CRSP

EX-99.1

2 crsp-ex99_1.htm

EX-99.1

EX-99.1

Exhibit 99.1

CRISPR Therapeutics Provides Business Update and Reports Third Quarter 2026 Financial Results

– CASGEVY® revenue of $99 million in the third quarter of 2026, up 30% quarter-over-quarter and nearly six times the third quarter of 2025 –

– CASGEVY approved in the U.K. for children 5 years of age and older with severe SCD or TDT, and in the United Arab Emirates and Bahrain for children 2 years of age and older –

– FDA granted Regenerative Medicine Advanced Therapy (RMAT) designations to CTX310® for severe hypertriglyceridemia and elevated LDL-C –

– Phase 1b data for CTX310 in severe hypertriglyceridemia to be presented in a late-breaking oral presentation at AHA Scientific Sessions 2026; Phase 1 data for zugo-cel in systemic sclerosis to be presented at ACR Convergence 2026 –

– Completed enrollment in the Phase 2 clinical trial of CTX611 in patients undergoing total knee arthroplasty –

– Appointed Siva Sakhamuri, Ph.D., as Chief Technical Officer and A.J. Silver as Chief Financial Officer –

ZUG, Switzerland and BOSTON, November 2, 2026 – CRISPR Therapeutics (Nasdaq: CRSP) today reported financial results for the third quarter ended September 30, 2026.

“The third quarter marked meaningful progress for CRISPR Therapeutics,” said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. “CASGEVY is reaching more patients, including younger children following new approvals in the U.K. and the Middle East, and the FDA’s RMAT designations for CTX310 underscore its potential to address significant unmet needs in cardiovascular disease. With new clinical data from CTX310 and zugo-cel in the coming weeks and enrollment complete in our Phase 2 trial of CTX611, we are entering a pivotal period for our pipeline.”

Recent Highlights and Outlook

Hemoglobinopathies and CASGEVY® (exagamglogene autotemcel)

• CASGEVY, the world’s first approved CRISPR-based therapy, has been shown to reduce or eliminate vaso-occlusive crises (VOCs) in eligible patients with sickle cell disease (SCD) and transfusion requirements in eligible patients with transfusion-dependent beta thalassemia (TDT). CASGEVY is approved in 39 countries across North America, Europe, and the Middle East for eligible patients 12 years of age and older, with approvals for younger children in five of those countries.

• CASGEVY generated third quarter 2026 revenue of $99 million, representing 30% growth quarter-over-quarter and nearly six times the revenue reported in the third quarter of 2025. Twice as many in the first nine months of 2026 as in all of 2025, and more than 750 patients have initiated treatment worldwide.

• CASGEVY’s availability for younger patients continues to expand. In July, the FDA approved CASGEVY for children 2 years of age and older with SCD or TDT, making it the first genetic therapy for SCD and TDT approved for this age group, and multiple children have since initiated treatment in the U.S. The Kingdom of Saudi Arabia, the United Arab Emirates, and the Kingdom of Bahrain have also approved CASGEVY for children 2 years of age and older, and the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) approved CASGEVY for children 5 years of age and older with severe SCD or TDT.

• Building on CASGEVY, CRISPR Therapeutics is advancing an in vivo hematopoietic stem cell editing approach that could extend gene editing to many more patients with SCD and TDT. Using lipid nanoparticle (LNP)-mediated delivery, the approach is designed to edit stem cells directly in the body, potentially simplifying treatment and broadening global access.

In Vivo Liver Editing

CRISPR Therapeutics continues to advance a diversified portfolio of in vivo gene editing programs leveraging its proprietary liver-directed LNP delivery platform.

• The Phase 1 clinical trial of CTX310®, targeting angiopoietin-related protein 3 (ANGPTL3) is ongoing.

• CRISPR Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designations to CTX310 for the treatment of severe hypertriglyceridemia (sHTG) and for the treatment of patients with elevated LDL-C. RMAT designation provides the benefits of Fast Track and Breakthrough Therapy designations, including early interactions with the FDA and potential eligibility for priority review and accelerated approval.

• In August, CRISPR Therapeutics presented Phase 1a durability data for CTX310 in a late-breaking session at the European Society of Cardiology (ESC) Congress 2026 and simultaneously published in The New England Journal of Medicine. A single course treatment of CTX310 produced deep and durable reductions in circulating ANGPTL3, triglycerides (TG), and low-density lipoprotein (LDL) that were sustained through one year of follow-up. At the highest dose, the mean reductions from baseline were 79% in ANGPTL3, 48% in triglycerides, and 53% in LDL-C in a mi

EX-99.1

2 crsp-ex99_1.htm

EX-99.1

EX-99.1

Exhibit 99.1

CRISPR Therapeutics Provides Business Update and Reports Second Quarter 2026 Financial Results

ZUG, Switzerland and BOSTON, August 3, 2026 – CRISPR Therapeutics (Nasdaq: CRSP) today reported financial results for the second quarter ended June 30, 2026.

