Editas Medicine announces advancements in in vivo CRISPR gene editing, optimizing costs, and reducing workforce while focusing on hematologic diseases.
Quiver AI Summary
Editas Medicine, Inc. has announced a strategic shift towards focusing on in vivo CRISPR-edited therapies following significant pre-clinical advancements in treating sickle cell disease and beta thalassemia. The company achieved proof of concept for high levels of HBG1/2 promoter editing and hemoglobin-F induction using a targeted lipid nanoparticle formulation in a humanized mouse model. Additionally, successful editing in the liver was confirmed in non-human primates. To streamline operations and extend cash resources into Q2 2027, Editas will significantly reduce its workforce by approximately 65% and discontinue the development of its reni-cel program. Management changes will accompany this transition, with a new Board Chair appointed. Editas remains committed to advancing its in vivo pipeline and will share further development details in early 2025.
Potential Positives
- Achieved pre-clinical in vivo proof of concept for high level HBG1/2 promoter editing and HbF induction in a humanized mouse model, demonstrating potential for treating sickle cell disease and beta thalassemia.
- In vivo proof of concept for high efficiency editing in the liver in non-human primates indicates strong progress in the development of gene editing technologies.
- Transitioning to a fully in vivo company strategically positions Editas Medicine to expand its therapeutic possibilities and aims for human proof of concept within approximately two years.
- Extending cash runway into Q2 2027 suggests improved financial stability and a proactive approach to cost management during this transition phase.
Potential Negatives
- Ending development of reni-cel after an extensive search for a commercial partner indicates difficulties in securing collaboration, which may affect the company's credibility and future partnerships.
- The planned reduction in workforce of approximately 65% raises concerns about the company’s stability and ability to execute its strategy effectively moving forward.
- The necessity of cost-saving measures and restructuring may signal underlying financial instability or operational challenges within the company.
FAQ
What recent scientific breakthroughs has Editas Medicine achieved?
Editas Medicine has achieved pre-clinical in vivo proof of concept for HBG1/2 promoter editing and HbF induction in humanized mouse models.
How does Editas Medicine plan to extend its cash runway?
The company is initiating cost-saving measures and reducing headcount to extend its cash runway into Q2 2027.
What does the transition to an in vivo company mean for Editas Medicine?
This transition focuses on developing in vivo programmable gene editing medicines, enhancing therapeutic possibilities for CRISPR-based treatments.
What are the implications of the headcount reduction at Editas Medicine?
Editas Medicine will reduce its workforce by approximately 65%, impacting various staff and management members to realign resources.
When will Editas Medicine provide updates on pre-clinical data?
Editas Medicine plans to share pre-clinical data and development timelines for its programs in the first quarter of 2025.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
$EDIT Insider Trading Activity
$EDIT insiders have traded $EDIT stock on the open market 6 times in the past 6 months. Of those trades, 0 have been purchases and 6 have been sales.
Here’s a breakdown of recent trading of $EDIT stock by insiders over the last 6 months:
- BAISONG MEI (EVP, CHIEF MEDICAL OFFICER) has traded it 3 times. They made 0 purchases and 3 sales, selling 7,678 shares.
- GILMORE NEIL O'NEILL (CEO) has traded it 2 times. They made 0 purchases and 2 sales, selling 3,173 shares.
- LINDA BURKLY (EVP, CHIEF SCIENTIFIC OFFICER) sold 11,886 shares.
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We have seen 103 institutional investors add shares of $EDIT stock to their portfolio, and 132 decrease their positions in their most recent quarter.
Here are some of the largest recent moves:
- STATE STREET CORP removed 2,128,891 shares (-46.4%) from their portfolio in Q3 2024
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- UBS ASSET MANAGEMENT AMERICAS LLC added 818,056 shares (+978.9%) to their portfolio in Q2 2024
- TWO SIGMA INVESTMENTS, LP added 818,001 shares (+151.3%) to their portfolio in Q3 2024
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Full Release
Focus on
in vivo
CRISPR-edited medicines based on Editas researchers’ recent scientific progress in multiple tissues:
Achieved pre-clinical
in vivo
proof of concept of high level HBG1/2 promoter editing and HbF induction in a humanized mouse model for treatment of sickle cell disease and beta thalassemia with a single dose of an HSC-targeted lipid nanoparticle (tLNP) formulation
Achieved
in vivo
proof of concept of high efficiency editing in the liver in non-human primates
Ending development of reni-cel after extensive search did not yield a commercial partner
The Company will work closely with the clinical trial sites, regulators, and other parties to determine the path forward for patients enrolled in the RUBY and EdiTHAL trials
Initiating cost savings measures and reduction in headcount to align workforce and resources to
in vivo
pipeline, extending cash runway into Q2 2027
Conference call and webcast today at 5:00 p.m. ET
CAMBRIDGE, Mass., Dec. 12, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading gene editing company, today announced a critical pivot to optimize its cost structure, extend its cash runway into Q2 2027, and position the Company to accelerate its intent to achieve
in vivo
human proof of concept in approximately two years.
