Editas Medicine Announces Progress Towards 2024 Goals, Including Achievement of In Vivo Preclinical Proof of Concept and Strategic Update
Editas Medicine (Nasdaq: EDIT) announced significant progress towards its 2024 goals, including achieving in vivo preclinical proof of concept for hematopoietic stem and progenitor cell (HSPC) editing. The company utilized its proprietary targeted lipid nanoparticle (tLNP) to achieve a 29% editing level in HSPCs after a single dose, resulting in 20% HbF-expressing human red blood cells. This advancement is important for developing an in vivo treatment for sickle cell disease and beta thalassemia.
Editas also initiated a process to partner or out-license reni-cel, focusing resources on in vivo pipeline development. The company completed enrollment for the RUBY trial for severe sickle cell disease and the EdiTHAL trial for transfusion-dependent beta thalassemia. Additionally, Editas secured $57 million in non-dilutive capital through a sale agreement with DRI Healthcare Trust and ended Q3 2024 with approximately $265 million in cash and equivalents.
Editas Medicine (Nasdaq: EDIT) ha annunciato progressi significativi verso i suoi obiettivi del 2024, inclusa l'ottenimento di una prova di concetto preclinica in vivo per l'editing delle cellule staminali ematopoietiche (HSPC). L'azienda ha utilizzato il suo nanoparticella lipidica mirata proprietaria (tLNP) per raggiungere un livello di editing del 29% nelle HSPC dopo una singola dose, con il risultato di 20% di globuli rossi umani esprimenti HbF. Questo progresso è importante per sviluppare un trattamento in vivo per la malattia delle cellule falciformi e la beta talassemia.
Editas ha anche avviato un processo per partnerizzare o concedere in licenza reni-cel, concentrando le risorse sullo sviluppo della pipeline in vivo. L'azienda ha completato l'arruolamento per il trial RUBY per la malattia grave delle cellule falciformi e il trial EdiTHAL per la beta talassemia dipendente da trasfusioni. Inoltre, Editas ha assicurato 57 milioni di dollari in capitale non diluitivo attraverso un accordo di vendita con DRI Healthcare Trust e ha concluso il terzo trimestre del 2024 con circa 265 milioni di dollari in contante e liquidità equivalenti.
Editas Medicine (Nasdaq: EDIT) anunció avances significativos hacia sus objetivos de 2024, incluyendo la obtención de una prueba de concepto preclínica in vivo para la edición de células madre hematopoyéticas (HSPC). La empresa utilizó su nanopartícula lipídica dirigida propietaria (tLNP) para alcanzar un nivel de edición del 29% en HSPC después de una sola dosis, resultando en 20% de glóbulos rojos humanos expresando HbF. Este avance es importante para desarrollar un tratamiento in vivo para la enfermedad de células falciformes y la beta talasemia.
Editas también inició un proceso para asociarse o licenciar reni-cel, enfocando recursos en el desarrollo de la cartera in vivo. La empresa completó la inscripción para el ensayo RUBY para la enfermedad grave de células falciformes y el ensayo EdiTHAL para la beta talasemia dependiente de transfusiones. Además, Editas aseguró 57 millones de dólares en capital no dilutivo a través de un acuerdo de venta con DRI Healthcare Trust y cerró el tercer trimestre de 2024 con aproximadamente 265 millones de dólares en efectivo y equivalentes.
Editas Medicine (Nasdaq: EDIT)는 2024년 목표를 향한 중요한 진전을 발표했습니다. 여기에는 조혈모세포(HSPC) 편집을 위한 생체 내 전임상 개념 증명을 달성하는 것이 포함됩니다. 이 회사는 고유한 표적 지방 나노 입자(tLNP)를 사용하여 단일 투여 후 HSPC에서 29% 편집 수준을 달성했으며, 그 결과 20% HbF를 발현하는 인간 적혈구를 확보했습니다. 이 발전은 겸상 적혈구 질환과 베타 지중해빈혈에 대한 생체 내 치료법 개발에 중요합니다.
