A comprehensive list of current cell and gene therapy products available in different markets across the world.
This page lays out approved cell, gene, and tissue-engineered therapies, including approved regions and a summary of approved indications (diseases and conditions). Details such as pediatric vs. adult use, year approved, previous lines of treatment needed, and recent approvals/changes are not necessarily reflected. If you have questions about or suggested edits/additions to this list, please contact Adam Wolf.
This list, though not exhaustive, exclusively includes those products that have undergone the necessary scientifically-based, rigorous regulatory approval process, including in-human clinical trials where appropriate, overseen by internationally-recognized regulatory agencies, such as the U.S. FDA; the EU’s European Medicines Agency; Japan’s Pharmaceuticals and Medical Devices Agency; South Korea’s Ministry of Food and Drug Safety, among others.
Abecma is a chimeric antigen receptor (CAR) T-cell therapy for adults with relapsed or refractory multiple myeloma.
Approved in: The United States, Canada, Japan, and the European Union
Approved for: R/R multiple myeloma
Amtagvi is a tumor-infiltrating lymphocyte (TIL) cell therapy approved to treat metastatic melanoma.
Approved in: The United States
Approved for: Metastatic melanoma
APCeden is an autologous monocyte-derived mature dendritic cell vaccine.
Approved in: India
Approved for: Treatment of prostate cancer, ovarian cancer, colorectal cancer, and Non Small Cell Lung carcinoma
Breyanzi is a CAR T-cell therapy for adults with relapsed or refractory large B-cell lymphoma.
Approved in: The United States and the European Union
Approved for: R/R large B-cell lymphoma, R/R diffuse large B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B
Carteyva is an autologous anti-CD19 CAR-T cell immunotherapy drug.
Approved in: China
Approved for: R/R large B-cell lymphoma and R/R follicular lymphoma
Carvykti is a CAR-T cell therapy used for adult patients who have cancer of the bone marrow called multiple myeloma.
Approved in: Brazil, Canada, The United Kingdom, The United States and the European Union
Approved for: R/R multiple myeloma
CreaVax RCC is a dendritic cell based vaccine.
Approved in: Republic of Korea
Approved for: Treatment of metastatic renal cell carcinoma for which nephrectomy can be performed
Ebvallo is an allogeneic T-cell immunotherapy used to treat adult and pediatric patients two years of age and older with relapsed or refractory Epstein‑Barr virus positive post‑transplant lymphoproliferative disease. (EBV+ PTLD)
Approved in: The European Union
Approved for: Epstein‑Barr virus positive post‑transplant lymphoproliferative disease (EBV+ PTLD)
Immuncell-LC is a medicine for anticancer cell immunotherapy, made with T-lymphocyte incubated and activated after extraction from the blood of a patient.
Approved in: Republic of Korea
Approved for: Liver cancer (hepatocellular carcinoma)
Inaticabtagene Autoleucel is a CAR-T cell therapy for the treatment of relapsed and refractory B-cell acute lymphoblastic leukemia. (r/r B-ALL)
Approved in: China
Approved for: R/R B-cell acute lymphoblastic leukemia
Kymriah is a CAR T-cell therapy product to treat R/R follicular lymphoma.
Approved in: The United States, the European Union, Canada, Japan, and Singapore.
Approved for: B-cell acute lymphoblastic leukemia, chronic lymphoid leukemia, diffuse large B-cell lymphoma, R/R large B-cell lymphoma, and R/R follicular lymphoma
Provenge is an autologous cellular immunotherapy.
Approved in: The United States
Approved for: Asymptomatic or minimally symptomatic metastatic castrate resistant (hormone refractory) prostate cancer
Tecartus is a CAR-T therapy designed to treat mantle cell lymphoma.
Approved in: The United States and the European Union
Approved for: Mantle cell lymphoma and B-cell precursor acute lymphoblastic leukemia
Tecelra is a TCR cell therapy to treat adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA antigen(s) 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 authorized companion diagnostic devices.
Approved in: The United States
Approved for: Advanced synovial sarcoma
Yescarta is a CAR T-cell therapy product.
Approved in: The United States, the European Union, Canada, Japan, and China
Approved for: B cell malignancies such as non-Hodgkin lymphoma, R/R follicular lymphoma, acute lymphoblastic leukemia, mantle cell lymphoma, chronic lymphoid leukemia, R/R large B-cell lymphoma and diffuse large B-Cell lymphoma
Adstiladrin is a adenoviral vector-based gene therapy used to treat adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.
