Editas Medicine 株価情報 (NASDAQ: EDIT)

最新ニュース Editas Medicine (NASDAQ:EDIT) 株価情報

Editas Medicine

Editas Medicine Inc is a clinical-stage genome editing company dedicated to developing potentially transformative genomic medicines to treat a broad range of serious diseases. The company focuses on developing a proprietary genome editing platform based on clustered, regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated protein 9 (Cas9 technology). It has developed CRISPR/Cas9 technology which uses a protein-RNA (ribonucleic acid) complex composed of the Cas9 enzyme bound to a guide RNA molecule designed to recognize a DNA (deoxyribonucleic acid) sequence that requires repair. The company is engaged in developing and commercializing genome editing technology.

Editas Medicine Inc

<ul>
<li>Achieved in vivo preclinical proof of concept of editing hematopoietic stem cells in non-human primates as a key step toward developing a novel in vivo treatment for sickle cell disease and beta thalassemia</li>
<li>Achieved in vivo editing of liver cells in non-human primates and in vivo delivery to two additional cell types in humanized mice</li>
<li>Anticipated 2025 milestones include: declare two in vivo development candidates, one in HSCs and one in liver; present further in vivo HSC data; present in vivo data in one liver indication; establish one additional target cell type/tissue; and continue to derive revenue through sublicensing foundational IP</li>
<li>Strategic priorities through 2027 include: submit at least one IND/CTA; achieve human in vivo proof of concept in HSC editing for the treatment of sickle cell disease and beta thalassemia; and commence late-stage trial of at least one asset</li>
<li>Strong financial position with operational runway into Q2 2027</li>
<li>Company to present at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15 at 11:15 a.m. PST &nbsp;</li>
</ul>
CAMBRIDGE, Mass., Jan. 13, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>), a pioneering gene editing company focused on developing transformative medicines for serious diseases, today announced its three-year strategic priorities, anticipated 2025 key milestones, and new in vivo preclinical proof of concept data in non-human primates editing hematopoietic stem cells (HSCs) and liver cells and in vivo delivery data in humanized mice to two additional target cell types.

Biotech

News

General

EDIT

Editas Medicine Announces In Vivo Preclinical Proof Of Concept In HSC And Liver Cells, 2025 Milestones, And Strategic Priorities Through 2027 With Operational Runway Into Q2 2027

Editas Medicine Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>) announced last week a strategic realignment to focus on in <a href="https://www.benzinga.com/general/biotech/24/12/42480865/editas-medicine-realigns-strategy-to-focus-on-in-vivo-gene-editing-targets-human-proof-of-concept-by-2026" target="_blank" rel="noreferrer noopener">vivo gene editing</a>.
The shift is designed to optimize costs, extend its cash runway into the second quarter of 2027, and accelerate its progress toward achieving human proof of concept within two years.
Also Read: <a target="_blank" href="https://www.benzinga.com/24/11/42170987/editas-medicine-downgraded-analyst-sees-limited-near-term-catalysts" rel="noreferrer noopener">Editas Medicine Downgraded: Analyst Sees Limited Near-Term Catalysts</a>
JPMorgan spoke with Editas management. The analyst said that, overall, they were surprised by the timing of the restructuring. While there are personnel and legal considerations, the pivot would be better received if delivered in a single step, the analyst wrote.
JPMorgan highlighted the strategic move to prioritize in vivo programs, but noted that clarity is needed on the next steps and the timeline for initial human proof-of-concept results.
Until more visibility is gained, investors may remain cautious, keeping Editas Medicine shares trading within a limited range compared to peers in the coverage.
The analyst saw significant value embedded in its technical expertise and IP estate. However, the next major inflection point in the stock is still a way away, and investors will want to see the first clinical proof of concept before returning to the story.
As such, Editas Medicine shares will underperform relative to other names in JPMorgan&#8217;s coverage universe for now. The analyst downgraded the rating to Underweight from Neutral.
The recent strategic updates, including workforce cuts and management/board departures, may raise concerns about Editas Medicine&#8217;s shift toward in vivo development. JP Morgan added that these changes could suggest further adjustments to its development strategy in the future.
EDIT Price Action: Editas Medicine stock is up 2.36% at $1.52 at publication Tuesday.
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Photo: Gorodenkoff/Shutterstock.com

Analyst Color

Downgrades

Health Care

Analyst Ratings

JPMorgan Downgrades Editas Medicine, Says Focus Shift Sparks Investor Caution

Evercore ISI Group analyst Liisa Bayko maintains Editas Medicine (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>) with a Outperform and lowers the price target from $7 to $5.

