Genome editing therapies like CRISPR technologies can mainly correct point mutations. An editing system that could restore function of an entire gene (with multiple mutations) would be beneficial. We present a mammalian transposase that delivers large DNA pieces without inducing double-strand breaks, enhancing safety. Key challenges with this technology are the delivery of twopayloads, minimizing off- target biodistribution, while ensuring a high cellular uptake/expression.  Lipid nanoparticles (LNPs) were engineered to co-deliver DNA and transposase mRNA. Optimized LNPs exhibited improved tolerability, efficient uptake, and considerable ABCA4 expression in Abca4⁻/⁻ mice photoreceptors, suggesting therapeutic efficacy in retinal diseases.

Chair:
- Mathew Cherian PhD, Consultant, Member IPS ExCo, USA

Co-moderator: -
Qurrotul Aini Shodiquna, Erasmus Mundus Student in Sustainable Drug Discovery, Indonesia

Panellists:
- Sandeep Nema PhD, President, BioPharm CMC Advisors, USA
- Subhro Guhathakurta PhD, Consultant, USA