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Editas Medicine to Present Pre-clinical Data Demonstrating Progression of in vivo Medicines Pipeline at the American Society of Gene and Cell Therapy Annual Meeting

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Editas Medicine, a clinical-stage gene editing company, is set to present pre-clinical data at the ASGCT Annual Meeting. The company will showcase advancements in in vivo gene editing technologies, including the delivery of CRISPR/Cas12a for treating Myocilin-Associated Glaucoma. Editas aims to develop transformative in vivo gene editing medicines, with a focus on optimizing guide RNA modifications and identifying potent recombinases. The presentations will demonstrate the potential for high-potency gene editing in various cell types and the development of novel gene editing technologies for targeted gene insertion. Overall, Editas is making progress towards confirming in vivo proof of concept by the end of the year.
Editas Medicine, un'azienda biomedica in fase di sviluppo clinico specializzata nell'editing genetico, è pronta a presentare dati preclinici all'incontro annuale dell'ASGCT. L'azienda metterà in evidenza i progressi nelle tecnologie di editing genetico in vivo, inclusa la consegna di CRISPR/Cas12a per il trattamento del glaucoma associato a Myocilin. Editas mira a sviluppare farmaci innovativi per l'editing genetico in vivo, concentrandosi sulla ottimizzazione delle modifiche all'RNA guida e sull'individuazione di ricombinasi potenti. Le presentazioni dimostreranno il potenziale dell'editing genetico ad alta potenza in vari tipi cellulari e lo sviluppo di nuove tecnologie di editing genetico per l'inserimento mirato di geni. Complessivamente, Editas sta facendo progressi verso la conferma in vivo del concetto di prova entro la fine dell'anno.
Editas Medicine, una compañía de edición genética en etapa clínica, está preparada para presentar datos preclínicos en la Reunión Anual de ASGCT. La compañía destacará avances en tecnologías de edición génica in vivo, incluyendo la entrega de CRISPR/Cas12a para tratar el Glaucoma Asociado a Myocilina. Editas tiene como objetivo desarrollar medicinas de edición genética in vivo transformadoras, enfocándose en la optimización de modificaciones de ARN guía e identificando recombinasas potentes. Las presentaciones demostrarán el potencial para la edición génica de alta potencia en diversos tipos de células y el desarrollo de nuevas tecnologías de edición genética para la inserción dirigida de genes. En general, Editas está avanzando hacia la confirmación del concepto de prueba in vivo para finales de año.
임상 개발 단계에 있는 유전자 편집 회사인 에디타스 메디슨은 ASGCT 연례 미팅에서 전임상 데이터를 발표할 예정입니다. 이 회사는 생체 내 유전자 편집 기술의 발전을 소개하며, 특히 마이오실린 연관 녹내장 치료를 위한 CRISPR/Cas12a의 전달에 주목할 것입니다. 에디타스는 가이드 RNA 수정 최적화와 강력한 리콤비나제 식별에 중점을 둔 혁신적인 생체 내 유전자 편집 의약품 개발을 목표로 하고 있습니다. 발표는 다양한 세포 유형에서의 고강도 유전자 편집 가능성과 표적 유전자 삽입을 위한 새로운 유전자 편집 기술 개발을 보여줄 것입니다. 전반적으로 에디타스는 올해 말까지 생체 내 개념 증명을 확립하는 데 진전을 보이고 있습니다.
Editas Medicine, une entreprise de modification génétique en phase clinique, est prête à présenter des données précliniques lors de la réunion annuelle de l'ASGCT. L'entreprise mettra en avant les avancées dans les technologies d'édition génique in vivo, incluant l'apport de CRISPR/Cas12a pour le traitement du glaucome associé à Myocilin. Editas vise à développer des médicaments de modification génique in vivo transformateurs, en se concentrant sur l'optimisation des modifications de l'ARN guide et l'identification de recombinases puissantes. Les présentations montreront le potentiel pour une édition génique de haute puissance dans différents types de cellules et le développement de nouvelles technologies d'édition génique pour l'insertion ciblée de gènes. Globalement, Editas progresse vers la confirmation in vivo de la preuve de concept d'ici la fin de l'année.
Editas Medicine, ein Unternehmen für Genschnitt-Technologie in der klinischen Entwicklungsphase, steht kurz davor, präklinische Daten auf der jährlichen ASGCT-Konferenz zu präsentieren. Das Unternehmen wird Fortschritte bei den in vivo-Genschnitttechnologien vorstellen, einschließlich der Verwendung von CRISPR/Cas12a zur Behandlung von mit Myozilin assoziiertem Glaukom. Editas zielt darauf ab, transformative in vivo-Genschnittmedikamente zu entwickeln, wobei der Schwerpunkt auf der Optimierung von Guide-RNA-Modifikationen und der Identifizierung potenten Rekombinasen liegt. Die Präsentationen werden das Potenzial für eine hochwirksame Genschnitt in verschiedenen Zelltypen und die Entwicklung neuer Genschnitttechnologien für die gezielte Geninsertion demonstrieren. Insgesamt macht Editas Fortschritte auf dem Weg zur Bestätigung eines in vivo-Beweises des Konzepts bis Ende des Jahres.
Positive
  • Editas Medicine to present pre-clinical data at the ASGCT Annual Meeting
  • Focus on in vivo gene editing technologies and potential treatments for serious diseases
  • Advancements in guide RNA modifications and recombinase proteins for improved gene editing outcomes
  • Progress towards confirming in vivo proof of concept by the end of the year
Negative
  • None.

CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage gene editing company, today announced that three abstracts have been accepted for presentation, including one oral presentation, at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) being held May 7 – 11, 2024, in Baltimore, MD, and virtually. The Company is presenting pre-clinical data to support its development of transformative in vivo gene editing medicines.  

Editas Medicine presentations at ASGCT include:

  • Oral presentation of in vivo pre-clinical data from mouse studies using lipid nanoparticle (LNP)-mediated delivery of an optimized guide RNA (gRNA) and engineered AsCas12a messenger RNA (mRNA).
  • Pre-clinical data demonstrating AsCas12a gRNA modifications that enable high-potency gene editing in multiple cell types and improve gene editing outcomes in vivo, enabling the development of in vivo gene editing medicines.
  • Research on identifying potent large serine recombinases (LSRs) as a foundation to develop novel in vivo gene editing technologies for whole gene knock-in, expanding potential in vivo gene editing targets for developing medicines.

“Editas Medicine is making strong progress towards the clinic with our technology optimization to develop transformative in vivo medicines for people living with serious diseases,” said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. “We look forward to sharing compelling data at ASGCT next month, including data demonstrating the delivery of AsCas12a mRNA using LNPs in vivo and guide RNA modifications to increase potency. These in vivo data are an important step towards confirming in vivo proof of concept by the end of the year.”

The complete list of Editas Medicine presentations is below. Abstracts can be accessed on the ASGCT website, and the presentations will be posted on the Editas Medicine website during the conference.

Oral Presentation:
Title: LNP-Based Delivery of CRISPR/Cas12a for the Potential Treatment of Myocilin-Associated Glaucoma
Session Date and Time: Friday, May 10, 2024, 3:45 – 5:30 p.m. ET
Presentation Time: 4:00 - 4:15 p.m.
Session title: Advancements in Technologies for In Vivo Gene Therapies 
Room: Room 324-326 
Final Abstract Number: 276

Poster Presentations:
Title: Chemically Modified AsCas12a Guide RNAs Improve Lipid Nanoparticle-Mediated In Vivo Gene Editing in Different Tissues
Session Date and Time: Thursday, May 9, 2024, 12:00 p.m. ET
Session Title: Thursday Posters: Gene Disruption and Excision
Presentation Room: Exhibit Hall
Final Abstract Number: 1182

Title: Metagenomic Discovery and Screening of Novel Recombinase Proteins for Targeted Integration
Session Date and Time: Friday, May 10, 2024, 12:00 p.m. ET
Session Title: Friday Posters: Targeted Gene Insertion 
Presentation Room: Exhibit Hall
Final Abstract Number: 1681

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 initiation, timing, progress and results of the Company’s preclinical studies and its research and development programs, and the timing for the Company’s receipt and presentation of data from its preclinical studies, including confirming in vivo proof-of-concept by the end of 2024. 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 pre-clinical studies and availability and timing of results from pre-clinical studies. 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.


FAQ

What is Editas Medicine presenting at the ASGCT Annual Meeting?

Editas Medicine is presenting pre-clinical data, including advancements in in vivo gene editing technologies and potential treatments for serious diseases.

When is the ASGCT Annual Meeting taking place?

The ASGCT Annual Meeting is being held from May 7 – 11, 2024, in Baltimore, MD, and virtually.

What is the focus of Editas Medicine's presentations at ASGCT?

The focus is on optimizing guide RNA modifications and identifying potent recombinase proteins for improved gene editing outcomes.

What is the goal of Editas Medicine in developing in vivo gene editing medicines?

The goal is to develop transformative in vivo gene editing medicines for people living with serious diseases.

Who will be presenting at the ASGCT Annual Meeting on behalf of Editas Medicine?

Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer of Editas Medicine, will be presenting the data.

What is the significance of the presentations at ASGCT for Editas Medicine?

The presentations demonstrate progress towards confirming in vivo proof of concept by the end of the year.

Editas Medicine, Inc.

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