Approved cell and gene therapy (CGT) products

The FDA’s drug approval process is designed to ensure that drugs are safe and effective for their intended use before they are made available to the public. Within the FDA, the Center for Biologics Evaluation and Research (CBER) reviews and 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. There are currently 11 gene and cell therapy products available in the US approved for treatment of 16 indications. The table below presents cell therapies as red; gene therapies as blue.


Approved indication


Kymriah (tisagenlecleucel)

Relapsed or refractory acute lymphoblastic leukemia

Yescarta (axicabtagene ciloleucel)
Kite Pharma, Inc.

Large B cell lymphoma

Luxturna (voretigene neparvovec-rzyl)
Spark Therapeutics, Inc.

Leber’s congenital amaurosis

Retinitis pigmentosa


Kymriah (tisagenlecleucel)
Novartis Pharmaceuticals

Large B cell lymphoma


Zolgensma (onasemnogene abeparvovedxiol)
Novartis Pharmaceuticals

Spinal Muscular Atrophy (SMA) Type 1


Tecartus (brexucabtageneciloleucel)
Kite Pharma, Inc.

Relapsed or refractory mantle cell lymphoma


Breyanzi (lisocabtagene maraleucel)
Bristol-Myers Squibb

Relapsed or refractory large B cell lymphoma

Abecma (Idecabtagene vicleucel)
Bristol-Myers Squibb

Relapsed or refractory multiple myeloma

Yescarta (axicabtagene ciloleucel)
Kite Pharma, Inc.

Follicular lymphoma

Tecartus (brexucabtageneciloleucel)
Kite Pharma, Inc.

Relapsed or refractory adult B-cell lymphoblastic leukemia


Carvykti (ciltacabtagene autoleucel)
Janssen Pharmaceuticals

Relapsed or refractory multiple myeloma

Kymriah (tisagenlecleucel)
Novartis Pharmaceuticals

Refractory follicular lymphoma

Zynteglo (betibeglogene autotemcel)
bluebird bio, Inc.


Skysona (elivaldogene autotemcel)
bluebird bio, Inc.

Cerebral adrenoleukodystrophy (CALD)

Hemgenix (etranacogene dezaparvovecdrib)
CSL Behring

Hemophilia B


Elevidys (delandistrogene moxeparvovec-rokl)
Sarepta Therapeutics

Duchenne muscular dystrophy

Roctavian (valoctocogene roxaparvovec)

Hemophilia A

NEWDIGS FoCUS maintains a pipeline analysis of clinical trials and FDA approvals of CGTs. Greater detail on therapies in the pipeline is detailed in the Pipeline Deep Dive. While clinical trial outcomes and regulatory approvals are never guaranteed, by 2026 we expect the following types of products may be available to patients:

  • Oncology
    • Additional therapies for B-cell leukemia and lymphoma
  • Rare disease treatments
    • Hematological conditions
      —Hemophilia A
      —Sickle cell anemia
    • Additional treatments for ophthalmological conditions
    • Neurological conditions
  • Higher prevalence disease treatments
    • Age-related wet macular degeneration
    • Osteoarthritis (knee)