[PART I. FINANCIAL INFORMATION](index=3&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements](index=3&type=section&id=Item%201.%20Financial%20Statements) This section presents Editas Medicine, Inc.'s unaudited condensed consolidated financial statements as of June 30, 2024, showing a net loss of $129.6 million for the six months ended June 30, 2024, and a decrease in total assets and stockholders' equity [Condensed Consolidated Balance Sheets](index=3&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of June 30, 2024, total assets decreased to $384.8 million from $499.2 million, primarily due to reduced cash, while total stockholders' equity declined to $232.0 million from $349.1 million Condensed Consolidated Balance Sheets (in thousands) | Account | June 30, 2024 | Dec 31, 2023 | | :--- | :--- | :--- | | **Total Assets** | **$384,801** | **$499,153** | | Total current assets | $286,354 | $340,829 | | **Total Liabilities** | **$152,792** | **$150,056** | | Total current liabilities | $74,306 | $63,217 | | **Total Stockholders' Equity** | **$232,009** | **$349,097** | [Condensed Consolidated Statements of Operations](index=4&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) For the three and six months ended June 30, 2024, the company reported increased net losses of $67.6 million and $129.6 million, respectively, driven by higher R&D expenses and reduced collaboration revenue Statements of Operations Highlights (in thousands, except per share data) | Metric | Q2 2024 | Q2 2023 | 6 Months 2024 | 6 Months 2023 | | :--- | :--- | :--- | :--- | :--- | | Collaboration and other revenues | $513 | $2,887 | $1,649 | $12,738 | | Research and development | $54,210 | $29,779 | $102,997 | $67,583 | | General and administrative | $18,206 | $17,202 | $37,545 | $40,211 | | **Operating loss** | **$(71,903)** | **$(44,094)** | **$(138,893)** | **$(95,056)** | | **Net loss** | **$(67,607)** | **$(40,290)** | **$(129,557)** | **$(89,326)** | | **Net loss per share** | **$(0.82)** | **$(0.56)** | **$(1.58)** | **$(1.27)** | [Condensed Consolidated Statements of Cash Flows](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) For the six months ended June 30, 2024, net cash used in operating activities increased to $106.9 million, resulting in a net decrease in cash of $59.2 million, significantly impacted by lower financing proceeds Cash Flow Summary for the Six Months Ended June 30 (in thousands) | Activity | 2024 | 2023 | | :--- | :--- | :--- | | Net cash used in operating activities | $(106,934) | $(74,479) | | Net cash provided by investing activities | $47,209 | $36,249 | | Net cash provided by financing activities | $514 | $117,525 | | **Net (decrease) increase in cash** | **$(59,211)** | **$79,295** | [Notes to Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) These notes detail the company's business, liquidity, and accounting policies, highlighting its accumulated deficit, expected cash runway, and the amended BMS collaboration agreement * The company is a clinical-stage gene editing company that has incurred net operating losses every year since inception and had an accumulated deficit of **$1.4 billion** as of June 30, 2024[14](index=14&type=chunk)[18](index=18&type=chunk) * Management expects existing cash, cash equivalents, and marketable securities will be sufficient to fund operations and capital expenditures for at least **twelve months** from the issuance date of the financial statements (August 7, 2024)[18](index=18&type=chunk) * In March 2024, the collaboration agreement with BMS was amended, extending the term to November 2026 and providing BMS the ability to select new gene targets, with a total transaction price of approximately **$56.7 million** to be recognized prospectively[33](index=33&type=chunk)[34](index=34&type=chunk)[35](index=35&type=chunk) Stock-Based Compensation Expense (in thousands) | Category | Q2 2024 | Q2 2023 | 6 Months 2024 | 6 Months 2023 | | :--- | :--- | :--- | :--- | :--- | | Research and development | $2,125 | $2,525 | $5,033 | $4,611 | | General and administrative | $2,885 | $2,690 | $7,562 | $5,111 | | **Total** | **$5,010** | **$5,215** | **$12,595** | **$9,722** | [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=18&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides management's analysis of financial condition and operations, detailing progress on the reni-cel program, trends in revenues and expenses, and liquidity, with current cash expected to fund operations into 2026 [Overview](index=18&type=section&id=Overview) Editas Medicine, a clinical-stage gene editing company, is advancing its lead reni-cel program for SCD and TDT with positive clinical data, while also developing in vivo gene editing medicines * The lead program, reni-cel, is being evaluated in the RUBY trial for severe SCD and the EdiTHAL trial for TDT, with **enrollment complete** for adult cohorts in both trials and the adolescent cohort in RUBY[49](index=49&type=chunk)[50](index=50&type=chunk) * In