WIPO logo
Mobile | Deutsch | Español | Français | 日本語 | 한국어 | Português | Русский | 中文 | العربية |
PATENTSCOPE

Search International and National Patent Collections
World Intellectual Property Organization
Options
Query Language
Stem
Sort by:
List Length
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.: WO/2018/165257 International Application No.: PCT/US2018/021292
Publication Date: 13.09.2018 International Filing Date: 07.03.2018
IPC:
C07C 305/06 (2006.01) ,A61K 47/20 (2006.01) ,C07C 317/18 (2006.01) ,C08G 65/00 (2006.01) ,A61K 47/54 (2017.01) ,A61K 9/127 (2006.01)
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
C
ACYCLIC OR CARBOCYCLIC COMPOUNDS
305
Esters of sulfuric acids
02
having oxygen atoms of sulfate groups bound to acyclic carbon atoms of a carbon skeleton
04
being acyclic and saturated
06
Hydrogenosulfates
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47
Medicinal preparations characterised by the non-active ingredients used, e.g. carriers, inert additives
06
Organic compounds
20
containing sulfur
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
C
ACYCLIC OR CARBOCYCLIC COMPOUNDS
317
Sulfones; Sulfoxides
16
having sulfone or sulfoxide groups and singly-bound oxygen atoms bound to the same carbon skeleton
18
with sulfone or sulfoxide groups bound to acyclic carbon atoms of the carbon skeleton
C CHEMISTRY; METALLURGY
08
ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
G
MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
65
Macromolecular compounds obtained by reactions forming an ether link in the main chain of the macromolecule
[IPC code unknown for A61K 47/54]
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
10
Dispersions; Emulsions
127
Liposomes
Applicants: TRANSLATE BIO, INC.[US/US]; 200 Sidney Street Cambridge, MA 02139, US
Inventors: HEARTLEIN, Michael; US
DEROSA, Frank; US
KARVE, Shrirang; US
Agent: ESPINO, Christine, G.; US
MORIN, Randall; US
SHALEK, James; US
COUSINS, Abigail; US
BAUER, Steven; US
Priority Data:
62/468,24407.03.2017US
Title (EN) POLYANIONIC DELIVERY OF NUCLEIC ACIDS
(FR) ADMINISTRATION POLYANIONIQUE D'ACIDES NUCLÉIQUES
Abstract:
(EN) Disclosed are dendritic anionic lipids which are compounds of Formula (I) : wherein R and R1 are non-polar groups, L is a linking moiety, and Dm is a dendritic moiety of m generations, each as defined herein. These dendritic anionic lipids are useful for delivery and expression of m RNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.
(FR) L'invention concerne des lipides anioniques dendritiques qui sont des composés de formule (I) : dans laquelle R et R1 sont des groupes non polaires, L représente une fraction de liaison, et Dm représente une fraction dendritique de m générations, chacune est telle que définie dans la description. Les lipides anioniques dendritiques de la présente invention sont utiles pour l'administration et l'expression d'ARNm et de protéine codée, par exemple, en tant que composant d'un véhicule d'administration liposomal, et peuvent par conséquent être utiles pour traiter diverses maladies, troubles et états pathologiques, tels que ceux associés à une déficience d'une ou de plusieurs protéines.
front page image
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, JO, 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 Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (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)