Editas Medicine Progresses Towards Two In Vivo Editing Development Candidates by Mid-2025 and Expands Research Pipeline

Editas Medicine reports advancements in in vivo gene editing, financial results, and plans for upcoming development candidates by mid-2025.

Quiver AI Summary

Editas Medicine, Inc. announced its progress in developing in vivo gene editing therapies, with plans to declare two development candidates for gene upregulation in hematopoietic stem cells (HSCs) and liver by mid-2025. The company aims to present additional preclinical data for these programs by the end of 2025 while also establishing a new target cell type. Despite a significant net loss in 2024 and the discontinuation of the reni-cel program due to lack of commercial partnership, Editas reported a strong cash position that extends its runway into the second quarter of 2027. The CEO emphasized the potential of the gene upregulation platform to address serious diseases with a single-dose strategy, marking a significant step toward clinical development.

Potential Positives

• Announced plans to declare two in vivo editing development candidates, one in HSCs and one in liver, by mid-2025, indicating progress in their gene editing pipeline.
• Demonstrated preclinical proof of concept in non-human primates and humanized mice, validating the effectiveness of their gene upregulation strategy across multiple tissues.
• Strong cash position of $269.9 million, providing operational runway into the second quarter of 2027, which supports ongoing research and development efforts.
• On track to establish one additional target cell type/tissue by year-end, showcasing the company's commitment to expanding its therapeutic possibilities in gene editing.

Potential Negatives

• Significant increase in net loss of $237.1 million for 2024 compared to $153.2 million in 2023, raising concerns about financial sustainability.
• Collaboration and research revenues decreased sharply from $78.1 million in 2023 to $32.3 million in 2024, indicating potential challenges in securing partnerships.
• Restructuring charges of $12.2 million due to the discontinuation of the reni-cel program and workforce reduction, which could affect employee morale and company culture.

FAQ

What are Editas Medicine's upcoming in vivo editing development candidates?

Editas Medicine is on track to declare two in vivo editing development candidates in mid-2025, one in HSCs and one in the liver.

When will Editas present additional preclinical data?

The company plans to present further in vivo HSC preclinical data and liver data by year-end 2025.

What is the current financial position of Editas Medicine?

Editas Medicine has a strong cash position with funding into the second quarter of 2027, totaling approximately $269.9 million.

What recent achievements has Editas Medicine accomplished?

Editas achieved preclinical proof of concept for its in vivo gene editing strategy, demonstrating effectiveness in hematopoietic stem cells and liver cells.

What impact did the reni-cel program have on Editas's expenses?

The discontinuation of the reni-cel program led to a $12.2 million restructuring charge in 2024, aligning resources with the in vivo pipeline.

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 4 times in the past 6 months. Of those trades, 0 have been purchases and 4 have been sales.

Here’s a breakdown of recent trading of $EDIT stock by insiders over the last 6 months:

• GILMORE NEIL O'NEILL (CEO) has made 0 purchases and 2 sales selling 3,173 shares for an estimated $8,686.
• BAISONG MEI (EVP, CHIEF MEDICAL OFFICER) has made 0 purchases and 2 sales selling 1,059 shares for an estimated $2,897.

To track insider transactions, check out Quiver Quantitative's insider trading dashboard.

$EDIT Hedge Fund Activity

We have seen 85 institutional investors add shares of $EDIT stock to their portfolio, and 151 decrease their positions in their most recent quarter.

Here are some of the largest recent moves:

• DEEP TRACK CAPITAL, LP removed 4,900,334 shares (-100.0%) from their portfolio in Q4 2024, for an estimated $6,223,424
• AVIDITY PARTNERS MANAGEMENT LP removed 1,465,000 shares (-100.0%) from their portfolio in Q4 2024, for an estimated $1,860,550
• RENAISSANCE TECHNOLOGIES LLC added 1,451,049 shares (+inf%) to their portfolio in Q4 2024, for an estimated $1,842,832
• PICTET ASSET MANAGEMENT HOLDING SA removed 1,155,937 shares (-100.0%) from their portfolio in Q4 2024, for an estimated $1,468,039
• BALYASNY ASSET MANAGEMENT L.P. added 1,126,569 shares (+647.3%) to their portfolio in Q4 2024, for an estimated $1,430,742
• MONACO ASSET MANAGEMENT SAM added 1,069,732 shares (+inf%) to their portfolio in Q4 2024, for an estimated $1,358,559
• BLACKROCK, INC. removed 716,636 shares (-8.9%) from their portfolio in Q4 2024, for an estimated $910,127

To track hedge funds' stock portfolios, check out Quiver Quantitative's institutional holdings dashboard.

