US010266485B2 (12 ) United States Patent (10 ) Patent No. : US 10 , 266 , 485 B2 Benenato ( 45 ) Date of Patent: * Apr. 23, 2019 ( 54 ) COMPOUNDS AND COMPOSITIONS FOR ( 2013. 01 ) ; C07C 227/ 16 (2013 . 01 ) ; C07C INTRACELLULAR DELIVERY OF 227/ 18 ( 2013 .01 ) ; C07C 229 / 16 (2013 . 01 ) ; THERAPEUTIC AGENTS C07C 233 / 36 ( 2013 .01 ) ; C07C 235 / 10 (2013 .01 ) ; C07C 255 /24 ( 2013 . 01 ) ; C07C (71 ) Applicant: Moderna TX , Inc ., Cambridge , MA 271/ 20 ( 2013 . 01 ) ; C07C 275 / 14 ( 2013 . 01 ) ; (US ) C07C 279 /12 ( 2013 .01 ) ; C07C 279 / 24 ( 2013 . 01 ) ; C07C 279 /28 ( 2013 .01 ) ; C07C ( 72 ) Inventor: Kerry E . Benenato , Sudbury , MA (US ) 279 / 32 ( 2013 . 01 ) ; C07C 311 / 05 ( 2013 . 01 ) ; C07C 335 / 08 (2013 . 01 ) ; C070 207 / 27 (73 ) Assignee : ModernaTX , Inc. , Cambridge, MA ( 2013 .01 ) ; CO7D 233 / 72 ( 2013 . 01 ) ; C07D (US ) 249 /04 (2013 .01 ) ; C07D 263 / 20 ( 2013 .01 ) ; C07D 265 / 33 ( 2013 .01 ) ; C07D 271 / 06 ( * ) Notice : Subject to any disclaimer, the term of this ( 2013 .01 ) ; C07D 271/ 10 (2013 .01 ) ; C07D patent is extended or adjusted under 35 277 / 38 ( 2013 . 01 ) ; C07F 9 / 091 ( 2013 .01 ) ; U . S . C . 154 ( b ) by 0 days . CO7K 14 /505 ( 2013. 01 ) ; A61K 9 /1271 This patent is subject to a terminal dis (2013 .01 ) ; A61K 48 / 00 ( 2013 .01 ) ; C07C claimer . 2601/ 02 (2017 .05 ) ; C07C 2601 / 04 ( 2017 . 05 ) ; C07C 2601/ 14 ( 2017 .05 ) ; C07C 2601 / 18 (21 ) Appl . No. : 16 / 005 , 286 (2017 . 05 ) (58 ) Field of Classification Search (22 ) Filed : Jun . 11 , 2018 CPC .. A61K 39 /00 ; A61K 51 /08 ; CO7K 14 /81 ; C07H 21 /00 (65 ) Prior Publication Data USPC .. .. 424 / 9 . 1 ; 435 /91 . 1 , 91. 31 , 458 ; 514 /44 ; US 2018 / 0273467 A1 Sep . 27, 2018 536 /23 . 1 , 24 . 5 See application file for complete search history . Related U . S . Application Data (63 ) Continuation of application No. 15 /846 , 084, filed on ( 56 ) References Cited Dec . 18 , 2017 , which is a continuation of application U . S . PATENT DOCUMENTS (Continued ) 3 , 872 , 171 A 3 / 1975 Cronin et al. (51 ) Int . Ci. 4 , 957 , 735 A 9 / 1990 Huang A6IK 48 /00 ( 2006 .01 ) (Continued ) A61K 9 / 127 ( 2006 . 01 ) C07C 229 / 12 ( 2006 . 01 ) FOREIGN PATENT DOCUMENTS C07C 271/ 20 ( 2006 .01 ) C07C 279 / 12 ( 2006 .01 ) AU 652831 B2 9 / 1994 C07C 279 / 28 ( 2006 .01 ) CN 102068701 A 5 / 2011 C07C 279 /32 ( 2006 .01 ) (Continued ) C07D 233 / 72 ( 2006 .01 ) C07D 249 /04 ( 2006 .01 ) OTHER PUBLICATIONS CO7D 263/ 20 ( 2006 .01 ) CO7D 265 /33 ( 2006 .01 ) U . S . Appl. No . 15 / 846 ,084 . * C07D 271 /06 ( 2006 . 01 ) (Continued ) C07D 271 / 10 ( 2006 .01 ) CO7D 207/ 27 ( 2006 .01 ) Primary Examiner — Jane J Zara C07D 277 / 38 ( 2006 .01 ) (74 ) Attorney, Agent, or Firm — Cooley LLP ; Heidi A . C07C 275 / 14 ( 2006 . 01 ) Erlacher ; Christine C . Pemberton C07C 279 /24 ( 2006 . 01 ) CO7C 311/ 05 ( 2006 .01 ) C07C 229 / 16 (2006 .01 ) (57 ) ABSTRACT (Continued ) The disclosure features novel lipids and compositions (52 ) U .S . CI. involving the same. Nanoparticle compositions include a CPC .. .. .. C07C 229 / 12 ( 2013 .01 ) ; A61K 9 / 0019 novel lipid as well as additional lipids such as phospholipids , ( 2013 .01 ) ; A61K 9 / 0043 ( 2013 . 01 ) ; A6IK structural lipids, and PEG lipids . 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