocyte Pyroptosis Promotes Progression of Murine Autoimmune Liver Disease

November 4, 2024

[Advanced Science] Researchers identified a specific expanded pathogenic subset of PD-1+CD8+ T cells in the liver, exhibiting strong functional activity and cytotoxicity against target cells.

Engraftment of a Surrogate Antigen onto Tumor Cell Surface via pHLIP Peptide to Universally Target CAR-T Cell Therapy to Solid Tumors

November 4, 2024

[Cancer Letters] The authors evaluated the efficiency of Myc-pH-low insertion peptide (pHLIP) in inserting the artificial c-Myc-tag onto the plasma membrane of malignant cells and determined if this engraftment could convert it into a therapeutic target for CAR-T cells or monoclonal antibodies.

Large DNA Deletions Occur During DNA Repair at 20-Fold Lower Frequency for Base Editors and Prime Editors than for Cas9 Nucleases

November 4, 2024

[Nature Biomedical Engineering] Scientists showed that Cas9-mediated double-strand breaks induced large deletions at varying frequencies in cancer cell lines, human embryonic stem cells, and human primary T cells, and that most deletions were produced by two repair pathways.

Phenotypic Insights into Genetic Risk Factors for Immune-Related Adverse Events in Cancer Immunotherapy

November 4, 2024

[Cancer Immunology Immunotherapy] Utilizing publicly accessible genome-wide association study data, this investigation evaluated the impact of over 5000 exposure variables on susceptibility to irAEs using univariate Mendelian randomization.

Navigating the Roadblocks: Progress and Challenges in Cell-Based Therapies for Human Immunodeficiency Virus

November 4, 2024

[Journal of Cellular Biochemistry] The authors address the challenges to cell-based therapies against HIV and discusses the recent advancements aimed at overcoming them.

CAR T Cells and T Cell Therapies for Cancer

November 4, 2024

[Jama-Journal of The American Medical Association] CAR T-cell therapy is an FDA-approved therapy that has improved progression-free survival for multiple myeloma, improved overall survival for large B-cell lymphoma, and attained high rates of cancer remission for other hematologic malignancies.

Focusing on Exosomes to Overcome the Existing Bottlenecks of CAR-T Cell Therapy

November 4, 2024

[Inflammation and Regeneration] Investigators delve into the effect of tumor-derived exosomes on the CAR-T cell function and discuss how inhibiting their secretion can enhance the efficacy of CAR-T cell therapy.

Allogene Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ALLO-316, an AlloCAR T™ Investigational Product for Adult Patients with Advanced or Metastatic...

November 4, 2024

[Allogene Therapeutics, Inc. (GlobenewsWire)] Allogene Therapeutics, Inc. announced that the US FDA granted RMAT designation to ALLO-316 for the treatment of adult patients with CD70 positive advanced or metastatic renal cell carcinoma.

‘It Was a Good Lesson’: FDA Reconsiders CAR-T Boxed Warning on Secondary Cancers, Peter Marks Says

November 4, 2024

[Fierce Pharma] An FDA announcement last year led to classwide black boxed warnings on existing CAR-T products’ labels. Now FDA is reconsidering those serious safety warnings with plans to change them potentially, Dr. Peter Marks, said in an interview with Fierce Pharma.

Published since 2002, Cell Therapy News is our longest running online publication. Our editorial team curates the top publications, reviews, and news on in vivo gene therapy, cell therapy, and immunotherapy research. With over 15,000 subscribers and followers across our website, email newsletter, and Twitter feed, we are proud to facilitate the communication of cutting-edge cell therapy innovations.