LNP for metabolic diseases
In metabolic diseases, we focus on targeting adipose tissue and other key metabolic organs. We design LNPs that can deliver both siRNA and mRNA selectively to adipocytes and related cell types to modulate gene expression in vivo. These approaches enable the silencing of disease-associated pathways and the expression of therapeutic proteins, with the goal of reprogramming tissue function, improving metabolic homeostasis, and treating disorders such as obesity and associated metabolic dysfunction.
LNP for neurological diseases
For neurological diseases, we develop LNP formulations capable of delivering RNA to the brain. Because the central nervous system is a major barrier for nucleic acid delivery, we focus on creating materials that can efficiently transfect neurons and other relevant cell types. These systems are applied to diseases such as glioblastoma and neurodegeneration.
High-Throughput Screening
The therapeutic potential of LNPs is fundamentally constrained by the range of organs and tissues they can efficiently transfect. To overcome this bottleneck, we developed next-generation high-throughput screening technologies capable of identifying LNPs that deliver therapeutic mRNA payloads to diverse disease-relevant tissues. Our platform, CYBR (Cytosolic-sensing Barcoded RNA), enables precise identification of LNPs that transfect historically hard-to-reach organs, including the heart, kidneys, and brain, through sequencing of target tissues. Leveraging CYBR, we have identified distinct LNP classes, including non-immunogenic and acid-degradable formulation variants, optimized for delivery to specific disease tissues and therapeutic strategies previously considered out of reach.