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Sana Biotechnology Announces Publication in Nature Biotechnology of in vivo Gene Editing of Human Hematopoietic Stem Cells in Preclinical Models Using the Fusogen Platform
Data Demonstrate Potent in vivo Gene Editing of Hematopoietic Stem Cells (HSCs) in the Bone Marrow with Systemic Delivery in Preclinical Murine Models Using
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[{"type":"text","content":"Data Demonstrate Potent in vivo Gene Editing of Hematopoietic Stem Cells (HSCs) in the Bone Marrow with Systemic Delivery in Preclinical Murine Models Using Fusogen Technology Broadens Application of Fusogen Technology Beyond T Cells to Second Cell Type, HSCs, Showing Potent and Specific in vivo Delivery Underscores Ability of Fusogen Technology to Deliver Diverse Payloads, including CRISPR Gene-Editing and Base-Editing Machinery Highlights Potential of Fusogen Platform in Diseases like Sickle Cell Disease and Beta Thalassemia Without the Need for Conditioning Chemotherapy Sana is Incorporating its Fusogen Technology to Develop SG293, a CD8-targeted Fusosome that Makes CD19-directed CAR T Cells in vivo; Expects to File IND for SG293 in B-cell Cancers and/or B-cell Mediated Autoimmune Diseases as Early as 2027 SEATTLE, Dec. 08, 2025 (GLOBE NEWSWIRE) -- Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, today announced that Nature Biotechnology has published a journal article titled “In vivo gene editing of human hematopoietic stem and progenitor cells using envelope-engineered virus-like particles” (DOI: 10.1038/s41587-025-02915-2). The article evaluated a systemically delivered virus-like particle (VLP) using Sana’s fusogen technology to target and gene edit hematopoietic stem cells (HSCs) in vivo. Results show potent and cell-specific in vivo gene editing of HSCs in the bone marrow in several murine models, with stable gene-editing of long-term HSCs. In vivo gene editing of HSCs in the bone marrow has the potential to transform the treatment of many diseases involving these important progenitor cells, including sickle cell disease and beta thalassemia. In vivo delivery can overcome many of the limitations of bone marrow transplants and current ex vivo gene editing approaches, including the need for high doses of conditioning chemotherapy with the risk of severe infections and secondary cancers, complex manufacturing, and prolonged hospitalization. An ideal in vivo delivery system should be capable of efficiently reaching long-term multipotent HSCs in their natural bone marrow niche, avoiding off-target cells in organs such as the liver, and delivering the reagents necessary for gene editing or base editing of clinically relevant loci in human HSCs. “The fus...