Invention Grant
- Patent Title: Delivery of structurally diverse polypeptide cargo into mammalian cells by a bacterial toxin
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Application No.: US15827595Application Date: 2017-11-30
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Publication No.: US10597663B2Publication Date: 2020-03-24
- Inventor: Roman A. Melnyk , Anick Auger , Greg Beilhartz , Berge Minassian , Seiji Sugiman-Marangos
- Applicant: The Hospital for Sick Children
- Applicant Address: CA Toronto
- Assignee: THE HOSPITAL FOR SICK CHILDREN
- Current Assignee: THE HOSPITAL FOR SICK CHILDREN
- Current Assignee Address: CA Toronto
- Agency: Borden Ladner Gervais LLP
- Agent Graeme R. Boocock
- Main IPC: A61K39/00
- IPC: A61K39/00 ; A61K39/02 ; C07H21/04 ; C12N15/62 ; A61K35/74 ; C07K14/34 ; C07K14/28 ; C07K14/33 ; C07K14/47 ; C07K14/475 ; C12N9/10 ; C12N9/22 ; C12N9/28 ; C12N9/52 ; A61K38/45 ; A61K38/48 ; A61K47/68 ; A61K47/42 ; C07K7/08 ; C07K14/00 ; C07K16/12 ; C12N11/06 ; A61K38/00

Abstract:
There is a need for delivery platforms with robust capacity that offer the possibility to deliver diverse protein-based therapeutics into specific cells. Described herein is a platform for delivering cargo polypeptides into cells, which is based on a recombinant molecule comprising: a cargo polypeptide, a diphtheria toxin enzymatic fragment (DTA), and a diphtheria toxin translocation fragment (DTB). The platform has been employed to deliver diverse cargo into cells, including those having low or high molecular weights. A hyper-stable cargo polypeptide has been delivered, as well as proteins of therapeutic significance (e.g, MecP2, SMN, FMRP, PNP, alpha-amylase, and RRSP). The platform is also useful for delivering genome-modifying proteins, such as the CRISPR protein, Cas9. Associated nucleic acids, pharmaceutical compositions, methods, uses, and kits are also described, including those of therapeutic significance aimed at treating diseases or disorders caused by enzyme or protein deficiency.
Public/Granted literature
- US20180080033A1 DELIVERY OF STRUCTURALLY DIVERSE POLYPEPTIDE CARGO INTO MAMMALIAN CELLS BY A BACTERIAL TOXIN Public/Granted day:2018-03-22
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