Financial Performance - Cash and cash equivalents increased to $128.5 million as of June 30, 2023, up from $47.7 million at the end of 2022, representing a growth of 168%[15] - Total current assets decreased to $251.2 million from $275.4 million, a decline of 8.8%[15] - Total operating expenses for the three months ended June 30, 2023, were $58.2 million, compared to $26.8 million for the same period in 2022, an increase of 117%[17] - Net loss for the three months ended June 30, 2023, was $55.3 million, compared to a net loss of $25.9 million in the same period of 2022, reflecting a 113% increase in losses[17] - The company reported a comprehensive loss of $55.1 million for the three months ended June 30, 2023, compared to a comprehensive loss of $26.7 million in the same period of 2022, an increase of 106%[17] - For the six months ended June 30, 2023, the net loss was $79.248 million, compared to a net loss of $51.771 million for the same period in 2022, representing a 53.2% increase in losses year-over-year[25] Assets and Liabilities - Total liabilities increased to $62.9 million as of June 30, 2023, from $25.6 million at the end of 2022, a rise of 145%[15] - The accumulated deficit grew to $321.7 million as of June 30, 2023, compared to $242.4 million at the end of 2022, an increase of 32.7%[15] - The company had cash, cash equivalents, and marketable securities totaling $246.7 million as of June 30, 2023, which is expected to fund operations for at least the next 12 months[32] - As of June 30, 2023, the total fair value of cash equivalents and marketable securities was $246.7 million, compared to $283.6 million as of December 31, 2022, reflecting a decrease of approximately 13%[49][51] - The company held $128.5 million in money market funds as of June 30, 2023, up from $45.7 million at the end of 2022, indicating a significant increase in liquidity[49][51] Research and Development - Research and development expenses were $13.5 million for the three months ended June 30, 2023, down from $17.8 million in the same period of 2022, a decrease of 24.8%[17] - The company plans to continue research activities associated with its pre-clinical non-genotoxic conditioning program, aiming to advance toward potential development candidates[29] - The Company recognized $1.1 million in research and development expense related to the LCGM MSA during the six months ended June 30, 2023, compared to $2.8 million during the same period in 2022[67] Corporate Actions and Restructuring - The company anticipates ongoing restructuring initiatives to improve operational efficiency and financial position[11] - The company executed a corporate restructuring plan resulting in a total reduction in force of 71.2% to reduce operational cash burn[28] - The Company recorded restructuring charges of $3.3 million during the six months ended June 30, 2023, related to the elimination of approximately 71.2% of its workforce[116] Stock and Shareholder Information - The company had 57,989,273 shares outstanding as of June 30, 2023, a slight decrease from 58,221,760 shares at the end of 2022[15] - As of June 30, 2023, the Company reserved a total of 17,448,042 shares for future issuance, an increase from 13,893,161 shares as of December 31, 2022[91] - The 2021 Stock Option and Incentive Plan has 7,188,823 shares available for future issuance as of June 30, 2023[104] - The Company issued 65,222 shares under the Employee Stock Purchase Plan (ESPP) during the three and six months ended June 30, 2023[109] - The Company repurchased 152,694 shares of founders' common stock awards during the three and six months ended June 30, 2023[97] Lease and Asset Management - As of June 30, 2023, the Company had operating lease liabilities of $53.4 million related to office suite leases recorded on its condensed balance sheet[82] - The Company recorded a non-cash impairment of $35.0 million to the right-of-use asset and related leasehold improvements due to the Restructuring Plan[81] - Future minimum lease payments for operating leases total $88.356 million, with $4.024 million due in the remaining six months of 2023[86] - Lease expense for the three and six months ended June 30, 2023, was $2.3 million and $4.0 million, respectively, compared to $1.6 million and $3.3 million for the same periods in 2022[86] Cash Flow and Investment Activities - The company reported net cash used in operating activities of $34.729 million for the six months ended June 30, 2023, down from $43.334 million in the same period of 2022[25] - The company generated net cash provided by investing activities of $115.389 million for the six months ended June 30, 2023, compared to a net cash used of $258.883 million in the same period of 2022[25] Licensing and Agreements - In August 2023, the company entered into a license and option agreement to grant a third party the option to acquire certain technology related to its nula-cel program[29] - The Company entered into an exclusive license agreement with Stanford University to develop human prophylactic and therapeutic products, enhancing its product development capabilities[57] - The Company entered into the IDT License Agreement with an upfront payment of $3.0 million and potential regulatory milestone payments of up to $5.3 million, totaling $8.8 million if expanding the licensed field[70] - The company entered into an asset purchase agreement on August 1, 2023, for the sale of certain assets related to its non-genotoxic conditioning technology, with total payments potentially reaching $1.5 million[123]

