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     117  0 Kommentare Generation Bio Announces the Presentation of Preclinical Data on iqDNA and ctLNP Platforms at the ASGCT 27th Annual Meeting

    • Data presented show immune-quiet DNA (iqDNA) is a partially single-stranded DNA cargo that maintains expression while evading innate immune sensors
    • Rapid enzymatic synthesis continues to improve iqDNA performance through process development and the identification of novel structural elements
    • Data on cell-targeted LNP (ctLNP) in vivo delivery of therapeutic transgenes to T cells to be discussed in an oral presentation on Saturday, May 11

    CAMBRIDGE, Mass., May 09, 2024 (GLOBE NEWSWIRE) --  Generation Bio Co. (Nasdaq:GBIO) a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, today presented five posters on its immune-quiet DNA (iqDNA) and cell-targeted lipid nanoparticle (ctLNP) platforms as well as its enzymatic synthesis manufacturing at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting. A sixth presentation on the company’s ctLNP platform will be discussed in an oral presentation on Saturday, May 11.

    “The data we’re presenting at ASGCT showcase some important mechanistic underpinnings of our novel platforms, which we believe have the potential to overcome the core challenges facing the field of genetic medicine,” said Matt Stanton, Ph.D., chief scientific officer of Generation Bio. “We believe immune-quiet DNA cargo and highly selective, targeted LNP delivery are the tools required to unlock the full potential of non-viral genetic medicines, and we are applying them to our current portfolio of programs in T cells, hematopoietic stem cells, and hepatocytes.”

    Data included in the posters describe iqDNA as a modified, structured, partially single-stranded DNA that evades innate immunity while remaining transcriptionally active in the cell. In mouse studies, the immune profile of iqDNA is consistent with avoidance of key innate immune pathways such as cGAS-STING, which is known to have a significantly lower binding affinity for single-stranded DNA than double-stranded DNA. Given the non-immunogenic nature of iqDNA, Generation Bio believes it can now create novel therapies with a wide therapeutic index, which has been a major challenge to the development of in vivo DNA-based genetic medicines.

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    Generation Bio Announces the Presentation of Preclinical Data on iqDNA and ctLNP Platforms at the ASGCT 27th Annual Meeting Data presented show immune-quiet DNA (iqDNA) is a partially single-stranded DNA cargo that maintains expression while evading innate immune sensorsRapid enzymatic synthesis continues to improve iqDNA performance through process development and the …