IP Library Patent Application 17863885
Patent Application
App. No. 17/863,885

LIPID CONTAINING FORMULATIONS

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Quick Facts
Patent No.
US None
App. No.
17/863,885
Abstract

Compositions and methods useful in administering nucleic acid based therapies, for example association complexes such as liposomes and lipoplexes are described.

Claims (56)

1 . A preparation comprising one or more compounds, each individually having a structure defined by formula (I) or a pharmaceutically acceptable salt thereof,

wherein

each X a and X b , for each occurrence, is independently C 1-6 alkylene;

n is 0, 1, 2, 3, 4, or 5; each R is independently H,

wherein at least n+2 of the R moieties in at least about 80% of the molecules of the compound of formula (I) in the preparation are not H;

m is 1, 2, 3 or 4; Y is O, NR 2 , or S;

R 1 is alkyl alkenyl or alkynyl; each of which is optionally substituted with one or more substituents; and

R 2 is H, alkyl alkenyl or alkynyl; each of which is optionally substituted each of which is optionally substituted with one or more substituents;

provided that, if n=0, then at least n+3 of the R moieties are not H.

2 - 138 . (canceled)

139 . A compound of formula (X),

wherein

R 1 and R 2 are each independently H, C 1 -C 6 alky], optionally substituted with 1-4 R 5 , C 2 -C 6 alkenyl, optionally substituted with 1-4 R 5 , or C(NR 6 )(NR 6 ) 2 ;

R 3 and R 4 are each independently alkyl, alkenyl, alkynly, each of which is optionally substituted with fluoro, chloro, bromo, or iodo;

L 1 and L 2 are each independently —NR 6 C(O)—, —C(O)NR 6 —, —OC(O)—, —C(O)O—, —S—S—, —N(R 6 )C(O)N(R 6 )—, —OC(O)N(R 6 )—, —N(R 6 )C(O)O—, —O—N═C—, OR, —OC(O)NH—N═C—, or —NHC(O)NH—N═C—,

L 1 -R 3 and L 2 -R 4 can be taken together to form an acetal, a ketal, or an orthoester, wherein

R 3 and R 4 are defined as above and can also be H or phenyl;

R 5 is fluoro, chloro, bromo, iodo, —OR 7 , —N(R 8 )(R 9 ), —CN, SR 10 , S(O)R 10 , S(O) 2 R 10

R 6 is H, C 1 -C 6 alkyl,

R 7 is H or C 1 -C 6 alkyl;

each R 8 and R 9 are independently H or C 1 -C 6 alkyl;

R 10 is H or C 1 -C 6 alkyl;

m is 1, 2, 3, 4, 5, or 6;

n is 0, 1, 2, 3, 4, 5, or 6;

and pharmaceutically acceptable salts thereof.

140 - 324 . (canceled)

325 . A compound of formula (XV)

wherein;

each L 1 and L 2 are independently a bond or C(O);

each R 1 and R 2 are independently alkyl alkenyl or alkynyl; each of which is optionally substituted with one or more substituents;

X is —C(O)NH—, —C(S)NH—, —C(O)C 1-3 alkylC(O)NH—; or —C(O)C 1-3 alkylC(O)O—; m is an integer from 0-11 and

n is an integer from 1-500.

326 . The compound of claim 325 , wherein L 1 and L 2 are both a bond.

327 . The compound of claim 325 , wherein L 1 and L 2 are both C(O).

328 . The compound of claim 325 , wherein each R 1 and R 2 are independently alkyl.

329 . The compound of claim 328 , wherein each R 1 and R 2 are independently C 6 -C 28 alkyl.

330 . The compound of claim 325 , wherein both R 1 and R 2 are straight chain alkyl having the same length.

331 . The compound of claim 330 , wherein both R 1 and R 2 are C 14 alkyl.

332 - 334 . (canceled)

335 . The compound of claim 325 , wherein each R 1 and R 2 are independently alkenyl.

336 . (canceled)

337 . The compound of claim 335 , wherein each R 1 and R 2 includes two double bond moieties.

338 . The compound of claim 325 , wherein X is —C(O)NH—, providing a compound of formula (XV′) below:

339 - 341 . (canceled)

342 . The compound of claim 325 , wherein the compound is a compound of formula (XV),

wherein both L 1 and L 2 are a bond.

343 . The compound of claim 342 , wherein each R 1 and R 2 are independently alkyl.

344 . The compound of claim 343 , wherein, both R 1 and R 2 are straight chain alkyl having the same length.

345 - 346 . (canceled)

347 . The compound of claim 342 , wherein, the compound is a compound of formula (XV), wherein L 1 and L 2 are both bonds, R 1 and R 2 are both alkyl, and n is an integer from about 40-400.

348 . The compound of claim 325 , wherein, the compound has a formula (XVI) below:

formula (XVI), wherein the repeating PEG moiety has an average molecular weight of 2000 with n value between 42 and 47.

349 . (canceled)

350 . A lipid nanoparticle comprising a compound of claim 325 .

351 . The lipid nanoparticle of claim 350 , which further comprises a therapeutic agent.

352 . A method for treating a patient having a disease or disorder comprising administering a therapeutically effective amount of the lipid nanoparticle of claim 351 to the patient.

Assignments (4)
MERGER Recorded Sep 15, 2022
From: PROTIVA BIOTHERAPEUTICS, INC.
To: ARBUTUS BIOPHARMA CORPORATION
Reel/Frame 061434/0749 →
CHANGE OF NAME Recorded Sep 12, 2022
From: TEKMIRA PHARMACEUTICALS CORPORATION
To: ARBUTUS BIOPHARMA CORPORATION
Reel/Frame 061063/0970 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 1, 2022
From: ALNYLAM PHARMACEUTICALS, INC.
To: TEKMIRA PHARMACEUTICALS CORPORATION
Reel/Frame 060964/0421 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 9, 2022
From: MANOHARAN, MUTHIAH; RAJEEV, KALLANTHOTTATHIL G.; AKINC, AKIN; NAIR, JAYAPRAKASH K.; JAYARAMAN, MUTHUSAMY; MAIER, MARTIN
To: ALNYLAM PHARMACEUTICALS, INC.
Reel/Frame 060760/0007 →