Editas Medicine to Present Preclinical Data Demonstrating Progress in the Development of an in vivo Gene Editing Pipeline at the American Society of Gene and Cell Therapy Annual Meeting
Editas Medicine (Nasdaq: EDIT) announced the acceptance of five abstracts for presentation at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), scheduled for May 13-17, 2025, in New Orleans.
The presentations include:
- An oral presentation on in vivo preclinical data using targeted lipid nanoparticles for HBG1/2 promoter editing
- Preclinical proof of concept for a liver target using CRISPR editing
- Results from mouse and non-human primate studies showing high levels of target gene editing in the liver
- Data on guide modification and targeting improvements for better gene editing outcomes
Linda C. Burkly, Executive Vice President and Chief Scientific Officer, highlighted the company's progress in developing in vivo medicines. The research demonstrates their ability to increase protein levels to address diseases caused by loss of function or mutations through gene upregulation editing strategy. Their targeted lipid nanoparticle delivery system shows promise for multiple tissue applications using a 'plug 'n play' approach.
Editas Medicine (Nasdaq: EDIT) ha annunciato l'accettazione di cinque abstract per la presentazione al 28° Congresso Annuale della American Society of Gene and Cell Therapy (ASGCT), previsto dal 13 al 17 maggio 2025 a New Orleans.
Le presentazioni includono:
- Una presentazione orale su dati preclinici in vivo riguardanti l'editing del promotore HBG1/2 utilizzando nanoparticelle lipidiche mirate
- La prova di concetto preclinica per un target epatico tramite editing CRISPR
- Risultati da studi su topi e primati non umani che mostrano alti livelli di editing genico nel fegato
- Dati su modifiche della guida e miglioramenti nel targeting per ottenere risultati migliori nell'editing genico
Linda C. Burkly, Vicepresidente Esecutiva e Direttrice Scientifica, ha sottolineato i progressi dell'azienda nello sviluppo di terapie in vivo. La ricerca dimostra la loro capacità di aumentare i livelli proteici per trattare malattie causate da perdita di funzione o mutazioni tramite una strategia di editing per la regolazione al rialzo dei geni. Il loro sistema di somministrazione tramite nanoparticelle lipidiche mirate promette applicazioni su diversi tessuti con un approccio 'plug 'n play'.
Editas Medicine (Nasdaq: EDIT) anunció la aceptación de cinco resúmenes para presentación en la 28ª Reunión Anual de la American Society of Gene and Cell Therapy (ASGCT), programada del 13 al 17 de mayo de 2025 en Nueva Orleans.
Las presentaciones incluyen:
- Una presentación oral sobre datos preclínicos in vivo utilizando nanopartículas lipídicas dirigidas para la edición del promotor HBG1/2
- Prueba de concepto preclínica para un objetivo hepático usando edición CRISPR
- Resultados de estudios en ratones y primates no humanos que muestran altos niveles de edición del gen objetivo en el hígado
- Datos sobre modificaciones de guías y mejoras en el direccionamiento para mejores resultados en la edición génica
Linda C. Burkly, Vicepresidenta Ejecutiva y Directora Científica, destacó el progreso de la compañía en el desarrollo de medicamentos in vivo. La investigación demuestra su capacidad para aumentar los niveles de proteínas y tratar enfermedades causadas por pérdida de función o mutaciones mediante una estrategia de edición para la regulación al alza del gen. Su sistema de entrega con nanopartículas lipídicas dirigidas muestra potencial para aplicaciones en múltiples tejidos con un enfoque 'plug 'n play'.
Editas Medicine (나스닥: EDIT)는 2025년 5월 13일부터 17일까지 뉴올리언스에서 개최되는 제28회 미국 유전자 및 세포 치료학회 연례회의(ASGCT)에 5개의 초록이 채택되었다고 발표했습니다.
발표 내용은 다음과 같습니다:
- HBG1/2 프로모터 편집을 위한 표적 지질 나노입자를 이용한 생체 내 전임상 데이터 구두 발표
- CRISPR 편집을 사용한 간 표적에 대한 전임상 개념 증명
- 생쥐 및 비인간 영장류 연구에서 간 내 목표 유전자 편집 수준이 높은 결과
- 더 나은 유전자 편집 결과를 위한 가이드 수정 및 표적화 개선 데이터
Linda C. Burkly 전무이사 겸 최고과학책임자는 생체 내 의약품 개발에서 회사의 진전을 강조했습니다. 이 연구는 유전자 상향 조절 편집 전략을 통해 기능 상실 또는 돌연변이로 인한 질환을 해결하기 위해 단백질 수치를 증가시킬 수 있는 능력을 보여줍니다. 그들의 표적 지질 나노입자 전달 시스템은 '플러그 앤 플레이' 방식으로 여러 조직에 적용 가능성을 보여줍니다.
