CRISPR Therapeutics and Vertex post $43 million CASGEVY revenue in Q1 2026 as pediatric submission lands with FDA Priority Voucher
First-quarter results from both partners show CASGEVY past 500 treatment starts, a US filing for ages 5 to under 12, and CRISPR Therapeutics' in vivo pipeline advancing across four liver-directed programs.
Overview
CRISPR Therapeutics and Vertex Pharmaceuticals each reported first-quarter 2026 financial results within 24 hours of one another, both attributing $43 million in revenue to CASGEVY, the gene-edited therapy the two companies co-developed for severe sickle cell disease and transfusion-dependent beta thalassemia. The matching figure, disclosed by CRISPR Therapeutics on May 4 according to GlobeNewswire and confirmed in Vertex’s release the next day according to BioSpace, arrived alongside fresh regulatory and pipeline milestones for both partners.
What we know
CASGEVY, the first FDA-approved CRISPR therapy, generated $43 million in first-quarter 2026 revenue, and “more than 500 people globally have now initiated the CASGEVY treatment journey,” according to the CRISPR Therapeutics release published on GlobeNewswire. The therapy is approved in the United States, Canada, the United Kingdom, the European Union, Switzerland, the Kingdom of Saudi Arabia, the Kingdom of Bahrain, Qatar, the United Arab Emirates, and Kuwait for patients 12 years and older with severe sickle cell disease or transfusion-dependent beta thalassemia, BioSpace’s mirror of the CRISPR Therapeutics release reports.
Vertex, which leads the commercial rollout, said it completed the U.S. regulatory submission for CASGEVY in children ages 5 to less than 12 years with sickle cell disease or transfusion-dependent beta thalassemia, and that the FDA awarded a Commissioner’s National Priority Voucher signaling expedited review, according to BioSpace. The company also disclosed a pricing agreement for CASGEVY in Germany and said it is implementing long-term reimbursed access there.
The addressable population now covers more than 60,000 eligible patients across approved markets, including approximately 37,000 in North America and Europe and more than 23,000 in the Middle East, BioSpace reports from the Vertex release.
For CRISPR Therapeutics, Q1 2026 net loss narrowed to $122.9 million from $136.0 million a year earlier, with cash, cash equivalents, and marketable securities of $2,441.8 million as of March 31, 2026, up from $1,975.8 million at year-end 2025, according to GlobeNewswire. The increase came primarily from $585.4 million in net proceeds from a convertible senior notes issuance in March 2026.
Vertex reported total Q1 2026 revenue of $2.99 billion, an 8% increase from the prior-year quarter, and reiterated its full-year 2026 revenue guidance of $12.95 billion to $13.1 billion, including more than $500 million in non-cystic-fibrosis revenue, according to BioSpace. Reshma Kewalramani, M.D., Vertex’s CEO and President, said the company is “off to a strong start in 2026, driven by leadership in cystic fibrosis; growth in sickle cell disease, beta thalassemia, and acute pain; as well as rapid pipeline progress.”
In vivo pipeline advances
Beyond CASGEVY, CRISPR Therapeutics highlighted progress on four in vivo liver-directed programs in its release on GlobeNewswire:
- CTX310, an investigational therapy targeting angiopoietin-related protein 3 (ANGPTL3), is progressing in a Phase 1b clinical trial. The company said it “recently received FDA clearance of its Investigational New Drug (IND) application,” supporting expansion of the ongoing trial into the United States. BioSpace notes the trial is prioritizing severe hypertriglyceridemia and refractory hypercholesterolemia.
- CTX340, targeting angiotensinogen, is in the IND/CTA-enabling phase, with clinical trial initiation expected in the first half of 2026.
- CTX460, targeting SERPINA1 for alpha-1 antitrypsin deficiency, is in IND/CTA-enabling studies, with clinical trial initiation expected in mid-2026.
- CTX321, targeting LPA, is also progressing through IND/CTA-enabling studies and incorporates an optimized guide RNA that the company said “delivered approximately two-fold greater potency” relative to the prior candidate.
The company also reported progress on zugo-cel (CTX112), its CD19-directed CAR-T therapy. According to GlobeNewswire, more than 14 patients have been dosed across systemic sclerosis, inflammatory myositis, and systemic lupus erythematosus, with over 10 clinical trial sites activated globally. The first and second SLE patients “remained in DORIS remission through Month 12 and Month 6, respectively.”
Samarth Kulkarni, Ph.D., CRISPR Therapeutics’ Chairman and CEO, framed the quarter in his prepared statement: “The first quarter reflected continued execution across CRISPR Therapeutics’ platform. We expanded zugo-cel into new autoimmune indications and advanced multiple in vivo liver-directed programs toward the clinic,” according to BioSpace.
What we don’t know
Neither release breaks out CASGEVY revenue by geography or by indication, leaving open how much of the $43 million reflects U.S. demand versus the newer Middle East and European launches. The releases also do not specify a target action date for the FDA pediatric submission; the Commissioner’s National Priority Voucher signals expedited review but does not, on its face, fix a deadline. Updates on the in vivo cardiovascular pipeline are expected in the second half of 2026, according to GlobeNewswire.
Context
The Machine Herald previously reported that CASGEVY remains the only CRISPR-based therapy approved by the FDA, with all other in vivo CRISPR programs still in earlier stages of clinical development. With more than 500 patients now initiated on CASGEVY and the pediatric submission under priority review, the Q1 2026 results offer one of the clearest readouts to date on whether ex vivo gene editing can scale into a durable commercial product.