the RUBY trial, all **18 treated SCD patients** were free of vaso-occlusive events post-infusion, and in the EdiTHAL trial, all **7 treated TDT patients** were transfusion-free[51](index=51&type=chunk)[52](index=52&type=chunk) * The company is advancing its in vivo gene editing platform and is on track to establish preclinical proof-of-concept for an undisclosed indication by **year-end 2024**[53](index=53&type=chunk) [Results of Operations](index=23&type=section&id=Results%20of%20Operations) This section analyzes the three and six months ended June 30, 2024, showing significant revenue decreases of 82% and 87%, respectively, alongside substantial increases in R&D expenses driven by the reni-cel program Comparison of Results for the Three Months Ended June 30 (in thousands) | Item | 2024 | 2023 | Change ($) | Change (%) | | :--- | :--- | :--- | :--- | :--- | | Revenues | $513 | $2,887 | $(2,374) | (82)% | | R&D Expenses | $54,210 | $29,779 | $24,431 | 82% | | G&A Expenses | $18,206 | $17,202 | $1,004 | 6% | | **Net Loss** | **$(67,607)** | **$(40,290)** | **$(27,317)** | **68%** | * The **$24.4 million** increase in Q2 2024 R&D expenses was primarily due to a **$19.9 million** rise in external costs for the reni-cel program's clinical and manufacturing activities and in vivo research[72](index=72&type=chunk)[73](index=73&type=chunk) Comparison of Results for the Six Months Ended June 30 (in thousands) | Item | 2024 | 2023 | Change ($) | Change (%) | | :--- | :--- | :--- | :--- | :--- | | Revenues | $1,649 | $12,738 | $(11,089) | (87)% | | R&D Expenses | $102,997 | $67,583 | $35,414 | 52% | | G&A Expenses | $37,545 | $40,211 | $(2,666) | (7)% | | **Net Loss** | **$(129,557)** | **$(89,326)** | **$(40,231)** | **45%** | * The **$35.4 million** increase in six-month R&D expenses was driven by a **$25.1 million** rise in external costs for the reni-cel program and a **$4.4 million** increase in sublicense and license fees[78](index=78&type=chunk)[79](index=79&type=chunk) [Liquidity and Capital Resources](index=28&type=section&id=Liquidity%20and%20Capital%20Resources) As of June 30, 2024, Editas held $318.3 million in cash, cash equivalents, and marketable securities, with management projecting these resources, combined with Vertex payments, will fund operations into 2026 * The company had cash, cash equivalents, and marketable securities of **$318.3 million** as of June 30, 2024[84](index=84&type=chunk) * Management projects its current cash position, along with expected payments from Vertex, will be sufficient to fund operations and capital expenditures **into 2026**[91](index=91&type=chunk) Cash Flow Summary for the Six Months Ended June 30 (in thousands) | Activity | 2024 | 2023 | | :--- | :--- | :--- | | Operating activities | $(106,934) | $(74,479) | | Investing activities | $47,209 | $36,249 | | Financing activities | $514 | $117,525 | [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=31&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk is interest rate sensitivity on its short-term, low-risk investment portfolio, with no material exposure to foreign currency exchange rate risk * The company's main market risk is interest rate sensitivity on its **$64.4 million** in cash and cash equivalents and **$253.9 million** in marketable securities[98](index=98&type=chunk) * Due to the short-term maturities and low risk of its investments, a **1% change** in interest rates is not expected to have a material effect on their fair market value[98](index=98&type=chunk) * The company does not believe it has any material exposure to foreign currency exchange rate risk as liabilities are substantially denominated in U.S. dollars[99](index=99&type=chunk) [Item 4. Controls and Procedures](index=31&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of June 30, 2024, with no material changes to internal control over financial reporting during the quarter * As of June 30, 2024, the CEO and CFO concluded that the company's disclosure controls and procedures were **effective** at the reasonable assurance level[100](index=100&type=chunk) * No material changes to the company's internal control over financial reporting occurred during the **second quarter of 2024**[101](index=101&type=chunk) [PART II. OTHER INFORMATION](index=33&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=33&type=section&id=Item%201.%20Legal%20Proceedings) The company may be involved in ordinary course legal proceedings, with intellectual property rights subject to validity disputes, as detailed in its Annual Report's Risk Factors * The company may become involved in litigation from the ordinary course of business and notes that its intellectual property rights are subject to **validity disputes**[103](index=103&type=chunk) [Item 1A. Risk Factors](index=33&type=section&id=Item%201A.