CRISPR Therapeutics — Third Quarter 2026 Earnings Summary

Financial Performance:

• Total revenue for the third quarter of 2026 was $12.7 million, compared to $0.9 million in the same period of 2025, driven by $12.3 million in collaboration revenue and $0.3 million in grant revenue.
• CASGEVY revenue specifically reached $99 million in Q3 2026, representing 30% growth quarter-over-quarter and nearly six times the revenue reported in Q3 2025.
• Net loss for the third quarter of 2026 was $111.4 million, compared to a net loss of $106.4 million in Q3 2025.
• Research and development expenses were $71.9 million in Q3 2026, up from $58.9 million in Q3 2025, primarily due to increased external R&D expenses and sublicense fees.
• General and administrative expenses were $17.9 million in Q3 2026, compared to $16.9 million in Q3 2025.
• Collaboration expense, net, decreased to $41.3 million in Q3 2026 from $57.1 million in Q3 2025, primarily attributable to an increase in the Company's share of CASGEVY revenue.
• Acquired in-process R&D expenses were $11.7 million in Q3 2026, consisting of a development milestone under the Sirius Agreement, compared to $0 in Q3 2025.
• Cash, cash equivalents, and marketable securities totaled $2,276.6 million as of September 30, 2026, an increase from $1,975.8 million as of December 31, 2025, driven by $585.4 million in net proceeds from convertible senior notes issued in March 2026.
• Comprehensive loss for the third quarter of 2026 was $120.9 million, compared to $105.5 million in Q3 2025.
• Net loss per common share (basic and diluted) was $1.15 for Q3 2026, compared to $1.17 in Q3 2025.

Guidance and Future Outlook:

• Phase 1b data for CTX310 in severe hypertriglyceridemia is scheduled for presentation at AHA Scientific Sessions 2026 on November 9, 2026.
• Phase 1 data for zugo-cel in systemic sclerosis is scheduled for presentation at ACR Convergence 2026 on November 8, 2026.
• An update on the Phase 2 clinical trial of CTX611 in patients undergoing total knee arthroplasty is expected in the second half of 2026.
• Updates on the Phase 1/2 clinical trial of zugo-cel in B-cell malignancies are anticipated in the second half of 2026.
• The Company expects to receive updates on its option to nominate up to two additional siRNA targets in 2027.
• Management indicated the Company is entering a "pivotal period" for its pipeline with new clinical data and enrollment completions.

Business Segments and Product Lines:

• CASGEVY is approved in 39 countries for patients 12 years and older, with approvals for younger children in five countries.
• More than 750 patients have initiated CASGEVY treatment worldwide, with twice as many initiations in the first nine months of 2026 compared to all of 2025.
• FDA approval for CASGEVY was granted for children 2 years and older with SCD or TDT in the U.S., and approvals for this age group were also received in Saudi Arabia, UAE, and Bahrain.
• The U.K. MHRA approved CASGEVY for children 5 years and older with severe SCD or TDT.
• The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designations to CTX310 for severe hypertriglyceridemia and elevated LDL-C.
• CTX310 Phase 1a data showed mean reductions of 79% in ANGPTL3, 48% in triglycerides, and 53% in LDL-C at the highest dose, with effects sustained through one year.
• Enrollment was completed in the Phase 2 clinical trial of CTX611 (siRNA targeting Factor XI) in patients undergoing total knee arthroplasty.
• The Phase 1 clinical trial of CTX340 (targeting angiotensinogen for refractory hypertension) is ongoing.
• The Phase 1 clinical trial of CTX460 (targeting SERPINA1 for alpha-1 antitrypsin deficiency) is ongoing; it is the first candidate from the SyNTase editing platform.
• CTX321 (next-generation LPA program) is progressing through IND/CTA-enabling studies.
• Zugo-cel is being evaluated in three ongoing Phase 1 autoimmune disease trials (rheumatology, hematology, and neurologic baskets) and a Phase 1/2 immuno-oncology trial in B-cell malignancies.
• A combination study evaluating zugo-cel with pirtobrutinib in aggressive B-cell lymphomas is ongoing under a collaboration with Lilly.
• The proprietary in vivo CAR-T platform is in the IND/CTA-enabling phase, focusing on transient mRNA-based and non-viral integrating CAR-T approaches.
• CTX213, a deviceless beta cell replacement candidate for Type 1 diabetes, is progressing toward the clinic following compelling preclinical efficacy.

Market and Competitive Landscape:

• CASGEVY is the world's first approved CRISPR-based therapy.
• The Company is advancing an in vivo hematopoietic stem cell editing approach using lipid nanoparticle (LNP) delivery to potentially simplify treatment and broaden global access for SCD and TDT.
• The in vivo liver editing portfolio leverages a proprietary LNP delivery platform utilizing both CRISPR/Cas9 and SyNTase editing technology.
• The CTX611 program is being co-developed with Sirius Therapeutics on an equal cost-and-profit-sharing basis, with CRISPR Therapeutics leading U.S. commercialization and Sirius leading in greater China.
• The CASGEVY collaboration with Vertex involves a 60/40 profit and program cost sharing arrangement, with Vertex leading global development, manufacturing, and commercialization.

Risks and Challenges:

• Forward-looking statements regarding clinical trials, regulatory approvals, and financial performance are subject to risks and uncertainties, including the possibility that actual results may differ materially from expectations.
• There is no guarantee that any investigational therapy will successfully complete clinical development or gain regulatory approval.
• The Company references "Risk Factors" in its most recent Form 10-K and subsequent SEC filings for detailed risks regarding the business.

Management Commentary and Tone:

• Samarth Kulkarni, Ph.D., Chairman and CEO, stated that the third quarter marked "meaningful progress," noting CASGEVY is reaching more patients including younger children.
• Management highlighted that FDA RMAT designations for CTX310 underscore its potential to address significant unmet needs in cardiovascular disease.
• The tone indicates confidence in the pipeline, describing the current period as "pivotal" due to upcoming data presentations and trial completions.

Other Key Points:

• Siva Sakhamuri, Ph.D., was appointed Chief Technical Officer effective immediately, bringing over 30 years of experience in technical operations and quality leadership.
• A.J. Silver was appointed Chief Financial Officer effective October 19, 2026, succeeding Raju Prasad, Ph.D., who stepped down for external opportunities.
• The Company's in vivo and cell therapy programs are supported by a wholly-owned GMP manufacturing facility in Framingham, Massachusetts, which enables an industry-leading cost of goods.
• The Company issued convertible senior notes in March 2026, generating $585.4 million in net proceeds.
• The Company is advancing a novel SyNTase editing platform designed to enable precise, efficient, and scalable gene correction.