Vertex PharmaceuticalsActive
The first approved CRISPR/Cas9 gene-editing medicine (exagamglogene autotemcel), a one-time therapy for sickle cell disease and transfusion-dependent beta thalassemia, developed and commercialized with CRISPR Therapeutics.
Latest update Jul 22, 2026
01
Approved across multiple jurisdictions and priced at ~$2.2M per one-time treatment. Vertex books the revenue (~$116M in FY2025) and is scaling a global network of authorized treatment centers; on July 1, 2026 the US label expanded to patients ages 2+ (from 12+) for both sickle cell disease and transfusion-dependent beta thalassemia - the first gene therapy cleared for children as young as two - and Vertex guides Casgevy revenue to roughly triple in 2026.
02
03
The US Court of Appeals for the District of Columbia Circuit on September 4, 2026 affirmed a lower-court finding that Vertex's program offering fertility-preservation services to Casgevy patients violates the federal Anti-Kickback Statute. The court said the perk was remuneration meant to induce purchase of the multimillion-dollar sickle-cell gene therapy. Bloomberg Law reported the panel stopped short of affirming a separate lower-court conclusion, leaving Vertex to recast how it supports patients through Casgevy's fertility risks.
The U.S. FDA on July 1 approved expanded use of Casgevy (exagamglogene autotemcel) - the world's first approved CRISPR/Cas9 gene-editing medicine, developed by Vertex Pharmaceuticals with CRISPR Therapeutics - for patients ages 2 and older with sickle cell disease or transfusion-dependent beta thalassemia, lowering the prior age floor of 12. Vertex said the expansion makes roughly 5,500 additional U.S. children newly eligible for the one-time therapy. CRISPR shares rose about 8% and Vertex about 6% on the news; CRISPR retains roughly 40% of Casgevy's economics under the partnership.
04
Vertex's flagship cystic fibrosis triple-combination CFTR modulator (elexacaftor/tezacaftor/ivacaftor), effective for roughly 90% of CF patients and the company's dominant revenue engine - now complemented by the once-daily next-generation Alyftrek.
A first-in-class oral, non-opioid pain medicine (VX-548) that selectively inhibits the NaV1.8 sodium channel to block pain signals in the peripheral nervous system before they reach the brain - the first new class of pain drug in over 20 years.
An allogeneic, stem-cell-derived, fully differentiated islet cell therapy for type 1 diabetes designed to restore the body's own insulin production - a potential functional cure delivered as a one-time infusion.
An oral, once-daily somatostatin receptor agonist for acromegaly, acquired through the Crinetics Pharmaceuticals deal - Vertex's first marketed product outside CF, pain, and gene therapy, and the anchor of its new endocrine-disease business.
05
Oklo
Oklo's fast-fission powerhouse product line is designed for build-own-operate deployment, targeting customers that want firm clean power in a smaller footprint than traditional large reactors.
Oklo
Oklo is developing an advanced fuel center to recycle used nuclear fuel and fabricate metal fuel for Aurora and other fast-reactor applications.
Oklo
Oklo's isotope business combines the Idaho Radiochemistry Laboratory, a planned pilot reactor in Texas, and a future multi-reactor foundry to supply critical medical, industrial, research, defense, and space isotopes.
TerraPower
Sodium-cooled fast reactor with integrated molten salt energy storage, enabling flexible output from 345 MWe to 500 MWe peak.