CRISPR Therapeutics posts $99m CASGEVY quarter as pipeline advances
CRISPR Therapeutics has reported third-quarter 2026 revenue attributable to CASGEVY of $99 million, a 30% rise on the prior quarter and almost six times the figure recorded in Q3 2025. The company said more than 750 patients have initiated treatment with the gene-edited cell therapy worldwide, with the patient volume in the first nine months of 2026 already doubling the full-year 2025 total.
CASGEVY (exagamglogene autotemcel), developed and commercialised in partnership with Vertex Pharmaceuticals on a 60/40 profit-and-cost-sharing arrangement, is approved in 39 countries for eligible patients aged 12 and over with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). Its footprint among younger patients is expanding: the FDA approved it in July for children aged two and over, the MHRA granted approval for children five and over in the UK, and regulators in Saudi Arabia, the UAE, and Bahrain have similarly approved it for the two-and-over age group.
Pipeline highlights
Beyond CASGEVY, the company's in vivo liver-editing programme is gaining regulatory traction. The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designations to CTX310, its ANGPTL3-targeting candidate, for both severe hypertriglyceridemia and elevated LDL-C. RMAT status carries the interaction and priority-review benefits of Fast Track and Breakthrough Therapy designations, which could shorten the path to a potential approval. Phase 1a durability data published in the New England Journal of Medicine in August showed a single course of CTX310 produced mean reductions from baseline of 79% in ANGPTL3, 48% in triglycerides, and 53% in LDL-C at the highest dose, sustained at one year. Phase 1b data in patients with severe hypertriglyceridemia are set to be presented at the American Heart Association Scientific Sessions on 9 November 2026.
Enrolment in the Phase 2 trial of CTX611, a long-acting siRNA targeting Factor XI developed with Sirius Therapeutics, is complete in patients undergoing total knee arthroplasty, with a readout expected in the second half of 2026. In autoimmune disease, allogeneic CAR-T candidate zugocabtagene geleucel (zugo-cel) is being evaluated across basket trials covering lupus, systemic sclerosis, inflammatory myositis, and several neuroimmune conditions; Phase 1 data in systemic sclerosis will be presented at ACR Convergence on 8 November.
Market context and financial position
The cardiovascular gene-editing space is intensifying. Several companies, including Intellia Therapeutics and Verve Therapeutics, are pursuing in vivo liver-directed approaches to lipid lowering, with ANGPTL3, PCSK9, and other targets under investigation. CTX310's RMAT designations and New England Journal of Medicine publication will increase scrutiny of its longer-term safety and durability profile relative to these programmes. The FDA's willingness to grant dual RMAT designations across two distinct cardiovascular indications signals meaningful early confidence in the modality.
On the financials, CRISPR Therapeutics held $2.28 billion in cash, equivalents, and marketable securities at 30 September 2026, up from $1.98 billion at year-end 2025, partly reflecting $585 million in convertible senior notes issued in March. Net loss for the quarter was $111.4 million, broadly flat year-on-year despite higher R&D spending of $71.9 million. The company also announced two C-suite appointments: Sivakesava Sakhamuri joins as Chief Technical Officer, bringing prior experience at Orna Therapeutics, and A.J. Silver was confirmed as Chief Financial Officer following the departure of Raju Prasad.
With multiple data packages due before year-end and its in vivo CAR-T platform progressing through IND-enabling studies, CRISPR Therapeutics is entering what chief executive Samarth Kulkarni described as "a pivotal period" for the broader pipeline.