Generation Bio Co. 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. Concurrent with extending its cash runway, the company is increasing investment in its iqDNA and ctLNP platforms. Systemic dosing in mouse and NHP studies demonstrates that iqDNA delivered via LNP evades detection by the key innate immune pathways that are reactive to DNA.

Due to the superior tolerability profile of iqDNA, Generation Bio is discontinuing development of prior ceDNA constructs and is advancing iqDNA as the cargo for its lead program in hemophilia A as well as for all other programs. Scaling of iqDNA factor VIII for NHP testing is underway, leveraging existing RES systems and processes.