MA45052A - Polynucléotides codant pour jagged1 pour le traitement du syndrome d'alagille - Google Patents
Polynucléotides codant pour jagged1 pour le traitement du syndrome d'alagilleInfo
- Publication number
- MA45052A MA45052A MA045052A MA45052A MA45052A MA 45052 A MA45052 A MA 45052A MA 045052 A MA045052 A MA 045052A MA 45052 A MA45052 A MA 45052A MA 45052 A MA45052 A MA 45052A
- Authority
- MA
- Morocco
- Prior art keywords
- alagillus
- jagged1
- syndrome
- treatment
- polynucleotides coding
- Prior art date
Links
Classifications
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
-
- 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
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/14—Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
-
- 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
-
- 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
- A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
-
- 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
- A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
- A61P35/02—Antineoplastic agents specific for leukemia
-
- 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
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
-
- 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
- A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
-
- 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
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Liposomes
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Hematology (AREA)
- Oncology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662338170P | 2016-05-18 | 2016-05-18 |
Publications (1)
Publication Number | Publication Date |
---|---|
MA45052A true MA45052A (fr) | 2019-03-27 |
Family
ID=60326440
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MA045052A MA45052A (fr) | 2016-05-18 | 2017-05-18 | Polynucléotides codant pour jagged1 pour le traitement du syndrome d'alagille |
Country Status (4)
Country | Link |
---|---|
US (1) | US20190275170A1 (fr) |
EP (1) | EP3458107B1 (fr) |
MA (1) | MA45052A (fr) |
WO (1) | WO2017201342A1 (fr) |
Families Citing this family (63)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9464124B2 (en) | 2011-09-12 | 2016-10-11 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
WO2015105955A1 (fr) | 2014-01-08 | 2015-07-16 | Flodesign Sonics, Inc. | Dispositif d'acoustophorèse avec double chambre acoustophorétique |
RU2746406C2 (ru) | 2014-04-23 | 2021-04-13 | МОДЕРНАТиЭкс, ИНК. | Вакцины на основе нуклеиновых кислот |
US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
US11364292B2 (en) | 2015-07-21 | 2022-06-21 | Modernatx, Inc. | CHIKV RNA vaccines |
US11007260B2 (en) | 2015-07-21 | 2021-05-18 | Modernatx, Inc. | Infectious disease vaccines |
US11564893B2 (en) | 2015-08-17 | 2023-01-31 | Modernatx, Inc. | Methods for preparing particles and related compositions |
SI3350157T1 (sl) | 2015-09-17 | 2022-04-29 | Modernatx, Inc. | Sestave za doziranje terapevtskih sredstev v celice |
WO2017070624A1 (fr) | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Vaccins contre des maladies tropicales |
PE20181529A1 (es) | 2015-10-22 | 2018-09-26 | Modernatx Inc | Vacunas de acido nucleico para el virus varicela-zoster (vzv) |
EP3364981A4 (fr) | 2015-10-22 | 2019-08-07 | ModernaTX, Inc. | Vaccin contre le cytomégalovirus humain |
WO2017070616A2 (fr) | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Vaccins contre les maladies sexuellement transmissibles |
PT3718565T (pt) | 2015-10-22 | 2022-07-20 | Modernatx Inc | Vacinas contra vírus respiratórios |
