Editas Medicine reports successful gene editing levels in stem cells, supporting potential treatment for sickle cell disease and beta thalassemia.
Quiver AI Summary
Editas Medicine announced promising new data on May 14, 2025, demonstrating effective gene editing levels for HBG1/2 promoters in hematopoietic stem cells (HSCs) through a single dose of their proprietary targeted lipid nanoparticle (tLNP). This innovative approach aims to increase fetal hemoglobin levels as a treatment for sickle cell disease and beta thalassemia. In studies with humanized mice and non-human primates, the tLNP achieved up to 48% and 47% editing levels respectively, exceeding the therapeutic threshold of 25%. These findings suggest Editas' gene editing method could significantly improve treatment outcomes for patients. The data were presented during the 28th Annual Meeting of the American Society of Gene and Cell Therapy in New Orleans.
Potential Positives
- New data demonstrate high levels of HBG1/2 promoter editing in hematopoietic stem cells using a proprietary targeted lipid nanoparticle, suggesting significant therapeutic potential for sickle cell disease and beta thalassemia.
- The studies exceeded the predicted editing threshold of ≥25% required for therapeutic benefit, indicating a promising pathway to commercially viable treatment options.
- Significant de-targeting of the liver was observed with the proprietary tLNP, contrasting with standard lipid nanoparticles, potentially improving patient outcomes and safety profiles.
Potential Negatives
- Potential risks associated with reliance on preclinical data for future clinical applications, which may result in setbacks or disappointments in translating results to human patients.
- Uncertainty regarding the effectiveness and safety of the proprietary tLNP formulation in diverse patient populations, as current findings are based on animal studies.
- The challenge of achieving regulatory approval for new gene editing therapies can be a lengthy and complex process, potentially delaying the availability of treatments to patients in need.
FAQ
What are the key findings from Editas Medicine's recent data?
Editas Medicine demonstrated therapeutically relevant editing levels in HBG1/2 promoters using targeted lipid nanoparticles in preclinical studies.
How does Editas Medicine's approach treat sickle cell disease?
The approach involves upregulating fetal hemoglobin using gene editing to potentially transform treatment for sickle cell disease and beta thalassemia.
What are the editing levels achieved in the studies?
Editas reported up to 48% editing in humanized mice and 47% in non-human primates, exceeding the therapeutic threshold of 25%.
What technology does Editas use for gene editing?
Editas uses proprietary AsCas12a technology for high-efficiency editing while minimizing off-target effects in hematopoietic stem cells.
When will Editas present additional findings?
Editas will present further data at the 28th Annual Meeting of the American Society of Gene and Cell Therapy on May 14, 2025.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
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Full Release
Data demonstrate therapeutically relevant editing levels using a clinically validated strategy, supporting its development as a novel, 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 shared new in vivo data demonstrating therapeutically relevant levels of HBG1/2 promoter editing in hematopoietic stem cells (HSCs) with a single dose of proprietary targeted lipid nanoparticle (tLNP) in humanized mice and non-human primates (NHPs). This clinically validated approach targeting HBG1/2 promoters to upregulate fetal hemoglobin (HbF) is in pre-clinical development as a potential transformative in vivo gene editing medicine for the treatment of sickle cell disease and beta thalassemia. The Company reported these data in a presentation available today and will detail the data in an oral presentation today at 1:30 p.m. CT/2:30 p.m. ET at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in New Orleans, LA, and virtually.
In these studies, the Company’s proprietary tLNP formulation delivered HBG1/2 promoter editing cargo to HSPCs and/or HSCs in humanized mice (mice engrafted with human CD34+ cells) and in NHPs. In an ongoing NHP study, administration of a single intravenous dose of Editas Medicine’s proprietary tLNP demonstrated high efficiency HSC delivery and achieved up to 47% HBG1/2 editing levels. In a study with humanized mice, administration of a single dose achieved 48% editing of HBG1/2 in long-term HSCs. Both studies exceeded the predicted editing threshold of ≥25% required for therapeutic benefit. In addition to achieving therapeutically relevant editing levels, preliminary biodistribution data in NHPs with Editas’ tLNP shows significant de-targeting of the liver in contrast to standard LNPs.
“These findings are very encouraging and further support our approach to developing a potentially first- and best-in-class in vivo gene edited medicine for the treatment of sickle cell disease and beta thalassemia,” said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. “We believe that translating these preclinical results to the clinic will address the continuing significant unmet need for a transformative gene edited medicine with the potential to improve the lives of people living with sickle cell disease and beta-thalassemia around the world.”
Editas Medicine’s in vivo HSC program targets HBG1/2 promoters to mimic naturally occurring mechanisms of hereditary persistence of fetal hemoglobin (HPFH) and utilizes proprietary AsCas12a to edit with high efficiency and minimize off-target editing. Editing the HBG1/2 promoters with AsCas12a with the investigational medicine reni-cel led to robust increases in fetal hemoglobin (HbF) and total hemoglobin (Hb) in clinical trials.
Oral Presentation Details:
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
Additional Editas Medicine presentations are below. Abstracts can be accessed on the ASGCT website , and the presentations will be posted on the Editas Medicine website during the conference.
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
.
