Financial Performance - The company reported net losses of 237.1million,153.2 million, and 220.4millionfortheyearsendedDecember31,2024,2023,and2022respectively,withanaccumulateddeficitof1.5 billion as of December 31, 2024[587]. - The net loss for the year ended December 31, 2024, was 237.1million,comparedtoanetlossof153.2 million in 2023, reflecting a 55% increase in losses[608]. - The net loss for 2024 was 237.1million,comparedtoanetlossof153.2 million in 2023, indicating a worsening of approximately 55%[662]. - The company reported a net loss per share of 2.88for2024,comparedto2.02 for 2023, indicating a deterioration in per-share performance[662]. - Total liabilities rose from 150.1millionin2023to207.3 million in 2024, an increase of approximately 38%[659]. - The accumulated deficit increased to 1.47billionin2024from1.23 billion in 2023, reflecting a growth of about 19%[659]. - The total stockholders' equity decreased significantly from 349.1millionin2023to134.3 million in 2024, a decline of approximately 62%[659]. Revenue Generation - The company has not generated any revenue from product sales to date and does not expect to do so for the foreseeable future[588]. - In 2024, the company recognized 18.1millionofrevenuerelatedtoitscollaborationwithBMS,with6.5 million being previously deferred revenue[589]. - Collaboration and other research and development revenues decreased by 45.8million,to32.3 million for the year ended December 31, 2024, from 78.1millionfortheyearendedDecember31,2023,adeclineof5921.6 million, to 199.2millionfortheyearendedDecember31,2024,representinga12177.7 million in 2023[610]. - Employee-related expenses within research and development increased by 15%, from 47.4millionin2023to54.2 million in 2024[610]. - External research and development expenses surged by 47%, from 53.3millionin2023to78.5 million in 2024[610]. - The company anticipates a decrease in expenses over the next few years due to the discontinuation of the reni-cel program, but will continue to incur significant expenses for research and development[631]. Cash Flow and Liquidity - Net cash used in operating activities was approximately 210.3millionfortheyearendedDecember31,2024,comparedto132.2 million in 2023, primarily due to increased research efforts and clinical activities[624][625]. - The company expects its existing cash and marketable securities will fund operations into the second quarter of 2027, but significant additional funding will be needed for ongoing research and development[632]. - The company had cash, cash equivalents, and marketable securities totaling 269.9millionasofDecember31,2024[678].−Thecompanyraisedanaggregateof1.0 billion in net proceeds through public offerings and had cash, cash equivalents, and marketable securities of 269.9million[618][620].−Cashflowfrominvestingactivitiesfor2024was162.1 million, compared to a net cash used of 3.7millionin2023[671].ExpensesandCharges−Generalandadministrativeexpensesrosebyapproximately2.3 million, to 72.0millionfortheyearendedDecember31,2024,a369.7 million in 2023[611]. - Restructuring charges amounted to 12.2millionfortheyearendedDecember31,2024,withnoequivalentchargesin2023,markinga10021,000,000 in operating lease costs for the year ended December 31, 2024, compared to 15,200,000in2023,markinga38.056.7 million, with no milestone or extension term fees included as of December 31, 2024[771]. - The Company has granted Beam Therapeutics an exclusive license for the development and commercialization of base editing therapies, with options for additional licenses[775]. Market and Risk Factors - The company is exposed to market risk related to changes in interest rates, with cash and cash equivalents of 131.5millionandmarketablesecuritiesof138.4 million as of December 31, 2024[642]. - The Company has not experienced any credit losses and did not have an allowance for credit losses as of December 31, 2024 and 2023[691]. - The Company has not had any write-offs of bad debt and did not have an allowance for doubtful accounts as of December 31, 2024 and 2023[692]. Asset Management - Total current assets decreased from 340.8millionin2023to289.3 million in 2024, a decline of approximately 15%[659]. - The Company recorded cash equivalents and marketable securities totaling 269,913,000asofDecember31,2024,comparedto427,135,000 as of December 31, 2023, indicating a decrease of approximately 37%[740]. - The Company holds no securities with remaining maturities greater than one year as of December 31, 2024[742].