Cell Therapy News
Cell therapy is one of the fastest-growing areas in modern medicine, with breakthroughs happening across research, clinical trials, and regulatory approvals. This page brings you a curated selection of the most important news shaping the future of cell therapy drug development. From scientific advances to key updates from regulatory agencies, we make it easy to stay informed on the developments that matter most.
bluebird bio has announced the the U.S. Food and Drug Administration (FDA) has approved ZYNTEGLO® (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. “The FDA approval of ZYNTEGLO offers people with beta-thalassemia the possibility of freedom from burdensome regular red blood cell transfusions and iron chelation, and unlocks new possibilities in their daily lives,” said Andrew Obenshain, chief executive officer, bluebird bio. “After more than a decade of research and clinical development, and through the perseverance [...]
Effort to address challenges to the manufacturing scale-up of cell-based therapies follows release of Project A-Gene for gene therapy in 2021 The Alliance for Regenerative Medicine (ARM) and the National Institute for Innovation in Manufacturing BioPharmaceuticals (NIIMBL) today released Project A-Cell, a multistakeholder collaboration to incorporate Quality by Design (QbD) principles into a manufacturing case study of a Chimeric Antigen Receptor T-cell (CAR-T) therapy. A-Cell brings best practices and a standard methodology for Chemistry, Manufacturing and Controls (CMC) to the cell-based therapy field, which has produced six FDA-approved CAR-T therapies to treat blood cancers but still faces challenges to manufacturing [...]
Scientists at St. Jude Children’s Research Hospital have developed a method that may be able to improve CAR T-cell therapies by identifying the early cells that become effective at killing cancer. St. Jude Children’s Research Hospital scientists have demonstrated that the long-term performance of cancer-killing chimeric antigen receptor (CAR) T cells can, in some cases, be predicted before treatment. The research provides a unique cell sequencing-based framework to measure and improve CAR T–cell immunotherapy performance. The report appears online early today in Cancer Discovery, a journal of the American Association for Cancer Research. CAR T cells work by harnessing a patient’s immune [...]
Graphite Bio is a clinical-stage, next-generation gene editing company harnessing the power of high-efficiency precision gene repair to develop therapies with the potential to treat or cure serious diseases. The FDA has just granted Fast Track Designation to GPH101 for the treatment of sickle cell disease (SCD). GPH101 is an investigational next-generation gene-edited autologous hematopoietic stem cell (HSC) therapy designed to directly correct the genetic mutation that causes SCD. GPH101 is the first investigational therapy to use a highly differentiated gene correction approach that seeks to efficiently and precisely correct the mutation in the beta-globin gene to decrease sickle [...]
The Center for Biologics Evaluation and Research (CBER) regulates cellular therapy products, human gene therapy products, and certain devices related to cell and gene therapy. CBER uses both the Public Health Service Act and the Federal Food Drug and Cosmetic Act as enabling statutes for oversight. Cellular therapy products include cellular immunotherapies, cancer vaccines, and other types of both autologous and allogeneic cells for certain therapeutic indications, including hematopoetic stem cells and adult and embryonic stem cells. Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells [...]