Vertex, CRISPR Set Lofty Goal for Casgevy Gene Therapy as Patient Starts Ramp

Why It Matters

The forecast marks the first sizable profit trajectory for a CRISPR‑based gene therapy, proving the commercial viability of genome‑editing medicines. Rapid uptake and expanding reimbursement are reshaping the sickle‑cell and beta‑thalassemia markets.

Key Takeaways

• Casgevy projected $500M revenue with Journavx in 2026.
• Q4 2025 sales hit $54.8M, beating expectations.
• 111 patients started infusions in 2025, triple 2024.
• 90% US patients have reimbursed insurance coverage.
• Label expansion to ages 5‑11 filing underway.

Pulse Analysis

The approval of Casgevy in late 2023 marked a watershed moment for CRISPR‑based therapeutics, positioning Vertex and CRISPR Therapeutics at the forefront of a nascent market that has long struggled to translate scientific breakthroughs into commercial success. As investors watch the broader gene‑editing landscape, the duo’s ability to move from a $10 million first‑year revenue to a projected $500 million combined sales figure within three years underscores the scaling potential of precision medicine when paired with robust commercial infrastructure.

Vertex’s fourth‑quarter report revealed $54.8 million in sales, surpassing consensus expectations, and a full‑year revenue of $115.8 million for Casgevy. The therapy’s patient pipeline accelerated dramatically, with 111 individuals initiating infusions in 2025—nearly three times the 2024 cohort. Reimbursement dynamics are also improving; roughly 90 % of U.S. patients now have covered access, and the drug is approved in ten international markets, including a recent Scottish indication for beta‑thalassemia. A pending label expansion to the 5‑11 age group could further broaden the addressable population and reinforce the therapy’s market position.

The broader implication for the biotech sector is clear: a successful, revenue‑generating CRISPR product validates the economic model for genome‑editing platforms, encouraging capital inflows and accelerating pipeline development across the industry. While short‑term revenue volatility persists due to the multi‑step treatment journey, analysts anticipate smoother cash flows post‑2027 as patient volumes mature. Stakeholders—from investors to payers—will closely monitor Casgevy’s trajectory as a bellwether for the commercial viability of next‑generation gene therapies.

Vertex, CRISPR Set Lofty Goal for Casgevy Gene Therapy as Patient Starts Ramp

September 11, 2024 · 7 min read · Therapeutic Growth

Vertex believes the gene therapy will join with new pain medication Journavx for $500 million in combined revenue for 2026—a 185 % increase.

This prognostication marks a change in fortune for the gene therapy, which has been dogged by slow patient uptake, as no patients had even received the therapy more than half a year after its approval.

Growth has come in dribs and drabs so far, but analysts are evidently encouraged by fourth‑quarter reports of revenue for the sickle‑cell disease and transfusion‑dependent beta thalassemia gene therapy from both companies.

Still, what makes Vertex executives so confident about a jump to $500 million in combined Casgevy/Journavx sales?

“We do feel very confident about that number and have great line of sight to the year,” said COO and CFO Charles Wagner, during Vertex’s fourth‑quarter earnings call on Thursday afternoon. “With Casgevy, we had a strong year with 300 or so patients initiating—150 or so having first cell collections. And given the length of the patient journey, that gives us great visibility into the year. So we’re very confident that Casgevy will ramp up nicely compared to 2025.”

No patients have received Casgevy, CRISPR Therapeutics and Vertex Pharmaceuticals’ recently approved sickle‑cell gene therapy. Experts weigh in on the path to profit for the treatment and the therapeutic class in general.

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Casgevy brought in a total of $54.8 million in the fourth quarter of 2025 for both partners, beating consensus expectations of $38 million. William Blair said that they view Casgevy, as well as Vertex’s non‑opioid painkiller Journavx, “at relative inflection points.”

Casgevy’s full‑year earnings for 2025 were $115.8 million. Casgevy made $10 million total in 2024, its first full year of availability after getting its first approval for sickle‑cell disease in December 2023.

“We successfully moved from a foundational year in 2024 to a year of building significant momentum in 2025,” said Duncan McKechnie, Vertex’s chief commercial officer.

William Blair modeled Casgevy’s 2026 earnings at $344 million, $132 million of which will go to CRISPR with Vertex taking the remaining $212 million.

Expectations for Casgevy’s growth are based on 111 new patients beginning infusions in 2025, nearly tripling the number of patients treated in 2024, according to Vertex’s earnings report. The 2025 number includes 37 initial cell collections and 30 edited cell transfusions.

Now about 90 % of U.S. patients have reimbursed insurance access to treatment. Casgevy is also covered in 10 ex‑U.S. countries, including a 2025 approval in Scotland for use in beta thalassemia. Casgevy has been made available in other European nations and the Middle East, with a “robust flow of patients” so far, according to McKechnie.

Vertex is also filing to expand Casgevy’s label into patients aged 5 to 11 in the first half of the year, CEO Reshma Kewalramani said on the company’s fourth‑quarter and full‑year earnings call Thursday. Vertex received a Commissioner’s National Priority Review Voucher for Casgevy and expects a quick review for its label expansion.

Vertex recently presented Phase 3 data for this younger cohort, both in sickle‑cell and in beta thalassemia, at the American Society for Hematology conference in December 2025. Those results, William Blair wrote in a note at the time, demonstrated Casgevy’s efficacy “in young pediatric patients for the first time.”

Nevertheless, Vertex executives on the earnings call acknowledged “quarter‑to‑quarter variability” with Casgevy revenue. This is because of “the duration of the patient journey and given the fact that patients themselves dictate when they wish to receive their infusion,” McKechnie said.

“We anticipate this will smooth out in 2027 and beyond as the number of patients at all stages of the treatment journey continues to build.”

Vertex executives also noted that Casgevy revenue could remain volatile in the near term. Revenue from the therapy isn’t realized until a patient is infused with an edited batch of cells, which can come months after patients initiate treatment.

The sentiments around Casgevy’s growth echo comments made by CRISPR CEO Samarth Kulkarni at the J.P. Morgan Healthcare Conference in January:

“We’re very happy that we’ve reached the goal of over $100 million in revenue,” she said. “The revenues are ramping up, and we feel very bullish about Casgevy and its trajectory. And we want the momentum to continue in ’26.”