Compact proteins

WO2024238296A3 Publication Date: 2025-04-03VERTEX PHARMACEUTICALS INC
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Patent Information

Application Number
PCT/US2024/028693
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2023-05-12
Filing Date
2024-05-10
Publication Date
2025-04-03

AI Technical Summary

Technical Problem

Current CRISPR-based genome editing technologies, specifically base editors, face challenges due to their large size, which exceeds the cargo capacity of single AAV vectors, necessitating the use of two AAVs and increasing viral dosage, raising safety concerns and complicating manufacturing processes.

Method used

Development of truncated nucleobase editors comprising a nucleotide deaminase, a Cas9 nuclease, and a linker between them, optimized to fit within a single AAV vector, with specific amino acid lengths and sequences that maintain or enhance base editing activity.

Benefits of technology

The compact nucleobase editors achieve significant base editing activity while reducing the need for multiple AAV vectors, enhancing safety and simplifying manufacturing by maintaining or exceeding the activity of full-length editors within a smaller genetic footprint.

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Abstract

Disclosed herein are compositions of compact proteins such as compact nucleobase editors that may be used, in some embodiments, for genome editing technologies.
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