Approved in: The United States
Approved for: High-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ
Beqvez is a AAV-based gene therapy to treat adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.
Approved in: The United States and the European Union
Approved for: Hemophilia B
Casgevy is a CRISPR gene editing therapy for the treatment of sickle cell disease patients with recurrent vaso-occlusive crises or transfusion-dependent beta-thalassemia (TDT).
Approved in: The United States, the European Union, the United Kingdom, Saudi Arabia, and Bahrain
Approved for: Sickle-cell disease and transfusion-dependent beta-thalassaemia
Collategene is a gene therapy consisting of an intramuscular injection of plasmid (DNA) coding for Hepatocyte Growth Factor (HGF), intended to grow blood vessels.
Approved in: Japan
Approved for: Critical limb ischemia
Elevidys is an adeno-associated virus-based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.
Approved in: The United States
Approved for: Duchenne Muscular Dystrophy in patients aged 4 through 5.
Gendicine is a recombinant adenovirus engineered to express wildtype-p53 (rAd-p53), designed to treat patients with tumors which have mutated p53 genes. Gendicine is the first gene therapy product approved for clinical use in humans.
Approved in: China
Approved for: Head and neck squamous cell cancer
Hemgenix is an adeno-associated virus vector-based gene therapy to treat adults with Hemophilia B.
Approved in: The European Union, Switzerland, the United Kingdom, Canada, and the United States
Approved for: Hemophilia B
Imlygic is a weakened form of Herpes Simplex Virus Type 1, which is commonly called the cold sore virus.
Approved in: The United States, the European Union, and Australia
Approved for: Unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery
A lentiviral vector-based gene therapy for the treatment of metachromatic leukodystrophy (MLD). It is approved as “Lenmeldy” in the United States.
Approved in: The European Union, the United States, and Switzerland
Approved for: Metachromatic leukodystrophy (MLD)
Luxturna is an adeno-associated viral vector gene therapy.
Approved in: The United States, the European Union, Japan, and Canada
Approved for: RPE65-mediated inherited retinal dystrophies
Lyfgenia is a lentiviral vector gene therapy to treat sickle cell disease in patients ages 12 and older who have a history of vaso-occlusive events.
Approved in: The United States
Approved for: Sickle-cell disease
Roctavian is a gene therapy for the treatment of severe hemophilia A in adult patients.
Approved in: The European Union and the United States
Approved for: Hemophilia A
Skysona is gene therapy made using a genetically modified autologous CD34+ cell-enriched population that contains hematopoietic stem cells.
Approved in: The United States
Approved for: Active cerebral adrenoleukodystrophy (CALD)
Strimvelis is an ex-vivo stem cell gene therapy which uses retroviral vector encoding adenosine deaminase gene transfer into hematopoietic stem/progenitor cells.
Approved in: The European Union
Approved for: Adenosine deaminase severe combined immune deficiency
Upstaza is a gene therapy used to treat severe aromatic L-amino acid decarboxylase (AADC) deficiency.
Approved in: The European Union
Approved for: Aromatic L‑amino acid decarboxylase (AADC) deficiency
VYJUVEK is a redosable gene therapy to treat dystrophic epidermolysis bullosa.
Approved in: The United States
Approved for: Dystrophic epidermolysis bullosa
Zolgensma (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based gene therapy.
Approved in: The United States, Japan, the European Union, Canada, Australia, and the Republic of Korea
Approved for: Spinal muscular atrophy (SMA)
Zynteglo is an ex-vivo lentiviral based gene therapy used to provide a functional copy of the the β-globin gene into a patient’s hematopoietic (blood) stem cells.
Approved in: The United States (Withdrawn in the European Union in 2021)
Approved for: ß-thalassemia who require regular red blood cell (RBC) transfusions
Alofisel is an allogeneic stem cell therapy to treat complex perianal fistulas in patients with Crohn’s disease.
Approved in: The European Union
Approved for: Complex perianal fistulas in patients with Crohn’s disease
Cartistem is a cellular therapeutic agent containing allogeneic human umbilical cord blood-derived mesenchymal stem cells.
Approved in: Republic of Korea
Approved for: Knee cartilage defects such as traumatic articular cartilage, degenerative arthritis and rheumatoid arthritis
Cellgram-AMI is an autologous intracoronary bone marrow-derived mesenchymal stem cell injection for the treatment acute myocardial infarction.