Editas는 또한 reni-cel의 파트너십 또는 라이센스 아웃을 위한 절차를 시작했습니다, 자원을 생체 내 파이프라인 개발에 집중하고 있습니다. 이 회사는 겸상 적혈구 질환의 RUBY 임상 시험과 수혈 의존성 베타 지중해빈혈의 EdiTHAL 임상 시험에 대한 등록을 완료했습니다. 게다가, Editas는 DRI Healthcare Trust와의 판매 계약을 통해 5천700만 달러의 비희석 자본을 확보했으며, 2024년 3분기를 약 2억6500만 달러의 현금 및 동등액으로 종료했습니다.
Editas Medicine (Nasdaq: EDIT) a annoncé des progrès significatifs vers ses objectifs 2024, y compris l'obtention d'une preuve de concept préclinique in vivo pour l'édition des cellules souches hématopoïétiques (HSPC). L'entreprise a utilisé sa nanoparticule lipidique ciblée propriétaire (tLNP) pour atteindre un niveau d'édition de 29 % dans les HSPC après une seule dose, ce qui a abouti à 20 % de globules rouges humains exprimant HbF. Ce progrès est important pour développer un traitement in vivo pour la drépanocytose et la bêta-thalassémie.
Editas a également lancé un processus pour partenariater ou accorder une licence à reni-cel, en concentrant les ressources sur le développement de la pipeline in vivo. L'entreprise a achevé l'inscription pour l'essai RUBY pour la drépanocytose sévère et l'essai EdiTHAL pour la bêta-thalassémie dépendante des transfusions. De plus, Editas a sécurisé 57 millions de dollars en capital non dilutif grâce à un accord de vente avec DRI Healthcare Trust et a terminé le troisième trimestre 2024 avec environ 265 millions de dollars en liquidités et équivalents.
Editas Medicine (Nasdaq: EDIT) hat bedeutende Fortschritte in Richtung seiner Ziele für 2024 bekannt gegeben, darunter den Erhalt eines in vivo präklinischen Nachweises des Konzepts für die Bearbeitung hämatopoetischer Stamm- und Vorläuferzellen (HSPC). Das Unternehmen nutzte sein proprietäres zielgerichtetes liposomales Nanopartikel (tLNP), um ein Editierungsniveau von 29 % in HSPCs nach einer Einzelgabe zu erreichen, was zu 20 % HbF-expressierenden menschlichen roten Blutkörperchen führte. Dieser Fortschritt ist wichtig für die Entwicklung einer in vivo Behandlung von Sichelzellanämie und beta-Thalassämie.
Editas hat auch einen Prozess eingeleitet, um reni-cel zu partnern oder auszulizenzieren und Ressourcen auf die Entwicklung der in vivo-Pipeline zu konzentrieren. Das Unternehmen hat die Rekrutierung für die RUBY-Studie bei schwerer Sichelzellanämie und die EdiTHAL-Studie bei transfusionsabhängiger beta-Thalassämie abgeschlossen. Darüber hinaus sicherte sich Editas 57 Millionen Dollar an nicht verwässerndem Kapital durch einen Verkaufsvertrag mit DRI Healthcare Trust und schloss das dritte Quartal 2024 mit ungefähr 265 Millionen Dollar in bar und in liquiden Mitteln ab.
