Engineered Nucleases Targeting Factor VIII Genes for Hemophilia A Treatment
Summary
The USPTO granted Patent US12595470B2 to Precision BioSciences, Inc. covering engineered nucleases that recognize and cleave a recognition sequence within the int22h-1 sequence of the Factor VIII gene. The patent also covers methods of using such nucleases to create genetically-modified cells and their use in pharmaceutical compositions for treating hemophilia A. The patent contains 18 claims and names five inventors.
What changed
USPTO granted Patent US12595470B2 to Precision BioSciences covering engineered nucleases targeting the int22h-1 sequence of the Factor VIII gene. The patent encompasses methods of making genetically-modified cells using these nucleases, pharmaceutical compositions containing the nucleases or modified cells, and methods for treating hemophilia A using these compositions.
For pharmaceutical companies and biotechnology firms developing gene therapies for hemophilia A, this patent establishes intellectual property rights that may require licensing negotiations or careful design-around strategies. Precision BioSciences now holds exclusive rights to this specific nuclease approach, potentially affecting competitive dynamics in the gene therapy hemophilia treatment space.
What to do next
- Monitor for patent scope implications if developing hemophilia A gene therapies
- Review licensing opportunities with Precision BioSciences if operating in this therapeutic space
- Track related patent applications in the gene editing hemophilia field
Archived snapshot
Apr 7, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
Engineered nucleases that target human and canine factor VIII genes as a treatment for hemophilia A
Grant US12595470B2 Kind: B2 Apr 07, 2026
Assignee
Precision BioSciences, Inc.
Inventors
Armin Hekele, Clayton Beard, Derek Jantz, James Jefferson Smith, Victor Bartsevich
Abstract
The present invention encompasses engineered nucleases which recognize and cleave a recognition sequence within the int22h-1 sequence of a Factor VIII gene. The present invention also encompasses methods of using such engineered nucleases to make genetically-modified cells, and the use of such cells in a pharmaceutical composition and in methods for treating hemophilia A. Further, the invention encompasses pharmaceutical compositions comprising engineered nuclease proteins, nucleic acids encoding engineered nucleases, or genetically-modified cells of the invention, and the use of such compositions for treating of hemophilia A.
CPC Classifications
C12N 9/22
Filing Date
2024-02-23
Application No.
18586064
Claims
18
Named provisions
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