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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

1. Sana demonstrates potent gene editing of hematopoietic stem cells using fusogen technology. 2. Fusogen technology expands delivery capabilities beyond T cells to HSCs. 3. Sana plans IND filing for SG293 targeted CAR T cells by 2027. 4. Potential treatment advancements for sickle cell disease and beta thalassemia highlighted. 5. Research shows significant in vivo gene editing effectiveness in preclinical models.

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Why Bullish?

The introduction of fusogen technology suggests significant advancements in gene therapy for diseases, which may attract investment and increase stock value, similar to previous market reactions to breakthrough therapies.

How important is it?

The article provides crucial insights about Sana’s innovative technology, directly influencing investor confidence and market positioning in the biotech sector focused on gene therapy.

Why Long Term?

The effects of technological advancements and IND filings usually unfold over time, impacting SANA's growth and market positioning as developments progress through to clinical trials and eventual approval.

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Sana Biotechnology Advances Gene Editing Technology for Hematopoietic Stem Cells

SEATTLE, December 08, 2025 (GLOBE NEWSWIRE) – Sana Biotechnology, Inc. (NASDAQ: SANA) has recently announced a significant breakthrough in the field of gene editing. The company's innovative fusogen technology was highlighted in a publication by Nature Biotechnology, which details in vivo gene editing of human hematopoietic stem cells (HSCs) using systemically delivered virus-like particles (VLPs). This advancement showcases the promising potential of Sana's technology in treating diseases such as sickle cell disease and beta thalassemia.

Breakthrough Results in Hematopoietic Stem Cell Editing

The published study evaluated Sana's fusogen technology for its ability to achieve potent and cell-specific editing of HSCs in the bone marrow across various murine preclinical models. The results demonstrated stable gene editing of long-term HSCs, marking a critical advancement in the treatment of blood disorders.

  • Key Findings:
  • Optimized VLP facilitates effective editing in long-term human HSCs.
  • Targeted fusogen technology minimizes off-target effects in organs such as the liver.
  • The approach offers a substantial improvement over traditional bone marrow transplants and ex vivo editing methods, which often rely on high doses of conditioning chemotherapy.

Implications for Future Treatments

Sana Biotechnology's fusogen platform is poised to change the landscape of gene therapy by simplifying the delivery mechanism. Current challenges faced by conventional therapies—such as severe infections, secondary cancers, and complicated manufacturing processes—could potentially be alleviated through this innovative delivery system. Dr. Dhaval Patel, Chief Scientific Officer of Sana, emphasized the transformative implications of the fusogen technology, stating, “This publication underscores the ability of our platform to deliver diverse payloads for gene editing efficiently and specifically, thus broadening the possible applications for various diseases.”

Future Developments: Project SG293

In addition to the advancements in HSC editing, Sana is actively developing SG293, a CD8-targeted fusosome designed to create CD19-directed CAR T cells in vivo. The company plans to file an Investigational New Drug (IND) application for SG293 targeting B-cell cancers and autoimmune diseases as early as 2027.

About Sana Biotechnology, Inc.

Sana is focused on creating and delivering engineered cells as medicines to improve patient outcomes. With operations in Seattle, WA, Cambridge, MA, and South San Francisco, CA, the company aims to replace damaged cells and repair genes, making its innovative therapies accessible to a broad patient population.

Cautionary Note Regarding Forward-Looking Statements

This announcement contains forward-looking statements related to the company’s development programs and future strategies. Sana emphasizes that these statements are subject to risks and uncertainties that could cause actual results to vary significantly. Readers should not rely solely on these projections as indicators of future performance.

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