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1. WO2020223323 - COMPOSITIONALLY DEFINED PLASMID DNA/POLYCATION NANOPARTICLES AND METHODS FOR MAKING THE SAME

Publication Number WO/2020/223323
Publication Date 05.11.2020
International Application No. PCT/US2020/030429
International Filing Date 29.04.2020
IPC
A61K 47/61 2017.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51the non-active ingredient being a modifying agent
56the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
61the organic macromolecular compound being a polysaccharide or a derivative thereof
A61K 47/64 2017.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51the non-active ingredient being a modifying agent
62the modifying agent being a protein, peptide or polyamino acid
64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
A61K 9/51 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
48Preparations in capsules, e.g. of gelatin, of chocolate
50Microcapsules
51Nanocapsules
CPC
A61K 47/59
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51the non-active ingredient being a modifying agent
56the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
59obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
A61K 47/6929
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
69the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
6921the form being a particulate, a powder, an adsorbate, a bead or a sphere
6927the form being a solid microparticle having no hollow or gas-filled cores
6929the form being a nanoparticle, e.g. an immuno-nanoparticle
A61K 48/0091
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
0091Purification or manufacturing processes for gene therapy compositions
A61K 9/5146
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
48Preparations in capsules, e.g. of gelatin, of chocolate
50Microcapsules ; having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
51Nanocapsules; Nanoparticles;
5107Excipients; Inactive ingredients
513Organic macromolecular compounds; Dendrimers
5146obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
C12N 15/88
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
88using microencapsulation, e.g. using ; amphiphile; liposome vesicle
Applicants
  • THE JOHNS HOPKINS UNIVERSITY [US]/[US]
Inventors
  • MAO, Hai-Quan
  • HU, Yizong
  • POMPER, Martin Gilbert
  • LIU, Heng-wen
  • MINN, Il
  • ULLMAN, Christopher
  • CARRINGTON, Christine
Agents
  • CHILDERS, Jeffrey W.
Priority Data
62/840,15229.04.2019US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONALLY DEFINED PLASMID DNA/POLYCATION NANOPARTICLES AND METHODS FOR MAKING THE SAME
(FR) NANOPARTICULES D'ADN PLASMIDIQUE/POLYCATIONS DE CONSTITUTION DÉFINIE ET LEURS PROCÉDÉS DE PRÉPARATION
Abstract
(EN) The presently disclosed subject matter provides a kinetically controlled mixing process, referred to herein as "flash nanocomplexation" or "(FNC)," to accelerate the mixing of a polyanion solution, for example, a plasmid DNA solution, with a polycation solution to match the polyelectrolyte complex (PEC) assembly kinetics through turbulent mixing in a microchamber, thus achieving explicit control of the kinetic conditions for nanoparticle assembly as demonstrated by the tunability of nanoparticle size, composition, hydrodynamic size, hydrodynamic density, surface charge, and polyanion payload.
(FR) La présente invention concerne un processus de mélange à cinétique régulée, désigné dans la description de l'invention par « nanocomplexation flash » ou « (FNC) », pour accélérer le mélange d'une solution de polyanions, par exemple, une solution d'ADN plasmidique, avec une solution de polycations pour correspondre à la cinétique de l'ensemble complexe polyélectrolytique (PEC) par l'intermédiaire d'un mélange turbulent dans une microchambre, ce qui permet d'obtenir une régulation explicite des conditions cinétiques pour l'ensemble de nanoparticules comme démontré par la capacité d'ajustement de la taille des nanoparticules, de la composition, de la taille hydrodynamique, de la densité hydrodynamique, de la charge de surface et de la charge utile des polyanions.
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