MA43568A - Polynucléotides codant pour la méthylmalonyl-coa mutase - Google Patents

Polynucléotides codant pour la méthylmalonyl-coa mutase

Info

Publication number
MA43568A
MA43568A MA043568A MA43568A MA43568A MA 43568 A MA43568 A MA 43568A MA 043568 A MA043568 A MA 043568A MA 43568 A MA43568 A MA 43568A MA 43568 A MA43568 A MA 43568A
Authority
MA
Morocco
Prior art keywords
methylmalonyl
coa mutase
polynucleotides coding
polynucleotides
coding
Prior art date
Application number
MA043568A
Other languages
English (en)
French (fr)
Inventor
Paolo Martini
Vladimir Presnyak
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
Application filed by Modernatx Inc filed Critical Modernatx Inc
Publication of MA43568A publication Critical patent/MA43568A/fr

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/52Isomerases (5)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules 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
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/5123Organic compounds, e.g. fats, sugars
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52Genes encoding for enzymes or proenzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/90Isomerases (5.)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y504/00Intramolecular transferases (5.4)
    • C12Y504/99Intramolecular transferases (5.4) transferring other groups (5.4.99)
    • C12Y504/99002Methylmalonyl-CoA mutase (5.4.99.2)

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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Hematology (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)
MA043568A 2015-12-17 2016-12-16 Polynucléotides codant pour la méthylmalonyl-coa mutase MA43568A (fr)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201562269089P 2015-12-17 2015-12-17
US201562269092P 2015-12-17 2015-12-17
US201562273112P 2015-12-30 2015-12-30
US201562273108P 2015-12-30 2015-12-30
US201662274727P 2016-01-04 2016-01-04
US201662274722P 2016-01-04 2016-01-04
US201662274733P 2016-01-04 2016-01-04
US201662274726P 2016-01-04 2016-01-04
US201662338456P 2016-05-18 2016-05-18
US201662338478P 2016-05-18 2016-05-18
US201662409343P 2016-10-17 2016-10-17

Publications (1)

Publication Number Publication Date
MA43568A true MA43568A (fr) 2018-11-14

Family

ID=57738001

Family Applications (1)

Application Number Title Priority Date Filing Date
MA043568A MA43568A (fr) 2015-12-17 2016-12-16 Polynucléotides codant pour la méthylmalonyl-coa mutase

Country Status (8)

Country Link
US (5) US10406112B2 (https=)
EP (1) EP3390630B1 (https=)
JP (1) JP7065036B2 (https=)
AU (1) AU2016369612B2 (https=)
CA (1) CA3007108A1 (https=)
IL (2) IL259667B (https=)
MA (1) MA43568A (https=)
WO (1) WO2017106799A1 (https=)

