CA3007108A1 - Polynucleotides encoding methylmalonyl-coa mutase - Google Patents
Polynucleotides encoding methylmalonyl-coa mutase Download PDFInfo
- Publication number
- CA3007108A1 CA3007108A1 CA3007108A CA3007108A CA3007108A1 CA 3007108 A1 CA3007108 A1 CA 3007108A1 CA 3007108 A CA3007108 A CA 3007108A CA 3007108 A CA3007108 A CA 3007108A CA 3007108 A1 CA3007108 A1 CA 3007108A1
- Authority
- CA
- Canada
- Prior art keywords
- nucleotides
- polynucleotide
- utp
- group
- seq
- Prior art date
- Legal status (The legal status 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 status listed.)
- Pending
Links
Classifications
-
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/52—Isomerases (5)
-
- 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
-
- 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—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
- A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- 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
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/90—Isomerases (5.)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y504/00—Intramolecular transferases (5.4)
- C12Y504/99—Intramolecular transferases (5.4) transferring other groups (5.4.99)
- C12Y504/99002—Methylmalonyl-CoA mutase (5.4.99.2)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Gastroenterology & Hepatology (AREA)
- Dispersion Chemistry (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (23)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562269089P | 2015-12-17 | 2015-12-17 | |
| US201562269092P | 2015-12-17 | 2015-12-17 | |
| US62/269,089 | 2015-12-17 | ||
| US62/269,092 | 2015-12-17 | ||
| US201562273112P | 2015-12-30 | 2015-12-30 | |
| US201562273108P | 2015-12-30 | 2015-12-30 | |
| US62/273,112 | 2015-12-30 | ||
| US62/273,108 | 2015-12-30 | ||
| US201662274733P | 2016-01-04 | 2016-01-04 | |
| US201662274726P | 2016-01-04 | 2016-01-04 | |
| US201662274727P | 2016-01-04 | 2016-01-04 | |
| US201662274722P | 2016-01-04 | 2016-01-04 | |
| US62/274,726 | 2016-01-04 | ||
| US62/274,727 | 2016-01-04 | ||
| US62/274,722 | 2016-01-04 | ||
| US62/274,733 | 2016-01-04 | ||
| US201662338456P | 2016-05-18 | 2016-05-18 | |
| US201662338478P | 2016-05-18 | 2016-05-18 | |
| US62/338,478 | 2016-05-18 | ||
| US62/338,456 | 2016-05-18 | ||
| US201662409343P | 2016-10-17 | 2016-10-17 | |
| US62/409,343 | 2016-10-17 | ||
| PCT/US2016/067393 WO2017106799A1 (en) | 2015-12-17 | 2016-12-16 | POLYNUCLEOTIDES ENCODING METHYLMALONYL-CoA MUTASE |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3007108A1 true CA3007108A1 (en) | 2017-06-22 |
Family
ID=57738001
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3007108A Pending CA3007108A1 (en) | 2015-12-17 | 2016-12-16 | Polynucleotides encoding methylmalonyl-coa mutase |
Country Status (8)
| Country | Link |
|---|---|
| US (5) | US10406112B2 (enExample) |
| EP (1) | EP3390630B1 (enExample) |
| JP (1) | JP7065036B2 (enExample) |
| AU (1) | AU2016369612B2 (enExample) |
| CA (1) | CA3007108A1 (enExample) |
| IL (2) | IL259667B (enExample) |
| MA (1) | MA43568A (enExample) |
| WO (1) | WO2017106799A1 (enExample) |
Families Citing this family (94)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| CA2935960C (en) | 2014-01-08 | 2023-01-10 | Bart Lipkens | Acoustophoresis device with dual acoustophoretic chamber |
