Vertex PharmaceuticalsIn Development
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.
Latest update Jul 4, 2026
01
In a pivotal Phase 1/2/3 program with FDA RMAT and Fast Track, EMA PRIME, and a UK Innovation Passport. All 10 participants in the reported cohort became insulin-independent at one year with >90% time-in-range; global regulatory submissions are expected in 2026.
02
03
Announced next steps from Vertex Pharmaceuticals that mention Zimislecel (VX-880).
2026–2027
Target2026–2027 (at risk)
File global regulatory submissions for the stem-cell islet therapy that has produced insulin-independence - potential functional cures - in trial participants, a landmark for cell therapy. Vertex temporarily postponed completion of dosing in early 2026 pending an internal manufacturing analysis, has since completed that analysis and resumed dosing, and continues to enroll and dose patients in the Phase 1/2/3 study. In parallel, the FDA cleared an IND for VX-017, a 'type O' universal-donor islet cell therapy, and Vertex is now weighing bringing VX-017 to market at or near the same time as zimislecel rather than sequencing the two. The company says it will provide updated regulatory and commercial timelines for the whole type 1 diabetes portfolio later in 2026; no BLA/MAA has been filed for either program as of late August 2026.
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.
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.
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
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