FDA-Approved Cell & Gene Therapy
Revolutionary treatments using living cells and genetic modification to cure previously untreatable diseases. The first CAR-T therapy was approved in 2017, marking a new era in medicine.
Most Expensive Therapies
Gene therapies represent the most expensive drugs ever created. These one-time treatments aim to cure diseases previously considered untreatable, with prices reflecting R&D costs and small patient populations.
Recently Approved (4)
Cell and gene therapy approvals have accelerated in recent years as the field matures.
What is Cell & Gene Therapy?
Cell and gene therapies represent a paradigm shift from treating symptoms to curing diseases at their root cause. These advanced therapies modify the patient's own cells or deliver functional genes to fix genetic defects.
CGT Clinical Trial Pipeline
The CGT pipeline is rapidly expanding with over 681 actively recruiting trials. CAR-T therapies for solid tumors, next-gen gene editing (CRISPR, base editing), and in-vivo gene therapies represent the most active areas of development.
Explore CGT Trials on ClinicalTrials.govMolecular Targets & Vectors
Top Targets
CD19 dominates CAR-T (B-cell cancers), BCMA for myeloma. Gene therapies target specific mutated genes.
Delivery Vectors
AAV vectors dominate in-vivo gene therapy. Lentivirus used for ex-vivo modification. CRISPR is newest.
CAR-T Cell Therapies
(7 products)
CAR-T Cell Therapies
(7 products)CAR-T therapies reprogram the patient's own immune cells to fight cancer. T cells are extracted, genetically modified to express a chimeric antigen receptor (CAR), expanded, and infused back into the patient. All currently approved CAR-T therapies target blood cancers.
First FDA-approved CAR-T therapy, targeting CD19 for blood cancers
CAR-T therapy for aggressive lymphomas
CAR-T for mantle cell lymphoma and ALL
CAR-T with defined CD4/CD8 ratio for lymphoma
First CAR-T for multiple myeloma, targets BCMA
BCMA-targeting CAR-T for relapsed myeloma
CAR-T for adult B-cell ALL with novel binding domain
Gene Therapies
(17 products)
Gene Therapies
(17 products)Gene therapies deliver functional copies of genes using viral vectors (AAV, lentivirus) to fix genetic mutations causing inherited diseases. Many are one-time treatments with the potential to be curative for conditions previously considered untreatable.
First gene therapy for inherited blindness
One-time gene therapy for SMA, one of the most expensive drugs
Gene therapy for transfusion-dependent beta-thalassemia
Gene therapy for early cerebral adrenoleukodystrophy
Most expensive drug at launch, gene therapy for hemophilia B
Intravesical gene therapy delivering interferon to bladder
Gene therapy for severe hemophilia A
Gene therapy for DMD, delivers micro-dystrophin
Gene therapy for sickle cell disease with anti-sickling gene
First CRISPR-based gene therapy approved by FDA
Gene therapy for MLD, delivers arylsulfatase A gene
Gene therapy for hemophilia B
Topical gene therapy for wound healing in butterfly skin disease
First oncolytic virus therapy, injected directly into melanoma tumors
Gene therapy for aromatic L-amino acid decarboxylase deficiency
Intrathecal formulation of Zolgensma for older SMA patients
Encapsulated cell technology delivering growth factors to retina
Cell Therapies
(11 products)
Cell Therapies
(11 products)Cell therapies transplant living cells (stem cells, islet cells, fibroblasts, tumor-infiltrating lymphocytes) to restore biological functions the body has lost. Unlike CAR-T, these cells are not genetically modified.
First TIL therapy approved, uses patient's own tumor-infiltrating lymphocytes
First TCR therapy approved, targets MAGE-A4 in solid tumors
Expanded cord blood cells for faster engraftment in transplant
First cellular therapy for Type 1 diabetes using donor islet cells
First FDA-approved cancer immunotherapy (autologous dendritic cells)
Autologous chondrocyte implant for knee cartilage repair
Living cellular scaffold for gum tissue regeneration
Autologous fibroblast therapy for wrinkle improvement
Bioengineered skin substitute for burn treatment
Thymus tissue implant to regenerate immune system in athymic patients
Mesenchymal stromal cells for graft-vs-host disease
Cord Blood Products
(4 products)
Cord Blood Products
(4 products)Cord blood products contain hematopoietic progenitor cells (HPCs) derived from umbilical cord blood, used for stem cell transplants in patients with blood disorders, cancers, and immune deficiencies.
Cord blood stem cells for transplant in blood disorders
Cord blood stem cells for transplant in blood disorders
Cord blood stem cells for transplant in blood disorders
Cord blood stem cells for transplant in blood disorders
Other Advanced Therapies
(1 products)
Other Advanced Therapies
(1 products)Other FDA-approved advanced therapy products including tissue-engineered products and bioengineered materials.
Bioengineered human acellular vessel for dialysis access
CGT Approvals by Year
2017 marked the breakthrough year with the first CAR-T (Kymriah) and first gene therapy for inherited disease (Luxturna). Approvals have accelerated since, with 2023-2024 seeing record numbers of CGT approvals.
Resources
- FDA Approved CGT Products (Official List) - Source data
- ClinicalTrials.gov Gene Therapy Search - Pipeline trials
- Alliance for Regenerative Medicine (ARM) - Industry association
- American Society of Gene & Cell Therapy (ASGCT) - Scientific society
Data sourced from FDA CBER Approved Cellular and Gene Therapy Products list. Last updated: 2026-04-05.