Financial Performance - Net loss for the three months ended March 31, 2023, was $23,934,000, a decrease from a net loss of $25,835,000 in the same period of 2022, reflecting an improvement of 7.3%[17] - The company reported a comprehensive loss of $23,355,000 for the three months ended March 31, 2023, compared to a comprehensive loss of $26,144,000 in the same period of 2022, indicating a 10.8% improvement[17] - The net loss for the three months ended March 31, 2023, was $23.934 million, compared to a net loss of $25.835 million for the same period in 2022, representing a 7.3% improvement[25] - Basic and diluted net loss per share attributable to common stockholders for Q1 2023 was $(0.43), an improvement from $(0.48) in Q1 2022[119] - Net loss for the three months ended March 31, 2023, was $23,934,000, compared to a net loss of $25,835,000 for the same period in 2022, representing a decrease of approximately 7.3%[119] Cash and Liquidity - Cash and cash equivalents increased to $69,811,000 as of March 31, 2023, up from $47,730,000 at December 31, 2022, representing a 46.3% increase[15] - The company had cash, cash equivalents, and restricted cash totaling $71.527 million at the end of the period, compared to $186.786 million at the end of the same period in 2022, indicating a decrease of 61.7%[25] - The company has $264.1 million in cash, cash equivalents, and marketable securities as of March 31, 2023, which is expected to fund operations for at least the next 12 months[32] Operating Expenses - Total operating expenses for the three months ended March 31, 2023, were $26,450,000, compared to $25,958,000 for the same period in 2022, an increase of 1.9%[17] - Research and development expenses were $16,244,000 for the three months ended March 31, 2023, down from $18,246,000 in the prior year, a reduction of 11.0%[17] - The company incurred restructuring costs of $2,583,000 during the three months ended March 31, 2023[17] - The company recorded $2.5 million in charges related to employee termination benefits as part of a restructuring plan that eliminated approximately 50% of the workforce[116] - The company expects to incur approximately $3.4 million in total employee termination benefits expense to implement the restructuring plan[116] Assets and Liabilities - Total current assets decreased slightly to $270,307,000 from $275,365,000, a decline of 1.9%[15] - Total liabilities increased to $33,772,000 as of March 31, 2023, from $25,611,000 at December 31, 2022, an increase of 31.8%[15] - Stockholders' equity decreased to $276,224,000 as of March 31, 2023, down from $296,291,000 at December 31, 2022, a decline of 6.8%[15] - The total fair value of cash equivalents and marketable securities was $264.1 million, a decrease from $283.6 million as of December 31, 2022[49] - The company has a total liability for operating leases of $4.541 million as of March 31, 2023, with a weighted average remaining lease term of 17 months[80] Stock and Equity - The weighted-average shares used in computing net loss per share were 55,864,475 for the three months ended March 31, 2023, compared to 54,005,299 for the same period in 2022[17] - The number of shares of restricted stock vested as of March 31, 2023, was 604,734, compared to 553,443 shares as of December 31, 2022[106] - The number of shares granted under the 2021 Plan increased by 2,911,088 shares on January 1, 2023, due to the evergreen provision[102] - The company reserved a total of 17,395,191 shares for future issuance as of March 31, 2023, compared to 13,893,161 shares as of December 31, 2022[92] - Total outstanding options to purchase common stock rose to 9,840,427 in Q1 2023, up from 6,994,758 in Q1 2022[120] Future Outlook - As of March 31, 2023, the company had an accumulated deficit of $266.3 million and expects to continue incurring substantial losses[32] - The company plans to continue research activities associated with its early-stage non-genotoxic conditioning program, aiming to advance toward potential development candidates[29] - The company has future development and regulatory milestone payments totaling up to $5.3 million and sales-based milestone payments of up to $7.5 million related to its exclusive license agreement with Stanford[62] - The company has a tenant improvement allowance of up to $14.9 million related to the Bayside Lease Agreement, expected to be fully utilized[73] Miscellaneous - The company did not recognize any realized gains or losses on the sale or maturity of available-for-sale securities during the three months ended March 31, 2023[52] - The company has not issued any shares or received any proceeds from offerings under its 2022 Shelf registration statement through May 11, 2023[33] - The company recorded an upfront license fee of $50,000 and agreed to issue approximately 0.6 million shares of common stock to Stanford as part of the license agreement[58] - The company does not provide quantitative and qualitative disclosures about market risk as a smaller reporting company under SEC rules[181] - No subsequent events were reported through May 11, 2023, indicating stability in the company's operations post-reporting period[122]