- Achieved 29% editing level in HSPCs after a single dose using proprietary tLNP technology
- Completed enrollment for RUBY and EdiTHAL trials
- Secured $57 million in non-dilutive capital through agreement with DRI Healthcare Trust
- Ended Q3 2024 with approximately $265 million in cash, cash equivalents, and marketable securities
- Initiated process to partner or out-license reni-cel, potentially reducing direct control over the asset
- Focusing resources on in vivo pipeline development may limit resources for other projects
Insights
Editas Medicine's announcement marks a significant milestone in their gene editing pipeline. The achievement of in vivo preclinical proof of concept for hematopoietic stem and progenitor cell (HSPC) editing is a major step towards developing a novel treatment for sickle cell disease and beta thalassemia. Key highlights include:
- 29% editing level in HSPCs after a single dose using their proprietary targeted lipid nanoparticle (tLNP) delivery system
- 20% HbF-expressing human red blood cells observed after one month, indicating functional outcome
- Competitive positioning relative to other in vivo approaches in development
The company's strategic shift to focus on in vivo pipeline development, including the decision to partner or out-license reni-cel, aligns with their long-term vision. This move could potentially accelerate the development of their in vivo programs while optimizing capital allocation. The
While promising, investors should note that these are still early-stage results and the path to clinical trials and potential commercialization remains long and uncertain. The success of the reni-cel partnering process will be important for the company's near-term financial outlook and pipeline advancement.
Editas Medicine's strategic update reveals a significant pivot in their business model and capital allocation strategy. Key financial implications include:
- Cash position of approximately
$265 million at Q3 end, boosted to$320 million with the DRI Healthcare Trust deal - Potential reduction in 2025 spending through reni-cel partnership/out-licensing
- Shift in R&D focus towards in vivo pipeline development
The decision to seek a partner for reni-cel could significantly impact the company's burn rate and extend their cash runway. This move, combined with the non-dilutive capital from the DRI deal, strengthens Editas' financial position as they advance their in vivo programs.
Investors should closely monitor the outcome of the reni-cel partnering process, as it will be important for future revenue potential and R&D cost structure. The market's reaction to this strategic shift will likely depend on the terms of any potential reni-cel deal and the progress of the in vivo pipeline. With a market cap of
Achieved in vivo preclinical proof of concept of hematopoietic stem and progenitor cell editing by utilizing Editas Medicine’s proprietary targeted LNP as a key step forward toward developing a novel in vivo treatment for sickle cell disease and beta thalassemia
Initiated process to partner or out-license reni-cel to focus resources on in vivo pipeline development
Company to present data and discuss strategic update in a Company-sponsored Webinar today at 8:00 a.m. ET
CAMBRIDGE, Mass., Oct. 22, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage gene editing company, today announced its achievement of in vivo preclinical proof of concept of hematopoietic stem and progenitor cell (HSPC) editing and fetal hemoglobin (HbF) induction in humanized mice engrafted with human hematopoietic stem cells and lacking their own hematopoietic cells. The Company observed high levels of editing of the HBG1/2 promoter, leveraging our clinically validated upregulation strategy, utilizing a novel and Editas-proprietary targeted lipid nanoparticle (tLNP) formulation for extrahepatic tissue delivery. The Company is also providing business development and financial updates, including that it initiated a process to partner or out-license reni-cel.
“Achieving in vivo preclinical proof of concept in a desired, extrahepatic target cell type, delivered utilizing a proprietary Editas LNP that works outside the liver, puts us on a clear path to develop a potentially first- and best-in-class in vivo gene edited medicine for the treatment of sickle cell disease and beta thalassemia,” said Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine. “I am delighted with our concrete progress on in vivo functional upregulation as we drive towards our long-term vision to be a leader in in vivo programmable gene editing, specifically achieving in vivo preclinical proof of concept ahead of schedule.”
Dr. O’Neill added, “Simultaneously, we are laser focused on the optimal use of capital as we continue development of reni-cel, invest in our in vivo pipeline and vision, and map to the future. As part of that focus, we frequently evaluate opportunities to most efficiently advance reni-cel, a potentially best-in-class cell therapy for the treatment of sickle cell disease and beta thalassemia, towards commercialization and ultimately deliver it to patients in need. We believe that the best option for both patients and our shareholders is for us to seek an alternative such as a global partner or out-licensing, which would allow for further development and ultimately commercialization of reni-cel with or by another party and would allow us to substantially reduce spend in 2025.”
