CRISPR Therapeutics (CRSP)

• CRISPR Therapeutics' gene-editing therapy, Casgevy, achieved over $100 million in revenue and is projected for strong momentum in 2024 and 2025, with ongoing expansion into pediatric populations.
• The in vivo gene editing platform showed promising results with CTX310 for hypercholesterolemia, demonstrating a 50% reduction in LDL cholesterol and a 55% reduction in triglycerides at the highest dose, identified as a multi-billion dollar opportunity.
• Key clinical pipeline assets, CTX611 (siRNA for anticoagulation) and CTX112 (allogeneic CAR-T), are advancing, with CTX611 expecting top-line Phase 2 data by the end of 2026 and CTX112 showing a 70% complete response rate in oncology and sustained remission in autoimmune patients.
• The company views 2026 as a "stepping stone pivotal year" for determining regulatory paths and pivotal trial designs for its advanced clinical assets, while also developing preclinical programs like A1AT and CTX340 (hypertension) as future value drivers.
• CRISPR Therapeutics anticipates that its gene editing procedures, including cardiovascular programs and allogeneic CAR-Ts, could ultimately reduce healthcare costs, with some potentially priced below $100,000.

• CRISPR Therapeutics reported that Casgevy, an approved product for hemoglobinopathies, has achieved over $100 million in revenue and is showing strong commercial momentum, with plans to expand the addressable patient population through pediatric data and gentler conditioning agents.
• The company presented transformative data for CTX310 in hypercholesterolemia, showing a 50% reduction in LDL cholesterol and a 55% reduction in triglycerides at the highest dose, highlighting its potential as a multi-billion dollar opportunity.
• Significant progress was noted across its in vivo gene editing platform, including CTX320/321 for Lp(a), CTX340 for hypertension entering the clinic soon, and a best-in-class A1AT program also expected to advance to clinical trials.
• CRISPR Therapeutics' CAR-T platform, particularly CTX112, demonstrated encouraging response rates with a 70% CR rate in oncology (DLBCL) and sustained drug-free remission in autoimmune settings (SLE) for up to nine months, positioning it as a potent allogeneic therapy.
• The company also highlighted its siRNA platform, with CTX611 (targeting Factor XI) in a Phase 2 trial, with top-line data anticipated in the second half of 2026, addressing a multi-billion dollar anticoagulation market.

• CRISPR Therapeutics' approved product, Casgevy, has achieved over $100 million in revenue and shows strong momentum with a 3x increase in patient initiations and cell collections between 2024 and 2025, indicating multi-billion dollar revenue potential.
• The company reported transformative Phase 1 data for CTX310 (hypercholesterolemia) in November 2025, demonstrating a 50% reduction in LDL cholesterol and a 55% reduction in triglycerides at the highest dose, positioning it as a potential multi-billion dollar opportunity.
• CRISPR is advancing multiple pipeline assets, including CTX112 (CAR-T for oncology and autoimmune diseases) with encouraging response rates reported in December 2025, and CTX611 (siRNA for anticoagulation) with Phase 2 top-line data expected in the second half of 2026, both targeting significant markets.
• For 2026, the company's strategic focus includes commercializing Casgevy, progressing CTX310, CTX611, and CTX112 towards pivotal trials, and moving preclinical assets like A1AT and CTX340 (for hypertension) into clinical development, with potential for partnerships on certain programs.

• CRISPR Therapeutics AG has advanced CTX310 into Phase 1b clinical trials for severe hypertriglyceridemia and refractory hypercholesterolemia, following positive Phase 1 results in 2025.
• The company is progressing zugocabtagene geleucel (zugo-cel) in both autoimmune diseases and hematologic malignancies, reporting positive Phase 1 results in systemic lupus erythematosus (SLE) and a collaboration with Eli Lilly for aggressive B-cell lymphomas.
• CRISPR Therapeutics is also advancing CTX320 and a next-generation LPA program, CTX321, which demonstrates approximately two-fold greater potency in preclinical testing.
• In its regenerative medicine portfolio, the company is transitioning to a next-generation diabetes candidate, CTX213, which has demonstrated compelling preclinical efficacy, informed by promising clinical data from CTX211.