%20Risk%20Factors) This section refers to the comprehensive risk factors detailed in the company's 2023 Annual Report on Form 10-K, which could materially affect its business and financial condition * The company directs investors to the 'Risk Factors' section of its **2023 Annual Report on Form 10-K** for a comprehensive discussion of risks affecting the business[104](index=104&type=chunk) [Item 5. Other Information](index=33&type=section&id=Item%205.%20Other%20Information) This section confirms that no directors or officers adopted or terminated Rule 10b5-1 trading arrangements during the second quarter of 2024 * During the quarterly period ended June 30, 2024, none of the company's directors or officers adopted or terminated a **Rule 10b5-1 trading arrangement**[106](index=106&type=chunk) [Item 6. Exhibits](index=34&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Quarterly Report on Form 10-Q, including CEO and CFO certifications and financial statements in Inline XBRL format * The report includes certifications from the Principal Executive Officer and Principal Financial Officer as required by the **Sarbanes-Oxley Act**[108](index=108&type=chunk) * Financial statements are provided in **Inline XBRL format** as part of the filing[108](index=108&type=chunk)

Financial Data and Key Metrics Changes - As of June 30, 2024, the company's cash, cash equivalents, and marketable securities were $318 million, down from $377 million as of March 31, 2024, indicating a higher burn rate due to increased external research and development expenses [13][14]. Business Line Data and Key Metrics Changes - The RUBY trial for sickle cell disease included 18 patients with follow-up periods ranging from 2.4 to 22.8 months, while the EdiTHAL trial for beta thalassemia included 7 patients with follow-up periods from 4.1 to 12.8 months [7]. - All patients in the RUBY trial were free from vaso-occlusive events post-infusion, with a mean total hemoglobin level exceeding 14 grams per deciliter and high fetal hemoglobin levels above 40% from six months onward [7][8]. Market Data and Key Metrics Changes - The company is focused on rare and orphan diseases for its in-vivo pipeline, which is expected to allow it to be first or best-in-class for specific indications [12]. Company Strategy and Development Direction - The company's strategy is built on three pillars: advancing reni-cel towards BLA and commercialization, developing a differentiated in-vivo editing pipeline, and increasing business development activities to monetize its strong IP [5][6]. - The company aims to establish preclinical proof-of-concept for an undisclosed indication in its in-vivo pipeline by the end of 2024 [10]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the progress towards BLA submission, aligning with FDA expectations and focusing on robust clinical data [9][43]. - The management highlighted the importance of capital efficiency in developing reni-cel and the potential for partnerships to expedite commercialization [9][63]. Other Important Information - The company is evaluating opportunities for IP licensing, with a strong foundational position from its agreements with Harvard, MIT, and the Broad [39]. - The management is optimistic about the potential for non-dilutive capital through IP licensing agreements [39]. Q&A Session Summary Question: Can you elaborate on the preclinical proof-of-concept for the in-vivo program? - Management confirmed they are on track to establish preclinical proof-of-concept by the end of the year, focusing on biodistribution, editing efficiency, and tolerability [18][19]. Question: Will you have an LMP targeted for the tissue of interest for the proof-of-concept readout? - Management indicated they are evaluating LMPs with different partners for targeting various tissue types but have not disclosed specific details yet [20][22]. Question: What is the timeline for the BLA submission with the adolescent reni-cel cohort now completed? - Management stated they are optimistic about collecting data for the BLA package but have not provided a specific timeline pending FDA alignment [25][26]. Question: What learnings from Kaskavi and Liginia will be utilized for the reni-cel launch? - Management noted that they anticipate significant uptake and are encouraged by the enrollment progress, which will inform their launch strategy [28][30]. Question: What is the average process time from patient enrollment to dose? - Management explained that the time varies significantly, with some patients taking as little as three months and others up to ten months or longer [33][34]. Question: How does the indel technology work for upregulating genes? - Management clarified that their strategy involves disrupting the binding site for repressors to upregulate gene expression, differentiating it from knockdown strategies [35][36]. Question: What is the potential market impact if fertility support is not provided post-approval? - Management expressed disappointment over recent decisions affecting fertility support and emphasized their commitment to addressing these issues for patients [61][62].