Editas Medicine (Nasdaq : EDIT) a annoncé l'acceptation de cinq résumés pour présentation lors du 28e congrès annuel de l'American Society of Gene and Cell Therapy (ASGCT), prévu du 13 au 17 mai 2025 à la Nouvelle-Orléans.
Les présentations comprennent :
- Une présentation orale sur des données précliniques in vivo utilisant des nanoparticules lipidiques ciblées pour l'édition du promoteur HBG1/2
- Une preuve de concept préclinique pour une cible hépatique utilisant l'édition CRISPR
- Des résultats d'études chez la souris et le primate non humain montrant des niveaux élevés d'édition du gène cible dans le foie
- Des données sur la modification des guides et les améliorations du ciblage pour de meilleurs résultats en édition génique
Linda C. Burkly, vice-présidente exécutive et directrice scientifique, a souligné les progrès de l'entreprise dans le développement de médicaments in vivo. La recherche démontre leur capacité à augmenter les niveaux de protéines pour traiter des maladies causées par une perte de fonction ou des mutations via une stratégie d'édition pour l'augmentation génique. Leur système de délivrance par nanoparticules lipidiques ciblées montre un potentiel pour des applications multi-tissulaires avec une approche « plug 'n play ».
Editas Medicine (Nasdaq: EDIT) gab die Annahme von fünf Abstracts für Präsentationen auf dem 28. Jahrestreffen der American Society of Gene and Cell Therapy (ASGCT) bekannt, das vom 13. bis 17. Mai 2025 in New Orleans stattfindet.
Die Präsentationen umfassen:
- Eine mündliche Präsentation zu in vivo präklinischen Daten mit zielgerichteten Lipid-Nanopartikeln zur Bearbeitung des HBG1/2-Promotors
- Präklinischer Machbarkeitsnachweis für ein Leberziel mittels CRISPR-Editing
- Ergebnisse aus Studien an Mäusen und nicht-menschlichen Primaten, die hohe Zielgen-Bearbeitungsraten in der Leber zeigen
- Daten zu Leitfadenmodifikationen und Verbesserungen beim Targeting für bessere Ergebnisse beim Gen-Editing
Linda C. Burkly, Executive Vice President und Chief Scientific Officer, hob die Fortschritte des Unternehmens bei der Entwicklung von In-vivo-Medikamenten hervor. Die Forschung zeigt ihre Fähigkeit, Proteinspiegel zu erhöhen, um Krankheiten zu behandeln, die durch Funktionsverlust oder Mutationen verursacht werden, mittels einer Gen-Hochregulierungs-Editing-Strategie. Ihr zielgerichtetes Lipid-Nanopartikel-Transportsystem zeigt Potenzial für Anwendungen in verschiedenen Geweben mit einem 'Plug-and-Play'-Ansatz.
- Five abstracts accepted for presentation at ASGCT, including one oral presentation, demonstrating research progress
- Successful preclinical proof of concept showing high levels of target gene editing in liver with biomarker response
- Demonstrated capability to increase protein levels to address loss-of-function diseases via gene upregulation strategy
- Progress in targeted lipid nanoparticles (tLNP) delivery system showing potential for multi-tissue applications
- Positive preclinical data from both humanized mouse and non-human primate studies
- Still in preclinical stage with no immediate revenue generation
- No clinical trial data or timeline announcements provided
- Multiple competitors in the gene editing space could affect market position
Insights
Editas's preclinical data shows promising gene editing progress in animal models, advancing their in vivo delivery technology toward potential clinical applications.
Editas Medicine has made notable preclinical progress on their in vivo gene editing platform, with five abstract acceptances at the upcoming ASGCT conference. Their data demonstrates two key technological advancements: First, they're showing success with targeted lipid nanoparticles (tLNPs) delivering editing cargo directly to hematopoietic stem cells in bone marrow of both humanized mice and non-human primates – a challenging target that could eliminate the need for ex vivo cell processing used in current approaches. Second, they've demonstrated a novel gene upregulation strategy that increases protein expression by editing regulatory regions rather than replacing genes, showing disease-relevant biomarker reductions in mouse models.
The data with AsCas12a nuclease delivered via LNPs in non-human primates further validates their platform approach. Their mention of a
Editas's preclinical data demonstrates technological progress in their gene editing platform but remains years from potential commercialization.