Full Release

On track to declare two

in vivo

editing


development candidates via gene upregulation, one in HSCs and one in liver, in mid-2025




Company to present further

in vivo

HSC preclinical data and further

in vivo

preclinical


data in one liver indication by year-end




On track to establish one additional target cell type/tissue by year-end




Strong cash position with operational runway into the second quarter of 2027

CAMBRIDGE, Mass., March 05, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company focused on developing transformative medicines for serious diseases, today reported financial results for the fourth quarter and full year 2024 and provided business updates.

“Our objective and strategy to become a leader in

in vivo

gene editing accelerated in the fourth quarter after we achieved

in vivo

preclinical proof of concept ahead of schedule and shared positive preclinical

in vivo

data demonstrating the potential of our platform technology to achieve gene upregulation, or amplifying the expression of an existing protein to achieve clinically relevant levels that could potentially drive cures across tissues with a single dose,” said Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine. “We believe the ability to provide

in vivo

gene editing via gene upregulation holds the potential to significantly expand the addressable therapeutic possibilities for CRISPR-based gene editing, and we are poised to make meaningful progress towards the clinic in 2025.”

“The data we have shared over the last several months demonstrate our ability to attain

in vivo

gene editing via gene upregulation to increase the level of a functioning protein to address diseases caused by loss of function or deleterious mutations. Notably, our progress highlights the potential of our gene upregulation strategy across multiple tissues with our ‘plug ‘n play’ program,” said Linda C. Burkly, Ph.D., Chief Scientific Officer, Editas Medicine. “I am proud of our team’s progress, underscoring the therapeutic promise of our scientific work as a significant step towards the clinic as we develop our pipeline of potentially transformative

in vivo

gene editing medicines.”

Recent Achievements and Outlook

In Vivo



Medicines

• 
  Demonstrated preclinical proof of concept in non-human primates and humanized mice, highlighting the potential of Editas’ gene upregulation strategy across tissues, presented in
  
  December 2024
  
  and
  
  January 2025
  
  .
  
  
  

Hematopoietic Stem Cells

• 
  Achieved effective delivery and meaningful levels of
  
  in vivo
  
  editing of the
  
  HBG 1/2
  
  promoter, our therapeutic target, in HSCs with Editas’ proprietary targeted lipid nanoparticles (tLNPs) after a single dose of tLNP in non-human primates.
  
  
  
  • 
    Ongoing evaluation of further optimized LNP formulations expected to achieve even higher therapeutic editing levels.
    
  
  
  
  


• 
  The Company remains on track to declare an
  
  in vivo
  
  HSC development candidate in mid-2025 and to present additional preclinical
  
  in vivo
  
  HSC data by year-end.
  
  
  

Liver Cells

• 
  Achieved proof of concept in non-human primates, validating high efficiency
  
  in vivo
  
  gene editing in the liver with first use of AsCas12a delivery by LNP.
  


• 
  Demonstrated proof of upregulation strategy in mice by increasing clinically relevant target protein resulting in significant disease biomarker reduction for an undisclosed liver target.
  


• 
  The Company remains on track to declare an
  
  in vivo
  
  liver development candidate in mid-2025 and to present additional preclinical
  
  in vivo
  
  liver data by year-end.
  
  
  

Other Cells/Tissues

• 
  Demonstrated
  
  in vivo
  
  proof of concept for “plug ‘n play” delivery to extrahepatic cell types using the Company’s proprietary LNP targeting platform at high efficiency in humanized mice.
  


• 
  The Company remains on track to establish and disclose one additional target cell type/tissue beyond HSCs and liver by year-end.
  

Ex Vivo



Hemoglobinopathies

• 
  Reni-cel (renizgamglogene autogedtemcel, previously EDIT-301) for severe sickle cell disease and transfusion-dependent beta thalassemia.
  
  
  
  • 
    In December 2024, the Company announced that it ended development of reni-cel after an extensive search failed to yield a commercial partner.
    
  
  
  • 
    As a result of the decision to end development of reni-cel, the Company initiated cost savings measures, including an approximately 65% reduction in headcount to align workforce and resources to an
    
    in vivo
    
    pipeline.
    