“Recent scientific breakthroughs by the Editas team have convinced us that the timelines around the near-term viability of
in vivo
CRISPR-edited medicines have accelerated meaningfully. Two years ago, we laid out our strategy and objective to become a leader in
in vivo
programmable gene editing. Based on these advances, we are transitioning to a fully
in vivo
company. We believe the ability to provide
in vivo
gene editing that functions via gene upregulation across tissues holds the potential to significantly expand the addressable therapeutic possibilities for CRISPR-based gene editing and uniquely position Editas to be a leader in the field moving forward,” said Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine.
The Company transition follows the recent
in vivo
pre-clinical proof of concept in multiple tissues:
Hematopoietic Stem Cells (HSCs):
Editas achieved ~40% editing of the
HBG1/2
promoter site after using a novel, Editas-proprietary targeted lipid nanoparticle (tLNP) for extrahepatic tissue delivery to deliver a single dose of its clinically validated Cas12a editing machinery directly to human hematopoietic stem cells (HSCs) in mice engrafted with human HSCs.
1
HBG1/2
biology has been validated and derisked in patients with reni-cel in the RUBY trial.
The editing in HSCs with the Company’s proprietary tLNP formulation resulted in the meaningful functional outcome of HbF induction, indicated by the presence of HbF expressing human red blood cells (on average 20%) that populate in the host by one month.
Liver:
The Company achieved
in vivo
proof of concept of high efficiency editing in the liver in non-human primates under its collaboration with Genevant.
The Company intends to share pre-clinical data and further development timelines from these programs in the first quarter of 2025.
In vivo
HSC editing success is expected to enable extrahepatic tissues/cell types targeting beyond HSCs and demonstrates the potential of “plug ‘n play” in an
in vivo
extrahepatic LNP platform. The Company’s upregulation capability additionally enables a differentiated strategy for liver targets for diseases with high unmet need and first-in-class opportunities.
In connection with Editas Medicine’s transition to an
in vivo
company, the Company initiated a reduction in headcount that will eliminate approximately 65% of its workforce over the next six months. As part of this reduction in force, several members of the Editas management team will depart the company over the next six months, including Baisong Mei, M.D., Ph.D., the Company’s Chief Medical Officer.
Additionally, Emma Reeve and Meeta Chatterjee, Ph.D. are resigning from the Board of Directors, effective December 31, 2024. Jessica Hopfield, Ph.D., has been named Chair of the Board, effective December 31, 2024.
Dr. O’Neill added, “We want to extend our deepest appreciation to patients, investigators, clinical sites staff, and our employees who have shown tremendous dedication and commitment to developing a potentially transformational medicine like reni-cel. We also want to express specific gratitude to the patients in our clinical trials and their caregivers whose dedication to disease research for their community makes us even more committed to accelerating our efforts towards an
in vivo
program for sickle cell disease and beta thalassemia.”
Conference Call
The Editas Medicine management team will host a conference call and
webcast
today at 5:00 p.m. ET. To access the call, please dial 1-877-407-0989 (domestic) or +1 201-389-0921 (international) and ask for the Editas Medicine conference call. A live webcast of the call will also be available on the Investors section of the Editas Medicine website at
www.editasmedicine.com
, and a replay will be available approximately two hours after its completion.
About
Editas Medicine
As a leading gene editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of
in vivo
medicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision
in vivo
gene editing medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit
www.editasmedicine.com
.
Forward-Looking Statements
This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Company’s transition to a fully
in vivo
company, the intention to achieve human proof of concept in approximately two years, and the potential success of its
in vivo
gene editing programs, the timing for releasing additional pre-clinical data, the anticipated effects, including potential cost savings, of the Company’s decision to discontinue development of reni-cel and initiate the related reduction in headcount, the scope and timing of the reduction in headcount, and the expected extension of the Company’s cash runway. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies; availability and timing of results from preclinical studies; expectations for regulatory approvals to conduct trials; availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; and that the decision to discontinue clinical development of reni-cel and the related reduction in headcount may have unexpected consequences or not result in the expected cost savings. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, as updated by the Company’s subsequent filings with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.
1
Previously disclosed editing of 29% in hematopoietic stem and progenitor cell (HSPCs) at one week after a single dose in a
Strategic Update webinar
in October 2024.
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