Financial Data and Key Metrics Changes - The company reported cash, cash equivalents, and marketable securities of $427 million as of December 31, 2023, down from $446 million as of September 30, 2023 [46] - Revenue for the fourth quarter of 2023 was $60 million, primarily from a license agreement with Vertex [47] - R&D expenses increased by $18 million to $70 million compared to the fourth quarter of 2022, reflecting additional sub-license expenses [65] - G&A expenses for the fourth quarter of 2023 were $14 million, down from $18 million in the same quarter of 2022 [78] Business Line Data and Key Metrics Changes - The company has enrolled 40 sickle cell and 9 beta thalassemia patients in the RUBY and EdiTHAL studies, with 18 RUBY patients and 7 EdiTHAL patients dosed [27][41] - The RUBY trial is now considered a Phase 1, 2, 3 study for BLA submission, with alignment on study design and endpoints with the FDA [42][36] Market Data and Key Metrics Changes - The company is focused on the gene editing market, particularly in hemoglobinopathies, and has engaged with the FDA regarding the RUBY trial [34][55] - The competitive landscape includes a recently approved gene editing treatment for sickle cell disease, which the company is monitoring closely [104] Company Strategy and Development Direction - The company aims to drive reni-cel towards BLA and commercialization, strengthen its discovery organization, and increase business development activities [34] - The company has decided not to pursue internal development of a milder conditioning regimen, focusing instead on in vivo HSE medicines [55] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing clinical trials and the potential for reni-cel to provide a competitive edge in the market [62] - The company plans to provide clinical updates from the RUBY and EdiTHAL trials in mid-2024 and by year-end 2024 [39] Other Important Information - The company has strengthened its senior leadership team with experienced professionals in drug development and commercialization [34] - The company has activated over 20 sites for the RUBY trial, with ongoing patient recruitment and dosing [100] Q&A Session Summary Question: Can you elaborate on the hemolysis markers being tracked? - The company tracks multiple hemolysis markers including reticulocyte, LDH, and bilirubin, and uses various instruments to measure patient-reported outcomes related to quality of life [70] Question: Can you share insights into your FDA conversations regarding the RUBY trial? - The company has aligned with the FDA on the RUBY trial as a Phase 1, 2, 3 study, including endpoint, sample size, and study design [72][74] Question: How does the Phase 1, 2, 3 designation impact the BLA path? - The designation allows for a seamless transition to support the BLA, utilizing all patient data from the study [102][88] Question: What are the latest thoughts on how reni-cel fits in the market? - The company sees robust market development and initial interest from stakeholders, with ongoing engagement in clinical studies [105] Question: Can you provide an update on the CRISPR-Cas9 appeal case? - The Court of Appeals has yet to schedule an oral hearing, but it is expected to occur sometime in 2024 [96] Question: What is the status of the adolescent cohort in the RUBY study? - The adolescent cohort has been initiated, with high interest and demand from patients [59] Question: How is the company addressing the patient experience with reni-cel? - The company is pleased with the number of apheresis cycles required and reports that all patients have achieved engraftment within 30 days [167]

Editas Medicine (EDIT) came out with a quarterly loss of $0.23 per share versus the Zacks Consensus Estimate of a loss of $0.52. This compares to loss of $0.88 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 55.77%. A quarter ago, it was expected that this genome editing company would post a loss of $0.64 per share when it actually produced a loss of $0.55, delivering a surprise of 14.06%.Over the last four quarters, the compa ...

Editas Medicine Announces Fourth Quarter and Full Year 2023 Results and Business Updates Company aligned with FDA that RUBY is a single Phase 1/2/3 trial On track to present additional clinical data from the RUBY trial and the EdiTHAL trial of reni-cel in mid-2024 and additional updates by year-end 2024 Initiated enrollment in the adolescent cohort in the RUBY trial Entered into a license agreement providing Vertex Pharmaceuticals a non-exclusive license for Cas9 Strong financial position with operational r ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 __________________________________________________ FORM 10-K __________________________________________________ (Mark One) x ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 or o TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ____________ to ________ Commission File Nu ...

Shares of Editas Medicine (EDIT) have gained 0.7% over the past four weeks to close the last trading session at $8.15, but there could still be a solid upside left in the stock if short-term price targets of Wall Street analysts are any indication. Going by the price targets, the mean estimate of $14.67 indicates a potential upside of 80%.The average comprises 12 short-term price targets ranging from a low of $7 to a high of $27, with a standard deviation of $6.69. While the lowest estimate indicates a decl ...

