How do the 12 FDA-approved gene therapies break down by medical indication

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The 12 FDA-approved gene therapies cover a range of medical indications, primarily focusing on genetic disorders, cancers, and hematological conditions. Here's a breakdown of these therapies by their medical indications:

  1. Genetic Disorders:

    • Luxturna (voretigene neparvovec-rzyl): Approved for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, which leads to vision loss and may cause complete blindness 1.
    • Zolgensma (onasemnogene abeparvovec-xioi): Indicated for pediatric patients with spinal muscular atrophy (SMA), a genetic disorder that affects muscle control 3.
    • Strimvelis: For the treatment of patients with adenosine deaminase severe combined immunodeficiency (ADA-SCID), a rare genetic disorder that results in a severely compromised immune system 3.
    • Vyjuvek (beremagene geperpavec): Approved for the treatment of dystrophic epidermolysis bullosa, which affects the skin and other organs 6 8.

  2. Cancers:

    • Kymriah (tisagenlecleucel): Used for treating B-cell precursor acute lymphoblastic leukemia (ALL) and relapsed or refractory large B-cell lymphoma 1 4.
    • Yescarta (axicabtagene ciloleucel): Approved for the treatment of large B-cell lymphoma after two or more lines of systemic therapy 1 4.
    • Tecartus (brexucabtagene autoleucel): Indicated for mantle cell lymphoma and B-cell lymphoblastic leukemia 1 4.
    • Breyanzi (lisocabtagene maraleucel): Used for treating relapsed or refractory large B-cell lymphoma 4.
    • Imlygic (Talimogene laherparepvec): Approved for the treatment of melanoma lesions in the skin and lymph nodes 3.

  3. Hematological Conditions:

    • Zynteglo (betibeglogene autotemcel): Treats beta-thalassemia, a blood disorder that reduces the production of hemoglobin 6 8.
    • Libmeldy (atidarsagene autotemcel): Used for treating metachromatic leukodystrophy (MLD), a metabolic disorder 3.
    • Skysona (elivaldogene autotemcel): Approved for cerebral adrenoleukodystrophy, a genetic neurological disorder 3.

These therapies represent significant advancements in treating conditions that previously had limited or no effective treatments, offering new hope to patients with severe and often life-threatening diseases. Each therapy utilizes different mechanisms, such as replacing, inactivating, or introducing genes, to address the underlying causes of these conditions 10.

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