This technology is an evolved CRISPR-associated transposase system that facilitates highly efficient programmable DNA insertions in human cells without relying on homologous repair and double strand breaks.
Current gene-editing technologies are either non-specific or inefficient and fail to integrate large genetic payloads without generating double-stranded DNA breaks or host homologous repair. Furthermore, while current methods can correct disease-causing mutations, regulatory approval and design of mutation-specific strategies significantly limit the number of patients who can benefit from therapeutic gene editing. There is a need for efficient targeted genomic integration of kilobase-scale payloads with minimal off-target events.
This technology is a CRISPR-transposon system that can facilitate kilobase-scale gene insertions with high specificity, minimal indels, and low off-target effects. Unlike current CRISPR/Cas9 systems, this technology uses Tn7-like transposons to catalyze RNA-guided DNA integration at defined sites without requiring double-strand breaks or host homologous repair. This technology has been optimized to enable multiplexed insertions, with integration events shown across multiple genomic targets in diverse hosts.
This technology has been extensively validated in bacterial cells and has shown efficacy in human cells, including primary human fibroblasts.
Patents Issued (US 10,947,534, US 12,331,292)
Patents Pending (US 20220372521, US 20230147495, US 20240279629, US 20240209399, US 20250376701)
CU19096, CU21342, CU21338, CU21352, CU22030, CU23042, CU24383
Licensing Contact: Cynthia Lang
