Bluebird Bio, now rebranded as Genetix Biotherapeutics, is a commercial-stage biotechnology company.
They appear twice among the makers of the 10 most expensive drugs in the U.S.
Skysona, which treats cerebral adrenoleukodystrophy, costs $3 million, while Zynteglo, a treatment for beta-thalassemia, costs $2.8 million.
The FDA approved both therapies on September 16, 2022. Each is a one-time gene therapy made using the patient’s own stem cells.
They remove the cells, genetically modify them outside the body, and then infuse them back into the patient.
Having two of the 10 most expensive drugs come from the same company, both approved on the same day, highlights how concentrated the gene-therapy market is.
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| wdt_ID | wdt_created_by | wdt_created_at | wdt_last_edited_by | wdt_last_edited_at | Rank | Drug | Manufacturer | Cost | Condition | Simple terms |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | Patience | 22/09/2026 03:32 PM | Patience | 22/09/2026 03:32 PM | 1 | Lenmeldy | Orchard Therapeutics (Kyowa Kirin) | $4.25M one-time | Metachromatic leukodystrophy (MLD) in children | Inherited brain and nerve disease |
| 2 | Patience | 22/09/2026 03:32 PM | Patience | 22/09/2026 03:32 PM | 2 | Hemgenix | CSL Behring | $3.5M one-time | Hemophilia B | Blood-clotting disorder |
| 3 | Patience | 22/09/2026 03:32 PM | Patience | 22/09/2026 03:32 PM | 3 | Elevidys | Sarepta Therapeutics | $3.2M one-time | Duchenne muscular dystrophy | Muscle-wasting disease |
| 4 | Patience | 22/09/2026 03:32 PM | Patience | 22/09/2026 03:32 PM | 4 | Skysona | bluebird bio | $3M one-time | Cerebral adrenoleukodystrophy | Inherited brain disease |
| 5 | Patience | 22/09/2026 03:32 PM | Patience | 22/09/2026 03:32 PM | 5 | Zynteglo | bluebird bio | $2.8M one-time | Beta-thalassemia | Inherited blood disorder |
| 6 | Patience | 22/09/2026 03:32 PM | Patience | 22/09/2026 03:32 PM | 6 | Zolgensma | Novartis | $2.125M one-time | Spinal muscular atrophy | Muscle-weakening nerve disease |
| 7 | Patience | 22/09/2026 03:32 PM | Patience | 22/09/2026 03:32 PM | 7 | Myalept | Chiesi Farmaceutici | ~$1.3M annually | Lipodystrophy | Body-fat disorder |
| 8 | Patience | 22/09/2026 03:32 PM | Patience | 22/09/2026 03:32 PM | 8 | Danyelza | Y-mAbs Therapeutics | ~$1.2M annually | Neuroblastoma | Childhood nerve cancer |
| 9 | Patience | 22/09/2026 03:32 PM | Patience | 22/09/2026 03:32 PM | 9 | Zokinvy | Eiger BioPharma | ~$1.2M annually | Progeria | Rapid-aging disorder in children |
| 10 | Patience | 22/09/2026 03:32 PM | Patience | 22/09/2026 03:32 PM | 10 | Kimmtrak | Immunocore | ~$1.1M annually | Uveal melanoma | Eye cancer |
Source:
Drugs.com
