On August 3, 2026 CRISPR Therapeutics (Nasdaq: CRSP) reported financial results for the second quarter ended June 30, 2026.

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"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-associated thrombosis (CAT), thrombosis in chronic kidney disease (CKD), peripheral vascular disease (PVD), and chronic coronary artery disease (CAD), collectively representing a multi-billion-dollar market opportunity. CRISPR Therapeutics is expected to lead global Phase 3 development, with Sirius Therapeutics overseeing development activities in greater China.
CRISPR Therapeutics has the option to nominate up to two additional siRNA targets for research and development. An update is expected in 2026.

Autoimmune Disease and Immuno-Oncology

Zugocabtagene geleucel (zugo-cel; formerly CTX112) continues to advance across both autoimmune disease and hematologic malignancies.

In autoimmune disease, zugo-cel is currently being evaluated in two ongoing Phase 1 basket trials: a rheumatology basket including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis (IM); and a hematology basket in immune thrombocytopenic purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA).
In addition, a third Phase 1 trial has been initiated in autoimmune neurologic diseases. The trial, includes progressive multiple sclerosis (PMS), neuromyelitis optica spectrum disorder (NMOSD), myelin oligodendrocyte glycoprotein antibody-associated Disease (MOGAD), N-methyl-D-aspartate receptor (NMDAR) and leucine-rich glioma-inactivated Protein 1 (LGI1) autoimmune encephalitis (AIE), and stiff person syndrome (SPS).
Enrollment across the zugo-cel autoimmune clinical program continues in SSc, IIM, SLE, ITP and wAIHA with multiple clinical sites activated globally. The Company expects to provide further updates in the second half of 2026.
In immuno-oncology, the Phase 1/2 clinical trial of zugo-cel in B-cell malignancies is ongoing, with updates anticipated in the second half of 2026. The Company has also initiated a combination study evaluating zugo-cel with pirtobrutinib in aggressive B-cell lymphomas, under the Company’s existing collaboration with Lilly.
The Company’s autoimmune and immuno-oncology programs are supported by a wholly-owned GMP manufacturing facility in Framingham, Massachusetts. The facility provides end-to-end production capabilities across the cell therapy portfolio, supports both clinical and future commercial supply and enables an industry-leading cost of goods.

CRISPR Therapeutics is also advancing a proprietary in vivo CAR-T platform with potential applications across autoimmune disease and oncology.

The Company is pursuing two complementary modalities, supported by an antibody-conjugated LNP delivery system that enables targeted delivery to immune cells: a transient, re-dosable CAR-T leveraging engineered mRNA, and a non-viral, integrating CAR-T employing next-generation site-specific integration technologies.
Both programs are currently in the IND/CTA-enabling phase, focused on a best-in-class profile.

Regenerative Medicine

CRISPR Therapeutics continues to advance its regenerative medicine program in diabetes. The Company is developing CTX213, a deviceless beta cell replacement candidate for Type 1 diabetes, consisting of unencapsulated precursor islet cells derived from edited induced pluripotent stem cells (iPSCs). CTX213 has demonstrated compelling preclinical efficacy through direct administration and is progressing toward the clinic. The Company expects to provide additional updates as development progresses.

Second Quarter 2026 Financial Results

Cash Position: Cash, cash equivalents, and marketable securities were $2,364.4 million as of June 30, 2026, compared to $1,975.8 million as of December 31, 2025. The increase in cash was primarily driven by net proceeds of $585.4 million from the issuance of convertible senior notes in March 2026, offset by operating expenses.
R&D Expenses: R&D expenses were $67.2 million for the second quarter of 2026, compared to $69.9 million for the second quarter of 2025. The decrease in R&D expense was primarily attributable to a decrease in employee-related costs and facility-related expenses, offset by an increase in license fees.
Acquired In-Process R&D Expenses: Acquired in-process R&D expenses were $2.5 million for the second quarter of 2026, compared to $96.3 million for the second quarter of 2025. Acquired in-process R&D expenses for the second quarter of 2026 were not material. Acquired in-process R&D expenses for the second quarter of 2025 were related to costs incurred upon entering the Sirius Agreement in 2025.
G&A Expenses: General and administrative expenses were $17.6 million for the second quarter of 2026, compared to $18.9 million for the second quarter of 2025. The decrease in G&A expense was primarily attributable to a decrease in employee-related costs, including stock-based compensation expenses.
Collaboration Expense: Collaboration expense, net, was $40.3 million for the second quarter of 2026, compared to $45.2 million for the second quarter of 2025. The decrease was primarily attributable to an increase in the Company’s share of CASGEVY revenue.
Net Loss: Net loss was $91.2 million for the second quarter of 2026, compared to a net loss of $208.5 million for the second quarter of 2025.

About CASGEVY (exagamglogene autotemcel [exa-cel])

CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT), in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world.

(Press release, CRISPR Therapeutics, AUG 3, 2026, View Source [SID1234669631])