Editas Medicine (EDIT) came out with a quarterly loss of $0.82 per share versus the Zacks Consensus Estimate of a loss of $0.69. This compares to loss of $0.56 per share a year ago. These figures are adjusted for nonrecurring items. This quarterly report represents an earnings surprise of -18.84%. A quarter ago, it was expected that this genome editing company would post a loss of $0.63 per share when it actually produced a loss of $0.76, delivering a surprise of -20.63%. Over the last four quarters, the co ...

[Recent Achievements and Outlook](index=1&type=section&id=Recent%20Achievements%20and%20Outlook) Editas Medicine reported significant progress on its clinical programs, with clinical data for reni-cel in SCD and TDT expected by year-end 2024, alongside in vivo preclinical proof-of-concept for a new indication [Ex Vivo Hemoglobinopathies](index=1&type=section&id=Ex%20Vivo%20Hemoglobinopathies) The reni-cel program for severe sickle cell disease and beta thalassemia is rapidly advancing, with enrollment complete and substantial clinical data expected by year-end 2024 - The company is on track to present substantive clinical data from the RUBY trial (SCD) and additional data from the EdiTHAL trial (TDT) by the end of 2024[1](index=1&type=chunk)[2](index=2&type=chunk)[3](index=3&type=chunk) - Enrollment for the adolescent cohort of the RUBY trial (SCD) and the adult cohort of the EdiTHAL trial (TDT) has been completed[2](index=2&type=chunk)[3](index=3&type=chunk) - CEO Gilmore O'Neill expressed high confidence that reni-cel is positioned to be a differentiated, best-in-class product for treating sickle cell disease[2](index=2&type=chunk) [In Vivo Medicines](index=1&type=section&id=In%20Vivo%20Medicines) The company is advancing its in vivo strategy to upregulate gene expression, targeting preclinical proof-of-concept for an undisclosed indication by year-end 2024 - Editas is on track to establish in vivo preclinical proof-of-concept for an undisclosed indication by the end of 2024[1](index=1&type=chunk)[4](index=4&type=chunk) - The in vivo strategy aims to drive functional upregulation of gene expression to address loss of function or deleterious mutations[4](index=4&type=chunk) [Second Quarter 2024 Financial Results](index=2&type=section&id=Second%20Quarter%202024%20Financial%20Results) Editas Medicine reported a **net loss of $67.6 million** in Q2 2024, driven by increased R&D expenses and decreased collaboration revenue, maintaining a **cash position of $318.3 million** into 2026 Q2 2024 Financial Highlights (Three Months Ended June 30) | Metric | Q2 2024 (Millions USD) | Q2 2023 (Millions USD) | | :--- | :--- | :--- | | Collaboration & Other Revenues | 0.5 | 2.9 | | Research & Development Expenses | 54.2 | 29.8 | | General & Administrative Expenses | 18.2 | 17.2 | | Net Loss | 67.6 | 40.3 | | Net Loss Per Share (USD) | 0.82 | 0.56 | - The increase in R&D expenses was primarily due to clinical and manufacturing costs for the accelerated reni-cel program and costs for in vivo research[6](index=6&type=chunk) - The decrease in revenue was mainly attributable to reduced drug supply activity with collaborators[6](index=6&type=chunk) Cash Position | Date | Cash, Cash Equivalents, and Marketable Securities (Millions USD) | | :--- | :--- | | June 30, 2024 | 318.3 | | March 31, 2024 | 376.8 | - The company expects its existing cash, along with anticipated license fees, to fund operations and capital expenditures into 2026[5](index=5&type=chunk) [Financial Statements](index=4&type=section&id=Financial%20Statements) This section presents the detailed unaudited Consolidated Statement of Operations and Selected Consolidated Balance Sheet Items for the periods ended June 30, 2024 [Consolidated Statement of Operations](index=4&type=section&id=Consolidated%20Statement%20of%20Operations) For the six months ended June 30, 2024, the company's operating loss widened to **$138.9 million**, driven by increased R&D expenses and decreased revenues Consolidated Statement of Operations (Six Months Ended June 30, in thousands) | Metric | 2024 (Thousands USD) | 2023 (Thousands USD) | | :--- | :--- | :--- | | Collaboration and other revenues | 1,649 | 12,738 | | Research and development expenses | 102,997 | 67,583 | | General and administrative expenses | 37,545 | 40,211 | | Operating loss | (138,893) | (95,056) | | Net loss | (129,557) | (89,326) | | Net loss per share (USD) | (1.58) | (1.27) | [Selected Consolidated Balance Sheet Items](index=5&type=section&id=Selected%20Consolidated%20Balance%20Sheet%20Items) As of June 30, 2024, Editas Medicine's balance sheet shows **cash, cash equivalents, and marketable securities at $318.3 million**, a decrease from year-end 2023 Selected Consolidated Balance Sheet Items (in thousands) | Metric | June 30, 2024 (Thousands USD) | December 31, 2023 (Thousands USD) | | :--- | :--- | :--- | | Cash, cash equivalents, and marketable securities | 318,307 | 427,135 | | Working capital | 212,048 | 277,612 | | Total assets | 384,801 | 499,153 | | Total stockholders' equity | 232,009 | 349,097 |

