Editas Medicine to Present in vivo HSC Delivery, Editing, and Biodistribution Data at the European Hematology Association 2025 Congress in June
Editas Medicine (NASDAQ: EDIT) announced the acceptance of new preclinical data for presentation at the European Hematology Association (EHA) 2025 Congress in Milan. The study, conducted in non-human primates, demonstrates promising results for their in vivo hematopoietic stem cell (HSC) gene editing program targeting sickle cell disease and beta thalassemia.
Key findings include high-efficiency HSC delivery, therapeutically relevant editing levels in the HBG1/2 promoter region exceeding 25% threshold with a single dose, and favorable biodistribution using Editas' targeted lipid nanoparticle (tLNP) technology. The data shows significant liver de-targeting compared to standard LNPs, supporting further development of their proprietary HSC-tLNP platform.
Editas Medicine (NASDAQ: EDIT) ha annunciato l'accettazione di nuovi dati preclinici per la presentazione al Congresso 2025 della European Hematology Association (EHA) a Milano. Lo studio, condotto su primati non umani, mostra risultati promettenti per il loro programma di editing genico in vivo delle cellule staminali ematopoietiche (HSC) mirato alla cura dell'anemia falciforme e della beta-talassemia.
I risultati principali includono un'efficiente consegna delle HSC, livelli di editing terapeuticamente rilevanti nella regione promotrice HBG1/2 che superano la soglia del 25% con una singola dose, e una biodistribuzione favorevole grazie alla tecnologia di nanoparticelle lipidiche mirate (tLNP) di Editas. I dati evidenziano una significativa riduzione del targeting al fegato rispetto alle LNP standard, a supporto dello sviluppo ulteriore della loro piattaforma proprietaria HSC-tLNP.
Editas Medicine (NASDAQ: EDIT) anunció la aceptación de nuevos datos preclínicos para su presentación en el Congreso 2025 de la European Hematology Association (EHA) en Milán. El estudio, realizado en primates no humanos, muestra resultados prometedores para su programa de edición genética in vivo de células madre hematopoyéticas (HSC) dirigido a la enfermedad de células falciformes y la beta talasemia.
Los hallazgos clave incluyen una entrega eficiente de HSC, niveles de edición terapéuticamente relevantes en la región promotora HBG1/2 que superan el umbral del 25% con una sola dosis, y una biodistribución favorable utilizando la tecnología de nanopartículas lipídicas dirigidas (tLNP) de Editas. Los datos muestran una desorientación significativa del hígado en comparación con las LNP estándar, apoyando el desarrollo adicional de su plataforma propietaria HSC-tLNP.
Editas Medicine (NASDAQ: EDIT)는 밀라노에서 열리는 2025년 유럽혈액학회(EHA) 총회에서 발표할 새로운 전임상 데이터를 수락했다고 발표했습니다. 비인간 영장류를 대상으로 한 이번 연구는 겸상 적혈구 빈혈과 베타 지중해빈혈을 표적으로 하는 체내 조혈모세포(HSC) 유전자 편집 프로그램에서 유망한 결과를 보여줍니다.
주요 결과로는 높은 효율의 HSC 전달, 단일 투여로 25% 이상의 치료적 편집 수준을 보이는 HBG1/2 프로모터 영역 편집, Editas의 표적 지질 나노입자(tLNP) 기술을 활용한 유리한 생체 분포가 포함됩니다. 데이터는 표준 LNP에 비해 간 표적화가 크게 감소했음을 보여주며, 독자적인 HSC-tLNP 플랫폼의 추가 개발을 뒷받침합니다.
Editas Medicine (NASDAQ: EDIT) a annoncé l'acceptation de nouvelles données précliniques pour présentation au Congrès 2025 de l'European Hematology Association (EHA) à Milan. L'étude, réalisée sur des primates non humains, démontre des résultats prometteurs pour leur programme d'édition génique in vivo des cellules souches hématopoïétiques (HSC) ciblant la drépanocytose et la bêta-thalassémie.
Les résultats clés incluent une livraison efficace des HSC, des niveaux d'édition thérapeutiquement pertinents dans la région promotrice HBG1/2 dépassant le seuil de 25 % après une seule dose, ainsi qu'une biodistribution favorable grâce à la technologie des nanoparticules lipidiques ciblées (tLNP) d'Editas. Les données montrent une déviation significative du foie par rapport aux LNP standard, soutenant le développement ultérieur de leur plateforme propriétaire HSC-tLNP.
Editas Medicine (NASDAQ: EDIT) gab die Annahme neuer präklinischer Daten für eine Präsentation auf dem European Hematology Association (EHA) 2025 Kongress in Mailand bekannt. Die Studie, durchgeführt an nicht-menschlichen Primaten, zeigt vielversprechende Ergebnisse für ihr in vivo hämatopoetisches Stammzell-Geneditierungsprogramm zur Behandlung von Sichelzellanämie und Beta-Thalassämie.
Wesentliche Erkenntnisse umfassen eine hocheffiziente HSC-Lieferung, therapeutisch relevante Editierungsniveaus im HBG1/2-Promotorbereich, die mit einer einzigen Dosis die 25%-Schwelle überschreiten, sowie eine günstige Biodistribution unter Verwendung der zielgerichteten Lipid-Nanopartikel (tLNP)-Technologie von Editas. Die Daten zeigen eine signifikante Reduktion der Leberanreicherung im Vergleich zu Standard-LNPs, was die weitere Entwicklung ihrer proprietären HSC-tLNP-Plattform unterstützt.
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Preclinical studies achieved therapeutically relevant gene editing levels of the HBG1/2 promoter & favorable biodistribution profile in non-human primates using a clinically validated editing strategy
Data reinforces continued development as a potentially transformative, in vivo approach to treating sickle cell disease and beta thalassemia
CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today announced that new data from a study in non-human primates (NHPs) has been accepted for a poster presentation at the European Hematology Association (EHA) 2025 Congress being held June 12-15, 2025, in Milan, Italy.
Key delivery, editing, and biodistribution data from an ongoing study in NHPs will be shared, including:
- High efficiency HSC delivery of editing cargo.
- Therapeutically relevant editing levels in the HBG1/2 promoter region exceeding the predicted editing threshold of ≥
25% required for therapeutic benefit with a single dose. - Favorable biodistribution with Editas’ tLNP, demonstrating significant de-targeting of the liver in contrast to standard LNPs.
“We look forward to sharing further data from our in vivo HSC program at EHA, demonstrating therapeutically relevant editing levels and favorable biodistribution in NHPs. These data warrant the further development of Editas’ proprietary HSC-tLNP for editing of the HBG1/2 promoters for the treatment of sickle cell disease and beta thalassemia, and we are encouraged by our significant progress towards the clinic,” said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine.
The presentation details are listed below. Abstracts can be accessed on the EHA website, and the presentation will be posted on the Editas Medicine website during the conference.
Poster Presentation Details:
Title: Targeted Lipid Nanoparticle Delivery Enables In Vivo HBG1/2 Genome Editing In Non-Human Primates
Date/Time: Saturday, June 14, 2025, 6:30 - 7:30 p.m. CEST/ 12:30 – 1:30 p.m. EDT
Location: Allianz MiCo, Milano Convention Centre
Session: Poster Session 2
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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