DK3386484T3 (da) | 2015-12-10 | 2022-07-04 | Modernatx Inc | Sammensætninger og fremgangsmåder til afgivelse af terapeutiske midler |
US10465190B1 (en) | 2015-12-23 | 2019-11-05 | Modernatx, Inc. | In vitro transcription methods and constructs |
US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
MA45051A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant la relaxine |
SG11201901941YA (en) | 2016-09-14 | 2019-04-29 | Modernatx Inc | High purity rna compositions and methods for preparation thereof |
WO2018075980A1 (fr) | 2016-10-21 | 2018-04-26 | Modernatx, Inc. | Vaccin contre le cytomégalovirus humain |
CA3041350A1 (fr) * | 2016-11-10 | 2018-05-17 | Translate Bio, Inc. | Administration sous-cutanee d'arn messager |
EP3538146A4 (fr) | 2016-11-11 | 2020-07-15 | ModernaTX, Inc. | Vaccin antigrippal |
EP3551193A4 (fr) | 2016-12-08 | 2020-08-19 | Modernatx, Inc. | Vaccins à acide nucléique contre des virus respiratoires |
EP3555289A1 (fr) | 2016-12-13 | 2019-10-23 | ModernaTX, Inc. | Purification par affinité d'arn |
EP3582790A4 (fr) | 2017-02-16 | 2020-11-25 | ModernaTX, Inc. | Compositions immunogènes très puissantes |
WO2018157009A1 (fr) * | 2017-02-24 | 2018-08-30 | Modernatx, Inc. | Thérapie de dystrophies musculaires à base d'acide nucléique |
US11576961B2 (en) | 2017-03-15 | 2023-02-14 | Modernatx, Inc. | Broad spectrum influenza virus vaccine |
JP7332478B2 (ja) | 2017-03-15 | 2023-08-23 | モデルナティエックス インコーポレイテッド | 脂質ナノ粒子製剤 |
US11045540B2 (en) | 2017-03-15 | 2021-06-29 | Modernatx, Inc. | Varicella zoster virus (VZV) vaccine |
CA3056133A1 (fr) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Formes cristallines d'aminolipides |
US11752206B2 (en) | 2017-03-15 | 2023-09-12 | Modernatx, Inc. | Herpes simplex virus vaccine |
EP3595713A4 (fr) | 2017-03-15 | 2021-01-13 | ModernaTX, Inc. | Vaccin contre le virus respiratoire syncytial |
MA47790A (fr) | 2017-03-17 | 2021-05-05 | Modernatx Inc | Vaccins à base d'arn contre des maladies zoonotiques |
SG10202110491PA (en) | 2017-03-24 | 2021-11-29 | Curevac Ag | Nucleic acids encoding crispr-associated proteins and uses thereof |
MA48047A (fr) | 2017-04-05 | 2020-02-12 | Modernatx Inc | Réduction ou élimination de réponses immunitaires à des protéines thérapeutiques administrées par voie non intraveineuse, par exemple par voie sous-cutanée |
US12077501B2 (en) | 2017-06-14 | 2024-09-03 | Modernatx, Inc. | Compounds and compositions for intracellular delivery of agents |
WO2018232357A1 (fr) | 2017-06-15 | 2018-12-20 | Modernatx, Inc. | Formulations d'arn |
EP3668971B8 (fr) | 2017-08-18 | 2024-05-29 | ModernaTX, Inc. | Variants d'arn polymérase |
EP3668977A4 (fr) | 2017-08-18 | 2021-04-21 | Modernatx, Inc. | Procédés analytiques par hplc |
US11912982B2 (en) | 2017-08-18 | 2024-02-27 | Modernatx, Inc. | Methods for HPLC analysis |
WO2019046809A1 (fr) | 2017-08-31 | 2019-03-07 | Modernatx, Inc. | Procédés de fabrication de nanoparticules lipidiques |
US10653767B2 (en) | 2017-09-14 | 2020-05-19 | Modernatx, Inc. | Zika virus MRNA vaccines |
WO2019118921A1 (fr) | 2017-12-14 | 2019-06-20 | Flodesign Sonics, Inc. | Circuit d'excitation et circuit de commande de transducteur acoustique |
MA54676A (fr) | 2018-01-29 | 2021-11-17 | Modernatx Inc | Vaccins à base d'arn contre le vrs |
JP2021521101A (ja) * | 2018-04-06 | 2021-08-26 | キャンプ4 セラピューティクス コーポレイション | 遺伝子シグナル伝達ネットワークの標的調整を介した疾患の治療 |
JP2021526792A (ja) | 2018-06-06 | 2021-10-11 | マサチューセッツ インスティテュート オブ テクノロジー | 真核細胞における翻訳のための環状rna |
US12090235B2 (en) | 2018-09-20 | 2024-09-17 | Modernatx, Inc. | Preparation of lipid nanoparticles and methods of administration thereof |