Price Target

Evercore ISI Group Maintains Outperform on Editas Medicine, Lowers Price Target to $5

JP Morgan analyst Brian Cheng downgrades Editas Medicine (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>) from Neutral to Underweight.

JP Morgan Downgrades Editas Medicine to Underweight

RBC Capital analyst Luca Issi maintains Editas Medicine (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>) with a Sector Perform and lowers the price target from $5 to $4.

RBC Capital Maintains Sector Perform on Editas Medicine, Lowers Price Target to $4

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<li>JP Morgan cut D.R. Horton, Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/DHI#NYSE">DHI</a>) price target from $188 to $156. JP Morgan analyst Michael Rehaut downgraded the stock from Neutral to Underweight. D.R. Horton shares closed at $151.21 on Thursday.  <a href="https://www.benzinga.com/quote/DHI/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>Stifel cut Editas Medicine, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>) price target from $11 to $3. Stifel analyst Dae Gon Ha downgraded the stock from Buy to Hold. Editas Medicine shares closed at $1.89 on Thursday. See how other analysts view this stock.</li>
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Top Wall Street analysts changed their outlook on these top names. For a complete view of all analyst rating changes, including upgrades and downgrades, please see our <a href="https://www.benzinga.com/analyst-stock-ratings">analyst ratings page</a>.
<ul>
<li>Stifel analyst Dae Gon Ha downgraded the rating for Editas Medicine, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>) from Buy to Hold and lowered the price target from $11 to $3. Editas Medicine shares closed at $1.89 on Thursday. See how other analysts view this stock.</li>
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<li>JP Morgan analyst Michael Rehaut downgraded Toll Brothers, Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/TOL#NYSE">TOL</a>) from Overweight to Neutral and lowered the price target from $166 to $150. Toll Brothers shares closed at $138.46 on Thursday. <a href="https://www.benzinga.com/quote/TOL/analyst-ratings">See how other analysts view this stock.</a></li>
<li>Baird analyst Michael Halloran downgraded the rating for Nordson Corporation (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/NDSN#NASDAQ">NDSN</a>) from Outperform to Neutral and slashed the price target from $294 to $237. Nordson shares closed at $229.11 on Thursday. <a href="https://www.benzinga.com/quote/NDSN/analyst-ratings">See how other analysts view this stock</a>.</li>
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NDSN

This Toll Brothers Analyst Is No Longer Bullish; Here Are Top 5 Downgrades For Friday

WIIM

Editas Medicine shares are trading lower after Stifel and Chardan Capital downgraded the stock and multiple firms cut their respective price targets.

Barclays analyst Gena Wang maintains Editas Medicine (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>) with a Equal-Weight and lowers the price target from $5 to $3.

Barclays Maintains Equal-Weight on Editas Medicine, Lowers Price Target to $3

Chardan Capital analyst Geulah Livshits downgrades Editas Medicine (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>) from Buy to Neutral.

Chardan Capital Downgrades Editas Medicine to Neutral

Baird analyst Joel Beatty maintains Editas Medicine (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>) with a Outperform and lowers the price target from $10 to $8.

