Six tightly-coupled research programs — the chemistry and biology of editing, the engineering of delivery (AAV and LNP), and the systems-level study of the nervous system.
We work across the CRISPR toolkit — Cas9 nucleases for double-strand breaks, base editors for single-nucleotide changes, and prime editing for templated edits without DSBs. Our focus is edit specificity, off-target profiling, and durable correction in clinically relevant cell types.
Engineered mRNA constructs optimized for stability, translation, and reduced innate-immune activation. We design 5'/3' UTRs, evaluate codon strategies, and screen modified nucleotides against expression and immunogenicity readouts.
Capsid engineering and tropism redesign for tissue-targeted delivery. We combine library-based directed evolution with structure-guided rational design, and characterize biodistribution and neutralizing-antibody escape.
Ionizable-lipid design and four-component LNP formulation for nucleic-acid delivery — mRNA, siRNA, and gene-editing payloads. We optimise the lipid mix for tissue tropism (liver, lung, immune cells), apparent pKa, and endosomal escape, and pair formulation work with in-house microfluidic mixing for reproducible particle size and PDI.
Rationally designed bioactive peptides — receptor agonists and antagonists, cell-penetrating peptides, and stapled or cyclic scaffolds with improved metabolic stability. Computation drives sequence design; in-house synthesis and assays close the loop.
We study the cellular and circuit biology underlying cognition and neurodegenerative disease, and explore how CRISPR, mRNA, and AAV tools can be applied responsibly to the brain. Strong emphasis on translational rigor and CNS-specific delivery constraints.
Genome editing, AAV delivery, and CNS-targeted therapeutics demand disproportionate care. We work within established biosafety frameworks, publish methods openly, and do not make therapeutic claims beyond the supporting evidence.