CRISPR Therapeutics Provides Business Update and Reports Third Quarter 2025 Financial Results

Core Insights - CRISPR Therapeutics has reported strong progress across its portfolio, particularly with CASGEVY® and CTX310®, indicating a robust pipeline and potential for significant revenue growth in the coming years [2][3] CASGEVY® Developments - CASGEVY® is a CRISPR/Cas9 gene-edited cell therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), approved in multiple regions including the U.S. and Europe [3][13] - Nearly 300 patients have been referred to Authorized Treatment Centers (ATCs), with approximately 165 completing their first cell collection and 39 receiving infusions [5][3] - The company anticipates over $100 million in total CASGEVY revenue for this year, with significant growth expected in 2026 [5][3] Clinical Trials and Pipeline - Positive Phase 1 data for CTX310®, targeting ANGPTL3, was presented at the AHA Scientific Sessions, showing potential to lower triglycerides and LDL [5][3] - Enrollment in two global Phase 3 studies for pediatric development of exa-cel has been completed, with initial data to be presented at the ASH annual meeting [5][3] - Ongoing clinical trials for CTX112™, targeting CD19, are expected to provide broad updates by year-end [5][10] Financial Performance - As of September 30, 2025, the company reported approximately $1.9 billion in cash, cash equivalents, and marketable securities [5][12] - R&D expenses decreased to $58.9 million for Q3 2025 from $82.2 million in Q3 2024, indicating improved cost management [12][26] - The net loss for Q3 2025 was $106.4 million, compared to a net loss of $85.9 million in Q3 2024, reflecting ongoing investment in R&D [12][26] Innovative Technologies - The company is advancing its proprietary SyNTase™ editing platform, which aims to enable precise in vivo gene correction, with CTX460™ expected to enter clinical trials in mid-2026 [5][3] - CTX340™, targeting AGT for refractory hypertension, has shown promising preclinical results, including significant liver editing and blood pressure reduction [6][5] Strategic Collaborations - CRISPR Therapeutics has a strategic collaboration with Vertex Pharmaceuticals for the development of CASGEVY, sharing program costs and profits [14][3] - A partnership with Sirius Therapeutics is focused on developing SRSD107, a long-acting siRNA targeting Factor XI for thromboembolic disorders [19][18]