Baird Maintains Outperform on Editas Medicine, Lowers Price Target to $8

On Thursday, Editas Medicine Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>) announced a strategic realignment to focus on in <a target="_blank" href="https://www.benzinga.com/pressreleases/24/12/g42471563/editas-medicine-announces-strategic-transition-to-in-vivo-gene-editing-company-with-intent-to-achi" rel="noreferrer noopener">vivo gene editing</a>.
In vivo refers to studies or experiments conducted on living organisms or cells instead of dead organisms or tissue extracts.&nbsp;The opposite of in vivo is in vitro, which means outside the body or in a laboratory.
The shift is designed to optimize costs, extend its cash runway into the second quarter of 2027, and accelerate its progress toward achieving human proof of concept within two years.
The transition follows recent in vivo preclinical research advancements, including proof of concept in multiple tissue types.
For hematopoietic stem cells (HSCs), Editas demonstrated ~40% editing of the HBG1/2 promoter using a proprietary targeted lipid nanoparticle (tLNP).
Also Read: <a target="_blank" href="https://www.benzinga.com/24/11/42170987/editas-medicine-downgraded-analyst-sees-limited-near-term-catalysts" rel="noreferrer noopener">Editas Medicine Downgraded: Analyst Sees Limited Near-Term Catalysts</a>
The single-dose delivery of Cas12a editing machinery directly to human HSCs in mice yielded significant functional outcomes. Within one month, human red blood cells expressed fetal hemoglobin (HbF) at an average of 20%.
The achievement builds on validated HBG1/2 biology from the RUBY trial with Editas&#8217; <a target="_blank" href="https://www.benzinga.com/pressreleases/24/12/g42394977/editas-medicine-reports-updated-clinical-data-from-the-ruby-trial-of-reni-cel-in-patients-with-sev" rel="noreferrer noopener">reni-cel program</a>.
In liver applications, Editas achieved high-efficiency gene editing in non-human primates in <a target="_blank" href="https://www.benzinga.com/pressreleases/24/10/g41439513/editas-medicine-and-genevant-sciences-to-collaborate-to-develop-novel-mrna-lnp-gene-editing-therap" rel="noreferrer noopener">collaboration with Genevant</a>.
The company will share detailed preclinical data and development timelines in early 2025.
As part of its transition, Editas announced a workforce reduction, eliminating approximately 65% of its employees over the next six months.
This includes several leadership team members, such as Chief Medical Officer Baisong Mei. The company stated that these changes align with its renewed focus on in vivo therapies.
By restructuring operations and prioritizing its in vivo platform, Editas aims to create a more streamlined and efficient organization.
Cash, cash equivalents, and marketable securities as of September&nbsp;30, 2024, <a target="_blank" href="https://www.benzinga.com/pressreleases/24/11/g41710488/editas-medicine-announces-third-quarter-2024-results-and-business-updates" rel="noreferrer noopener">were $265.1&nbsp;million</a>.
The company expects the existing cash, cash equivalents, and marketable securities, the upfront cash payment from DRI, and the retained portions of the payments payable under the license agreement with Vertex to fund operating expenses and capital expenditures into the&nbsp;<a target="_blank" href="https://www.benzinga.com/pressreleases/24/10/g41176304/editas-medicine-announces-50-million-monetization-financing-with-dri-healthcare-trust" rel="noreferrer noopener">second quarter&nbsp;of&nbsp;2026</a>.
Price Action: At last check on Friday, EDIT stock was down 8.99% at $1.72 during the premarket session.
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Movers

Editas Medicine Realigns Strategy to Focus on In Vivo Gene Editing, Targets Human Proof Of Concept By 2026

Stifel analyst Dae Gon Ha downgrades Editas Medicine (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>) from Buy to Hold and lowers the price target from $11 to $3.

Stifel Downgrades Editas Medicine to Hold, Lowers Price Target to $3

Editas Medicine shares are trading higher after the company announced plans to transition to an in vivo focus and initiated a reduction in headcount, aiming to eliminate approximately 65% of its workforce over the next six months.

Editas Medicine Initiates Cost Savings Measures And Reduction In Headcount To Align Workforce And Resources To In Vivo Pipeline, Extending Cash Runway Into Q2 2027

Editas Medicine Says Ending Development Of Reni-Cel After Extensive Search Did Not Yield A Commercial Partner

Editas Medicine  Plans Transition To An In Vivo Company;  Initiates Reduction In Headcount That Will Eliminate Approximately 65% Of Its Workforce Over Next Six Months

