The definitive guide to gene editing.
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Nasdaq: VRTX · Boston, Massachusetts

Vertex Pharmaceuticals

The established biotechnology company that partnered on, and commercialises, the first approved CRISPR medicine.

Approved Large biotechnology

Pipeline

Where each disclosed programme stands. Stages come from the company's own disclosures on the date shown.

ProgrammeDiseaseTechnologyStage
Casgevy Sickle cell disease / beta thalassemia CRISPR-Cas9 DiscoveryPreclinicalPhase IPhase IIPhase IIIApproved

Pipeline as recorded in the Atlas on Aug 25, 2026. Companies revise pipelines frequently — verify against the company's own materials and regulatory filings.

In one paragraph

Vertex is a large, profitable biotechnology company best known for transforming cystic fibrosis treatment. It partnered with CRISPR Therapeutics on Casgevy and leads its commercialisation — the part of bringing a gene-editing medicine to patients that requires global regulatory, manufacturing and reimbursement capability rather than laboratory science.

Vertex Pharmaceuticals, founded in 1989, is a commercial-stage biotechnology company with a franchise in cystic fibrosis modulators. It collaborated with CRISPR Therapeutics on exagamglogene autotemcel and leads its development and commercialisation. Its presence in the field illustrates a structural point: the scientific origin of an editing medicine and the organisation capable of delivering it globally are frequently not the same company.

Sources

Not investment advice This profile is educational information compiled from public disclosures. It is not a recommendation to buy, sell or hold any security, and it is not a view on the company's prospects. Biotechnology is highly speculative and most clinical programmes fail. Full disclaimers →

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