CTX310 is an investigational in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3 for severe or refractory dyslipidemias. In Phase 1, single-course IV dosing showed dose-dependent reductions in ANGPTL3, triglycerides, and LDL cholesterol with supportive safety data. The therapy remains unapproved and in clinical development.
CRISPR Therapeutics said CASGEVY launch momentum is building and multiple pipeline assets could generate data in the next 12 to 18 months. The company also highlighted zugo-cel progress in oncology and autoimmune disease.
CRISPR Therapeutics reported a $581.6 million net loss in 2025 with revenue of just $3.5 million, while continuing to advance its gene-editing pipeline including the approved CASGEVY therapy and next-generation CAR T cell programs. The company maintains strategic partnerships and expects current funds to support operations for at least 24 months.
CRISPR Therapeutics reported a Q4 2025 loss of $1.37 per share, missing estimates, with revenues of $0.9 million falling short of expectations. The company's partner Vertex recorded $54 million in Casgevy sales for the quarter, with regulatory submissions for pediatric label expansion planned for early 2026.
The cell therapy manufacturing market is projected to reach $14.01 billion by 2035, with CAR-T therapies dominating at 65% market share. Recent FDA approvals for new CAR-T indications and Japanese regulatory acceleration highlight growing clinical adoption, while research advances include new anti-aging protein platforms and CRISPR-based treatments.
The FDA has issued draft guidance that may speed approval pathways for rare disease therapies using genome editing technologies, potentially benefiting CRISPR Therapeutics' pipeline of six clinical-stage candidates.
Switzerland-based CRISPR Therapeutics stands to benefit from increased Casgevy sales following partner Vertex Pharmaceuticals' fourth-quarter results, with five additional gene-editing therapies in clinical trials targeting larger patient populations.
Vertex Pharmaceuticals and CRISPR Therapeutics reported $116 million in full-year 2025 sales for gene-editing therapy Casgevy, with 64 patients receiving infusions and patient initiations nearly tripling compared to 2024.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07491172 |
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias |
RECRUITING | PHASE1 |
