CRISPR Therapeutics reported its financial results for the fourth quarter and full year of 2025, alongside a detailed business update highlighting progress across its clinical and preclinical pipeline. The company emphasized growth in its approved gene-editing therapy CASGEVY® for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), advances in in vivo gene editing programs, and momentum in its siRNA collaboration with Sirius Therapeutics. Additionally, CRISPR Therapeutics provided updates on its autoimmune disease and immuno-oncology programs.

Financial Performance and CASGEVY Commercial Update

  • CASGEVY, CRISPR’s exagamglogene autotemcel therapy, is approved in multiple major markets including the U.S., EU, UK, Canada, Switzerland, and several Middle Eastern countries for patients aged 12 and older with severe SCD or TDT.
  • The therapy generated $54 million in revenue in Q4 2025 and $116 million for the full year, reflecting steady growth driven by increased patient treatments.
  • In 2025, 64 patients received CASGEVY infusions, with 30 treated in the fourth quarter alone.
  • Globally, 147 patients initiated the treatment process by undergoing their first cell collection during 2025, nearly tripling the number from 2024.
  • Access and reimbursement for CASGEVY expanded, with approximately 90% of eligible U.S. patients having reimbursed access by year-end.
  • Reimbursement was also secured or maintained in the UK, Italy, Austria, Denmark, Luxembourg, Saudi Arabia, Bahrain, UAE, Kuwait, and most recently Scotland, where Vertex secured reimbursement for eligible SCD patients.
  • Positive pediatric data for children aged 5-11 with SCD or TDT were presented at the American Society of Hematology (ASH) meeting in December 2025, earning a spot in the “Best of ASH” program.
  • Regulatory submissions for pediatric use are planned to begin in the first half of 2026, with the FDA granting a National Priority Voucher to expedite review.

Pipeline Progress: In Vivo Liver Editing Programs

  • CRISPR is advancing its in vivo gene editing portfolio using lipid nanoparticle (LNP) delivery technology.
  • CTX310®, targeting ANGPTL3 for severe hypertriglyceridemia and refractory hypercholesterolemia, is in Phase 1b clinical trials, with an update expected in the second half of 2026.
  • The next-generation LPA-targeting program, CTX321™, features an improved guide RNA with roughly double the potency in preclinical tests while using the same LNP delivery system. CTX321 is currently in IND/CTA-enabling studies, with a program update anticipated in 2026.
  • Several preclinical candidates are advancing:
    • CTX460™, targeting SERPINA1 for alpha-1 antitrypsin deficiency (AATD), is the first candidate from CRISPR’s SyNTase™ editing platform, with clinical trials expected to start mid-2026.
    • CTX340™, targeting angiotensinogen (AGT) for refractory hypertension, is in IND/CTA-enabling studies, with clinical trials planned for the first half of 2026.

siRNA Collaboration with Sirius Therapeutics

  • CRISPR’s siRNA portfolio includes cardiovascular and thromboembolic disease programs developed with Sirius Therapeutics.
  • CTX611 (SRSD107), a long-acting siRNA targeting Factor XI (FXI), is in an ongoing Phase 2 trial for patients undergoing total knee arthroplasty.
  • An update on CTX611 is expected in the second half of 2026.
  • The program targets a broad range of thromboembolic and clotting disorders, including atrial fibrillation, venous thromboembolism, ischemic stroke, cancer-associated thrombosis, thrombosis in chronic kidney disease, peripheral vascular disease, and chronic coronary artery disease.
  • CRISPR will lead global Phase 3 development, while Sirius will manage development activities in Greater China.
  • CRISPR has the option to nominate up to two additional siRNA targets for research and development, with plans to provide updates in 2026.
  • For each nominated target, CRISPR will fund research and retain rights to lead clinical development and commercialization, while Sirius will receive milestone and contingent payments plus tiered royalties.

Autoimmune Disease and Immuno-Oncology Programs

  • Zugocabtagene geleucel (zugo-cel; formerly CTX112™), CRISPR’s cell therapy candidate, continues to advance in clinical development for autoimmune diseases and oncology.
  • The company highlighted encouraging clinical data from zugo-cel programs, though specific trial results or timelines were not detailed in this update.

Implications for Investors

  • CASGEVY’s expanding global reimbursement and increasing patient uptake underpin growing revenue and market penetration in gene-editing therapies for hemoglobinopathies.
  • The pediatric data and upcoming regulatory submissions could broaden the eligible patient population, potentially accelerating future revenue growth.
  • Progress in in vivo liver editing programs and siRNA collaborations diversifies CRISPR’s pipeline beyond cell therapies, targeting large markets in cardiovascular, metabolic, and rare diseases.
  • The advancement of multiple candidates toward clinical trials in 2026 signals a maturing pipeline with several potential catalysts ahead.
  • The partnership with Sirius Therapeutics positions CRISPR to leverage external expertise and share development risks while maintaining leadership in key siRNA programs.

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