"The second quarter reflected strong execution across CRISPR Therapeutics' portfolio and platform," said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. "CASGEVY's momentum continued to build, highlighted by the FDA's approval for children as young as 2 years old, while we expanded our in vivo pipeline with the initiation of Phase 1 clinical trials for CTX340 for refractory hypertension and CTX460 for alpha-1 antitrypsin deficiency. We enter the second half of 2026 well positioned, with a number of important milestones ahead across our pipeline."

Recent Highlights and Outlook

Hemoglobinopathies and CASGEVY® (exagamglogene autotemcel)

CASGEVY is a non-viral, ex vivo, CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT that has been shown to reduce or eliminate vaso-occlusive crises (VOCs) for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved in 39 countries across North America, Europe, and the Middle East.

• CASGEVY generated second quarter 2026 revenue of $76 million, representing 78% growth quarter-over-quarter and 151% growth year-over-year.

• The U.S. FDA recently approved CASGEVY in children 2 years of age and older with SCD or TDT, the first genetic therapy indicated for children as young as 2 years for both SCD and TDT. With this approval, achieved in just 53 days post filing, approximately 5,500 patients with SCD or TDT may be eligible for treatment with CASGEVY for the first time. Regulatory submissions have also been completed for CASGEVY in the Kingdom of Saudi Arabia (KSA) and the United Kingdom (U.K.) for the treatment of children 5 to 11 years of age.

• In May, Vertex secured reimbursement for CASGEVY for eligible patients 12 years and older with SCD or TDT in Germany. Efforts remain underway with government and reimbursement authorities globally to ensure sustainable access for eligible patients.

• CRISPR Therapeutics continues to advance its in vivo hematopoietic stem cell editing approach using lipid nanoparticle (LNP)-mediated delivery. This approach has the potential to expand the addressable patient populations for SCD and TDT.

In Vivo Liver Editing

CRISPR Therapeutics continues to advance a diversified portfolio of in vivo gene editing programs leveraging its proprietary liver-directed LNP delivery platform.

• Development of CTX310®, an investigational therapy targeting angiopoietin-related protein 3 (ANGPTL3), continues in a Phase 1b clinical trial, with the Company prioritizing indications in severe hypertriglyceridemia (sHTG) and refractory hypercholesterolemia. U.S. trials have been initiated, with ex-U.S. trials ongoing.

• CRISPR Therapeutics announced that a late-breaking abstract entitled "Durability of Effects of CTX310, a CRISPR-Cas9 Gene Editing Targeting ANGPTL3" has been accepted for presentation at the European Society of Cardiology (ESC) Congress 2026 in Munich, Germany, on August 28 at 4:30 p.m. CET to discuss a clinical update of the ongoing Phase 1a trial. The Company also expects to provide a Phase 1b clinical update in the second half of 2026.

• CTX340™, targeting angiotensinogen (AGT), has received IND clearance from the FDA. The Company has initiated a Phase 1 clinical trial for patients with refractory hypertension.

• CTX460™, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD), is the first investigational candidate generated from the Company's SyNTase™ editing platform. The Company has initiated a Phase 1 clinical trial for CTX460.

• CRISPR Therapeutics' pipeline of preclinical in vivo gene editing candidates includes CTX321™, the Company's next-generation LPA program, which is progressing through IND/CTA-enabling studies. The candidate incorporates an optimized guide RNA that delivered approximately two-fold greater potency in preclinical models, paired with the same LNP delivery system used previously. An Lp(a) program update is anticipated in 2026.

siRNA-based Programs

CRISPR Therapeutics' small interfering RNA (siRNA)-based portfolio includes clinical-stage programs targeting cardiovascular and thromboembolic diseases, developed in collaboration with Sirius Therapeutics.

• CTX611 (SRSD107), a long-acting siRNA therapeutic targeting Factor XI (FXI), is advancing through a Phase 2 clinical trial in patients undergoing total knee arthroplasty (TKA). The Company expects to provide an update in the second half of 2026.

• CTX611 has the potential to address a broad range of thromboembolic and clotting-related indications, including atrial fibrillation (AF), venous thromboembolism (VTE), ischemic stroke, cancer-assoc

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