WO2017049245A3 - Compounds and compositions for intracellular delivery of therapeutic agents - Google Patents

Compounds and compositions for intracellular delivery of therapeutic agents Download PDF

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Publication number
WO2017049245A3
WO2017049245A3 PCT/US2016/052352 US2016052352W WO2017049245A3 WO 2017049245 A3 WO2017049245 A3 WO 2017049245A3 US 2016052352 W US2016052352 W US 2016052352W WO 2017049245 A3 WO2017049245 A3 WO 2017049245A3
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WO
WIPO (PCT)
Prior art keywords
lipids
compositions
compounds
therapeutic agents
intracellular delivery
Prior art date
Application number
PCT/US2016/052352
Other languages
French (fr)
Other versions
WO2017049245A2 (en
Inventor
Kerry E. BENENATO
Ellalahewage Sathyajith Kumarasinghe
Mark Cornebise
Original Assignee
Modernatx, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority to EP20180006.7A priority Critical patent/EP3736261B1/en
Priority to ES16781895T priority patent/ES2910425T3/en
Priority to EP16781895.4A priority patent/EP3350157B1/en
Priority to SI201631463T priority patent/SI3350157T1/en
Priority to EP23202539.5A priority patent/EP4286012A2/en
Priority to JP2018514267A priority patent/JP6948313B6/en
Priority to LTEPPCT/US2016/052352T priority patent/LT3350157T/en
Priority to HRP20220156TT priority patent/HRP20220156T1/en
Application filed by Modernatx, Inc. filed Critical Modernatx, Inc.
Priority to DK16781895.4T priority patent/DK3350157T3/en
Priority to RS20220262A priority patent/RS63030B1/en
Priority to AU2016324310A priority patent/AU2016324310B2/en
Priority to PL16781895T priority patent/PL3350157T3/en
Priority to CA2998810A priority patent/CA2998810A1/en
Publication of WO2017049245A2 publication Critical patent/WO2017049245A2/en
Priority to US15/476,253 priority patent/US9868691B2/en
Priority to US15/476,263 priority patent/US9868692B2/en
Priority to US15/493,281 priority patent/US9867888B2/en
Priority to US15/498,698 priority patent/US9868693B2/en
Publication of WO2017049245A3 publication Critical patent/WO2017049245A3/en
Priority to US15/846,084 priority patent/US10442756B2/en
Priority to US16/005,286 priority patent/US10266485B2/en
Priority to US16/148,822 priority patent/US10392341B2/en
Priority to US16/555,045 priority patent/US11220476B2/en
Priority to AU2021204763A priority patent/AU2021204763B2/en
Priority to JP2021151758A priority patent/JP7326395B2/en
Priority to US17/538,217 priority patent/US20220380299A1/en
Priority to CY20221100215T priority patent/CY1125154T1/en
Priority to JP2023125995A priority patent/JP2023164798A/en
Priority to US18/467,190 priority patent/US20240124388A1/en

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    • C07C229/02—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/06—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
    • C07C229/10—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings
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Abstract

The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include a novel lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
PCT/US2016/052352 2015-09-17 2016-09-16 Compounds and compositions for intracellular delivery of therapeutic agents WO2017049245A2 (en)

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LTEPPCT/US2016/052352T LT3350157T (en) 2015-09-17 2016-09-16 Compounds and compositions for intracellular delivery of therapeutic agents
EP16781895.4A EP3350157B1 (en) 2015-09-17 2016-09-16 Compounds and compositions for intracellular delivery of therapeutic agents
SI201631463T SI3350157T1 (en) 2015-09-17 2016-09-16 Compounds and compositions for intracellular delivery of therapeutic agents
EP23202539.5A EP4286012A2 (en) 2015-09-17 2016-09-16 Compounds and compositions for intracellular delivery of therapeutic agents
JP2018514267A JP6948313B6 (en) 2015-09-17 2016-09-16 Compounds and compositions for intracellular delivery of therapeutic agents
EP20180006.7A EP3736261B1 (en) 2015-09-17 2016-09-16 Compounds and compositions for intracellular delivery of therapeutic agents
HRP20220156TT HRP20220156T1 (en) 2015-09-17 2016-09-16 Compounds and compositions for intracellular delivery of therapeutic agents
ES16781895T ES2910425T3 (en) 2015-09-17 2016-09-16 Compounds and compositions for the intracellular delivery of therapeutic agents
DK16781895.4T DK3350157T3 (en) 2015-09-17 2016-09-16 COMPOUNDS AND COMPOSITIONS FOR INTRACELLUAL DELIVERY OF THERAPEUTIC AGENTS
RS20220262A RS63030B1 (en) 2015-09-17 2016-09-16 Compounds and compositions for intracellular delivery of therapeutic agents
AU2016324310A AU2016324310B2 (en) 2015-09-17 2016-09-16 Compounds and compositions for intracellular delivery of therapeutic agents
PL16781895T PL3350157T3 (en) 2015-09-17 2016-09-16 Compounds and compositions for intracellular delivery of therapeutic agents
CA2998810A CA2998810A1 (en) 2015-09-17 2016-09-16 Compounds and compositions for intracellular delivery of therapeutic agents
US15/476,253 US9868691B2 (en) 2015-09-17 2017-03-31 Compounds and compositions for intracellular delivery of therapeutic agents
US15/476,263 US9868692B2 (en) 2015-09-17 2017-03-31 Compounds and compositions for intracellular delivery of therapeutic agents
US15/493,281 US9867888B2 (en) 2015-09-17 2017-04-21 Compounds and compositions for intracellular delivery of therapeutic agents
US15/498,698 US9868693B2 (en) 2015-09-17 2017-04-27 Compounds and compositions for intracellular delivery of therapeutic agents
US15/846,084 US10442756B2 (en) 2015-09-17 2017-12-18 Compounds and compositions for intracellular delivery of therapeutic agents
US16/005,286 US10266485B2 (en) 2015-09-17 2018-06-11 Compounds and compositions for intracellular delivery of therapeutic agents
US16/148,822 US10392341B2 (en) 2015-09-17 2018-10-01 Compounds and compositions for intracellular delivery of therapeutic agents
US16/555,045 US11220476B2 (en) 2015-09-17 2019-08-29 Compounds and compositions for intracellular delivery of therapeutic agents
AU2021204763A AU2021204763B2 (en) 2015-09-17 2021-07-07 Compounds and compositions for intracellular delivery of therapeutic agents
JP2021151758A JP7326395B2 (en) 2015-09-17 2021-09-17 Compounds and compositions for intracellular delivery of therapeutic agents
US17/538,217 US20220380299A1 (en) 2015-09-17 2021-11-30 Compounds and compositions for intracellular delivery of therapeutic agents
CY20221100215T CY1125154T1 (en) 2015-09-17 2022-03-15 COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF THERAPEUTIC AGENTS
JP2023125995A JP2023164798A (en) 2015-09-17 2023-08-02 Compounds and compositions for intracellular delivery of therapeutic agents
US18/467,190 US20240124388A1 (en) 2015-09-17 2023-09-14 Compounds and compositions for intracellular delivery of therapeutic agents

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US201562252316P 2015-11-06 2015-11-06
US62/252,316 2015-11-06
US201562253433P 2015-11-10 2015-11-10
US62/253,433 2015-11-10
US201562266460P 2015-12-11 2015-12-11
US62/266,460 2015-12-11
US201662333557P 2016-05-09 2016-05-09
US62/333,557 2016-05-09
US201662382740P 2016-09-01 2016-09-01
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