| DK3134131T3 (en) | 2014-04-23 | 2022-02-07 | Modernatx Inc | Nucleic acid vaccines |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11364292B2 (en) | 2015-07-21 | 2022-06-21 | Modernatx, Inc. | CHIKV RNA vaccines |
| WO2017020026A1 (en) | 2015-07-30 | 2017-02-02 | Modernatx, Inc. | Concatemeric peptide epitopes rnas |
| US11564893B2 (en) | 2015-08-17 | 2023-01-31 | Modernatx, Inc. | Methods for preparing particles and related compositions |
| RS63030B1 (sr) | 2015-09-17 | 2022-04-29 | Modernatx Inc | Jedinjenja i kompozicije za intracelularno isporučivanje terapeutskih sredstava |
| EP3364950A4 (en) | 2015-10-22 | 2019-10-23 | ModernaTX, Inc. | VACCINES AGAINST TROPICAL DISEASES |
| MD3718565T2 (ro) | 2015-10-22 | 2022-09-30 | Modernatx Inc | Vaccinuri împotriva virusului respirator |
| CN108430456B (zh) | 2015-10-22 | 2022-01-18 | 摩登纳特斯有限公司 | 癌症疫苗 |
| WO2017070601A1 (en) | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Nucleic acid vaccines for varicella zoster virus (vzv) |
| EP3364981A4 (en) | 2015-10-22 | 2019-08-07 | ModernaTX, Inc. | VACCINE AGAINST THE HUMAN CYTOMEGALOVIRUS |
| CA3007955A1 (en) | 2015-12-10 | 2017-06-15 | Modernatx, Inc. | Lipid nanoparticles for delivery of therapeutic agents |
| EP3390630B1 (en) | 2015-12-17 | 2025-01-22 | ModernaTX, Inc. | Polynucleotides encoding methylmalonyl-coa mutase |
| JP7114465B2 (ja) | 2015-12-22 | 2022-08-08 | モデルナティエックス インコーポレイテッド | 薬剤の細胞内送達のための化合物および組成物 |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| MA45032A (fr) | 2016-05-18 | 2019-03-27 | Fundacion Para La Investig Medica Aplicada | Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë |
| US20190298657A1 (en) * | 2016-05-18 | 2019-10-03 | Modernatx, Inc. | Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency |
| US12128113B2 (en) | 2016-05-18 | 2024-10-29 | Modernatx, Inc. | Polynucleotides encoding JAGGED1 for the treatment of Alagille syndrome |
| US12123030B2 (en) | 2016-05-18 | 2024-10-22 | Modernatx, Inc. | Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia |
| US11001861B2 (en) | 2016-05-18 | 2021-05-11 | Modernatx, Inc. | Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1 |
| MA45053A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique |
| WO2017201349A1 (en) | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 |
| JP6983455B2 (ja) | 2016-09-14 | 2021-12-17 | モデルナティーエックス， インコーポレイテッド | 高純度ｒｎａ組成物及びその調製のための方法 |
| AU2017345766A1 (en) | 2016-10-21 | 2019-05-16 | Modernatx, Inc. | Human cytomegalovirus vaccine |
| US12491261B2 (en) | 2016-10-26 | 2025-12-09 | Acuitas Therapeutics, Inc. | Lipid nanoparticle formulations |
| US11583504B2 (en) | 2016-11-08 | 2023-02-21 | Modernatx, Inc. | Stabilized formulations of lipid nanoparticles |
| CN110167587A (zh) | 2016-11-11 | 2019-08-23 | 摩登纳特斯有限公司 | 流感疫苗 |
| EP3551193A4 (en) | 2016-12-08 | 2020-08-19 | Modernatx, Inc. | NUCLEIC ACID VACCINES AGAINST RESPIRATORY VIRUSES |
| EP3555289A1 (en) | 2016-12-13 | 2019-10-23 | ModernaTX, Inc. | Rna affinity purification |
| MA47787A (fr) | 2017-03-15 | 2020-01-22 | Modernatx Inc | Vaccin contre le virus respiratoire syncytial |
| US11752206B2 (en) * | 2017-03-15 | 2023-09-12 | Modernatx, Inc. | Herpes simplex virus vaccine |
