Sana Biotechnology announced that a paper published in Nature Biotechnology shows its “fusogen” virus‑like particle (VLP) platform successfully achieved in vivo gene editing of human hematopoietic stem and progenitor cells (HSPCs) in preclinical murine models.
The study demonstrated that systemic delivery of optimized fusogen‑VLPs led to substantial, cell‑specific editing in long-term human HSCs residing in bone marrow. In particular, the VLPs edited loci relevant to hemoglobinopathies—including BCL11A and HBG1/2—with editing efficiencies of ~26% and ~7.5% at early time points, and also achieved ~31% editing of β2‑microglobulin at 8 weeks post dosing, indicating durable editing in long‑term HSCs.
Importantly, the edited cells retained their long-term engraftment potential and multipotency in vivo, suggesting that the procedure did not impair their capacity to regenerate diverse blood cell lineages. The VLP design also avoided significant off‑target delivery to hepatocytes in the liver, a common safety concern in systemic gene delivery.
According to Sana, the result expands the fusogen platform’s scope beyond T‑cells (its previously demonstrated target) to HSCs—potentially enabling in vivo treatment of a broad range of blood and genetic disorders such as sickle‑cell disease and beta‑thalassemia, without the need for conditioning chemotherapy or ex vivo cell manipulation.
The company said it plans to leverage the same technology to develop an in vivo CAR‑T therapy (named SG293), with the first investigational new‑drug (IND) filing targeted as early as 2027.