• Zugo-cel, an investigational allogeneic CAR T therapy, demonstrated encouraging preliminary data in autoimmune diseases, with four patients showing deep B-cell depletion and significant clinical improvement, including one systemic lupus erythematosus (SLE) patient achieving DORIS remission through Month 6.
• In relapsed or refractory (R/R) large B-cell lymphoma (LBCL), zugo-cel showed an overall response rate (ORR) of 90% and a complete response rate (CRR) of 70% at the 600 million cell dose, with 67% of patients remaining in complete response after one year.
• CRISPR Therapeutics initiated a new Phase 1 basket trial for zugo-cel in refractory primary immune thrombocytopenic purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA) and established a collaboration with Lilly to evaluate zugo-cel with pirtobrutinib for aggressive B-cell lymphomas.
• Additional updates on zugo-cel across autoimmune disease and hematological malignancies are anticipated in the second half of 2026.

• CRISPR Therapeutics reported a strong launch for CASGEVY, with Vertex projecting over $100 million in revenues in 2025 and significant growth in 2026, and approximately 300 patients initiating the patient journey.
• The company maintains a strong financial position with $1.94 billion on its balance sheet and anticipates 2025 to be the peak spending year for the CASGEVY program.
• Key pipeline programs are advancing, including CTX310, which showed a 55% reduction in triglycerides and 50% reduction in LDL, and the Factor XI program with a major de-risking study readout expected in the second half of 2026.
• An update on cell therapy, including initial data for CTX112 in autoimmune disease and additional oncology data, is expected by year-end.

• CRISPR Therapeutics reported a strong financial position with $1.94 billion on its balance sheet.
• The CASGEVY launch is progressing well, with Vertex projecting over $100 million in revenues in 2025 and significant growth in 2026, with nearly 300 patients initiating the journey.
• Key pipeline updates include the CTX310 program showing 55% reduction in triglycerides and 50% reduction in LDL , and the Factor XI program expecting several de-risking readouts in the next 12 months, with a TKA study readout anticipated in the second half of 2026.
• The company anticipates a cell therapy update by year-end 2025 for oncology and autoimmune disease, including initial results for CTX112 in autoimmune disease.
• The CTX320 program for Lp(a) is awaiting the Horizon trial readout next year to inform its development path.

• CASGEVY is performing well, with Vertex reporting over $100 million in revenues in 2025 and significant growth in 2026, with nearly 300 patients initiating the patient journey. CRISPR anticipates a multi-billion dollar opportunity and expects revenues from the program to buffer company spend long-term.
• The pipeline is progressing, with the CTX310 program showing 55% reduction in triglycerides and 50% reduction in LDL in the overall cohort. A Factor XI TKA study readout is expected in the second half of 2026 , and initial results for CTX112 in autoimmune disease are anticipated by year-end.
• The CTX320 (Lp(a)) program is ongoing, but its advancement is paused pending the Horizon trial readout next year to inform optimal dosing and patient populations.
• CRISPR Therapeutics maintains a strong financial position with $1.94 billion on the balance sheet , and 2025 is expected to be the max spend year for the CASGEVY program.

CRISPR Therapeutics AG announced its financial results for the third quarter ended September 30, 2025, reporting a net loss and maintaining a strong cash position. CASGEVY® is showing accelerating momentum, with nearly 300 patients referred, approximately 165 patients completing their first cell collection, and 39 patients having received infusions globally as of September 30, 2025. Vertex expects over $100 million in total CASGEVY revenue this year and significant growth in 2026. The company provided updates on its diverse pipeline, including positive Phase 1 data for CTX310®, ongoing clinical trials for CTX112™ and SRSD107, and plans to initiate a clinical trial for CTX460™ in mid-2026.