PH12014501901A1 - Trialkyl cationic lipids and methods of use thereof - Google Patents
Trialkyl cationic lipids and methods of use thereofInfo
- Publication number
- PH12014501901A1 PH12014501901A1 PH12014501901A PH12014501901A PH12014501901A1 PH 12014501901 A1 PH12014501901 A1 PH 12014501901A1 PH 12014501901 A PH12014501901 A PH 12014501901A PH 12014501901 A PH12014501901 A PH 12014501901A PH 12014501901 A1 PH12014501901 A1 PH 12014501901A1
- Authority
- PH
- Philippines
- Prior art keywords
- des
- compositions
- efficace
- nucl
- une
- Prior art date
Links
- 125000002091 cationic group Chemical group 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 abstract 4
- 238000005538 encapsulation Methods 0.000 abstract 2
- 238000001727 in vivo Methods 0.000 abstract 2
- 102000039446 nucleic acids Human genes 0.000 abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 238000012384 transportation and delivery Methods 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 1
- 239000003814 drug Substances 0.000 abstract 1
- 238000003197 gene knockdown Methods 0.000 abstract 1
- 230000002779 inactivation Effects 0.000 abstract 1
- 239000002245 particle Substances 0.000 abstract 1
- 230000003389 potentiating effect Effects 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 229940124597 therapeutic agent Drugs 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C219/00—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
- C07C219/02—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
- C07C219/20—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being unsaturated
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
- A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
- A61K9/145—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C217/00—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton
- C07C217/02—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
- C07C217/04—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
- C07C217/06—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one etherified hydroxy group and one amino group bound to the carbon skeleton, which is not further substituted
- C07C217/08—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one etherified hydroxy group and one amino group bound to the carbon skeleton, which is not further substituted the oxygen atom of the etherified hydroxy group being further bound to an acyclic carbon atom
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C217/00—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton
- C07C217/02—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
- C07C217/46—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and unsaturated
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C219/00—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
- C07C219/02—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
- C07C219/04—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
- C07C219/06—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having the hydroxy groups esterified by carboxylic acids having the esterifying carboxyl groups bound to hydrogen atoms or to acyclic carbon atoms of an acyclic saturated carbon skeleton
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C229/00—Compounds containing amino and carboxyl groups bound to the same carbon skeleton
- 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
- C07C229/12—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 to carbon atoms of acyclic carbon skeletons
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C271/00—Derivatives of carbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
- C07C271/06—Esters of carbamic acids
- C07C271/08—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms
- C07C271/10—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms
- C07C271/20—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms to carbon atoms of hydrocarbon radicals substituted by nitrogen atoms not being part of nitro or nitroso groups
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C2601/00—Systems containing only non-condensed rings
- C07C2601/02—Systems containing only non-condensed rings with a three-membered ring
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
Landscapes
- Chemical & Material Sciences (AREA)
- Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Communicable Diseases (AREA)
- Oncology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261602990P | 2012-02-24 | 2012-02-24 | |
| PCT/US2013/027469 WO2013126803A1 (en) | 2012-02-24 | 2013-02-22 | Trialkyl cationic lipids and methods of use thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| PH12014501901B1 PH12014501901B1 (en) | 2014-11-24 |
| PH12014501901A1 true PH12014501901A1 (en) | 2014-11-24 |
Family
ID=47884528
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PH12014501901A PH12014501901A1 (en) | 2012-02-24 | 2014-08-22 | Trialkyl cationic lipids and methods of use thereof |
Country Status (27)
| Country | Link |
|---|---|
| US (4) | US9352042B2 (enExample) |
| EP (3) | EP2817287B1 (enExample) |
| JP (4) | JP6275655B2 (enExample) |
| KR (4) | KR102384791B1 (enExample) |
| CN (2) | CN110003030A (enExample) |
| AU (1) | AU2013222179B2 (enExample) |
| BR (1) | BR112014020824B1 (enExample) |
| CA (1) | CA2865412C (enExample) |
| CY (1) | CY1121232T1 (enExample) |
| DK (1) | DK2817287T3 (enExample) |
| ES (2) | ES2702874T3 (enExample) |
| HR (1) | HRP20182156T1 (enExample) |
| HU (1) | HUE041494T2 (enExample) |
