Generation Bio Announces Breakthrough in its Non-Viral Genetic Medicine Platform with Novel “Immune-Quiet” DNA

– Immune-quiet DNA (iqDNA) is a novel variant of closed-ended DNA (ceDNA) that evades host innate immune detection in both mice and non-human primates (NHPs) with a systemic cytokine profile and tolerability comparable to mRNA

– Company is advancing iqDNA in lieu of prior ceDNA constructs across all programs, including its lead program in hemophilia A

– Company is extending cash runway guidance into 2026 through collaboration income and expense control while increasing investment in its iqDNA and cell-targeted lipid nanoparticle (ctLNP) platforms

– Company will present data on iqDNA and ctLNP platforms at upcoming scientific meetings today and on October 26, and will host a webcast R&D event on November 1

CAMBRIDGE, Mass., Oct. 18, 2023 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq:GBIO), a biotechnology company developing genetic medicines for people living with rare and prevalent diseases, today announced the development of a proprietary, novel DNA called immune-quiet DNA (iqDNA). iqDNA is an optimized variant of the company’s closed-ended DNA (ceDNA) that upon systemic administration with Generation Bio’s lipid nanoparticle (LNP) delivery has shown cytokine levels and tolerability comparable to chemically modified messenger RNA (mRNA) in mice and non-human primates (NHPs).

Generation Bio’s non-viral genetic medicine platform aims to advance durable, redosable, and titratable therapies to reach potentially hundreds of millions of patients worldwide. The company has made deep investments in its three proprietary platform technologies, comprising novel DNA cargos, rapid enzymatic synthesis (RES) manufacturing, and cell-targeted LNP (ctLNP) delivery.

“Developing solutions that allow genetic medicines to avoid innate immune stimulation is a shared and fundamental requirement for enabling non-viral therapeutics, as exemplified by the development of modified mRNA to enable a global modality,” said Matt Stanton, Ph.D., chief scientific officer of Generation Bio. “Our development of iqDNA is a similar step toward realizing the full potential of non-viral DNA therapeutics. I’m excited to further advance our platform as we seek to create safe and effective medicines that could marry the scalability and drug-like properties of mRNA with the durability of DNA-based genetic medicines.”