Table 3.
US FDA approved cellular and gene therapy products
| Name | Approval year | Category | Therapeutic use | Formulation | Trade name |
|---|---|---|---|---|---|
| Idecabtagene vicleucel | 2021 | B-cell maturation antigen (BCMA)-directed genetically modified autologous T cell immunotherapy | 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 | Suspension for intravenous infusion | ABECMA® |
| Ciltacabtagene autoleucel | 2022 | B-cell maturation antigen (BCMA)-directed chimeric antigen receptor t-cell therapy | Relapsed or refractory multiple myeloma (RRMM) after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. | Infusion | CARVYKTI™ |
| HPC, Cord Blood | 2013 | HPC (Hematopoietic Progenitor Cell), Cord Blood, is an allogeneic cord blood hematopoietic progenitor cell therapy | Unrelated donor hematopoietic progenitor cell transplantation procedures in conjunction with an appropriate preparative regimen for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment | Suspension for Intravenous Use | ALLOCORD |
| 2016 | CLEVECORD™ | ||||
| 2011 | HEMACORD | ||||
| 2012 | DUCORD | ||||
| Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen |
2012 | Allogeneic cellularized scaffold produc | Topical (non-submerged) application to a surgically created vascular wound bed in the treatment of mucogingival conditions in adults. | Cellular Sheet for Topical Oral Application | GINTUIT |
| Talimogene laherparepvec | 2015 | A genetically modified oncolytic viral therapy | Local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery | Suspension for intralesional injection | IMLYGIC |
| Azficel-T | 2011 | Autologous cellular product | For improvement of the appearance of moderate to severe nasolabial fold wrinkles in adults. | Suspension for Intradermal Injection | LAVIV® |
| Voretigene neparvovec-rzyl | 2017 | Adeno-associated virus vector-based gene therapy | Confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician | Intraocular suspension for subretinal injection | LUXTURNA |
| Autologous Cultured Chondrocytes on a Porcine Collagen Membrane | 2016 | Autologous cellularized scaffold product | Repair of symptomatic, single or multiple full-thickness cartilage defects of the knee with or without bone involvement in adults. | Cellular sheet for autologous implantation | MACI |
| Sipuleucel-T | 2010 | Autologous cellular immunotherapy | Treatment of asymptomatic or minimally symptomatic metastatic castrate resistant (hormone refractory) prostate cancer | Suspension for Intravenous Infusion | PROVENGE® |
| Allogeneic processed thymus tissue–agdc | 2021 | Allogeneic processed thymus tissue | Immune reconstitution in pediatric patients with congenital athymia | For surgical implantation | RETHYMIC |
| Allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen-dsa | 2021 | Allogeneic cellularized scaffold product (allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen-dsat) | Treatment of adults with thermal burns containing intact dermal elements for which surgical intervention is clinically indicated (deep partial-thickness burns) | Topical us | StrataGraft® |
| Onasemnogene abeparvovec-xioi | 2019 | Adeno-associated virus vector-based gene therapy | Treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene | Suspension, for intravenous infusion | ZOLGENSMA |
| Tisagenlecleucel | 2017 | CD19-directed genetically modified autologous T-cell immunotherapy | B-cell precursor acute lymphoblastic leukemia (ALL) | Suspension for intravenous infusion | Kymriah |
| Axicabtagene ciloleucel | 2017 | CD19-directed genetically modified autologous T cell immunotherapy | Relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy | Suspension for intravenous infusion | Yescarta |
| Brexucabtagene autoleucel | 2020 | CD19-directed genetically modified autologous T cell immunotherapy | Adult patients with relapsed or refractory mantle cell lymphoma (MCL) | Suspension for intravenous infusion | Tecartus |
| Lisocabtagene maraleucel | 2021 | CD19-directed genetically modified autologous T cell immunotherapy | 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) | Suspension for intravenous infusion | Breyanzi |