During the last three months, 12 analysts shared their evaluations of Editas Medicine (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>), revealing diverse outlooks from bullish to bearish.
The table below provides a concise overview of recent ratings by analysts, offering insights into the changing sentiments over the past 30 days and drawing comparisons with the preceding months for a holistic perspective.
<table>
<thead>
<tr>
<th></th>
<th align="right">Bullish</th>
<th align="right">Somewhat Bullish</th>
<th align="right">Indifferent</th>
<th align="right">Somewhat Bearish</th>
<th align="right">Bearish</th>
</tr>
</thead>
<tbody>
<tr>
<td>Total Ratings</td>
<td align="right">5</td>
<td align="right">2</td>
<td align="right">5</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>Last 30D</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>1M Ago</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>2M Ago</td>
<td align="right">4</td>
<td align="right">2</td>
<td align="right">3</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>3M Ago</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
</tbody>
</table>
The 12-month price targets, analyzed by analysts, offer insights with an average target of $8.08, a high estimate of $13.00, and a low estimate of $3.00. A negative shift in sentiment is evident as analysts have decreased the average price target by 33.66%. 
<img src="https://www.benzinga.com/files/images/story/2024/1733936445_0.png" alt="price target chart" />
<h2>Investigating Analyst Ratings: An Elaborate Study</h2>
The analysis of recent analyst actions sheds light on the perception of Editas Medicine by financial experts. The following summary presents key analysts, their recent evaluations, and adjustments to ratings and price targets.
<table>
<thead>
<tr>
<th>Analyst</th>
<th>Analyst Firm</th>
<th>Action Taken</th>
<th>Rating</th>
<th>Current Price Target</th>
<th>Prior Price Target</th>
</tr>
</thead>
<tbody>
<tr>
<td>Yanan Zhu</td>
<td>Wells Fargo</td>
<td>Lowers</td>
<td>Equal-Weight</td>
<td>$4.00</td>
<td>$7.00</td>
</tr>
<tr>
<td>Geulah Livshits</td>
<td>Chardan Capital</td>
<td>Maintains</td>
<td>Buy</td>
<td>$12.00</td>
<td>$12.00</td>
</tr>
<tr>
<td>Luca Issi</td>
<td>RBC Capital</td>
<td>Lowers</td>
<td>Sector Perform</td>
<td>$5.00</td>
<td>$8.00</td>
</tr>
<tr>
<td>Dae Gon Ha</td>
<td>Stifel</td>
<td>Lowers</td>
<td>Buy</td>
<td>$11.00</td>
<td>$17.00</td>
</tr>
<tr>
<td>Joon Lee</td>
<td>Truist Securities</td>
<td>Lowers</td>
<td>Buy</td>
<td>$8.00</td>
<td>$12.00</td>
</tr>
<tr>
<td>Yanan Zhu</td>
<td>Wells Fargo</td>
<td>Lowers</td>
<td>Overweight</td>
<td>$7.00</td>
<td>$9.00</td>
</tr>
<tr>
<td>Gena Wang</td>
<td>Barclays</td>
<td>Lowers</td>
<td>Equal-Weight</td>
<td>$5.00</td>
<td>$7.00</td>
</tr>
<tr>
<td>Liisa Bayko</td>
<td>Evercore ISI Group</td>
<td>Lowers</td>
<td>In-Line</td>
<td>$3.00</td>
<td>$7.00</td>
</tr>
<tr>
<td>Yanan Zhu</td>
<td>Wells Fargo</td>
<td>Lowers</td>
<td>Overweight</td>
<td>$9.00</td>
<td>$27.00</td>
</tr>
<tr>
<td>Geulah Livshits</td>
<td>Chardan Capital</td>
<td>Lowers</td>
<td>Buy</td>
<td>$12.00</td>
<td>$20.00</td>
</tr>
<tr>
<td>Luca Issi</td>
<td>RBC Capital</td>
<td>Maintains</td>
<td>Sector Perform</td>
<td>$8.00</td>
<td>$8.00</td>
</tr>
<tr>
<td>Soumit Roy</td>
<td>Jones Trading</td>
<td>Announces</td>
<td>Buy</td>
<td>$13.00</td>
<td>-</td>
</tr>
</tbody>
</table>
<h3>Key Insights:</h3>
<ul>
<li>Action Taken: Analysts frequently update their recommendations based on evolving market conditions and company performance. Whether they 'Maintain', 'Raise' or 'Lower' their stance, it reflects their reaction to recent developments related to Editas Medicine. This information provides a snapshot of how analysts perceive the current state of the company.</li>
<li>Rating: Analysts unravel qualitative evaluations for stocks, ranging from 'Outperform' to 'Underperform'. These ratings offer insights into expectations for the relative performance of Editas Medicine compared to the broader market.</li>
