Gene therapy can cure some diseases with a single treatment, yet the companies that make these drugs are still searching for a business model that pays. The problem is simple to state and hard to solve: a cure ends the need for further treatment, which also ends the revenue that would have paid for the next drug. The result has been high prices, slow sales and a string of companies pulling back from the field.
Prices show the scale of the problem. Every gene therapy on the market in the United States is a one-time treatment, with list prices from $500,000 to $4.25 million per patient. Lenmeldy, for metachromatic leukodystrophy, carries the highest list price in the country at $4.25 million. Fayuvi, approved by the Food and Drug Administration on 17 September 2026 for Sanfilippo syndrome type A, was priced at $3.95 million, level with Kebilidi for AADC deficiency.
Other approved treatments sit in the same range. Hemgenix, for haemophilia B, costs $3.5 million, Lyfgenia for sickle cell disease costs $3.1 million, and Casgevy, the first therapy using CRISPR gene editing, approved in December 2023 for sickle cell disease, launched at $2.2 million. Zolgensma, for spinal muscular atrophy, costs $2.125 million. Makers say a single infusion replaces a lifetime of care, such as years of factor replacement for haemophilia or repeated spinal injections.
Why gene therapy economics are hard
The warning came early. In 2018, Goldman Sachs published a report, The Genome Revolution, asking whether curing patients was a sustainable business model. Its analysts argued that a therapy that permanently removes a disease shrinks the pool of people who need it, and that Gilead Sciences’ hepatitis C drugs showed the pattern: highly effective cures cut the number of untreated patients, and sales fell after the franchise peaked.
Eight years later, the prediction looks partly right, though not for the reasons Goldman Sachs gave. Several gene therapies have struggled to find a market, and some treat only a handful of patients each year, too few to turn a profit. The Institute for Clinical and Economic Review, an independent group that estimates what a drug’s benefit is worth, has usually priced these treatments below their list prices. Its benchmark for a sickle cell gene therapy was $1.35 million to $2.05 million, against Casgevy’s $2.2 million launch price.
Payment rules are adding to the pressure. The Centers for Medicare and Medicaid Services said in a final rule filed on 5 November that a drug given once, such as a gene therapy, will count as 12 thirty-day supplies when Medicare sets prices in negotiation. The rule takes effect on 5 December. Industry groups said the method makes durable cures look far more expensive, because a single dose is spread across a year of monthly units.
How gene therapy makers are responding
Some companies are changing how they are organised rather than what they charge. Fondazione Telethon, an Italian nonprofit, picked up a gene therapy for Wiskott-Aldrich syndrome, etu-cel, after commercial developers stepped back, and won market approval in the United States and Europe in January. The first American patient was due to receive the treatment, sold as Waskyra, at the University of California, San Francisco, in the first week of November. The price was set at $3.25 million, which the groups said reflected their work to keep costs down.
Smaller health plans face the same strain from the other side. At the Academy of Managed Care Pharmacy meeting in November, Carmen Witsken of the Alliance of Community Health Plans said upfront claims of $2 million to $4 million strain smaller community-based plans, which may struggle to absorb a one-time claim while waiting for savings that arrive over years. She said no national system exists to finance gene therapy, and that access can depend on whether a plan can manage the expense.
What gene therapy needs next
The Centers for Medicare and Medicaid Services is opening its Cell and Gene Therapy Access Model, so far limited to sickle cell disease, to other cell and gene therapies. The Goldman Sachs report suggested three routes for companies: target large markets, target diseases with high incidence, and keep expanding their portfolios. UniQure, one of the companies still in the field, reported a third-quarter net loss of $61.2 million while its Huntington’s disease therapy waits for a Food and Drug Administration decision due by 1 May 2027.