Sana Biotechnology Inc. (NASDAQ:SANA) is one of the best emerging technology stocks to invest in. On December 8, Sana Biotechnology announced the publication of groundbreaking preclinical data in the journal Nature Biotechnology. The study, titled “In vivo gene editing of human hematopoietic stem and progenitor cells using envelope-engineered virus-like particles,” details the successful use of Sana’s Fusogen platform to perform gene editing directly within the body.
Using systemic delivery in murine models, the company showed that its virus-like particles/VLPs could precisely target and edit hematopoietic stem cells/HSCs inside the bone marrow, achieving stable results in long-term HSCs. The publication highlights the versatility of the Fusogen technology, which has now proven capable of delivering diverse payloads (including CRISPR gene-editing and base-editing machinery) to two distinct cell types: T cells and HSCs.
A critical finding of the study was the platform’s high specificity; the targeted particles successfully avoided off-target delivery to other organs, such as the liver (hepatocytes) or gonadal tissues. By editing two loci relevant to hemoglobinopathies, including fetal hemoglobin, the platform shows immense promise for treating diseases like sickle cell disease and beta thalassemia.
This in vivo approach aims to revolutionize the standard of care by eliminating the need for traditional bone marrow transplants and ex vivo gene editing. Current methods require conditioning the patient with high-dose chemotherapy, which carries risks of severe infection, secondary cancers, and prolonged hospitalization. Sana’s technology could bypass these toxicities, offering a simplified supply chain and a safer treatment profile.
Sana Biotechnology Inc. (NASDAQ:SANA) is a biotechnology company that utilizes engineered cells as medicines in the US. It develops ex vivo and in vivo cell engineering platforms for therapeutic areas with unmet treatment needs, including type 1 diabetes, B-cell-mediated autoimmune diseases, and oncology.
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Disclosure: None. This article is originally published at Insider Monkey.
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