Cell Therapies
Mary Johnson (han at labome dot com)
Synatom Research, Princeton, New Jersey, United States
DOI
//dx.doi.org/10.13070/mm.en.14.3201
Date
last modified : 2025-05-30; original version : 2024-05-12
Cite as
MATER METHODS 2024;14:3201
Abstract

A select list of FDA-approved cell therapies.

2025
  • April 28, ZEVASKYN prademagene zamikeracel from Abeona Therapeutics. Indicated for treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa. ZEVASKYN consists of a patient's own cells that have been genemodified through RVV transduction to express the COL7A1 gene to produce the C7 protein.
2024
  • December 18, RYONCIL remestemcel-L-rknd from Mesoblast. Indicated for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older. The mechanism of action for RYONCIL is not clear.
  • November 8, AUCATZYL obecabtagene autoleucel from Autolus Inc. Indicated for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. AUCATZYL is a CD19-directed genetically modified autologous T cell immunotherapy consisting of the patient's own T cells expressing an anti-CD19 chimeric antigen receptor.
  • August 1, TECELRA afamitresgene autoleucel from Adaptimmune. Indicated for the treatment of adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive and whose tumor expresses the MAGE-A4 antigen as determined by FDA-approved or cleared companion diagnostic devices. TECELRA contains CD4+ and CD8+ T cells transduced with a self-inactivating lentiviral vector to express an affinity-enhanced T cell receptor specific for human MAGE-A4 on the cell surface.
  • March 18, LENMELDY atidarsagene autotemcel from Orchard Therapeutics / Kyowa Kirin. Indicated for treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy. LENMELDY contains autologous CD34+ cells ( hematopoietic stem cells) transduced with a lentiviral vector encoding the human arylsulfatase A (ARSA) gene.
  • February 16, AMTAGVI lifileucel from Iovance Biotherapeutics. Indicated for treatment of adults with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor. AMTAGVI (lifileucel) contains autologous T cells derived from resected patient tumor tissue prosected from one or more tumor lesions.
  • January 16, CASGEVY exagamglogene autotemcel (exa-cel) from Vertex Pharmaceuticals. Indicated for treatment of patients aged 12 years and older with transfusion-dependent beta-thalassemia. CASGEVY (exagamglogene autotemcel) contains autologous CD34+ hematopoietic stem cells edited by CRISPR/Cas9-technology at the erythroid specific enhancer region of the BCL11A gene to reduce BCL11A expression in erythroid lineage cells, leading to increased fetal hemoglobin protein production.
2023
  • December 8, CASGEVY exagamglogene autotemcel (exa-cel) from Vertex Pharmaceuticals. Indicated for treatment of sickle cell disease in patients 12 years of age or older with recurrent vaso-occlusive crises. CASGEVY (exagamglogene autotemcel) contains autologous CD34+ hematopoietic stem cells edited by CRISPR/Cas9-technology at the erythroid specific enhancer region of the BCL11A gene to reduce BCL11A expression in erythroid lineage cells, leading to increased fetal hemoglobin protein production.
  • December 8, LYFGENIA lovotibeglogene autotemcel (lovo-cel) from bluebird bio. Indicated for treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events. LYFGENIA (lovotibeglogene autotemcel) contains autologous CD34+ hematopoietic stem cells from patients with sickle cell disease transduced with BB305 lentiviral vector encoding beta-globin (A-T87Q).
  • June 28, LANTIDRA donislecel-jujn from CellTrans. Indicated for treatment of adults with Type 1 diabetes who are unable to approach target HbA1c because of current repeated episodes of severe hypoglycemia despite intensive diabetes management and education. LANTIDRA consists of a suspension of allogeneic pancreatic islets derived from a donor pancreas.
  • April 17, OMISIRGE omidubicel-onlv from Gamida Cell. Indicated for use in patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection. OMISIRGE (omidubicel-onlv) contains nicotinamide modified allogeneic hematopoietic progenitor cells derived from cord blood.
2022
  • September 16, SKYSONA elivaldogene autotemcel from bluebird bio. Indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy. SKYSONA contains autologous CD34+ hematopoietic stem cells transduced ex vivo with Lenti-D lentiviral vector carrying ABCD1 cDNA.
  • August 17, ZYNTEGLO betibeglogene autotemcel from bluebird bio. Indicated for treatment of patients with beta-thalassemia who require regular red blood cell transfusions. ZYNTEGLO contains autologous CD34+ hematopoietic stem cells transduced with BB305 lentiviral vector encoding beta-globin (A-T87Q).
  • February 28, CARVYKTI ciltacabtagene autoleucel from Janssen Biotech. Indicated for treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. CARVYKTI contains autologous mononuclear cells transduced with a lentiviral vector to express a chimeric antigen receptor comprising an anti- BCMA targeting domain, which consists of two single-domain antibodies linked to a 4-1BB costimulatory domain and a CD3-zeta signaling domain.
2021
  • March 26, ABECMA idecabtagene vicleucel from Celgene / Bristol-Myers Squibb. Indicated for treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody. ABECMA contains autologous T cells transduced with an anti-BCMA02 chimeric antigen receptor lentiviral vector.
  • February 5, BREYANZI lisocabtagene maraleucel from Juno Therapeutics / Bristol-Myers Squibb. Indicated for treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B. BREYANZI contains autologous T cells transduced with a chimeric antigen receptor comprised of the FMC63 monoclonal antibody-derived single-chain variable fragment (against CD19), IgG4 hinge region, CD28 transmembrane domain, 4-1BB costimulatory domain, and CD3 zeta activation domain.
2020
  • July 24, TECARTUS brexucabtagene autoleucel from Kite Pharmaceuticals / Gilead Sciences. Indicated for treatment of adult patients with relapsed or refractory mantle cell lymphoma. TECARTUS contains autologous T cells transduced to express a chimeric antigen receptor comprising a murine anti-CD19 single-chain variable fragment linked to CD28 and CD3-zeta co-stimulatory domains.
2017
  • October 18, YESCARTA axicabtagene ciloleucel from Kite Pharma / Gilead Sciences. Indicated for treatment of adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy, adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma, and adult patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy. YESCARTA contains autologous T cells genetically modified ex vivo by retroviral transduction to express a chimeric antigen receptor comprising a murine anti-CD19 single chain variable fragment linked to CD28 and CD3-zeta co-stimulatory domains.
  • August 30, KYMRIAH tisagenlecleucel from Novartis. Indicated for treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse, adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma, and adult patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy. KYMRIAH contains autologous T cells transduced with a lentiviral vector encoding an anti-CD19 chimeric antigen receptor.
ISSN : 2329-5139