Internal Reference: 2020-042

Market Need聽

CRISPR enzymes suffer from several disadvantages in use, such as potential off-target effects that can be exacerbated when uncontrolled. The safety of CRISPR remains an important concern for practical drug development. To fully implement safe CRISPR-based therapeutics, it may be necessary to develop 鈥渒ill switch鈥� inhibitors that can halt activity of CRISPR. There is an urgent need to develop strategies and molecules that can inhibit CRISPR either for safety concerns or to optimize specificity of enzymes.

Technology Overview

成人VR视频 has invented nucleic acid-based CRISPR inhibitor molecules composed of an artificial nucleic acid construct having a first polynucleotide. The inhibitor molecule is capable of establishing several points of contact with a CRISPR protein with high binding affinity. The first polynucleotide is comprised of a sequence selected from the group consisting of: a poly颅nucleotide that interacts with a protospacer adjacent motif (PAM)-interaction (PI) domain of a CRISPR-associated (Cas) protein, a polynucleotide that interacts with a guide sequence of a crRNA or an equivalent position of a single-颅guide RNA, and a polynucleotide that interacts with a repeat region of a tracrRNA or an equivalent position of a single-颅guide RNA. The CRISPR inhibitor molecule may also include a second polynucleotide and a linker.

Commercial Advantages

• Our inhibitors are substantially smaller than anti-CRISPR proteins which can be further engineered with additional chemical modifications
• Our inhibitors allow for tuning drug-like properties and makes them better drug candidates than potentially competing anti-CRISPR proteins

Additional Information

• Researcher: Masad Damha
• Patents:聽US 17/280,994 (Granted), Canada: CA 3,114,405 (Filed)
• Keywords:聽Research Tool, Platform, Gene Therapy