Sana Biotechnology announced that its fusogen platform study has been published in Nature Biotechnology on December 8 2025, marking the first peer‑reviewed demonstration of in‑vivo gene editing in human hematopoietic stem and progenitor cells (HSCs). The publication confirms that the platform can deliver CRISPR or base‑editing tools directly to long‑term HSCs in preclinical murine models, achieving stable edits that persist in the stem cell compartment.
The study shows that the fusogen‑mediated delivery system can edit two hemoglobinopathy loci, including the fetal hemoglobin promoter, in a cell‑specific manner without the need for conditioning chemotherapy or ex‑vivo cell manufacturing. By avoiding the intensive conditioning regimens that currently accompany bone‑marrow transplants, the technology could reduce hospital stays and lower the risk of graft‑versus‑host disease, offering a potentially safer and more scalable approach to treating sickle cell disease and beta‑thalassemia.
Sana’s fusogen platform uses envelope‑engineered virus‑like particles (VLPs) to ferry gene‑editing payloads into target cells. The new data extend the platform’s reach beyond its original focus on CD8+ T cells to include HSCs, demonstrating the technology’s versatility and reinforcing the company’s strategy of developing in‑vivo cell‑engineering solutions for a broad range of diseases.
The publication strengthens Sana’s competitive position in the gene‑therapy market by providing a proof of concept that could accelerate the company’s pipeline. Management indicated that the next milestone will be an Investigational New Drug (IND) filing for SG293, a CD8‑targeted fusosome designed to generate in‑vivo CAR‑T cells, slated for submission as early as 2027. The successful HSC study supports the broader applicability of the fusogen platform and may broaden the company’s therapeutic portfolio beyond blood disorders.
Dr. Dhaval Patel, Sana’s Chief Scientific Officer, said, “The fusogen technology has now shown the potential to offer cell‑specific, in‑vivo delivery of various payloads into multiple cell types, and we believe it can be an important technology to treat a variety of diseases.” He added that the HSC data “demonstrate the platform’s versatility and open new therapeutic avenues for serious genetic disorders.”
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