Core Insights - Editas Medicine is making significant progress in its clinical trials, particularly with its product reni-cel for sickle cell disease, and is on track to present additional clinical data from the RUBY and EdiTHAL trials by year-end 2024 [1][3][4] - The company maintains a strong financial position with cash reserves expected to fund operations into 2026 [6][13] - The net loss for the second quarter of 2024 was $67.6 million, an increase from $40.3 million in the same period in 2023, indicating rising operational costs [7][12] Clinical Development - Reni-cel is positioned as a best-in-class treatment for sickle cell disease, with substantial clinical updates shared mid-year and ongoing patient enrollment in the RUBY trial [2][3] - The adolescent cohort of the RUBY trial has completed enrollment, and the company continues to dose adult patients [3] - The EdiTHAL trial for transfusion-dependent beta thalassemia has also completed enrollment for the adult cohort, with ongoing patient dosing [4] Financial Performance - As of June 30, 2024, the company reported cash, cash equivalents, and marketable securities totaling $318.3 million, down from $376.8 million as of March 31, 2024 [6][13] - Collaboration and other research and development revenues decreased to $0.5 million in Q2 2024 from $2.9 million in Q2 2023, primarily due to reduced drug supply activity [7][12] - Research and development expenses increased significantly to $54.2 million in Q2 2024, up from $29.8 million in Q2 2023, driven by costs associated with the accelerated progression of the reni-cel program [7][8][12] Strategic Outlook - The company is on track to establish in vivo preclinical proof-of-concept for an undisclosed indication by year-end 2024, indicating ongoing innovation in its research strategy [5] - Editas Medicine aims to develop transformative genomic medicines for serious diseases, leveraging its expertise in CRISPR technology [10]

Core Insights - Editas Medicine has reported positive data from its RUBY and EdiTHAL studies, demonstrating the efficacy and safety of its lead candidate, reni-cel, in treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) [6][12][20] Company Progress - Editas has dosed over 20 SCD patients and completed adult cohort enrollment while also enrolling patients in the adolescent cohort of the RUBY study, indicating significant progress [2] - All patients in both studies showed successful neutrophil and platelet engraftment after treatment with reni-cel [3] Efficacy Results - In the RUBY study, SCD patients experienced a sustained increase in the mean percentage of HbF-containing red cells, remaining above 90% from month 4 onward for 12 patients with at least four months of follow-up [7] - The mean corpuscular fetal hemoglobin of F-cells remained above the anti-sickling threshold of 10 pg/F-cell by month 3 post-reni-cel infusion for 14 patients with at least three months of follow-up [7] - In the EdiTHAL study, all TDT patients experienced early increases in total hemoglobin (Hb) levels, rising above the transfusion independence threshold of 9.0 g/dL, with all seven patients remaining transfusion-free at last follow-up for 4.1 to 12.8 months [14] Safety Profile - Safety data from both studies indicated that reni-cel was well-tolerated, with a safety profile consistent with myeloablative conditioning and autologous hematopoietic stem cell transplant, and no serious treatment-related adverse events reported [20] Competitive Landscape - Vertex and CRISPR Therapeutics are conducting new phase III studies for SCD and TDT, and their gene therapy, Casgevy, has been approved in multiple regions, including the US and EU [4][9][21]

EHA RUBY oral presentation on Saturday, June 15 at 11:30 a.m. CEST/5:30 a.m. EDT "I am encouraged by these results from the RUBY trial, demonstrating this investigational gene editing medicine has been well-tolerated and shows promising efficacy for people living with sickle cell disease. Treatment with reni-cel showed a favorable safety profile and promising preliminary efficacy, supporting further investigation as a differentiated gene-edited medicine for patients with SCD. We look forward to continuing t ...