US11351242B1 (en) | 2019-02-12 | 2022-06-07 | Modernatx, Inc. | HMPV/hPIV3 mRNA vaccine composition |
EP3927821A4 (fr) | 2019-02-20 | 2023-01-25 | ModernaTX, Inc. | Variants d'arn polymérase pour le coiffage co-transcriptionnel |
US11851694B1 (en) | 2019-02-20 | 2023-12-26 | Modernatx, Inc. | High fidelity in vitro transcription |
WO2020190750A1 (fr) | 2019-03-15 | 2020-09-24 | Modernatx, Inc. | Vaccins à base d'arn contre le vih |
SG11202112922WA (en) | 2019-05-22 | 2021-12-30 | Massachusetts Inst Technology | Circular rna compositions and methods |
US11066355B2 (en) | 2019-09-19 | 2021-07-20 | Modernatx, Inc. | Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents |
EP3920976B1 (fr) | 2019-12-04 | 2023-07-19 | Orna Therapeutics, Inc. | Méthodes et compositions d'arn circulaire |
IL297084A (en) | 2020-04-09 | 2022-12-01 | Suzhou Abogen Biosciences Co Ltd | Lipid nanoparticle compounds |
TW202204622A (zh) | 2020-04-09 | 2022-02-01 | 大陸商蘇州艾博生物科技有限公司 | 針對冠狀病毒之核酸疫苗 |
MX2022013254A (es) | 2020-04-22 | 2023-01-24 | BioNTech SE | Vacuna contra el coronavirus. |
US11406703B2 (en) | 2020-08-25 | 2022-08-09 | Modernatx, Inc. | Human cytomegalovirus vaccine |
US11524023B2 (en) | 2021-02-19 | 2022-12-13 | Modernatx, Inc. | Lipid nanoparticle compositions and methods of formulating the same |
CN115745819A (zh) * | 2021-09-03 | 2023-03-07 | 广州谷森制药有限公司 | 新型阳离子脂质化合物 |
US11878055B1 (en) | 2022-06-26 | 2024-01-23 | BioNTech SE | Coronavirus vaccine |
WO2024107928A1 (fr) * | 2022-11-16 | 2024-05-23 | Propella Therapeutics, Inc. | Composition de décanoate d'abiratérone et utilisation en thérapie |
Family Cites Families (45)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5082767A (en) | 1989-02-27 | 1992-01-21 | Hatfield G Wesley | Codon pair utilization |
EP1030925A1 (fr) | 1997-11-12 | 2000-08-30 | Brigham & Women's Hospital, Inc. | Element amplificateur de traduction du gene de la proteine precurseur amyloide humaine |
AU6270499A (en) | 1998-09-29 | 2000-04-17 | Phylos, Inc. | Synthesis of codon randomized nucleic acids |
WO2001055369A1 (fr) | 2000-01-28 | 2001-08-02 | The Scripps Research Institute | Sites d'entree internes du ribosome de synthese et procedes d'identification correspondants |
ES2340532T3 (es) | 2001-06-05 | 2010-06-04 | Curevac Gmbh | Arnm con un contenido g/c aumentado que codifica para un antigeno bacteriano y utilizacion del mismo. |
EP2385123B1 (fr) | 2001-09-28 | 2018-04-25 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | Molécules de micro arn |
EP1490494A1 (fr) | 2002-04-01 | 2004-12-29 | Walter Reed Army Institute of Research | Procede pour concevoir des sequences d'acides nucleiques de synthese pour l'expression optimale de proteines dans une cellule hote |
DE10260805A1 (de) | 2002-12-23 | 2004-07-22 | Geneart Gmbh | Verfahren und Vorrichtung zum Optimieren einer Nucleotidsequenz zur Expression eines Proteins |
CA2533701A1 (fr) | 2003-07-31 | 2005-02-17 | Isis Pharmaceuticals, Inc. | Composes oligomeres et compositions utilisables pour moduler des petits arn non-codants |
US8101385B2 (en) | 2005-06-30 | 2012-01-24 | Archemix Corp. | Materials and methods for the generation of transcripts comprising modified nucleotides |
RU2008103346A (ru) | 2005-06-30 | 2009-08-10 | Аркемикс Корп. (Us) | Материалы и способы для получения полностью 2-модифицированных транскриптов нуклеиновых кислот |
EP4332227A1 (fr) | 2005-08-23 | 2024-03-06 | The Trustees of the University of Pennsylvania | Arn contenant des nucléosides modifiées et leurs procédés d'utilisation |
CA2620420A1 (fr) | 2005-08-24 | 2007-03-01 | The Scripps Research Institute | Systemes de vecteurs dependant d'elements d'activation traductionnelle |
