Title:
MUSCLE CELL-TARGETING NANOPARTICLES FOR VACCINATION AND NUCLEIC ACID DELIVERY, AND METHODS OF PRODUCTION AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2015/116568
Kind Code:
A3
Abstract:
Described herein are conjugates (e.g., nanoparticles), compositions (e.g., nanoparticle-based compositions), kits, methods and platforms for muscle-specific targeting of vaccines and gene therapies. The nanoparticles are composed of charged polymers or charged particles (e.g., polyamidoamide dendrimers) surface-modified with a muscle cell ligand (a targeting moiety) and an anionic enhancer (e.g. a phospholipid) resulting in a multinucleotide-carrying conjugate for introducing vaccines or genes into skeletal muscle cells of a subject (e.g., human and other mammals). The novel compositions described herein find use in muscle-targeted vaccination and gene therapy delivery (e.g., delivery of gene-based vaccines) for the prevention and treatment of disease (e.g., skeletal muscle atrophy, cancer, autoimmune disease, infection).
Inventors:
DAFTARIAN PIROUZ (US)
DAUNERT SYLVIA (US)
DEO SAPNA (US)
DIKICI EMRE (US)
KAIFER ANGEL (US)
JATIVA SAMUEL (US)
DAUNERT SYLVIA (US)
DEO SAPNA (US)
DIKICI EMRE (US)
KAIFER ANGEL (US)
JATIVA SAMUEL (US)
Application Number:
PCT/US2015/013024
Publication Date:
November 12, 2015
Filing Date:
January 27, 2015
Export Citation:
Assignee:
UNIV MIAMI (US)
International Classes:
A61K9/14; A61K47/48
Domestic Patent References:
| WO2007062000A2 | 2007-05-31 |
Foreign References:
| US20120093761A1 | 2012-04-19 | |||
| US20120283410A1 | 2012-11-08 | |||
| US20100297007A1 | 2010-11-25 |
Attorney, Agent or Firm:
DOBBELAERE, Amy A. (525 Okeechobee Blvd. 15th FloorWest Palm Beach, Florida, US)
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