CRISPR Therapeutics AG

What they do

CRISPR Therapeutics develops gene-based medicines across four franchises: hemoglobinopathies, in vivo editing, CAR T, and regenerative medicine. Its lead product CASGEVY is a non-viral, ex vivo CRISPR/Cas9 therapy that edits the BCL11A enhancer in a patient's own hematopoietic stem cells and is advanced with Vertex Pharmaceuticals. The company also runs liver-directed in vivo programs using lipid nanoparticle delivery and siRNA programs with Sirius Therapeutics, and is developing the SyNTase editing platform.

Revenue drivers

• CASGEVY (exagamglogene autotemcel) — Approved CRISPR/Cas9 therapy for SCD and TDT, developed and commercialized with Vertex. CASGEVY generated $76 million in Q2 2026 revenue, up 78% quarter-over-quarter and 151% year-over-year, and is approved in 39 countries. This is the company's only commercial product.
• Collaboration revenue — Historically included amounts from the Vertex partnership and other arrangements. Total annual revenue declined from $371.2 million in 2023 to $37.3 million in 2024 and $3.5 million in 2025.
• Pipeline and platform programs — Pre-commercial assets include CTX310 (ANGPTL3), CTX340 (AGT, refractory hypertension), CTX460 (SERPINA1, AATD), CTX321 (next-generation LPA), and CTX611 (FXI siRNA with Sirius Therapeutics). These are in Phase 1, Phase 1b, or Phase 2 and do not currently generate product revenue.

Recent performance

Second quarter 2026 revenue was $10.2 million, according to reported quarterly figures, versus $1.5 million in Q1 2026, $864,000 in Q4 2025, and $889,000 in Q3 2025. The company reported that CASGEVY product revenue specifically was $76 million in Q2 2026, though the XBRL-reported quarterly revenue figure differs from that release figure. Annual revenue fell from $371.2 million in 2023 to $37.3 million in 2024 and $3.5 million in 2025. Net loss was $581.6 million in 2025 with diluted EPS of -$6.47, and operating cash flow was -$345.0 million in 2025. At June 30, 2026, total assets were $2.65 billion, total liabilities $904.2 million, shareholders' equity $1.75 billion, cash and equivalents $291.3 million, and long-term debt $586.2 million.

Strategy

CRISPR Therapeutics is expanding CASGEVY access, including the July 2026 FDA approval for patients ages 2 years and older with SCD or TDT, and pursuing targeted conditioning and in vivo hematopoietic stem cell editing to broaden the eligible population. It is advancing a liver-directed in vivo portfolio, with CTX310 in Phase 1b for severe hypertriglyceridemia and refractory hypercholesterolemia, CTX340 in Phase 1 for refractory hypertension, and CTX460 in Phase 1 for alpha-1 antitrypsin deficiency. It is also developing next-generation technologies such as SyNTase and optimized guide RNAs, and running siRNA programs with Sirius Therapeutics. Reimbursement and regulatory efforts continue globally, including Germany and submissions in Saudi Arabia and the United Kingdom for children ages 5 to 11.

Risks

• Persistent losses and cash use — The company has incurred significant operating losses since inception and reported a net loss of $581.6 million and operating cash outflow of $345.0 million in 2025.
• Revenue concentration in CASGEVY — CASGEVY is the only commercial product, and total reported annual revenue fell from $371.2 million in 2023 to $3.5 million in 2025, leaving results dependent on a single therapy and its Vertex collaboration.
• Dependence on the Vertex partnership — CASGEVY is advanced under a joint development and commercialization agreement with Vertex, so development, reimbursement, and commercialization outcomes depend on that collaboration.
• Early-stage pipeline risk — In vivo, CAR T, regenerative medicine, and siRNA candidates remain in preclinical through Phase 2 testing and may not replicate the results seen in earlier studies or receive approval.

Outlook

Management stated it enters the second half of 2026 well positioned with important pipeline milestones ahead. Expected updates include a Phase 1b clinical update for CTX310 in the second half of 2026, an Lp(a) program update in 2026, and an update for CTX611 in the second half of 2026. Regulatory reviews continue for CASGEVY in children ages 5 to 11 in Saudi Arabia and the United Kingdom.