CRISPR-Cas Inhibiting Polypeptides

Tech ID: 32880 / UC Case 2018-166-0

Technology Description

The inventions introduce novel Cas nuclease inhibiting polypeptides with synthetic/ non-natural amino acids as well as techniques to conjugate it to an antibody or PEG for easy delivery to targeted cells. Bacterial cells respond to phage attack by activating CRISPR-Cas nuclease to digest phage genome; to escape it, phages encode anti-CRIPSR-Cas proteins. Broad-spectrum Cas enzyme inhibitors will allow for better post-translational regulation of CRISPR-Cas system for gene editing. These polypeptides were hitherto unknown and their use along with CRISPR-Cas system for genetic manipulation will improve its efficacy and potential for translation to human applications. 

Value Proposition

 

  • Anti-CRISPR polypeptides are the tools which will efficiently target the genes being edited
  • Large number of clinical trials are currently using CRISPR-Cas systems to treat diseases- from hematological malignancies to treating HIV via permanent genetic modifications.  However, a major risk involved in using CRISPR-Cas based gene therapy are off-target effects, which may lead to permanent changes in patient genome at non-target sites. 
  • Anti-CRISPR polypeptides allow for long-term regulation of CRISPR-Cas therapy without any off-target effects. They can be introduced independently of the CRISPR system, and they will maintain activity over longer durations.
  • Anti-CRISPR polypeptides act with a better specificity are easier to design and synthesize, when compared to small molecule inhibitors.

 

Related Materials

Patent Status

Country Type Number Dated Case
United States Of America Published Application 20210363206 11/25/2021 2018-166
United States Of America Published Application 20200087354 03/19/2020 2017-057
Japan Published Application 2019 535324 12/12/2019 2017-057
 

Additional Patents Pending

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Inventors

  • Bondy Denomy, Joseph W.
  • Doudna, Jennifer A.

Other Information

Keywords

CRISPR, gene editing, Cas, anti-CRISPR

Categorized As