| WO2018170270A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Varicella zoster virus (vzv) vaccine |
| WO2018170245A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Broad spectrum influenza virus vaccine |
| ES3020935T3 (en) | 2017-03-15 | 2025-05-23 | Modernatx Inc | Lipid nanoparticle formulation |
| CA3056133A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Crystal forms of amino lipids |
| KR20190132405A (ko) | 2017-03-15 | 2019-11-27 | 모더나티엑스, 인크. | 치료제의 세포내 전달을 위한 화합물 및 조성물 |
| WO2018170347A1 (en) | 2017-03-17 | 2018-09-20 | Modernatx, Inc. | Zoonotic disease rna vaccines |
| WO2018187590A1 (en) | 2017-04-05 | 2018-10-11 | Modernatx, Inc. | Reduction or elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins |
| US12077501B2 (en) | 2017-06-14 | 2024-09-03 | Modernatx, Inc. | Compounds and compositions for intracellular delivery of agents |
| US20200131498A1 (en) * | 2017-06-14 | 2020-04-30 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase |
| WO2018232357A1 (en) | 2017-06-15 | 2018-12-20 | Modernatx, Inc. | Rna formulations |
| CN111212905A (zh) | 2017-08-18 | 2020-05-29 | 摩登纳特斯有限公司 | Rna聚合酶变体 |
| WO2019036683A1 (en) | 2017-08-18 | 2019-02-21 | Modernatx, Inc. | ANALYTICAL METHODS BY HPLC |
| WO2019036685A1 (en) | 2017-08-18 | 2019-02-21 | Modernatx, Inc. | METHODS FOR HPLC ANALYSIS |
| WO2019046809A1 (en) | 2017-08-31 | 2019-03-07 | Modernatx, Inc. | METHODS OF MANUFACTURING LIPID NANOPARTICLES |
| EP3679379A2 (en) * | 2017-09-08 | 2020-07-15 | The U.S.A. as represented by the Secretary, Department of Health and Human Services | Biomarkers of organic acidemias |
| US10653767B2 (en) | 2017-09-14 | 2020-05-19 | Modernatx, Inc. | Zika virus MRNA vaccines |
| SG11202003907WA (en) | 2017-12-14 | 2020-05-28 | Flodesign Sonics Inc | Acoustic transducer drive and controller |
| JP7398115B2 (ja) | 2017-12-22 | 2023-12-14 | ノース カロライナ ステート ユニバーシティ | ポリマーフルオロフォア、それを含む組成物、ならびにその調製および使用方法 |
| WO2019148101A1 (en) | 2018-01-29 | 2019-08-01 | Modernatx, Inc. | Rsv rna vaccines |
| US20190314291A1 (en) * | 2018-01-30 | 2019-10-17 | Modernatx, Inc. | Compositions and methods for delivery of agents to immune cells |
| CN108704162A (zh) * | 2018-08-08 | 2018-10-26 | 中国科学院长春应用化学研究所 | 一种吸水敷料及其制备方法 |
| US12564645B2 (en) * | 2018-09-14 | 2026-03-03 | Translate Bio, Inc. | Methods for treatment of methylmalonic acidemia |
| AU2019345067A1 (en) | 2018-09-19 | 2021-04-08 | Modernatx, Inc. | High-purity peg lipids and uses thereof |
| WO2020061367A1 (en) | 2018-09-19 | 2020-03-26 | Modernatx, Inc. | Compounds and compositions for intracellular delivery of therapeutic agents |
| US12151029B2 (en) | 2018-09-19 | 2024-11-26 | Modernatx, Inc. | PEG lipids and uses thereof |
| WO2020061457A1 (en) | 2018-09-20 | 2020-03-26 | Modernatx, Inc. | Preparation of lipid nanoparticles and methods of administration thereof |
| US12331320B2 (en) | 2018-10-10 | 2025-06-17 | The Research Foundation For The State University Of New York | Genome edited cancer cell vaccines |
| US12583816B2 (en) | 2018-10-18 | 2026-03-24 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| EP3883592A1 (en) | 2018-11-21 | 2021-09-29 | Translate Bio, Inc. | Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr |
