Two University of Michigan cardiologists are asking whether medicine should be so quick to replace daily pills with one-time gene edits. In a Viewpoint in JAMA Internal Medicine, Eric Smith and Venkatesh Murthy note that such therapies permanently alter a person’s genetic activity, so they cannot be stopped or adjusted the way a prescription can. They also point out that some existing gene therapies carry list prices above $1 million per patient.
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Their question, as Michigan Medicine summarized it, is blunt: just because scientists can permanently change genetic activity, does that mean they should? The paper offers five tests for answering it.
Five questions to ask before a permanent genomic therapy
The Viewpoint, called “Clinical Framework for Durable Genomic Interventions” and listed on the journal’s site under DOI 10.1001/jamainternmed.2026.4690, lays out considerations for deciding when genomic therapy counts as a viable option. According to the ScienceDaily summary, the five questions are how severe the disease is, whether effective therapies exist that can be adjusted or stopped, whether the target tissue suits a one-dose intervention with lasting effects, whether the delivery risks are reasonable given the severity, and whether safety and effectiveness can be tracked for years, especially at large scale.
Smith, in the university’s account, called the monitoring question perhaps the most difficult and urgent of the five. Clinical trials of genomic therapies are small and short, so rare or delayed side effects can go undetected until thousands of people have been treated. The Viewpoint opens by observing that genomic therapy has moved into clinical practice, with hundreds of trials underway, which is the reason its authors want a shared yardstick now instead of after the first common-disease products arrive. They also stress that these treatments are somatic: they alter only the treated person’s own cells and are not meant to be inherited, so the concern is permanence within one patient rather than changes passed to children.
Casgevy, Lyfgenia and the million-dollar price tier
The authors write that some existing gene therapies carry list prices above $1 million per patient and that several cost substantially more. Their summary names no products, but public prices show the scale. Vertex Pharmaceuticals set a list price of $2.2 million for Casgevy and Bluebird bio set $3.1 million for Lyfgenia, both announced on Dec. 8, 2023, the day the FDA approved the two sickle cell treatments, according to Biopharma Dive.
Such therapies sit on the agency’s list of approved cellular and gene therapy products, which was current as of Oct. 30 and counts 55 licensed items, including cell-based, cord blood and tissue products as well as gene therapies. Gene therapy has helped treat spinal muscular atrophy and sickle cell disease, and the FDA list includes Zolgensma for the former alongside Casgevy and Lyfgenia for the latter. More than 6,000 rare genetic disorders have been identified, but only a small proportion have treatable targets.
Price is part of the authors’ argument about common disease, since a list price in the millions is a manageable outlier when a few thousand patients qualify and a very different proposition when the eligible population runs to millions. If a therapy for a widespread condition were priced the way rare disease products are, they warn, affordability and access could worsen health inequities.
When a gene edit competes with a daily pill
For rare, severe illnesses the case for a durable fix is more straightforward, because little else exists. The harder terrain is cardiovascular and metabolic medicine, where the Viewpoint points to durable silencing or editing of targets such as PCSK9, ANGPTL3 and TTR, with implications for atherosclerosis, dyslipidemia and amyloidosis. Early studies are testing whether a single treatment can durably lower LDL cholesterol, a condition patients manage today with daily medication that can be raised, lowered or stopped at any time.
That adjustability is the trade the authors want weighed. A daily medication works only while it is taken, which is a burden but also a safety valve. A permanent edit offers convenience for patients who struggle with chronic regimens and removes the valve. Benefits may not last, and immune reactions to viral delivery vehicles can block a second dose, according to Smith. Until biological off-switches are shown to be reliable, the authors argue, reversibility should count as a clinical benefit in its own right.
The reasoning also changes with scale. The authors note that rare side effects matter more when millions of people are treated, so a risk tolerable in a small group with a severe disease looks different when the same edit is offered for a common condition. Whether registries and long-term follow-up can be built to catch such events, and who will pay for them, is what the framework leaves to regulators and health systems to answer. The cardiology focus is no accident: Smith and Murthy are both in the University of Michigan’s Division of Cardiovascular Medicine, and the examples they raise come from the field they know best.
This article was produced with the assistance of AI and reviewed by Morning Overview editors prior to publication.
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