Approved in: Republic of Korea
Approved for: Acute myocardial infarction
Cupistem is an autologous adipose derived mesenchymal stem cell treatment to reduce inflammation and regenerate damage joint tissues.
Approved in: Republic of Korea
Approved for: Crohn’s fistula
CureSkin is an autologous dermal fibroblast cell-based therapy.
Approved in: Republic of Korea
Approved for: Treatment of depressed acne scars
FUCASO is a BCMA CAR-T therapy for the treatment of relapsed or refractory multiple myeloma.
Approved in: China
Approved for: Relapsed or refractory multiple myeloma.
Holoclar is a cell therapy based on autologous cultures of limbal stem cells. It regenerates a functional corneal epithelium allowing recovery of visual acuity.
Approved in: The European Union
Approved for: Moderate to severe limbal stem cell deficiency due to ocular burns
Kaloderm is an allogeneic cell therapy for deep second degree burns and diabetic foot ulcers.
Approved in: Republic of Korea
Approved for: Burns and diabetic foot ulcers
KeraHeal is an autologous keratinocyte based cell therapy.
Approved in: Republic of Korea
Approved for: Treatment of 2nd degree burns
KeraHeal-Allo is a hydrogel-type allogeneic keratinocyte therapy product.
Approved in: Republic of Korea
Approved for: Treatment of second-degree burns.
Lantidra is an allogeneic (donor) pancreatic islet cellular therapy made from deceased donor pancreatic cells for the treatment of type 1 diabetes.
Approved in: The United States
Approved for: Adults with type 1 diabetes who are unable to approach target glycated hemoglobin (average blood glucose levels)
LaViv is an autologous fibroblast-based cell therapy.
Approved in: United States
Approved for: Improvement of the appearance of moderate to severe nasolabial fold wrinkles in adults
Neuronata-r is an autologous bone marrow mesenchymal stem cell.
Approved in: Republic of Korea
Approved for: Amyotrophic lateral sclerosis (ALS)
Omisirge is a NAM-enabled stem cell therapy for hematologic malignancies that are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection.
Approved in: The United States
Approved for: Hematologic malignancies
Queencell is an autologous mesenchymal stem cell for the treatment of connective tissue disorders.
Approved in: Republic of Korea
Approved for: Connective tissue disorders
Rosmir is autologous cell therapy.
Approved in: Republic of Korea
Approved for: Treatment of under eye wrinkles
Spherox is a product containing spheroids of human autologous chondrocytes for use in cartilage defects.
Approved in: The European Union
Approved for: Cartilage defects
Stemirac is a mesenchymal stem cell therapy.
Approved in: Japan
Approved for: Treatment of spinal cord injury
Stempeucel is an ex-vivo cultured adult allogeneic mesenchymal stromal cell therapy.
Approved in: India
Approved for: Critical Limb Ischemia
TEMCELL is an allogeneic mesenchymal stem cell product.
Approved in: Japan, Canada and New Zealand
Approved for: Acute radiation injury, chronic obstructive pulmonary disease, Crohn’s disease, acute graft-versus-host disease, Type I diabetes and myocardial infarction
Vyznova is a cell therapy for the treatment of bullous keratopathy of the cornea.
Approved in: Japan
Approved for: Bullous keratopathy of the cornea
CLEVECORD is used for 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.
Approved in: The United States
Approved for: Unrelated donor hematopoietic progenitor cell transplantation procedures
HPC Cord Blood is used for 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.
Approved in: The United States
Approved for: Unrelated donor hematopoietic progenitor cell transplantation procedures
HPC Cord Blood is used in 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.
Approved in: The United States
Approved for: Unrelated donor hematopoietic progenitor cell transplantation procedures
Hemacord is used for 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.
Approved in: The United States
Approved for: Unrelated donor hematopoietic progenitor cell transplantation procedures
Allocord is an allogeneic cord blood hematopoietic progenitor cell therapy indicated for use in 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.
Approved in: The United States
Approved for: Unrelated donor hematopoietic progenitor cell transplantation procedures
Ducord is an allogeneic cord blood hematopoietic progenitor cell therapy indicated for use in 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.
Approved in: The United States
Approved for: Unrelated donor hematopoietic progenitor cell transplantation procedures
Clinimmune’s HPC Cord Blood is an allogeneic cord blood hematopoietic progenitor cell therapy indicated for use in 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.