| JP2022521094A (ja) | 2019-02-20 | 2022-04-05 | モデルナティエックス インコーポレイテッド | 共転写キャッピング用ｒｎａポリメラーゼバリアント |
| US11851694B1 (en) | 2019-02-20 | 2023-12-26 | Modernatx, Inc. | High fidelity in vitro transcription |
| US20220162278A1 (en) * | 2019-02-27 | 2022-05-26 | The Regents Of The University Of Michigan | Carbamoyl phosphate synthatase-1 for the treatment and prevention of liver injury |
| CN113874502A (zh) | 2019-03-11 | 2021-12-31 | 摩登纳特斯有限公司 | 补料分批体外转录方法 |
| WO2020190750A1 (en) | 2019-03-15 | 2020-09-24 | Modernatx, Inc. | Hiv rna vaccines |
| MX2022003269A (es) | 2019-09-19 | 2022-07-04 | Modernatx Inc | Compuestos lipidicos de cola ramificada y composiciones para la administracion intracelular de agentes terapeuticos. |
| WO2021055609A1 (en) | 2019-09-20 | 2021-03-25 | Translate Bio, Inc. | Mrna encoding engineered cftr |
| KR102924173B1 (ko) | 2020-04-09 | 2026-02-06 | 쑤저우 아보젠 바이오사이언시스 컴퍼니 리미티드 | 지질 나노입자 조성물 |
| WO2021204179A1 (en) | 2020-04-09 | 2021-10-14 | Suzhou Abogen Biosciences Co., Ltd. | Nucleic acid vaccines for coronavirus |
| CN113521269A (zh) | 2020-04-22 | 2021-10-22 | 生物技术Rna制药有限公司 | 冠状病毒疫苗 |
| JP7798855B2 (ja) | 2020-07-16 | 2026-01-14 | アクイタス セラピューティクス インコーポレイテッド | 脂質ナノ粒子に使用するためのカチオン性脂質 |
| US11406703B2 (en) | 2020-08-25 | 2022-08-09 | Modernatx, Inc. | Human cytomegalovirus vaccine |
| WO2022150717A1 (en) | 2021-01-11 | 2022-07-14 | Modernatx, Inc. | Seasonal rna influenza virus vaccines |
| US20240226025A1 (en) * | 2021-03-24 | 2024-07-11 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia |
| US20220363937A1 (en) | 2021-05-14 | 2022-11-17 | Armstrong World Industries, Inc. | Stabilization of antimicrobial coatings |
| US20240269248A1 (en) * | 2021-05-19 | 2024-08-15 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia |
| WO2022245980A1 (en) * | 2021-05-20 | 2022-11-24 | City Of Hope | Mir-142 compounds and uses thereof |
| IL309505A (en) | 2021-09-03 | 2024-02-01 | CureVac SE | Novel lipid nanoparticles for delivery of nucleic acids |
| US20250027108A1 (en) | 2021-10-29 | 2025-01-23 | CureVac SE | Improved circular rna for expressing therapeutic proteins |
| US12186387B2 (en) | 2021-11-29 | 2025-01-07 | BioNTech SE | Coronavirus vaccine |
| US12529047B1 (en) | 2021-12-21 | 2026-01-20 | Modernatx, Inc. | mRNA quantification methods |
| US20250099614A1 (en) | 2022-01-28 | 2025-03-27 | CureVac SE | Nucleic acid encoded transcription factor inhibitors |
| CA3248209A1 (en) | 2022-05-25 | 2023-11-30 | CureVac SE | Escherichia coli Antigenic Polypeptide-Based Nucleic Acid Vaccine (FIMH) |
| WO2024002985A1 (en) | 2022-06-26 | 2024-01-04 | BioNTech SE | Coronavirus vaccine |
| EP4608442A1 (en) | 2022-10-28 | 2025-09-03 | GlaxoSmithKline Biologicals S.A. | Nucleic acid based vaccine |
| JP2026509258A (ja) | 2023-03-08 | 2026-03-17 | キュアバック エスイー | 核酸送達のための新規な脂質ナノ粒子製剤 |
| WO2024230934A1 (en) | 2023-05-11 | 2024-11-14 | CureVac SE | Therapeutic nucleic acid for the treatment of ophthalmic diseases |
| WO2025064906A1 (en) * | 2023-09-21 | 2025-03-27 | The Board Of Regents Of The University Of Texas System | Endogenous mitochondria targeting signal peptides and uses thereof |
| WO2025126242A1 (en) * | 2023-12-11 | 2025-06-19 | Dr. Reddy's Institute Of Life Sciences | Nucleic acid sequence(s) and construct(s) for treating methylmalonic acidemia and method(s) thereof |
