Approved Gene Therapies: The FDA and EMA List

Gene therapy is no longer an experimental idea — there are now officially approved treatments being given to patients. This page renders approval records directly from the FollowGene database, and every row links to its official regulatory file.

What is gene therapy, and what does "approved" mean?

Gene therapy targets the genetic cause of a disease directly. A working copy of a missing or faulty gene can be delivered into cells, or the gene itself can be edited inside the cell. "Approved" means the therapy completed clinical trials and received authorisation from a regulator — the FDA in the United States, the EMA in Europe — meaning its safety and efficacy data were independently reviewed.

Approval does not mean the therapy is right for every patient, or available in every country. Authorisation is always granted for a specific indication (a disease and patient group), and reimbursement is a separate national decision.

List of approved gene therapies

The table below is generated from approval records in the FollowGene database and updated by scheduled syncs. Each entry is shown with its official approval number and source link.

The main technologies in use

AAV-based gene delivery

Adeno-associated virus (AAV) vectors are harmless, engineered viruses used to carry a healthy copy of a gene into cells. Zolgensma (SMA) and Luxturna (RPE65-related retinal dystrophy) both use this approach. They are given as a single dose, and the delivered gene copy tends to persist in non-dividing target cells.

CRISPR-Cas9 gene editing

CRISPR-Cas9 makes a targeted cut in the cell's own DNA to change how a gene behaves. In sickle cell disease and beta-thalassaemia, Casgevy targets a regulatory region of the BCL11A gene to restart fetal haemoglobin production — instead of repairing the faulty gene itself, it makes the body produce an alternative haemoglobin.

Ex vivo cell therapy

Here the patient's own stem cells are collected, modified genetically in the laboratory, and returned to the patient. Casgevy, Zynteglo and Skysona work this way. The process usually requires conditioning chemotherapy and is delivered at specialised centres.

Approved therapy vs. clinical trial

• Approved therapy: authorised by a regulator and can be given as standard care for its specific indication.
• Clinical trial: still under investigation; participation depends on eligibility criteria and follows the study protocol.
• Expanded access (compassionate use): an exceptional route before approval for patients without other options. Rules vary by country and company.

Where to go next

Each gene page brings together the approvals, active clinical trials, variants and recent publications for that gene. These genes have approved therapies: