CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2025 Financial Results

Core Insights - CRISPR Therapeutics reported financial results for Q4 and full year 2025, highlighting progress in its clinical and preclinical pipeline, including advancements in autoimmune diseases and oncology, as well as the global uptake of its therapy CASGEVY [1][2] Financial Performance - CASGEVY generated Q4 2025 revenue of $54 million and full year revenue of $116 million, with a significant increase in patient initiations and first cell collections, nearly tripling from 2024 to 2025 [5] - Cash, cash equivalents, and marketable securities totaled $1,975.8 million as of December 31, 2025, up from $1,903.8 million in 2024, primarily due to common share issuance and interest income [14] - R&D expenses for Q4 2025 were $83.5 million, an increase from $71.7 million in Q4 2024, driven by higher licensing fees [14] - The net loss for Q4 2025 was $130.6 million, compared to a net loss of $37.3 million in Q4 2024 [14] Pipeline Developments - The company is advancing its in vivo liver editing portfolio and has made progress with its siRNA collaboration with Sirius Therapeutics [2][4] - CTX310, targeting ANGPTL3, is in Phase 1b trials for severe hypertriglyceridemia and refractory hypercholesterolemia, with updates expected in H2 2026 [5] - CTX460, targeting SERPINA1 for alpha-1 antitrypsin deficiency, is expected to enter clinical trials in mid-2026 [5] - Zugo-cel is in ongoing Phase 1 trials for autoimmune diseases and hematologic malignancies, with updates anticipated in H2 2026 [10][17] Market Expansion - CASGEVY is approved in multiple regions, including the U.S., EU, and several Middle Eastern countries, with approximately 90% of U.S. patients having reimbursed access [5] - The company is expanding its regenerative medicine portfolio, including CTX213 for Type 1 diabetes, which has shown promising preclinical efficacy [9] Collaborations - CRISPR Therapeutics has a strategic collaboration with Vertex Pharmaceuticals for CASGEVY, with Vertex leading global development and commercialization [13] - The collaboration with Sirius Therapeutics focuses on developing siRNA therapies, with CTX611 being a key program targeting FXI for thromboembolic disorders [19]