In Vivo Proof of Concept Data Highlights
- Achieved highly competitive level of in vivo hematopoietic stem and progenitor cell (HSPC) editing utilizing a novel, Editas-proprietary targeted lipid nanoparticle (t-LNP) for extrahepatic tissue delivery in a humanized mouse model, mice engrafted with human hematopoietic stem cells.
- Enabled an editing level of
29% in HSPCs after a single dose by utilizing a hematopoietic stem cell targeting strategy and tLNP formulation optimization. - Further demonstrated that editing with the Company’s proprietary tLNP formulation resulted in the 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. - This level of in vivo editing in a humanized mouse model after a single dose constitutes a highly competitive dataset relative to data in the public domain for the development of an in vivo medicine for sickle cell disease and beta thalassemia.
Ex Vivo Hemoglobinopathies
- Reni-cel (renizgamglogene autogedtemcel, previously EDIT-301) for Severe Sickle Cell Disease (SCD)
- On-track to present a substantive clinical data set of sickle cell patients with considerable clinical follow-up in the RUBY trial at the American Society of Hematology (ASH) Annual Meeting and Exposition, December 7-10, 2024.
- As previously disclosed, the Company has completed enrollment of the adolescent and adult cohorts of the Phase 1/2/3 RUBY trial for SCD.
- Manufacturing drug product for the initial adolescent cohort patients.
- The Company continues to dose adult patients in the RUBY trial and has dosed 28 patients to date.
- Reni-cel for Transfusion-dependent Beta Thalassemia (TDT)
- On-track to present additional clinical data from the EdiTHAL trial by year-end 2024.
- The Company completed enrollment of the adult cohort of the EdiTHAL trial for TDT and continues to dose patients.
Other Corporate Highlights
- On October 3, 2024, Editas Medicine announced the sale of certain future license fees and other payments owed to the Company under its Cas9 license agreement with Vertex Pharmaceuticals to a wholly owned subsidiary of DRI Healthcare Trust (DRI) for an upfront cash payment of
$57 million . The upfront cash payment brings non-dilutive capital to Editas Medicine, helping enable further pipeline development and related strategic priorities. - The Company ended the third quarter 2024 with approximately
$265 million of cash, cash equivalents, and marketable securities, or approximately$320 million following receipt of the upfront cash payment from DRI. - The Company has engaged Moelis & Company LLC, a leading global independent investment bank, to lead the global process to partner or out-license reni-cel.
In light of this webinar, the Company does not plan to host a conference call when it announces third quarter 2024 financial results next month.
Webinar Presentation Details:
The live and archived webcast of the Company’s webinar presentation will be accessible through this webcast link, or through the Events & Presentations page of the “Investors” section of the Company’s website.
A replay of the webinar will be available upon conclusion of the webinar in the Investors section of the Editas Medicine website at https://www.editasmedicine.com/.
About renizgamglogene autogedtemcel (reni-cel)
Reni-cel, formerly known as EDIT-301, is an experimental gene editing medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Reni-cel consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by AsCas12a, a novel, proprietary, highly efficient, and specific gene editing nuclease. Red blood cells derived from reni-cel CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe SCD and TDT.
About Editas Medicine
As a clinical-stage 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 treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic 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 intent to partner or out-license reni-cel and any benefits resulting therefrom, the initiation, timing, progress and results of the Company’s preclinical and clinical studies and its research and development programs, the timing for the Company’s receipt and presentation of data from its clinical trials and preclinical studies, including presenting additional clinical data from the RUBY trial at the ASH Annual Meeting and Exposition and from the EdiTHAL trial by year-end 2024, the potential of, and expectations for, the Company’s product candidates, including any in vivo gene edited medicines the Company may develop, and the Company’s expectations regarding 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 and clinical trials, including the RUBY and EdiTHAL trials, and clinical development of the Company’s product candidates, including reni-cel; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from preclinical studies and clinical trials will be predictive of the final results of the study or trial or the results of any future clinical trials; expectations for regulatory approvals to conduct trials or to market products; and 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 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.
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