Sana Biotechnology announces publication of in vivo gene editing

Sana Biotechnology (SANA) 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.” 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. Key Findings from The Publication: Optimized VLP enables potent in vivo editing in long-term human HSCs and editing of two hemoglobinopathy-relevant loci, including fetal hemoglobin. Gene-editing VLP with targeted fusogen technology avoids off-target delivery to hepatocytes with systemic delivery.