Preclinical studies conducted with Sail’s eRNA demonstrate durable protein expression in vivo, enabling a prolonged therapeutic effect. This has a huge impact on how we think about biologic medicines, opening the door for RNA medicines to treat chronic diseases, especially those requiring intracellular or transmembrane protein replacement not amenable to biologics, with lower dose levels and less frequent administration.

eRNA is a form of translatable circular RNA where the contiguous ring of single-stranded RNA lacks the cap and poly-A tail that cells normally use to control mRNA degradation.

mRNA medicines have shown great promise, most notably in vaccines, but they have a relatively short duration of therapeutic protein expression, typically 1-2 days, which puts significant limitations on the types of disease that can be addressed and the performance of the medicines themselves. eRNA has the potential to overcome these challenges and unlock the full promise of RNA therapeutics.

Targeted nanoparticles (TNPs) extend the deployment of RNA medicines beyond the liver. By conjugating targeting ligands to a clinically validated lipid nanoparticle core, each TNP is designed to seek out a chosen cell type to deliver its eRNA cargo, minimizing off-target exposure.

Targeting ligands direct cargo to the intended cell population, reducing systemic exposure.

Optimized lipid composition supports robust cytoplasmic delivery.

Industry-standard unit operations and established conjugation process to enable efficient platform-based production

Sail’s TNPs have demonstrated unique cell tropism in multiple animal models. Modular, site-specific conjugation chemistry lets us swap in new targeting ligands without reformulating the core nanoparticle, opening a rapid path from idea to IND.

We’re using cutting-edge, proprietary AI techniques and rapid prototyping abilities to accelerate our efforts to revolutionize programmable RNA-based medicines.

With our programmable eRNA technology and proprietary atlas of programmable nanoparticle components, Sail has dramatically expanded the RNA medicines design space. To interrogate this vast design space, we have built a high-throughput system that generates functional data at remarkable pace to create iterative learning loops leverage machine intelligence to predictably program RNA medicines.

With unmatched data flow and proprietary techniques, we’re able to apply AI to program in an integrated way across the pharmacology and delivery components of the medicine – i.e., RNA and nanoparticle – with the aim of driving breakthroughs in durability, efficiency, safety/tolerability and targeting – and ultimately delivering therapeutic benefits to patients that have previously been unreachable.