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1. WO2017112865 - COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF AGENTS

Publication Number WO/2017/112865
Publication Date 29.06.2017
International Application No. PCT/US2016/068300
International Filing Date 22.12.2016
IPC
C07D 211/14 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
211Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
04with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
06having no double bonds between ring members or between ring members and non-ring members
08with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms
10with radicals containing only carbon and hydrogen atoms attached to ring carbon atoms
14with hydrocarbon or substituted hydrocarbon radicals attached to the ring nitrogen atom
C07D 211/16 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
211Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
04with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
06having no double bonds between ring members or between ring members and non-ring members
08with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms
10with radicals containing only carbon and hydrogen atoms attached to ring carbon atoms
16with acylated ring nitrogen atom
C07D 295/13 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
295Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
04with substituted hydrocarbon radicals attached to ring nitrogen atoms
12substituted by singly or doubly bound nitrogen atoms
125with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
13to an acyclic saturated chain
C07D 295/185 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
295Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
16acylated on ring nitrogen atoms
18by radicals derived from carboxylic acids, or sulfur or nitrogen analogues thereof
182Radicals derived from carboxylic acids
185from aliphatic carboxylic acids
A61K 31/495 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
495having six-membered rings with two nitrogen atoms as the only ring hetero atoms, e.g. piperazine
A61K 31/445 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
44Non-condensed pyridines; Hydrogenated derivatives thereof
445Non-condensed piperidines, e.g. piperocaine
CPC
A61K 38/1808
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
18Growth factors; Growth regulators
1808Epidermal growth factor [EGF] urogastrone
A61K 38/1816
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
18Growth factors; Growth regulators
1816Erythropoietin [EPO]
A61K 47/22
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
06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
22Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
A61K 48/0058
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
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
A61K 9/127
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
10Dispersions; Emulsions
127Liposomes
A61K 9/5123
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
5123Organic compounds, e.g. fats, sugars
Applicants
  • MODERNATX, INC. [US]/[US]
Inventors
  • BENENATO, Kerry E.
  • BUTCHER, William
Agents
  • OUYANG, Lian
  • ERLACHER, Heidi A.
  • ELRIFI, Ivor R.
  • PEMBERTON, Christine C.
Priority Data
62/271,13722.12.2015US
62/271,14622.12.2015US
62/271,16022.12.2015US
62/271,17922.12.2015US
62/271,20022.12.2015US
62/338,47418.05.2016US
62/413,34526.10.2016US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF AGENTS
(FR) COMPOSÉS ET COMPOSITIONS POUR L'ADMINISTRATION INTRACELLULAIRE D'AGENTS THÉRAPEUTIQUES ET/OU PROPHYLACTIQUES
Abstract
(EN) The disclosure features amino lipids and compositions involving the same. Nanoparticle compositions include an amino lipid as well as additional lipids such as phospholipids, structural lipids, PEG lipids, or a combination thereof. Nanoparticle compositions further including therapeutic and/or prophylactic agents such as RNA are useful in the delivery of therapeutic and/or prophylactic agents to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
(FR) La présente divulgation concerne des lipides aminés et des compositions les impliquant. Les compositions nanoparticulaires comprennent un lipide aminé ainsi que d'autres lipides tels que des phospholipides, des lipides structuraux, des lipides PEG, ou une combinaison de ceux-ci. Des compositions nanoparticulaires comprenant en outre des agents thérapeutiques et/ou prophylactiques tels que l'ARN qui sont utiles dans l'administration d'agents thérapeutiques et/ou prophylactiques à des cellules ou des organes de mammifères, par exemple, pour réguler l'expression de polypeptides, de protéines, ou de gènes sont en outre décrites.
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