Editas Medicine, Inc. (NASDAQ:EDIT) Oppenheimer 34th Annual Healthcare Life Sciences Conference February 13, 2024 8:40 AM ET Company Participants Dr. Baisong Mei - Senior Vice President and Chief Medical Officer Conference Call Participants Jay Olson - Oppenheimer Jay Olson Hello everyone and welcome to Oppenheimer’s 34th Annual Healthcare Conference. I am Jay Olson, one of the biotech analysts at Oppenheimer and I want to thank you for joining us. It's my pleasure to welcome Editas to our conference, and i ...

Quite a few bigwigs in the biotech sector have reported results for the fourth quarter and the picture is ordinary. While results have been mixed, the outlook provided by most of these companies is encouraging, indicating bright prospects driven by new drug approvals and positive pipeline updates. The macroeconomic environment, however, remains uncertain and growth might be sluggish.Meanwhile, biotech companies are in the spotlight as pharma/biotech giants are now looking to bolster their product portfolios ...

Editas Medicine, Inc. (NASDAQ:EDIT) Guggenheim 6th Annual Biotechnology Conference February 7, 2024 9:30 AM ET Company Participants Gilmore O'Neill - President and CEO Conference Call Participants Debjit Chattopadhyay - Guggenheim Debjit Chattopadhyay Good morning. Thank you for joining the Guggenheim Securities Healthcare team at our 6th Annual Conference. I'm Debjit and joining us from Editas Medicine is its President and CEO, Gilmore O'Neill. Thank you so much for your time Gilmore. And if we could start ...

Summary of Editas Medicine, Inc. Conference Call Company Overview - Editas Medicine is a clinical stage company focused on programmable CRISPR-mediated gene editing, with its lead program being reni-cel, a therapy for hemoglobinopathies including sickle cell disease and beta thalassemia [2][3] Strategic Transition - The company is transitioning from a technology platform to a commercial therapeutics company, focusing on three strategic pillars: 1. Driving the reni-cel asset towards BLA (Biologics License Application) and commercialization 2. Concentrating on the development of in vivo therapeutics 3. Increasing business development activities to exploit intellectual property (IP) for non-dilutive capital [2][3] Clinical Trials and Data - The RUBY trial for reni-cel has enrolled over 25 patients and is on track for full enrollment this year, with adolescent recruitment initiated [5] - A significant clinical data set is expected to be presented mid-year, focusing on vaso-occlusive events and hematological data [5][39] - The company has observed robust correction of anemia in patients after four to five months of follow-up [6][39] Market Dynamics - Following the approval of exa-cel, Editas has not faced challenges in enrollment; instead, there has been increased enthusiasm from clinical trial sites and patients [9][10] - The FDA granted RMAT (Regenerative Medicine Advanced Therapy) designation, indicating a continued unmet need in the market [10] Mechanism of Action - Editas utilizes a different enzyme (Cas12a) and targets the HBG1 promoter to upregulate fetal hemoglobin, which is believed to provide a differentiation advantage over competitors like exa-cel [12][13] Patient Considerations - The company is addressing patient concerns regarding fertility by implementing best practices for fertility preservation [18] - Efforts are being made to reduce the vein-to-vein time for patients, which currently averages three to six months [15][16] Future Pipeline and Development - Editas is focused on high conviction targets for its in vivo pipeline, avoiding areas where patients are already well served [22][23] - The company is exploring delivery mechanisms beyond AAV (adeno-associated virus) and is interested in nanoparticles for gene delivery [25][27] Licensing and Partnerships - Editas has a non-exclusive licensing agreement with Vertex for Cas9, which is seen as a template for future partnerships [30][31] Upcoming Milestones - The company plans to unveil a second program targeting non-human primate proof-of-concept for in vivo editing later this year [35][36] - A meaningful data set from the reni-cel trial is expected to support a BLA filing in 2025, contingent on discussions with the FDA [41][42] Capacity and Market Position - Editas believes being a fast follower in the market could provide advantages, allowing the company to benefit from the groundwork laid by competitors [47]

Editas Medicine (EDIT) could be a solid choice for investors given its recent upgrade to a Zacks Rank #2 (Buy). This upgrade is essentially a reflection of an upward trend in earnings estimates -- one of the most powerful forces impacting stock prices.A company's changing earnings picture is at the core of the Zacks rating. The system tracks the Zacks Consensus Estimate -- the consensus measure of EPS estimates from the sell-side analysts covering the stock -- for the current and following years.Since a cha ...