Key Takeaways
- Tessera Therapeutics presents preclinical breakthroughs in genome engineering for sickle cell disease and T-cell therapies.
- Gene Writing achieved high efficiency in rewriting the HBB gene, showing therapeutic potential for SCD.
- CAR-T cells were successfully generated in vivo, with promising applications in cancer and autoimmune diseases.
Tessera Therapeutics unveiled groundbreaking preclinical data at the 66th American Society of Hematology (ASH) Annual Meeting, showcasing advances in genome engineering for sickle cell disease (SCD) and T-cell therapies. Using its proprietary Gene Writing™ technology encapsulated in lipid nanoparticles (LNPs), Tessera demonstrated the potential to efficiently rewrite genes and generate therapeutic CAR-T cells in vivo.
In SCD studies, Tessera’s Gene Writers achieved significant HBB gene rewriting in humanized mice and non-human primates (NHPs). These results surpassed thresholds associated with reversing the sickle phenotype in patients and marked a milestone in advancing non-viral gene therapies.
Advancements in Sickle Cell and T-Cell Therapies
Tessera’s preclinical SCD data showed an average of 62% rewriting of the HBB gene in humanized mice following a single intravenous dose. Early NHP studies achieved 24% HBB rewriting, reaching therapeutic levels established in human mixed-donor chimerism research. Furthermore, Tessera’s proprietary LNP platform enhanced bone marrow delivery 52-fold compared to liver-tropic LNPs, ensuring precise targeting of hematopoietic stem cells.
For T-cell therapies, Gene Writers achieved up to 60% CAR integration in vitro and 30% CAR writing in vivo in naïve humanized mouse models. A single infusion generated functional CAR-T cells targeting CD19 and CD20, leading to tumor clearance in xenograft models and B-cell depletion, respectively. These findings highlight the therapeutic potential of Tessera’s platform in oncology and autoimmune diseases.
Unlocking Therapeutic Frontiers
Michael Severino, M.D., CEO of Tessera Therapeutics, emphasized the transformative potential of Gene Writing in delivering precise, non-viral genome edits. “Our results at ASH underscore the therapeutic possibilities of in vivo gene editing, providing hope for patients with genetic disorders and cancer,” he stated.
Tessera’s Gene Writing platform represents a new frontier in genome engineering, offering the ability to address inherited genetic conditions and engineer immune cells to combat cancer and autoimmune diseases. The company’s proprietary LNP technology ensures targeted delivery, reducing off-target effects and maximizing therapeutic outcomes.
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