Product Development and Clinical Trials - The company announced a voluntary pause of its Phase 1/2 CEDAR study of its lead product candidate, nula-cel, for sickle cell disease due to a serious adverse event in the first patient dosed[23]. - In February 2023, the company decided to discontinue the development of nula-cel and explore strategic alternatives[29]. - Nula-cel demonstrated approximately 70% gene correction efficiency in hematopoietic stem and progenitor cells (HSPCs) in ex vivo studies, exceeding the expected curative threshold of 20%[30]. - Under IND-enabling GMP manufacturing conditions, over 55% of treated cells had the SCD mutation corrected, supporting the potential for a functional cure[31]. - The company plans to continue research on early-stage non-genotoxic conditioning programs despite discontinuing the nula-cel development, resulting in a 50% workforce reduction[41]. - Nula-cel was designed to directly correct the mutation responsible for sickle cell disease (SCD), aiming to restore normal adult hemoglobin expression[75]. - Nula-cel Phase 1/2 clinical trial initiated in November 2021 with approximately 15 patients, focusing on safety and gene correction rates[99]. - A voluntary pause was announced in January 2023 due to a serious adverse event in the first patient dosed, leading to the decision to discontinue nula-cel development in February 2023[99]. Financial Performance and Expectations - The company has incurred significant losses since inception and expects to continue incurring losses for the foreseeable future[20]. - The company has never generated revenue from product sales and may never achieve profitability[20]. - The corporate restructuring announced in February 2023 may not achieve its intended objectives[20]. Gene Editing Technology - The gene editing technology builds on first-generation CRISPR technology, achieving high rates of targeted gene integration[24]. - The company’s approach to gene editing allows for precise correction of pathogenic genes, differing from first-generation technologies that focused on gene knockout[27]. - The gene editing platform technology allows for the targeted insertion of entire gene cassettes, which could have broad therapeutic applications[33]. - The approach aims to avoid the risk of insertional oncogenesis associated with random gene integration methods, enhancing safety[30]. - The company believes that high efficiency and precision in gene integration could expand the patient population eligible for one-time curative therapies[30]. - CRISPR-Cas9 technology is being developed to create INDELs that alter gene expression, with current approaches favoring non-homologous end joining (NHEJ) over homology-directed repair (HDR) due to technical challenges[47]. - The new gene editing approach aims for high-efficiency targeted gene integration, potentially correcting any disease-causing genetic lesion and expanding therapeutic applications[52]. - The technology utilizes HDR to achieve targeted gene integration with efficiencies of up to approximately 70% in human hematopoietic stem and progenitor cells (HSPCs) in ex vivo studies[61]. - The platform combines CRISPR and HDR with expertise in HSCs to achieve high-efficiency targeted gene integration, significantly improving upon previous methods[63]. Market Need and Competitive Landscape - The global incidence of SCD is estimated at over 300,000 births annually, with approximately 100,000 individuals living with SCD in the United States and 67,000 in the European Union[77]. - Current FDA-approved therapies for SCD require lifelong usage and do not eliminate serious symptoms or complications, highlighting the unmet need for more effective treatments[79]. - Allo-HSCT remains the only curative therapy for SCD, but is rarely used due to difficulties in finding matched donors and safety risks[81]. - The company believes there is substantial underlying demand for curative options, as indicated by over 150 HSCT procedures performed annually in the United States[82]. - The gene therapy and gene editing fields are characterized by intense competition from major pharmaceutical and biotechnology companies[133]. Regulatory Environment - The FDA regulates biological products under the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act, requiring compliance with various preclinical, clinical, and commercial approval processes[168]. - The Biologics License Application (BLA) process requires extensive documentation, including results from preclinical studies and clinical trials, and must be submitted to the FDA for approval before marketing[180]. - The FDA aims to review standard BLA applications within ten months and priority applications within six months after acceptance for filing[181]. - The FDA may impose clinical holds on product candidates at any time due to safety concerns or non-compliance during clinical trials[170]. - The FDA may grant regulatory approval for a product with specific indications and limitations on marketing uses[184]. - Orphan drug designation is available for products treating rare diseases affecting fewer than 200,000 individuals in the U.S., with exclusivity for seven years upon first approval[193]. Licensing and Agreements - The company has entered into a Master Development and Manufacturing Services Agreement with WuXi Advanced Therapies Inc. for a term of five years, extendable by mutual agreement, to provide product candidate development and manufacturing services[140]. - An exclusive license agreement with Stanford allows the company to develop, manufacture, and commercialize products for the treatment of SCD, XSCID, and beta thalassemia, with an upfront license fee of $50,000 and a total consideration of $2.8 million recorded as research and development expense[143][144]. - The company is obligated to share a portion of sublicense income with Stanford, which varies from mid-teen to low double-digit percentages based on clinical milestones achieved[146]. - The company has agreed to pay Stanford up to $12.8 million upon achieving certain development, regulatory, and commercial milestones for each licensed product[147].