Families Citing this family (94)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
WO2015105955A1 (en) 2014-01-08 2015-07-16 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
PL4023249T3 (pl) 2014-04-23 2025-03-10 Modernatx, Inc. Szczepionki z kwasem nukleinowym
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
MA42543A (fr) 2015-07-30 2018-06-06 Modernatx Inc Arn épitope peptidiques concatémériques
US11564893B2 (en) 2015-08-17 2023-01-31 Modernatx, Inc. Methods for preparing particles and related compositions
JP6948313B6 (ja) 2015-09-17 2022-01-14 モデルナティエックス インコーポレイテッド 治療剤の細胞内送達のための化合物および組成物
WO2017070624A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Tropical disease vaccines
WO2017070601A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Nucleic acid vaccines for varicella zoster virus (vzv)
AU2016341309A1 (en) 2015-10-22 2018-06-07 Modernatx, Inc. Cancer vaccines
EP4011451A1 (en) 2015-10-22 2022-06-15 ModernaTX, Inc. Metapneumovirus mrna vaccines
AU2016342045A1 (en) 2015-10-22 2018-06-07 Modernatx, Inc. Human cytomegalovirus vaccine
PL3386484T3 (pl) 2015-12-10 2022-07-25 Modernatx, Inc. Kompozycje i sposoby dostarczania środków terapeutycznych
MA43568A (fr) 2015-12-17 2018-11-14 Modernatx Inc Polynucléotides codant pour la méthylmalonyl-coa mutase
LT3394030T (lt) 2015-12-22 2022-04-11 Modernatx, Inc. Junginiai ir kompozicijos terapinei medžiagai teikti intraceliuliniu būdu
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
EP3458108A4 (en) 2016-05-18 2020-04-22 ModernaTX, Inc. POLYNUCLEOTIDES ENCODING A CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR FOR THE TREATMENT OF CYSTIC FIBROSIS
US11001861B2 (en) 2016-05-18 2021-05-11 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
MA45036A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2
US12123030B2 (en) 2016-05-18 2024-10-22 Modernatx, Inc. Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia
WO2017201342A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
JP2019519601A (ja) 2016-05-18 2019-07-11 モダーナティエックス・インコーポレイテッドModernaTX, Inc. 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド
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
CA3036831A1 (en) 2016-09-14 2018-03-22 Modernatx, Inc. High purity rna compositions and methods for preparation thereof
EP3528821A4 (en) 2016-10-21 2020-07-01 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
MA46766A (fr) 2016-11-11 2019-09-18 Modernatx Inc Vaccin antigrippal
WO2018107088A2 (en) 2016-12-08 2018-06-14 Modernatx, Inc. Respiratory virus nucleic acid vaccines
US11384352B2 (en) 2016-12-13 2022-07-12 Modernatx, Inc. RNA affinity purification
WO2018170270A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Varicella zoster virus (vzv) vaccine
CA3055653A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Lipid nanoparticle formulation
WO2018170322A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Crystal forms of amino lipids
EP3595713A4 (en) 2017-03-15 2021-01-13 ModernaTX, Inc. RESPIRATORY SYNCYTIAL VIRUS Vaccine
WO2018170256A1 (en) * 2017-03-15 2018-09-20 Modernatx, Inc. Herpes simplex virus vaccine
WO2018170245A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Broad spectrum influenza virus vaccine
EP4186888B1 (en) 2017-03-15 2025-11-26 ModernaTX, Inc. Compound and compositions for intracellular delivery of therapeutic agents
EP3595676A4 (en) 2017-03-17 2021-05-05 Modernatx, Inc. RNA VACCINES AGAINST ZOONOSES
US11905525B2 (en) 2017-04-05 2024-02-20 Modernatx, Inc. Reduction of elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins
WO2018232120A1 (en) 2017-06-14 2018-12-20 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
MA49421A (fr) 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
MA49914A (fr) 2017-08-18 2021-04-21 Modernatx Inc Procédés analytiques par hplc
EP3668979A4 (en) 2017-08-18 2021-06-02 Modernatx, Inc. METHOD OF HPLC ANALYSIS
WO2019036682A1 (en) 2017-08-18 2019-02-21 Modernatx, Inc. RNA VARIANTS POLYMERASE
EP3675817A1 (en) 2017-08-31 2020-07-08 Modernatx, Inc. Methods of making 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
WO2019055807A1 (en) 2017-09-14 2019-03-21 Modernatx, Inc. RNA VACCINES AGAINST ZIKA VIRUS
KR102439221B1 (ko) 2017-12-14 2022-09-01 프로디자인 소닉스, 인크. 음향 트랜스듀서 구동기 및 제어기
CN111712468A (zh) 2017-12-22 2020-09-25 北卡罗莱纳州立大学 聚合物荧光团、包含其的组合物及制备和使用其的方法
EP3746090A4 (en) 2018-01-29 2021-11-17 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
EP3849617A1 (en) * 2018-09-14 2021-07-21 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia
WO2020061295A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. High-purity peg lipids and uses thereof
CA3113025A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Peg lipids and uses thereof
WO2020061367A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
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
CA3116576A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
WO2020106946A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR
AU2020224103A1 (en) 2019-02-20 2021-09-16 Modernatx, Inc. Rna polymerase variants for co-transcriptional capping
US11851694B1 (en) 2019-02-20 2023-12-26 Modernatx, Inc. High fidelity in vitro transcription
EP3931312A4 (en) * 2019-02-27 2022-08-24 The Regents of The University of Michigan CARBAMOYL PHOSPHATE SYNTHETASE-1 FOR THE TREATMENT AND PREVENTION OF HEPATIC DAMAGE
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
US12070495B2 (en) 2019-03-15 2024-08-27 Modernatx, Inc. HIV RNA vaccines
AU2020350759A1 (en) 2019-09-19 2022-03-31 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
EP4031662A1 (en) 2019-09-20 2022-07-27 Translate Bio, Inc. Mrna encoding engineered cftr
WO2021204175A1 (en) 2020-04-09 2021-10-14 Suzhou Abogen Biosciences Co., Ltd. Lipid nanoparticle composition
EP4132576A1 (en) 2020-04-09 2023-02-15 Suzhou Abogen Biosciences Co., Ltd. Nucleic acid vaccines for coronavirus
IL297419B2 (en) 2020-04-22 2025-02-01 BioNTech SE Coronavirus vaccine
ES3054438T3 (en) 2020-07-16 2026-02-03 Acuitas Therapeutics Inc Cationic lipids for use in lipid nanoparticles
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
EP4274607A1 (en) 2021-01-11 2023-11-15 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
US20250195666A1 (en) * 2021-05-20 2025-06-19 City Of Hope Mir-142 compounds and uses thereof
US20240398940A1 (en) 2021-09-03 2024-12-05 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
EP4422698A1 (en) 2021-10-29 2024-09-04 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
US20250345407A1 (en) 2022-05-25 2025-11-13 CureVac SE Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
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
WO2024184500A1 (en) 2023-03-08 2024-09-12 CureVac SE Novel lipid nanoparticle formulations for delivery of nucleic acids
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
AU2024347117A1 (en) * 2023-09-21 2026-04-09 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)