This preclinical data announcement represents incremental scientific progress for Editas as they continue developing their in vivo gene editing platform. The company is demonstrating technical capabilities across multiple key areas: in vivo editing of hematopoietic stem cells, gene upregulation strategies for liver targets, and optimization of lipid nanoparticle delivery. This aligns with their strategic refocus toward in vivo editing announced previously and shows execution against their technological roadmap.
Particularly noteworthy is their demonstration of functional biomarker improvements in disease-relevant mouse models and the translation of their editing approach to non-human primates, essential steps toward potential clinical applications. Their work on targeted delivery to specific cell types could provide a competitive advantage in the crowded gene editing space.
However, investors should recognize that these programs remain in early preclinical stages with likely multiple years of development before human trials. The announcement lacks specifics on development timelines or prioritization among these programs. While scientifically meaningful, this news represents expected R&D progress rather than a fundamental catalyst that would significantly change Editas's near-term prospects or valuation. The data will be most useful in helping the company determine which programs to advance toward clinical development and potentially attract partnership interest.
CAMBRIDGE, Mass., April 28, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today announced that five abstracts have been accepted for presentation, including one oral presentation, at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) being held May 13 – 17, 2025, in New Orleans, LA, and virtually. The Company is presenting preclinical data to support its development of transformative in vivo gene editing medicines.
Editas Medicine presentations at ASGCT include:
- Oral presentation of in vivo preclinical data from humanized mouse and non-human primate (NHP) studies using targeted lipid nanoparticles (tLNPs) to deliver HBG1/2 promoter editing cargo to hematopoietic stem and progenitor cells (HSPCs) and/or hematopoietic stem cells (HSCs) in bone marrow.
- Preclinical proof of concept for an undisclosed liver target using in vivo CRISPR editing to upregulate target protein expression and reduce a disease-associated biomarker in a relevant mouse disease model.
- Proof of concept results from the first in vivo mouse and NHP studies demonstrating high levels of target gene editing in the liver and corresponding biomarker response following intravenous administration of AsCas12a messenger RNA (mRNA) and chemically modified guide RNAs (gRNAs) delivered using LNPs from Genevant.
- Additional preclinical data demonstrating in vivo gene editing capabilities towards developing transformative in vivo medicines, including guide modification and targeting moiety optimizations to increase potency and improve gene editing outcomes in vivo.
“Editas Medicine is making significant progress towards the clinic with our in vivo medicines in preclinical development 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 further proof of concept data at ASGCT, including preclinical data confirming our ability to increase the level of a protein to address diseases caused by loss of function or deleterious mutations via our differentiated gene upregulation editing strategy. Our progress with tLNP delivery highlights the potential to execute our gene upregulation strategy across multiple tissues with our ‘plug ‘n play’ approach.”
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: In Vivo Delivery of HBG1/2 Promoter Editing Cargo to HSC of Humanized Mouse and Non-Human Primate with Lipid Nanoparticles
Session Date and Time: Wednesday, May 14, 2025, 1:30 p.m. – 1:45 p.m. CT
Session Title: Translational Applications of Base and Prime Editors
Room: 265-268
Final Abstract Number: AMA353
Poster Presentations:
Title: Design and Development of Improved LNP Targeting Ligands for In Vivo Hematopoietic Stem Cell Editing
Session Date and Time: Tuesday, May 13, 2025, 6:00 p.m. – 7:30 p.m. CT
Session Title: Tuesday Poster Reception
Presentation Room: Poster Hall, Hall 12
Final Abstract Number: AMA245
Title: Design of Chemically Modified AsCas12a Guide RNAs for Increased Potency of LNP-Delivered Gene Editing Cargos
Session Date and Time: Tuesday, May 13, 2025, 6:00 p.m. – 7:30 p.m. CT
Session Title: Tuesday Poster Reception
Presentation Room: Poster Hall, Hall 12
Final Abstract Number: AMA420
Title: In Vivo Gene Editing and Disease-Associated Biomarker Reduction for Multiple Liver Targets in Non-human Primate Using AsCas12a Nuclease Delivered by LNP
Session Date and Time: Wednesday, May 14, 2025, 5:30 p.m. – 7:00 p.m. CT
Session Title: Wednesday Poster Reception
Presentation Room: Poster Hall, Hall 12
Final Abstract Number: AMA640
Title: In Vivo CRISPR Editing of Genetic Regulatory Regions Results in Functional Upregulation of Target Protein and Meaningful Reduction of Disease-Associated Biomarker in Mice
Session Date and Time: Wednesday, May 14, 2025, 5:30 p.m. – 7:00 p.m. CT
Session Title: Wednesday Poster Reception
Presentation Room: Poster Hall, Hall 12
Final Abstract Number: AMA351
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 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.
Media and Investor Contact: ir@editasmed.com
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