CRISPR Therapeutics(US:CRSP)2025-11-10 13:15Gene Editing and Therapies - CASGEVY became the first-ever approved CRISPR-based gene-editing therapy in 2023, approved in multiple regions including the U.S. and EU for treating eligible patients aged 12 and older with SCD or TDT [125]. - The company has recognized revenue of $205.0 million from its collaboration with Vertex for gene-edited stem cell therapies for diabetes, with potential for additional milestones and royalties [144]. - CTX310 and CTX320 are in ongoing Phase 1 clinical trials targeting ANGPTL3 and Lp(a) respectively, aiming to address cardiovascular diseases [134][135]. - SRSD107 demonstrated over 93% reduction in FXI levels and FXI activity in Phase 1 trials, with effects sustained for up to six months post-dosing [139]. - The company is advancing CTX211, an allogeneic, gene-edited, hypoimmune stem cell-derived product candidate for T1D, currently in a Phase 1 clinical trial [140]. - The strategic collaboration with Sirius Therapeutics includes a $25.0 million upfront cash payment and shared development costs for siRNA therapies [145][146]. - The company is developing next-generation CAR T therapies with enhanced potency and manufacturing robustness, targeting autoimmune and oncology indications [128]. - The CRISPR-X team is focused on innovating next-generation editing modalities to enable whole gene correction and insertion without viral delivery [141]. - The company has established a leading platform for in vivo gene editing, targeting major unmet needs in cardiovascular and metabolic diseases [132]. - The partnership with Vertex for hemoglobinopathies has evolved into a joint development and commercialization agreement for CASGEVY [143]. Financial Performance - Total revenue for the three months ended September 30, 2025, was $889,000, an increase of $287,000 compared to $602,000 in the same period of 2024 [162]. - Research and development expenses decreased to $58.9 million for the three months ended September 30, 2025, from $82.2 million in 2024, a reduction of approximately 28.3% [164]. - General and administrative expenses were $16.9 million for the three months ended September 30, 2025, down from $17.4 million in 2024, a decrease of about 2.9% [165]. - Collaboration expense, net, increased significantly to $57.1 million for the three months ended September 30, 2025, compared to $11.2 million in 2024, reflecting a rise of approximately 411.5% [166]. - Net loss for the three months ended September 30, 2025, was $106.4 million, compared to a net loss of $85.9 million in 2024, an increase of $20.5 million [162]. - For the nine months ended September 30, 2025, total revenue was $2.646 million, an increase of $1.023 million from $1.623 million in 2024 [169]. - Research and development expenses for the nine months ended September 30, 2025, were $201.3 million, down from $238.5 million in 2024, a decrease of approximately 15.6% [171]. - Acquired in-process research and development expenses were $96.3 million for the nine months ended September 30, 2025, with no comparable expenses in 2024 [169]. - Total operating expenses for the nine months ended September 30, 2025, were $512.5 million, an increase of $108.9 million from $403.6 million in 2024 [169]. - Net loss for the nine months ended September 30, 2025, was $451.0 million, compared to a net loss of $328.9 million in 2024, an increase of $122.1 million [169]. - General and administrative expenses increased to $55.1 million for the nine months ended September 30, 2025, from $54.9 million in 2024, primarily due to increased stock-based compensation [174]. - Collaboration expense, net, rose to $159.8 million for the nine months ended September 30, 2025, compared to $110.3 million in 2024, driven by increased operating expenses for CASGEVY [175]. - Other income decreased to $61.8 million for the nine months ended September 30, 2025, from $75.9 million in 2024, mainly due to lower interest income [176]. Cash Flow and Financial Position - As of September 30, 2025, the company had $1,944.1 million in cash, cash equivalents, and marketable securities, with an accumulated deficit of $1,816.9 million [177]. - Net cash used in operating activities was $252.5 million for the nine months ended September 30, 2025, an increase of $159.7 million from $92.7 million in 2024 [190]. - Net cash used in investing activities decreased to $69.0 million for the nine months ended September 30, 2025, from $386.3 million in 2024 [191]. - Net cash provided by financing activities was $309.6 million for the nine months ended September 30, 2025, compared to $315.1 million in 2024 [192]. - The company expects to fund its operating expenses and capital expenditures for at least the next 24 months with existing cash and marketable securities [186]. - The company anticipates continued losses for at least the next several years and will require additional capital to fund operations [177]. Economic and Market Conditions - The company faces exposure to foreign currency exchange rate movements, primarily with the Swiss Franc and British Pound against the U.S. dollar [201]. - Foreign currency transaction gains and losses have not been material to the company's financial statements [201]. - The company has not engaged in any foreign currency hedging transactions [201]. - Inflation has increased costs related to labor, clinical trials, and manufacturing [202]. - The company does not believe inflation had a material effect on its business or financial results during the three and nine months ended September 30, 2025 and 2024 [202].