| CN118360253A (zh) * | 2024-05-11 | 2024-07-19 | 中山大学附属第六医院 | 一种干扰甲基丙二酰辅酶a变位酶表达的细胞系及其构建方法与应用 |
| WO2026014954A1 (ko) * | 2024-07-10 | 2026-01-15 | 주식회사 엣진 | 개선된 염기 교정 효율을 갖는 미토콘드리아 dna 염기 에디터 |
Family Cites Families (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5602240A (en) | 1990-07-27 | 1997-02-11 | Ciba Geigy Ag. | Backbone modified oligonucleotide analogs |
| US5489677A (en) | 1990-07-27 | 1996-02-06 | Isis Pharmaceuticals, Inc. | Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms |
| WO1995027512A2 (en) | 1994-04-11 | 1995-10-19 | Baylor College Of Medicine | Compositions and methods for gene therapy to treat disease |
| US6652886B2 (en) | 2001-02-16 | 2003-11-25 | Expression Genetics | Biodegradable cationic copolymers of poly (alkylenimine) and poly (ethylene glycol) for the delivery of bioactive agents |
| WO2007024997A2 (en) | 2005-08-22 | 2007-03-01 | Fermalogic, Inc. | Methods of increasing production of secondary metabolites |
| HUE043492T2 (hu) | 2005-08-23 | 2019-08-28 | Univ Pennsylvania | Módosított nukleozidokat tartalmazó RNS és eljárások az alkalmazására |
| WO2009030254A1 (en) | 2007-09-04 | 2009-03-12 | Curevac Gmbh | Complexes of rna and cationic peptides for transfection and for immunostimulation |
| CN107028886A (zh) * | 2009-11-04 | 2017-08-11 | 不列颠哥伦比亚大学 | 含有核酸的脂质粒子及相关的方法 |
| PL3338765T3 (pl) | 2009-12-01 | 2019-06-28 | Translate Bio, Inc. | Pochodna steroidowa dla dostarczania mrna w ludzkich chorobach genetycznych |
| SE535869C2 (sv) * | 2010-04-01 | 2013-01-22 | Elways Ab | Ett för ett eller flera, elektriskt framdrivbara, fordon anpassat system (Laddningssystem). |
| WO2013086373A1 (en) * | 2011-12-07 | 2013-06-13 | Alnylam Pharmaceuticals, Inc. | Lipids for the delivery of active agents |
| SG11201404741VA (en) | 2012-02-07 | 2014-09-26 | Global Bio Therapeutics Usa Inc | Compartmentalized method of nucleic acid delivery and compositions and uses thereof |
| CN108949772A (zh) * | 2012-04-02 | 2018-12-07 | 现代泰克斯公司 | 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸 |
| WO2013151663A1 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of membrane proteins |
| US9255250B2 (en) * | 2012-12-05 | 2016-02-09 | Sangamo Bioscience, Inc. | Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene |
| JP2016504050A (ja) * | 2013-01-17 | 2016-02-12 | モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 細胞表現型の改変のためのシグナルセンサーポリヌクレオチド |
| WO2014151927A1 (en) * | 2013-03-14 | 2014-09-25 | Ipg Photonics Corporation | Universal fiber optic connector |
| ES2647832T3 (es) | 2013-03-14 | 2017-12-26 | Translate Bio, Inc. | Ácidos ribonucleicos con nucleótidos modificados con 4-tio y procedimientos relacionados |
| US9944918B2 (en) | 2013-03-15 | 2018-04-17 | The United States Of America, As Represented By The Secretary, Dept. Of Health & Human Services | Synthetic methylmalonyl-CoA mutase transgene for the treatment of MUT class methylmalonic acidemia (MMA) |
| EP2968602B1 (en) * | 2013-03-15 | 2017-08-30 | The United States of America, as represented by the Secretary, Department of Health and Human Services | Synthetic methylmalonyl-coa mutase transgene for the treatment of mut class methylmalonic acidemia (mma) |
