CRISPR Therapeutics(CRSP) - 2024 Q4 - Annual Report

Regulatory Approvals - CASGEVY became the first-ever approved CRISPR-based gene-editing therapy in 2023, approved in multiple regions including the US and EU for treating eligible patients aged 12 and older with SCD or TDT [599]. Financial Performance - Collaboration revenue for the years ended December 31, 2024, 2023, and 2022 was $35.0 million, $370.0 million, and $0.4 million, respectively, primarily from agreements with Vertex [617]. - Total revenue for 2024 was $37.3 million, down from $371.2 million in 2023, reflecting a decline of $333.9 million [640]. - The net loss for 2024 was $366.3 million, compared to a net loss of $153.6 million in 2023, an increase in loss of $212.6 million [640]. - Other income increased to $103.9 million in 2024 from $71.8 million in 2023, an increase of $32.1 million [640]. - Grant revenue for the year ended December 31, 2024, was $2.3 million, an increase from $1.2 million in 2023 [643]. Expenses - Research and development expenses were $320.7 million in 2024, a decrease of $66.7 million from $387.3 million in 2023 [640]. - General and administrative expenses decreased to $73.0 million in 2024 from $76.2 million in 2023, a reduction of $3.2 million [640]. - Total operating expenses for 2024 were $503.9 million, down from $593.7 million in 2023, representing a decrease of $89.9 million [640]. - Collaboration expense, net, was $110.3 million in 2024, down from $130.3 million in 2023, a decrease of $20.0 million [640]. - Research and development expenses decreased to $320.7 million in 2024 from $387.3 million in 2023, a reduction of approximately 17.2% [644]. - General and administrative expenses were $73.0 million in 2024, down from $76.2 million in 2023, reflecting a decrease of $3.2 million [645]. - Collaboration expense, net, decreased to $110.3 million in 2024 from $130.3 million in 2023, a decline of $20.0 million [646]. Cash and Financing - As of December 31, 2024, the company had $1,903.8 million in cash, cash equivalents, and marketable securities, with an accumulated deficit of $1,366.0 million [649]. - Net cash used in operating activities improved to $142.8 million in 2024 from $260.4 million in 2023 [657]. - Net cash provided by financing activities was $331.9 million in 2024, significantly higher than $62.7 million in 2023 [659]. - The company expects its existing cash and marketable securities to fund operations for at least the next 24 months [663]. - Future contractual payments on operating lease obligations due within one year are $29.4 million, and due greater than one year are $265.2 million [665]. - As of December 31, 2024, the company had cash, cash equivalents, and marketable securities totaling $1,903.8 million, primarily invested in U.S. Treasury securities and government agency securities [669]. Research and Development - The company is advancing several CAR T programs, including CTX112 and CTX131, targeting CD19 and CD70, respectively, with enhanced product profiles and broader patient access [602]. - CTX310 and CTX320 are in vivo programs targeting cardiovascular disease, focusing on ANGPTL3 and Lp(a), respectively, with ongoing clinical trials [607][608]. - The company has established a portfolio of therapeutic programs across four core franchises: hemoglobinopathies, CAR T, in vivo approaches, and type 1 diabetes [596]. - The company is developing gene-edited stem cell-derived therapies for type 1 diabetes, including CTX211, which is in an ongoing clinical trial [610]. - The company has recognized $205 million in revenue from upfront and milestone payments related to its collaboration with Vertex for diabetes therapies [613]. - The company expects to continue incurring research and development costs consistent with its size and stage, potentially increasing as development programs progress [622]. - The company anticipates ongoing general and administrative expenses to support continued research and development activities and potential commercialization of product candidates [624]. Market and Economic Factors - The company does not believe it has a material exposure to interest rate risk, as a 1% change in interest rates would result in an immaterial change in the fair value of its investment portfolio [669]. - The company faces exposure to foreign currency exchange rate movements, primarily with the Swiss Franc and British Pound against the U.S. dollar, but foreign currency transaction gains and losses have not been material to its financial statements [670]. - Inflation has generally increased the company's labor, clinical trial, and manufacturing costs, but it has not had a material effect on the company's business or financial results during the years ended December 31, 2024, 2023, and 2022 [671]. Innovation and Partnerships - The CRISPR-X team is focused on innovating next-generation editing modalities to enhance gene editing capabilities [611]. - The company maintains broad partnerships to develop gene editing-based therapeutics across various disease areas, including collaborations with Vertex and others [612]. - Collaboration revenue decreased to $35.0 million in 2024 from $370.0 million in 2023, primarily due to a $200.0 million milestone in 2023 related to the approval of CASGEVY [642].