DE102005046490A1 (de) | 2005-09-28 | 2007-03-29 | Johannes-Gutenberg-Universität Mainz | Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen |
US20080046192A1 (en) | 2006-08-16 | 2008-02-21 | Richard Lathrop | Polypepetide-encoding nucleotide sequences with refined translational kinetics and methods of making same |
CA2673029C (fr) | 2006-12-22 | 2017-03-28 | Archemix Corp. | Materiaux et procedes pour la generation de transcrits comprenant des nucleotides modifies |
EP3156414B1 (fr) | 2007-09-26 | 2019-12-04 | Intrexon Corporation | Vecteurs d'expression 5'utr synthétiques et procédés pour augmenter l'expression de transgènes |
EP2584038B1 (fr) | 2007-12-11 | 2014-06-25 | The Scripps Research Institute | Compositions et procédés concernant des éléments activateurs de traduction de l'ARNm |
US7561973B1 (en) | 2008-07-31 | 2009-07-14 | Dna Twopointo, Inc. | Methods for determining properties that affect an expression property value of polynucleotides in an expression system |
US8126653B2 (en) | 2008-07-31 | 2012-02-28 | Dna Twopointo, Inc. | Synthetic nucleic acids for expression of encoded proteins |
US8401798B2 (en) | 2008-06-06 | 2013-03-19 | Dna Twopointo, Inc. | Systems and methods for constructing frequency lookup tables for expression systems |
PL215513B1 (pl) | 2008-06-06 | 2013-12-31 | Univ Warszawski | Nowe boranofosforanowe analogi dinukleotydów, ich zastosowanie, czasteczka RNA, sposób otrzymywania RNA oraz sposób otrzymywania peptydów lub bialka |
US20110082055A1 (en) | 2009-09-18 | 2011-04-07 | Codexis, Inc. | Reduced codon mutagenesis |
US20130230884A1 (en) | 2010-07-16 | 2013-09-05 | John Chaput | Methods to Identify Synthetic and Natural RNA Elements that Enhance Protein Translation |
EP3202760B1 (fr) | 2011-01-11 | 2019-08-21 | Alnylam Pharmaceuticals, Inc. | Lipides pégylés et leur utilisation pour l'administration de médicaments |
AU2012236099A1 (en) | 2011-03-31 | 2013-10-03 | Moderna Therapeutics, Inc. | Delivery and formulation of engineered nucleic acids |
EP2718269B1 (fr) | 2011-06-08 | 2018-01-31 | Translate Bio, Inc. | Lipides clivables |
WO2013039857A1 (fr) | 2011-09-12 | 2013-03-21 | modeRNA Therapeutics | Acides nucléiques modifiés et leurs procédés d'utilisation |
EP3682905B1 (fr) | 2011-10-03 | 2021-12-01 | ModernaTX, Inc. | Nucléosides, nucléotides et acides nucléiques modifiés et leurs utilisations |
WO2013086354A1 (fr) | 2011-12-07 | 2013-06-13 | Alnylam Pharmaceuticals, Inc. | Lipides biodégradables pour l'administration d'agents actifs |
KR102075810B1 (ko) | 2012-02-24 | 2020-02-10 | 아뷰터스 바이오파마 코포레이션 | 트리알킬 양이온성 지질 및 그의 사용 방법 |
DE18200782T1 (de) | 2012-04-02 | 2021-10-21 | Modernatx, Inc. | Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen |
US9512456B2 (en) | 2012-08-14 | 2016-12-06 | Modernatx, Inc. | Enzymes and polymerases for the synthesis of RNA |
WO2014093924A1 (fr) | 2012-12-13 | 2014-06-19 | Moderna Therapeutics, Inc. | Molécules d'acide nucléique modifiées et leurs utilisations |
HRP20220607T1 (hr) * | 2012-11-26 | 2022-06-24 | Modernatx, Inc. | Terminalno modificirana rna |
WO2014089121A2 (fr) * | 2012-12-03 | 2014-06-12 | Thomas Ichim | Administration rétrograde de cellules et d'acides nucléiques pour le traitement de maladies cardiovasculaires |
US20150315541A1 (en) * | 2012-12-13 | 2015-11-05 | Moderna Therapeutics, Inc. | Modified polynucleotides for altering cell phenotype |
WO2014113089A2 (fr) | 2013-01-17 | 2014-07-24 | Moderna Therapeutics, Inc. | Polynucléotides capteurs de signal servant à modifier les phénotypes cellulaires |