| AU2014296278C1 (en) * | 2013-07-30 | 2023-02-02 | Genevant Sciences Gmbh | Block copolymers and their conjugates or complexes with oligonucleotides |
| IL316479A (en) * | 2014-06-25 | 2024-12-01 | Acuitas Therapeutics Inc | Novel lipids and lipid nanoparticle compositions for nucleic acid delivery |
| WO2016077125A1 (en) | 2014-11-10 | 2016-05-19 | Moderna Therapeutics, Inc. | Alternative nucleic acid molecules containing reduced uracil content and uses thereof |
| CN114642735A (zh) | 2015-01-21 | 2022-06-21 | 菲泽尔克斯公司 | 用于将治疗剂和诊断剂递送到细胞中的方法、组合物和系统 |
| WO2016172574A2 (en) * | 2015-04-24 | 2016-10-27 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Tagged form of mut enzyme, genetic constructs incorporating it, and its use in gene therapy |
| RS63030B1 (sr) | 2015-09-17 | 2022-04-29 | Modernatx Inc | Jedinjenja i kompozicije za intracelularno isporučivanje terapeutskih sredstava |
| RS63986B1 (sr) | 2015-10-28 | 2023-03-31 | Acuitas Therapeutics Inc | Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina |
| WO2017100551A1 (en) | 2015-12-09 | 2017-06-15 | Alexion Pharmaceuticals, Inc. | HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION |
| EP3390630B1 (en) | 2015-12-17 | 2025-01-22 | ModernaTX, Inc. | Polynucleotides encoding methylmalonyl-coa mutase |
| WO2017147720A1 (en) | 2016-03-04 | 2017-09-08 | Exerkine Corporation | Method for treating a central nervous system disorder |
| EP3426775A1 (en) | 2016-03-10 | 2019-01-16 | Novartis AG | Chemically modified messenger rna's |
| WO2017191274A2 (en) | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| US20180126003A1 (en) | 2016-05-04 | 2018-05-10 | Curevac Ag | New targets for rna therapeutics |
| US20190298657A1 (en) | 2016-05-18 | 2019-10-03 | Modernatx, Inc. | Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency |
| MA45053A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique |
| US12123030B2 (en) | 2016-05-18 | 2024-10-22 | Modernatx, Inc. | Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia |
| US11001861B2 (en) | 2016-05-18 | 2021-05-11 | Modernatx, Inc. | Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1 |
| MA45032A (fr) | 2016-05-18 | 2019-03-27 | Fundacion Para La Investig Medica Aplicada | Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë |
| WO2017201349A1 (en) | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 |
| US20200368162A1 (en) | 2017-02-24 | 2020-11-26 | Modernatx, Inc. | Nucleic Acid-Based Therapy of Muscular Dystrophies |
| US20200131498A1 (en) | 2017-06-14 | 2020-04-30 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase |
| US12564645B2 (en) | 2018-09-14 | 2026-03-03 | Translate Bio, Inc. | Methods for treatment of methylmalonic acidemia |
-
2016
- 2016-12-16 EP EP16822592.8A patent/EP3390630B1/en active Active
- 2016-12-16 WO PCT/US2016/067393 patent/WO2017106799A1/en not_active Ceased
- 2016-12-16 JP JP2018551890A patent/JP7065036B2/ja active Active
- 2016-12-16 CA CA3007108A patent/CA3007108A1/en active Pending
- 2016-12-16 MA MA043568A patent/MA43568A/fr unknown
- 2016-12-16 AU AU2016369612A patent/AU2016369612B2/en not_active Ceased
-
2018
- 2018-05-29 IL IL259667A patent/IL259667B/en unknown
- 2018-06-07 US US16/002,376 patent/US10406112B2/en active Active