<li>Price Targets: Analysts set price targets as an estimate of a stock's future value. Comparing the current and prior price targets provides insight into how analysts' expectations have changed over time. This information can be valuable for investors seeking to understand consensus views on the stock's potential future performance.</li>
</ul>
Considering these analyst evaluations in conjunction with other financial indicators can offer a comprehensive understanding of Editas Medicine's market position. Stay informed and make well-informed decisions with our Ratings Table.
<a href="https://www.benzinga.com/quote/EDIT/analyst-ratings?utm_source=Benzinga Insights">Stay up to date on Editas Medicine analyst ratings.</a>
If you are interested in following small-cap stock news and performance <a href="https://www.benzinga.com/news/small-cap">you can start by tracking it here</a>.
<h2>All You Need to Know About Editas Medicine</h2>
Editas Medicine Inc is a clinical-stage genome editing company dedicated to developing potentially transformative genomic medicines to treat a broad range of serious diseases. The company focuses on developing a proprietary genome editing platform based on clustered, regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated protein 9 (Cas9 technology). It has developed CRISPR/Cas9 technology which uses a protein-RNA (ribonucleic acid) complex composed of the Cas9 enzyme bound to a guide RNA molecule designed to recognize a DNA (deoxyribonucleic acid) sequence that requires repair. The company is engaged in developing and commercializing genome editing technology.
<h3>Editas Medicine: Financial Performance Dissected</h3>
Market Capitalization: Indicating a reduced size compared to industry averages, the company's market capitalization poses unique challenges.
Decline in Revenue: Over the 3 months period, Editas Medicine faced challenges, resulting in a decline of approximately -98.86% in revenue growth as of 30 September, 2024. This signifies a reduction in the company's top-line earnings. When compared to others in the Health Care sector, the company faces challenges, achieving a growth rate lower than the average among peers.
Net Margin: Editas Medicine's net margin is below industry standards, pointing towards difficulties in achieving strong profitability. With a net margin of -101870.49%, the company may encounter challenges in effective cost control.
Return on Equity (ROE): Editas Medicine's ROE lags behind industry averages, suggesting challenges in maximizing returns on equity capital. With an ROE of -30.49%, the company may face hurdles in achieving optimal financial performance.
Return on Assets (ROA): The company's ROA is below industry benchmarks, signaling potential difficulties in efficiently utilizing assets. With an ROA of -17.45%, the company may need to address challenges in generating satisfactory returns from its assets.
Debt Management: Editas Medicine's debt-to-equity ratio is below industry norms, indicating a sound financial structure with a ratio of 0.22.
<h2>The Core of Analyst Ratings: What Every Investor Should Know</h2>
Analysts are specialists within banking and financial systems that typically report for specific stocks or within defined sectors. These people research company financial statements, sit in conference calls and meetings, and speak with relevant insiders to determine what are known as analyst ratings for stocks. Typically, analysts will rate each stock once a quarter. 
Beyond their standard evaluations, some analysts contribute predictions for metrics like growth estimates, earnings, and revenue, furnishing investors with additional guidance. Users of analyst ratings should be mindful that this specialized advice is shaped by human perspectives and may be subject to variability.
Which Stocks Are Analysts Recommending Now?
Benzinga Edge gives you instant access to all major analyst upgrades, downgrades, and price targets. Sort by accuracy, upside potential, and more. <a href="https://www.benzinga.com/premium/ideas/benzinga-edge-checkout/?t=be8be9arja3be22bz21&amp;utm_source=benzinga-insights">Click here to stay ahead of the market</a>.
This article was generated by Benzinga's automated content engine and reviewed by an editor.

