CRISPR Therapeutics reports Q2 2026 results, expanded CASGEVY approval and new trial starts

CRISPR Therapeutics AG reported second quarter 2026 results on August 3, 2026, highlighting a broadened U.S. approval for CASGEVY (exagamglogene autotemcel) alongside progress across its in vivo and cell therapy pipeline.

The FDA extended CASGEVY's approval to children 2 years of age and older with sickle cell disease or transfusion-dependent beta thalassemia, making it the first genetic therapy indicated for children as young as 2 for both conditions, a decision reached 53 days after filing and opening eligibility to roughly 5,500 additional patients.¹ Regulatory submissions have also been completed in Saudi Arabia and the United Kingdom to extend CASGEVY to patients 5 to 11 years old.¹ CASGEVY generated $76 million in revenue for the quarter, up 78% from the prior quarter and 151% year over year.¹

In the pipeline, CTX340, targeting angiotensinogen for refractory hypertension, received FDA IND clearance and entered a Phase 1 trial.¹ CTX460, the company's first candidate from its SyNTase editing platform, targeting SERPINA1 for alpha-1 antitrypsin deficiency, also began a Phase 1 trial.¹ CRISPR Therapeutics said data from the Phase 1a trial of CTX310, its ANGPTL3 program, will be presented at the European Society of Cardiology Congress on August 28, with a Phase 1b update expected in the second half of 2026.¹ The company also initiated a combination study of zugocabtagene geleucel with pirtobrutinib in aggressive B-cell lymphomas under its Lilly collaboration, with further zugo-cel updates expected later in 2026.¹

On finances, cash, cash equivalents, and marketable securities totaled $2,364.4 million as of June 30, 2026, up from $1,975.8 million at year-end 2025, driven mainly by $585.4 million in net proceeds from convertible senior notes issued in March 2026, offset by operating expenses.¹ Net loss for the quarter was $91.2 million, compared with $208.5 million in the same period last year.¹