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Gene & Cell Therapies

Also called advanced therapy medicinal products, ATMPs, CAR-T

Gene and cell therapies treat disease by changing cells rather than by dosing the body with a drug. Some deliver a working copy of a faulty gene using a viral vector. Some edit the patient's own DNA. CAR-T therapies remove a patient's T-cells, re-engineer them in a lab to recognise cancer, and infuse them back. They are typically one-time treatments — and among the most expensive medicines ever launched.

What this class is used for
Spinal muscular atrophy, haemophilia B, sickle cell disease, B-cell lymphomas and leukaemias

5 Gene & Cell Therapies in this directory

Zolgensma
Onasemnogene Abeparvovec
A one-time gene therapy for spinal muscular atrophy in infants.
Yescarta
Axicabtagene Ciloleucel
An intravenous gene-modified cellular therapy for certain types of advanced lymphoma.
Kymriah
Tisagenlecleucel
An intravenous gene-modified cellular immunotherapy for leukemia and lymphoma.
Casgevy
Exagamglogene Autotemcel
A single-dose intravenous gene-edited cellular therapy for sickle cell disease and transfusion-dependent beta thalassemia.
Hemgenix
Etranacogene Dezaparvovec
A single-dose intravenous gene therapy used to treat adults with severe Hemophilia B.

Browse other drug classes

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Class descriptions are written by the Priya Life Science editorial team. Individual drug pages combine that summary with live label, approval, manufacturer and shortage data from the U.S. FDA via the openFDA API. This page is general information and is not medical advice — it is not exhaustive, drugs within a class are not automatically interchangeable, and approvals and brand names differ between the US, EU/Ireland (EMA/HPRA) and other regions. Always consult the official prescribing information and your clinician or pharmacist. Related: Drug Shortages Tracker · FDA Approvals · All drug comparisons