- 2018-06-07 US US16/002,472 patent/US10426738B2/en active Active
- 2018-08-27 US US16/113,814 patent/US20190022019A1/en not_active Abandoned
-
2019
- 2019-07-18 US US16/515,256 patent/US11504337B2/en active Active
-
2021
- 2021-12-19 IL IL289098A patent/IL289098A/en unknown
-
2022
- 2022-11-18 US US17/989,941 patent/US20230270685A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP3390630B1 (en) | 2025-01-22 |
| AU2016369612B2 (en) | 2023-06-01 |
| US20180271795A1 (en) | 2018-09-27 |
| US10426738B2 (en) | 2019-10-01 |
| US20180289838A1 (en) | 2018-10-11 |
| IL259667B (en) | 2022-01-01 |
| WO2017106799A1 (en) | 2017-06-22 |
| JP7065036B2 (ja) | 2022-05-11 |
| US20190022019A1 (en) | 2019-01-24 |
| IL259667A (en) | 2018-07-31 |
| US10406112B2 (en) | 2019-09-10 |
| US20230270685A1 (en) | 2023-08-31 |
| IL289098A (en) | 2022-02-01 |
| US20200078314A1 (en) | 2020-03-12 |
| MA43568A (fr) | 2018-11-14 |
| AU2016369612A1 (en) | 2018-06-14 |
| US11504337B2 (en) | 2022-11-22 |
| JP2019500430A (ja) | 2019-01-10 |
| EP3390630A1 (en) | 2018-10-24 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| AU2016369612B2 (en) | Polynucleotides encoding methylmalonyl-CoA mutase | |
| EP3458105B1 (en) | Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1 | |
| EP3169693B1 (en) | Chimeric polynucleotides | |
| AU2017268396B2 (en) | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 | |
| AU2017202228B2 (en) | Terminally modified RNA | |
| CA3024624A1 (en) | Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria | |
| EP3682905B1 (en) | Modified nucleosides, nucleotides, and nucleic acids, and uses thereof | |
| WO2018231990A2 (en) | Polynucleotides encoding methylmalonyl-coa mutase | |
| CA3024500A1 (en) | Polynucleotides encoding relaxin | |
| EP3052479A2 (en) | Polynucleotide molecules and uses thereof | |
| CA3024507A1 (en) | Polynucleotides encoding .alpha.-galactosidase a for the treatment of fabry disease | |
| EP3188749A1 (en) | Tolerogenic compositions and methods | |
| EP2946014A2 (en) | Signal-sensor polynucleotides for the alteration of cellular phenotypes | |
| WO2016011222A2 (en) | Circular polynucleotides | |
| EP3171895A1 (en) | Modified polynucleotides for the production of intrabodies | |
| US20220298516A1 (en) | Compositions and methods for delivery of nucleic acids | |
| HK40080438A (en) | Terminally modified rna | |
| HK40032374A (en) | Modified nucleosides, nucleotides, and nucleic acids, and uses thereof | |
| HK40032374B (en) | Modified nucleosides, nucleotides, and nucleic acids, and uses thereof | |
| HK1214515B (en) | Terminally modified rna |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20211208 |
|
| B12 | Application deemed to be withdrawn, abandoned or lapsed |
Free format text: ST27 STATUS EVENT CODE: N-6-6-B10-B12-B303 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO AN EXAMINER'S REQUISITION Effective date: 20240806 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20250912 |
|
| U13 | Renewal or maintenance fee not paid |
Free format text: ST27 STATUS EVENT CODE: N-2-6-U10-U13-U300 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO MAINTENANCE FEE NOTICE Effective date: 20251112 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: N-6-6-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20260128 |