US20160022840A1 (en) | 2013-03-09 | 2016-01-28 | Moderna Therapeutics, Inc. | Heterologous untranslated regions for mrna |
WO2014179562A1 (fr) * | 2013-05-01 | 2014-11-06 | Massachusetts Institute Of Technology | Dérivés de 1,3,5-triazinane-2,4,6-trione et leurs utilisations |
PL3766916T3 (pl) | 2014-06-25 | 2023-02-27 | Acuitas Therapeutics Inc. | Nowe lipidy i formulacje nanocząstek lipidowych do dostarczania kwasów nukleinowych |
JP2017523777A (ja) * | 2014-07-17 | 2017-08-24 | モデルナティエックス インコーポレイテッドModernaTX,Inc. | ポリヌクレオチドの末端修飾 |
WO2016061509A1 (fr) * | 2014-10-17 | 2016-04-21 | The Broad Institute, Inc. | Compositions et procédés de traitement de la dystrophie musculaire |
WO2017070616A2 (fr) * | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Vaccins contre les maladies sexuellement transmissibles |
WO2017075531A1 (fr) | 2015-10-28 | 2017-05-04 | Acuitas Therapeutics, Inc. | Nouveaux lipides et nouvelles formulations de nanoparticules de lipides pour l'administration d'acides nucléiques |
-
2017
- 2017-05-18 EP EP17800205.1A patent/EP3458107B1/fr active Active
- 2017-05-18 US US16/302,607 patent/US20190275170A1/en active Pending
- 2017-05-18 WO PCT/US2017/033413 patent/WO2017201342A1/fr unknown
- 2017-05-18 MA MA045052A patent/MA45052A/fr unknown
Also Published As
Publication number | Publication date |
---|---|
WO2017201342A1 (fr) | 2017-11-23 |
EP3458107B1 (fr) | 2024-03-13 |
US20190275170A1 (en) | 2019-09-12 |
EP3458107A4 (fr) | 2020-02-19 |
EP3458107A1 (fr) | 2019-03-27 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
MA45052A (fr) | Polynucléotides codant pour jagged1 pour le traitement du syndrome d'alagille | |
MA45539A (fr) | Dihydropyranopyrimidines pour le traitement d'infections virales | |
MA45036A (fr) | Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2 | |
MA49144A (fr) | Polythérapies pour le traitement du cancer | |
MA45192A (fr) | Traitement d'association | |
MA45041A (fr) | Polynucléotides codant pour la galactose-1-phosphate uridylyltransférase destinés au traitement de la galactosémie de type 1 | |
MA45481A (fr) | Utilisation d'exosomes pour le traitement de maladies | |
MA45050A (fr) | Polynucléotides codant pour la lipoprotéine lipase destinés au traitement de l'hyperlipidémie | |
MA50578A (fr) | Systèmes crispr/cas pour le traitement de dmd | |
MA49402A (fr) | Polynucléotides aadc pour le traitement de la maladie de parkinson | |
MA45037A (fr) | Polythérapie à base d'arnm pour le traitement du cancer | |
MA47719A (fr) | Esketamine pour le traitement de la dépression | |
MA45032A (fr) | Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë | |
MA54925A (fr) | Compositions pour le traitement de troubles acido-basiques | |
MA41449A (fr) | Polythérapies pour le traitement de cancers | |
MA48637A (fr) | Polythérapies pour le traitement du cancer | |
MA50409A (fr) | Polythérapies pour le traitement du cancer | |
MA50803A (fr) | Polynucléotides codant pour l'ornithine transcarbamylase pour le traitement de troubles du cycle de l'urée | |
MA44737A (fr) | Compositions pour le traitement de troubles liés à l'hyperkératose | |
MA43825A (fr) | Schémas posologiques pour le traitement d'infections fongiques | |
MA52252A (fr) | Formulation de poudre nasale pour le traitement de l'hypoglycémie | |
MA42999A (fr) | Polythérapie pour le traitement de malignités | |
MA53609A (fr) | Polynucléotides codant la glucose-6-phosphatase pour le traitement de la glycogénose | |
MA44864A (fr) | Trithérapie pour le traitement d'une maladie intestinale inflammatoire | |
MA49131A (fr) | Utilisation d'antagonistes de klk5 pour le traitement d'une maladie |