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description: Current gene therapies cost 2-4 million dollars per treatment using ex vivo editing but in vivo approaches like Verve's one-time PCSK9 base editing infusion showing 53 percent LDL reduction could reach 50-200K by 2035 making curative medicine scalable
type: claim
domain: health
created: 2026-02-17
source: "IGI CRISPR clinical trials update 2025; BioPharma Dive Verve PCSK9 data; BioInformant FDA-approved CGT database; GEN reimbursement outlook 2025; PMC gene therapy pipeline analysis"
confidence: likely
related:
- FDA is replacing animal testing with AI models and organ on chip as the default preclinical pathway which will compress drug development timelines and reduce the 90 percent clinical failure rate
reweave_edges:
- FDA is replacing animal testing with AI models and organ on chip as the default preclinical pathway which will compress drug development timelines and reduce the 90 percent clinical failure rate|related|2026-03-28
---

# gene editing is shifting from ex vivo to in vivo delivery via lipid nanoparticles which will reduce curative therapy costs from millions to hundreds of thousands per treatment

As of early 2026, 46 cell and gene therapies have FDA approval, with prices concentrated in the $2-4M range: Casgevy ($2.2M for sickle cell), Lyfgenia ($3.1M), Zolgensma ($2.1M for SMA), Hemgenix ($3.5M for hemophilia B). These are all ex vivo therapies -- harvest cells, edit them, reinfuse -- requiring complex per-patient manufacturing that drives costs.

The shift to in vivo delivery changes the economics entirely. Verve Therapeutics' VERVE-102 demonstrated the paradigm: a one-time IV infusion of lipid nanoparticle-delivered base editors targeting PCSK9 in the liver reduced LDL cholesterol by 53% and PCSK9 protein by 60% at the highest dose. If validated at scale, a single infusion could replace decades of statin therapy. Eli Lilly is collaborating on Phase 2 trials. Beyond cardiovascular disease, base editing showed >60% fetal hemoglobin induction in 7 sickle cell patients (Beam Therapeutics), and the first prime editing clinical trial was cleared for chronic granulomatous disease (Prime Medicine, May 2024).

The technology progression runs from CRISPR-Cas9 (double-strand breaks) to base editing (single letter changes without breaks) to prime editing (precise insertions, deletions, all 12 point mutations without breaks). Each generation increases precision and reduces off-target risk. The pipeline contains 2,500+ active CGT INDs and ~1,300 gene therapy INDs.

LNP-based in vivo therapies could reach the $50-200K range by 2032-2035, making them cost-competitive with lifetime chronic disease management. Diseases already functionally cured include sickle cell, beta thalassemia, SMA, hemophilia A and B, and Wiskott-Aldrich syndrome. By 2035, familial hypercholesterolemia, hereditary angioedema, glycogen storage disease, and select inherited retinal dystrophies will likely join the list.

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Relevant Notes:
- [[the healthcare cost curve bends up through 2035 because new curative and screening capabilities create more treatable conditions faster than prices decline]] -- gene therapy front-loading creates enormous single-year expenditures even as it eliminates lifetime chronic costs
- [[GLP-1 receptor agonists are the largest therapeutic category launch in pharmaceutical history but their chronic use model makes the net cost impact inflationary through 2035]] -- gene editing's one-time cure model is the structural opposite of GLP-1's chronic use model
- [[AI compresses drug discovery timelines by 30-40 percent but has not yet improved the 90 percent clinical failure rate that determines industry economics]] -- AI accelerates target identification but gene editing provides the delivery mechanism for curative interventions

Topics:
- health and wellness
