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1. (WO2017112865) COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF AGENTS
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/2017/112865 International Application No.: PCT/US2016/068300
Publication Date: 29.06.2017 International Filing Date: 22.12.2016
IPC:
C07D 211/14 (2006.01) ,C07D 211/16 (2006.01) ,C07D 295/13 (2006.01) ,C07D 295/185 (2006.01) ,A61K 31/495 (2006.01) ,A61K 31/445 (2006.01) ,A61K 31/45 (2006.01) ,A61K 9/14 (2006.01)
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
211
Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
04
with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
06
having no double bonds between ring members or between ring members and non-ring members
08
with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms
10
with radicals containing only carbon and hydrogen atoms attached to ring carbon atoms
14
with hydrocarbon or substituted hydrocarbon radicals attached to the ring nitrogen atom
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
211
Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
04
with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
06
having no double bonds between ring members or between ring members and non-ring members
08
with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms
10
with radicals containing only carbon and hydrogen atoms attached to ring carbon atoms
16
with acylated ring nitrogen atom
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
295
Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
04
with substituted hydrocarbon radicals attached to ring nitrogen atoms
12
substituted by singly or doubly bound nitrogen atoms
125
with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
13
to an acyclic saturated chain
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
295
Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
16
acylated on ring nitrogen atoms
18
by radicals derived from carboxylic acids, or sulfur or nitrogen analogues thereof
182
Radicals derived from carboxylic acids
185
from aliphatic carboxylic acids
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
33
Heterocyclic compounds
395
having nitrogen as a ring hetero atom, e.g. guanethidine, rifamycins
495
having six-membered rings with two nitrogen atoms as the only ring hetero atoms, e.g. piperazine
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
33
Heterocyclic compounds
395
having nitrogen as a ring hetero atom, e.g. guanethidine, rifamycins
435
having six-membered rings with one nitrogen as the only ring hetero atom
44
Non-condensed pyridines; Hydrogenated derivatives thereof
445
Non-condensed piperidines, e.g. piperocaine
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
33
Heterocyclic compounds
395
having nitrogen as a ring hetero atom, e.g. guanethidine, rifamycins
435
having six-membered rings with one nitrogen as the only ring hetero atom
44
Non-condensed pyridines; Hydrogenated derivatives thereof
445
Non-condensed piperidines, e.g. piperocaine
45
having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9
Medicinal preparations characterised by special physical form
14
Particulate form, e.g. powders
Applicants:
MODERNATX, INC. [US/US]; 200 Technology Square Cambridge, Massachusetts 02139, US
Inventors:
BENENATO, Kerry E.; US
BUTCHER, William; US
Agent:
OUYANG, Lian; US
ERLACHER, Heidi A.; US
ELRIFI, Ivor R.; US
PEMBERTON, Christine C.; US
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
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.
Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)
Also published as:
AU2016377681CA3007297EP3394030US20180369419