Targeted delivery to beta cells

US12208140B2 · kind B2 · utility

Assignees

• The Broad Institute, Inc. · US
• The Brigham and Women's Hospital, Inc. · US

Inventors

• Amit Choudhary · Boston, US
• Debasish MANNA · Boston, US
• Miseon Lee · Cambridge, US
• Bridget Wagner · Medford, US
• Basudeb Maji · Boston, US

Key dates

• Filing date: Apr 20, 2018
• Grant date: Jan 28, 2025
• Expiry date: May 11, 2039

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N15/63
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The disclosure includes zinc prodrugs for targeted delivery of therapeutic, diagnostic or imaging agents to β-cells and methods of use therefor. The disclosure also includes targeted delivery of small molecules to β-cells that stabilize and activate CRISPR effector proteins comprising at least one destabilization domain, to enable CRISPR-based genome editing and transcriptional activation or repression in β-cells.