Approved in: The United States
Approved for: Unrelated donor hematopoietic progenitor cell transplantation procedures
Life South’s HPC Cord Blood is an allogeneic cord blood hematopoietic progenitor cell therapy indicated for use in 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.
Approved in: The United States
Approved for: Unrelated donor hematopoietic progenitor cell transplantation procedures
Aurix is a biodynamic hematogel composed of platelet-rich plasma gel prepared from a small sample of a patient’s own platelets and plasma as a catalyst for healing indicated for the treatment of wounds.
Approved in: The United States
Approved for: Treatment of wounds
Apligraf is a bi-layered living skin substitute made from a dermal layer of human cells (fibroblasts) in a bovine type I collagen and an overlying cornified epidermal layer of living human keratinocytes.
Approved in: The United States
Approved for: Treatment of chronic venous leg ulcers and diabetic foot ulcer
Dermagraft is a dermal substitute used to help in the wound closure of diabetic foot ulcers. It is made from human cells (fibroblasts), placed on a dissolvable mesh material.
Approved in: The United States
Approved for: Chronic foot ulcers in patients with diabetes
Epicel is a permanent skin replacement product grown from a patient’s own skin cells. The autologous keratinocytes are co-cultured with irradiated murine cells to form cultured epidermal autografts (CEA).
Approved in: The United States
Approved for: Deep dermal or full thickness burns
Heart Sheet is an autologous skeletal myoblast preparation.
Approved in: Japan
Approved for: Treatment of patients with serious heart failure
Holloderm is a cultured epidermal autograft composed by culturing autologous keratinocytes. It is transplanted to the wound and aids in regeneration of the dermis and develops into new skin.
Approved in: Republic of Korea
Approved for: Treatment of skin disorders such as burns, vitiligo, nevi and scars
Hyalograft 3D is a cell therapy which cultivates autologous skin fibroblasts in 3D scaffolds formed of hyaluronic acid derivatives for the treatment of diabetic foot ulcers.
Approved in: Republic of Korea
Approved for: Diabetic foot ulcers
JACC is a combination product of autologous cultured chondrocytes and collagen gel.
Approved in: Japan
Approved for: Traumatic cartilage defect or osteochondritis dissecans of the knee
JACE is an epidermal cell sheet produced from keratinocytes isolated from a patient’s own skin tissue.
Approved in: Japan
Approved for: Deep dermal and full-thickness burns covering 30% or more of the total body surface area; and treatment of giant congenital melanocytic nevi
MACI is an autologous cellularized scaffold product, indicated for the repair of single or multiple symptomatic, full-thickness cartilage defects of the knee with or without bone involvement in adults.
Approved in: The United States
Approved for: Single or multiple symptomatic, full-thickness cartilage defects of the knee
Novocart 3D consists of three-dimensional collagen-chondroitin sulphate scaffolds with embedded autologous chondrocytes in a cell suspension.
Approved in: The European Union
Approved for: Articular cartilage repair
Ortho-ACI is an autologous chondroctye-based cellularized scaffold to treat symptomatic defects of the articulating cartilage of the joints, predominately the knee and ankle.
Approved in: Australia
Approved for: Symptomatic defects
Ossron is an autologous bone cell implantation for the treatment of bone defects in patients caused by degeneration, drugs, intense physical stress, diet, genetics, obesity, smoking, alcohol or disease.
Approved in: Republic of Korea and India
Approved for: Bone defects
An autologous cell harvesting device that enables a to create a regenerative epithelial suspension using the sample of the patient’s skin for the treatment of ulcers.
Approved in: The European Union
Approved for: Treatment of ulcers
ReNovaCell is an autologous cell harvesting device that enables a to create a regenerative epithelial suspension using the sample of the patient’s skin for the treatment of skin discoloration.
Approved in: The European Union
Approved for: Treatment of skin discoloration
Rethymic is a one-time tissue therapy to treat immune reconstitution in pediatric patients with congenital athymia.
Approved in: The United States
Approved for: Immune reconstitution in patients with congenital athymia
Startagraft (Allogeneic cultured keratinocytes and dermal fibroblast in murine collgen-dsat) is a allogeneic cellularized scaffold product.
Approved in: The United States
Approved for: Deep partial-thickness burns
Transcyte is a tissue-engineered skin substitute made from a nylon mesh and a silastic semi permissible and biocompatible layer.
Approved in: The United States
Approved for: The treatment of Epidermolysis Bullosa