12 Analysts Have This To Say About Editas Medicine

Wells Fargo analyst Yanan Zhu downgrades Editas Medicine (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>) from Overweight to Equal-Weight and lowers the price target from $7 to $4.

Wells Fargo Downgrades Editas Medicine to Equal-Weight, Lowers Price Target to $4

<h2>Editas Medicine Reports Updated Clinical Data from the RUBY Trial of Reni-cel in Patients with Severe Sickle Cell Disease at the American Society of Hematology (ASH) Annual Meeting</h2>
Globe Newswire
Poster presentation at ASH on Monday, December 9 at 6:00 p.m. PT / 9:00 p.m. ET
CAMBRIDGE, Mass., Dec. 09, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EDIT#NASDAQ">EDIT</a>), a leading gene editing company, will present updated safety and efficacy data in 28 patients living with severe sickle cell disease (SCD) treated with renizgamglogene autogedtemcel (reni-cel; formerly known as EDIT-301) in the Phase 1/2/3 RUBY clinical trial. The data will be presented by Dr. Rabi Hanna, M.D., Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic Children's, during a poster presentation at the American Society of Hematology (ASH) Annual Meeting in San Diego, CA, today at 6:00 p.m. PT (9:00 p.m. ET).
In the RUBY trial as of the data cutoff date (October 29, 2024), reni-cel was well-tolerated and continued to demonstrate a safety profile consistent with myeloablative busulfan conditioning and autologous hematopoietic stem cell transplant by all patients (N=28). Patients were at a median of 9.5 months post-reni-cel infusion, with 11 patients having &gt;1 year follow-up. Since reni-cel treatment, 27 of the 28 patients were free of vaso-occlusive events (VOEs). Patients were observed to have early normalization of total hemoglobin, with a mean total hemoglobin increasing from 9.8 g/dL at baseline to 13.8 g/dL at Month 6 (n=18). Patients were also observed to have rapid and sustained improvements in fetal hemoglobin (HbF) ≥40% and mean corpuscular concentration (MCH-F) of HbF per F-cells, well above the anti-sickling threshold. In addition, sustained clinically meaningful improvements were observed in patient-reported outcome domains for pain, physical function, and social roles and activities.
Efficacy data in Patients with Severe Sickle Cell Disease Patients were a median (range) of 9.5 (0.7–25.2) months post-reni-cel infusion, with 11 patients having &gt;1 year follow-up. Of 28 patients, 27 were VOE-free post-reni-cel infusion. Reni-cel administration led to early, robust increases and sustained levels of total Hb and HbF. At month 6, the mean total Hb was 13.8 g/dL with a mean HbF percentage of 48.1% (n=18).
The mean percentage of F-cells increased early and was sustained at &gt;90% from month 4 through last follow-up (n=20). MCH-F of HbF-containing red&nbsp;cells (F-cells) increased early, with mean value of 16.3 pg/F-cells at month 4 visit and sustained above the anti-sickling threshold of 10 pg/F-cell through last follow-up. Markers of hemolysis, including absolute reticulocyte count, indirect bilirubin, lactate dehydrogenase, and haptoglobin, improved or normalized by Month 6 and were generally maintained or improved as of last follow-up.
Sustained clinically meaningful improvements were observed in pain, physical, and social patient-reported outcome domains following treatment with reni-cel​.
Safety data in Patients with Severe Sickle Cell Disease Reni-cel was well-tolerated and demonstrated a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant by all evaluated RUBY trial patients (N=28). After reni-cel infusion, all evaluable patients (n=27) achieved successful engraftment; with median time to neutrophil engraftment of 23 days and median time to platelet engraftment of 25 days, which is important for limiting infection and bleeding risk. Two serious adverse events (SAEs) assessed by the investigators as possibly related to reni-cel treatment have been reported in the RUBY trial.
RUBY Poster Presentation Details: Title: Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Sustained Hemoglobin Normalization and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease Treated in the RUBY Trial Presenting Author: Rabi Hanna, M.D., Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic Children's, Cleveland, OH, United States Date/Time: Monday, December 9, 2024; 6:00 p.m. – 8:00 p.m. PT / 9:00 p.m. – 11:00 p.m. ET Location: San Diego Convention Center, Halls G-H Session: 801. Gene Therapies: Poster III

Editas Medicine Presents Data From RUBY Trial At ASH 2024, Showing Reni-Cel Achieves 96% VOE-Free Rate, Sustained Increases In Hemoglobin And Fetal Hemoglobin, And Clinically Meaningful Pain And Function Improvements In Severe Sickle Cell Patients; Safety Profile Consistent With Myeloablative Conditioning

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