| IL (3) | IL234266B (enExample) |
| LT (1) | LT2817287T (enExample) |
| MX (3) | MX356904B (enExample) |
| NZ (1) | NZ700075A (enExample) |
| PH (1) | PH12014501901A1 (enExample) |
| PL (1) | PL2817287T3 (enExample) |
| PT (1) | PT2817287T (enExample) |
| RS (1) | RS58077B1 (enExample) |
| RU (2) | RU2718053C2 (enExample) |
| SG (1) | SG11201405157PA (enExample) |
| SI (1) | SI2817287T1 (enExample) |
| UA (1) | UA120026C2 (enExample) |
| WO (1) | WO2013126803A1 (enExample) |
| ZA (1) | ZA201406896B (enExample) |
Families Citing this family (232)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NZ700688A (en) | 2009-12-01 | 2016-02-26 | Shire Human Genetic Therapies | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
| US8853377B2 (en) | 2010-11-30 | 2014-10-07 | Shire Human Genetic Therapies, Inc. | mRNA for use in treatment of human genetic diseases |
| CN103906527B (zh) | 2011-06-08 | 2020-07-10 | 川斯勒佰尔公司 | Mrna递送的脂质纳米颗粒组合物和方法 |
| EP3336082B1 (en) | 2011-06-08 | 2020-04-15 | Translate Bio, Inc. | Cleavable lipids |
| US9352042B2 (en) | 2012-02-24 | 2016-05-31 | Protiva Biotherapeutics, Inc. | Trialkyl cationic lipids and methods of use thereof |
| BR112014024131A2 (pt) | 2012-03-29 | 2017-07-25 | Shire Human Genetic Therapies | lipídios catiônicos ionizáveis |
| JP6211054B2 (ja) | 2012-03-29 | 2017-10-11 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | 脂質誘導される中性ナノ粒子 |
| EP3884949A1 (en) | 2012-06-08 | 2021-09-29 | Translate Bio, Inc. | Pulmonary delivery of mrna to non-lung target cells |
| US20150267192A1 (en) | 2012-06-08 | 2015-09-24 | Shire Human Genetic Therapies, Inc. | Nuclease resistant polynucleotides and uses thereof |
| EP2971010B1 (en) | 2013-03-14 | 2020-06-10 | ModernaTX, Inc. | Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions |
| ES2708561T3 (es) | 2013-03-14 | 2019-04-10 | Translate Bio Inc | Métodos para la purificación de ARN mensajero |
| BR112015022855A2 (pt) | 2013-03-14 | 2017-11-07 | Shire Human Genetic Therapies | composições e método para produção de um anticorpo in vitro |
| EP2968586B1 (en) | 2013-03-14 | 2018-07-25 | Translate Bio, Inc. | Cftr mrna compositions and related methods and uses |
| RS57316B1 (sr) | 2013-03-15 | 2018-08-31 | Translate Bio Inc | Sinergističko poboljšanje isporuke nukleinskih kiselina pomoću mešavina formulacija |
| CN105555757A (zh) * | 2013-07-23 | 2016-05-04 | 普洛体维生物治疗公司 | 用于递送信使rna的组合物和方法 |
| EP3041938A1 (en) | 2013-09-03 | 2016-07-13 | Moderna Therapeutics, Inc. | Circular polynucleotides |
| WO2015051214A1 (en) | 2013-10-03 | 2015-04-09 | Moderna Therapeutics, Inc. | Polynucleotides encoding low density lipoprotein receptor |
| EP3060258A1 (en) | 2013-10-22 | 2016-08-31 | Shire Human Genetic Therapies, Inc. | Mrna therapy for phenylketonuria |
| EP3060671B1 (en) | 2013-10-22 | 2021-12-29 | Translate Bio, Inc. | Cns delivery of mrna and uses thereof |
| KR101988552B1 (ko) | 2013-10-22 | 2019-09-25 | 샤이어 휴먼 지네틱 테라피즈 인크. | 메신저 rna의 전달을 위한 지질 제형 |
| EP4276176A3 (en) | 2013-10-22 | 2024-01-10 | Translate Bio, Inc. | Mrna therapy for argininosuccinate synthetase deficiency |
| CA2944800A1 (en) | 2014-04-25 | 2015-10-29 | Shire Human Genetic Therapies, Inc. | Methods for purification of messenger rna |
| EP3148552B1 (en) | 2014-05-30 | 2019-07-31 | Translate Bio, Inc. | Biodegradable lipids for delivery of nucleic acids |
| KR102511554B1 (ko) | 2014-06-24 | 2023-03-16 | 샤이어 휴먼 지네틱 테라피즈 인크. | 핵산의 전달용 입체화학적으로 풍부한 조성물 |
| LT3766916T (lt) | 2014-06-25 | 2023-01-10 | Acuitas Therapeutics Inc. | Nauji lipidai ir lipidų nanodalelių kompozicijos, skirtos nukleorūgščių tiekimui |
| CA2953265C (en) | 2014-07-02 | 2023-09-26 | Shire Human Genetic Therapies, Inc. | Encapsulation of messenger rna |
| CA2955250A1 (en) | 2014-07-16 | 2016-01-21 | Moderna Therapeutics, Inc. | Chimeric polynucleotides |
| US20170210788A1 (en) | 2014-07-23 | 2017-07-27 | Modernatx, Inc. | Modified polynucleotides for the production of intrabodies |
| KR101882634B1 (ko) | 2014-08-07 | 2018-07-26 | 다케다 야쿠힝 고교 가부시키가이샤 | 양이온성 지질 |
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| US9943595B2 (en) | 2014-12-05 | 2018-04-17 | Translate Bio, Inc. | Messenger RNA therapy for treatment of articular disease |
| ES2797085T3 (es) * | 2015-03-11 | 2020-12-01 | Inst Nat Sante Rech Med | Métodos y composiciones farmacéuticas para el tratamiento de infecciones por filovirus |
| JP6895892B2 (ja) | 2015-03-19 | 2021-06-30 | トランスレイト バイオ, インコーポレイテッド | ポンペ病のmRNA治療 |
| JP2018526321A (ja) | 2015-04-27 | 2018-09-13 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 適応免疫応答を誘導するためのヌクレオシド修飾rna |
| EP4420679A3 (en) * | 2015-06-29 | 2024-12-04 | Acuitas Therapeutics Inc. | Lipids and lipid nanoparticle formulations for delivery of nucleic acids |
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| AU2016338559B2 (en) | 2015-10-14 | 2022-11-24 | Translate Bio, Inc. | Modification of RNA-related enzymes for enhanced production |
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| PT3394093T (pt) | 2015-12-23 | 2022-05-30 | Modernatx Inc | Métodos de utilização de polinucleotídeos que codificam ligandos ox40 |
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| CN109072223B (zh) | 2016-04-08 | 2022-10-21 | 川斯勒佰尔公司 | 多聚体编码核酸及其用途 |
| MA45052A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant pour jagged1 pour le traitement du syndrome d'alagille |
| CN109312313A (zh) | 2016-06-13 | 2019-02-05 | 川斯勒佰尔公司 | 用于治疗鸟氨酸转氨甲酰酶缺乏症的信使rna疗法 |
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