    
    
  
  
  
  

Fourth Quarter and Full Year 2024 Financial Results



Cash, cash equivalents, and marketable securities as of December 31, 2024, were $269.9 million compared to $265.1 million as of September 30, 2024. The Company expects the existing cash, cash equivalents, and marketable securities, together with the retained portions of the payments payable under its license agreement with Vertex Pharmaceuticals, to fund operating expenses and capital expenditures into the second quarter of 2027. The Company’s cash runway includes total estimated expenses of approximately $45.0 million to $55.0 million related to ending development of reni-cel and related employee exit costs.

Fourth Quarter 2024

• 
  For the three months ended December 31, 2024, net loss attributable to common stockholders was $45.4 million, or $0.55 per share, compared to a net loss of $18.9 million, or $0.23 per share, for the same period in 2023.
  
  
  
  
  


• 
  Collaboration and other research and development revenues decreased to $30.6 million for the three months ended December 31, 2024, compared to $60.0 million for the same period in 2023. This decrease was primarily attributable to revenue recognized from the upfront payment under the Company’s license agreement with Vertex executed in December 2023.
  
  
  
  
  


• 
  Research and development expenses decreased by $21.0 million to $48.6 million for the three months ended December 31, 2024, compared to $69.6 million for the same period in 2023. The decrease was primarily attributable to sublicense payments made in connection with the Vertex license agreement in December 2023.
  
  
  
  
  


• 
  General and administrative expenses increased by $1.9 million to $16.4 million for the three months ended December 31, 2024, compared to $14.5 million for the same period in 2023. The increase was primarily driven by increased professional service expenses for strategic business initiatives.
  
  
  
  
  


• 
  Restructuring charges were $12.2 million for the three months ended December 31, 2024, compared to no restructuring charges for the same period in 2023. The restructuring charges were related to the discontinuation of the clinical development of the reni-cel program, initiated in December 2024, and the related workforce reduction.
  
  
  

Full Year 2024

• 
  For the full year 2024, net loss attributable to common stockholders was $237.1 million, or $2.88 per share, compared to net loss of $153.2 million, or $2.02 per share, for the same period in 2023.
  
  
  
  
  


• 
  Collaboration and other research and development revenues decreased to $32.3 million for 2024, compared to $78.1 million for the same period in 2023. The decrease was primarily attributable to revenue recognized from the upfront payment under the Company’s license agreement with Vertex executed in December 2023.
  
  
  
  
  


• 
  Research and development expenses increased by $21.5 million to $199.2 million for 2024, compared to $177.7 million for the same period in 2023. The increase was primarily related to clinical and manufacturing costs related to the progression of the Company’s former reni-cel program as well as costs attributable to
  
  in vivo
  
  research and discovery.
  
  
  
  
  


• 
  General and administrative expenses increased by $2.3 million to $72.0 million for 2024, compared to $69.7 million for the same period in 2023. The increase was primarily attributable to increased employee-related expenses related to increased headcount to support business operations.
  
  
  
  
  


• 
  Restructuring charges were $12.2 million for 2024, compared to no restructuring charges for the same period in 2023. The restructuring charges were related to the discontinuation of the clinical development of our reni-cel program, initiated in December 2024, and the related workforce reduction.
  
  
  

Upcoming Events



Editas Medicine plans to participate in the following investor events:

• 
  Leerink Partners Global Biopharma Conference
  
  
  March 10, 2024
  
  
  Miami Beach, FL
  
  
  
  
  


• 
  Barclays 27th Annual Global Healthcare Conference
  
  
  March 11, 2024
  
  
  Miami Beach, FL
  

No Conference Call



The Company is no longer hosting quarterly earnings conference calls.

About Editas Medicine



As a pioneering 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 transformative in vivo medicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize 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 initiation, timing, progress and results of the Company’s preclinical studies and its research and development programs, including the Company’s expectation to declare two

in vivo

development candidates in mid-2025 and establish an additional

in vivo

target cell type/tissue beyond HSCs and the liver by the end of 2025; the timing for the Company’s receipt and presentation of data from its preclinical studies, including presenting further

in vivo

HSC data and further

in vivo

data in one liver indication by the end of 2025; the potential of, and expectations for, the Company’s

in vivo

product candidates; the timing or likelihood of regulatory filings and approvals; the amount of anticipated costs related to ending development of reni-cel and related employee exit costs; and the Company’s expectations regarding cash runway into the second quarter of 2027. 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; and the availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. 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 represent the Company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, the Company explicitly disclaims any obligation to update any forward-looking statements.

This press release contains hyperlinks to information that is not deemed to be incorporated by reference in this press release.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.