* Cited by examiner, † Cited by third party
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
CA2188675A1 (en) 1994-04-11 1995-10-19 Savio L. C. Woo 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
US20070122885A1 (en) 2005-08-22 2007-05-31 Fermalogic, Inc. Methods of increasing production of secondary metabolites by manipulating metabolic pathways that include methylmalonyl-coa
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
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
EP3318248B1 (en) 2009-12-01 2019-04-10 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
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
JP6188728B2 (ja) 2012-02-07 2017-08-30 グローバル・バイオ・セラピューティクス・インコーポレイテッドGlobal Bio Therapeutics,Inc. 核酸送達の区画化方法ならびにその組成物および使用
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
CN104411338A (zh) * 2012-04-02 2015-03-11 现代治疗公司 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
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
AU2013374345A1 (en) 2013-01-17 2015-08-06 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
WO2014151927A1 (en) * 2013-03-14 2014-09-25 Ipg Photonics Corporation Universal fiber optic connector
EP3750903A1 (en) 2013-03-14 2020-12-16 Translate Bio, Inc. Ribonucleic acids with 4'-thio-modified nucleotides and related methods
ES2644468T3 (es) 2013-03-15 2017-11-29 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Transgén sintético de la metilmalonil-CoA mutasa para el tratamiento de la acidemia metilmalónica (MMA) de clase MUT
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)
AU2014296278C1 (en) * 2013-07-30 2023-02-02 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
HUE060907T2 (hu) 2014-06-25 2023-04-28 Acuitas Therapeutics Inc Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére
WO2016077125A1 (en) 2014-11-10 2016-05-19 Moderna Therapeutics, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
JP2018509387A (ja) 2015-01-21 2018-04-05 フェーズアールエックス インコーポレイテッド 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム
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
JP6948313B6 (ja) 2015-09-17 2022-01-14 モデルナティエックス インコーポレイテッド 治療剤の細胞内送達のための化合物および組成物
HRP20230209T1 (hr) 2015-10-28 2023-04-14 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
MA43568A (fr) 2015-12-17 2018-11-14 Modernatx Inc Polynucléotides codant pour la méthylmalonyl-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
US20180126003A1 (en) 2016-05-04 2018-05-10 Curevac Ag New targets for rna therapeutics
WO2017191274A2 (en) 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
US11001861B2 (en) 2016-05-18 2021-05-11 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
JP2019519601A (ja) 2016-05-18 2019-07-11 モダーナティエックス・インコーポレイテッドModernaTX, Inc. 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド
US12123030B2 (en) 2016-05-18 2024-10-22 Modernatx, Inc. Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia
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
EP3458108A4 (en) 2016-05-18 2020-04-22 ModernaTX, Inc. POLYNUCLEOTIDES ENCODING A CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR FOR THE TREATMENT OF CYSTIC FIBROSIS
MA45036A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2
WO2018157009A1 (en) 2017-02-24 2018-08-30 Modernatx, Inc. Nucleic acid-based therapy of muscular dystrophies
US20200131498A1 (en) 2017-06-14 2020-04-30 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
EP3849617A1 (en) 2018-09-14 2021-07-21 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia

Also Published As

Publication number Publication date
US20200078314A1 (en) 2020-03-12
AU2016369612A1 (en) 2018-06-14
JP7065036B2 (ja) 2022-05-11
EP3390630B1 (en) 2025-01-22
IL289098A (en) 2022-02-01
EP3390630A1 (en) 2018-10-24
CA3007108A1 (en) 2017-06-22
US11504337B2 (en) 2022-11-22
US20180289838A1 (en) 2018-10-11
US20190022019A1 (en) 2019-01-24
AU2016369612B2 (en) 2023-06-01
IL259667B (en) 2022-01-01
WO2017106799A1 (en) 2017-06-22
US10426738B2 (en) 2019-10-01
US10406112B2 (en) 2019-09-10
IL259667A (en) 2018-07-31
US20180271795A1 (en) 2018-09-27
JP2019500430A (ja) 2019-01-10
US20230270685A1 (en) 2023-08-31

Similar Documents

Publication Publication Date Title
IL289098A (en) Polynucleotides encoding methylmalonyl–coa mutase
MA45051A (fr) Polynucléotides codant la relaxine
EP3360001A4 (en) HEAD-MOUNTED DISPLAY DEVICE
DK3356636T3 (da) Slagindretning
EP3408858A4 (en) GRAPHIC FRAME FOR SUPERCONDENSATORS
PT3298027T (pt) Compostos depsipéptidos antelmínticos
BR112016021624A2 (pt) indexação de locais baseada em polígono
PT4197530T (pt) Comprimido de ribociclib
EP3337164A4 (en) HEAD-MOUNTED DISPLAY
EP3358200A4 (en) CONSTRUCTION MACHINE
EP3305993A4 (en) CONSTRUCTION MACHINE
CL2016002165A1 (es) Compuestos de aminocarbonilcarbamato
HUE054351T2 (hu) Vegyületek
DK3597189T3 (da) Krystallinske forbindelser
EP3359485C0 (en) Production of graphene
ES2789757T8 (es) Herramienta de conformado
DK3289490T3 (da) Flertrins cache-udfyldning
LT3253749T (lt) Junginiai, pasižymintys priešnavikiniu aktyvumu
FR3032494B1 (fr) Aube de soufflante
DK3325728T3 (da) Spildevandsbrønd
UY4467S (es) Frasco
LT3331885T (lt) Junginiai
ES2811901T8 (es) Cerradura de cilindro
EP3388149A4 (en) COLUMN STRUCTURE FOR BIOCHIP
FR3039220B1 (fr) Dipositif de postcombustion pour turboreacteur