All patients treated in the EdiTHAL trial maintained hemoglobin levels above the transfusion threshold and are transfusion-free post-renizgamglogene autogedtemcel (reni-cel) infusion In the EdiTHAL trial to date, reni-cel was well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all patients (N=7). Following treatment with reni-cel, all EdiTHAL patients had early and robust increase of total ...

Summary of Editas Medicine, Inc. Conference Call Company Overview - **Company**: Editas Medicine, Inc. (NASDAQ:EDIT) - **Industry**: Biotechnology, specifically focused on gene editing and therapies for genetic diseases Key Points and Arguments Progress on Reni-cel Asset - Reni-cel is now in late-stage pivotal studies, with FDA approval to transition the sickle cell RUBY study from Phase 1/2 to Phase 1/2/3 [3] - Enrollment for the adult cohort is complete, and the adolescent cohort is enrolling faster than expected [3] - Data presentation at the European Hematology Association (EHA) is scheduled, with at least 18 patients having 2 to 21 months of follow-up [3][5] In Vivo Delivery Technologies - Focus on nanoparticle delivery for in vivo applications, emphasizing functional upregulation rather than knockdown strategies [4] - Internal validation of this approach is ongoing, with future updates anticipated [4] Business Development and Financing - A significant deal with Vertex was executed to enable their launch of Cas JV, extending Editas' cash runway into 2026 [5] - This deal also validates the value of Editas' intellectual property (IP) [5] Clinical Data and Differentiation - Reni-cel shows an average of 45% upregulation of fetal hemoglobin in sickle cell patients, with no severe vaso-occlusive events reported post-treatment [8] - All patients have corrected their anemia to within normal ranges, which is a significant clinical improvement [9] Safety and Adverse Events - A previously reported case of Grade 2 polycythemia was determined to be unrelated to reni-cel and was transient, associated with iron therapy [11] BLA Filing Strategy - Editas is in discussions with the FDA regarding the Biologics License Application (BLA) strategy, with a focus on presenting a robust dataset [14] - The company is optimistic about the timing of the BLA filing, potentially in the second half of the next year [13] Pricing Strategy - Editas is conducting extensive work to understand pricing but has not finalized discussions [17] - The anticipated price by ICER is positioned in the middle of the pricing spectrum observed in the industry [18] Focus on In Vivo Pipeline - The company is pivoting towards in vivo therapies, moving away from minor conditioning strategies due to complexity and resource allocation [21][22] - Emphasis on targeting hematopoietic stem cells and other tissues for functional upregulation [22] International Market Opportunities - Editas is exploring partnerships outside the U.S., particularly in the Middle East, where there is a high prevalence of hemoglobinopathies [24] Intellectual Property Strategy - Future deals regarding IP can occur at any stage of a product's lifecycle, with flexibility in deal structure and timing [29] Additional Important Insights - The efficacy of gene editing technologies is highlighted as unprecedented, with a response rate approaching 100% [18] - The potential cost savings for patients with sickle cell disease, who may incur significant lifetime healthcare costs, is emphasized [19] - The company is focused on maximizing the probability of commercial success by differentiating its therapies from standard care [27]

Editas Medicine, Inc. (NASDAQ:EDIT) RBC Capital Markets Global Healthcare Conference Call May 15, 2024 2:05 PM ET Company Participants Gilmore O'Neill - Chief Executive Officer Conference Call Participants Luca Issi - RBC Capital Markets Luca Issi Thanks, everybody. Luca Issi, senior biotech analyst here at RBC Capital Markets. Today is our great privilege to have Editas for us with us with a fireside chat. Representing the company, we have Gilmore O'Neill, Chief Executive Officer. Gilmore thanks so much fo ...