MA45032A - Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë - Google Patents

Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë

Info

Publication number
MA45032A
MA45032A MA045032A MA45032A MA45032A MA 45032 A MA45032 A MA 45032A MA 045032 A MA045032 A MA 045032A MA 45032 A MA45032 A MA 45032A MA 45032 A MA45032 A MA 45032A
Authority
MA
Morocco
Prior art keywords
desaminase
intermitten
porphobilinogen
porphyria
acute
Prior art date
Application number
MA045032A
Other languages
English (en)
Inventor
Kerry Benenato
López Pedro Berraondo
Romá Antonio Fontanellas
Lin Tung Guey
Stephen Hoge
Lei Jiang
Ellalahewage Sathyajith Kumarasinghe
Paolo Martini
Iain Mcfadyen
Vladimir Presnyak
Staci Sabnis
Zaragozá Matías Antonio Ávila
Original Assignee
Fundacion Para La Investig Medica Aplicada
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 Fundacion Para La Investig Medica Aplicada, Modernatx Inc filed Critical Fundacion Para La Investig Medica Aplicada
Publication of MA45032A publication Critical patent/MA45032A/fr

Links

Classifications

    • 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
    • A61K38/45Transferases (2)
    • 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
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • A61K48/0033Medicinal 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 non-polymeric
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/08Plasma substitutes; Perfusion solutions; Dialytics or haemodialytics; Drugs for electrolytic or acid-base disorders, e.g. hypovolemic shock
    • 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
    • C12N9/10Transferases (2.)
    • C12N9/1085Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y205/00Transferases transferring alkyl or aryl groups, other than methyl groups (2.5)
    • C12Y205/01Transferases transferring alkyl or aryl groups, other than methyl groups (2.5) transferring alkyl or aryl groups, other than methyl groups (2.5.1)
    • C12Y205/01061Hydroxymethylbilane synthase (2.5.1.61)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
MA045032A 2016-05-18 2017-05-18 Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë MA45032A (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662338161P 2016-05-18 2016-05-18
EP17382259 2017-05-09

Publications (1)

Publication Number Publication Date
MA45032A true MA45032A (fr) 2019-03-27

Family

ID=67223874

Family Applications (1)

Application Number Title Priority Date Filing Date
MA045032A MA45032A (fr) 2016-05-18 2017-05-18 Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë

Country Status (3)

Country Link
US (2) US20200085916A1 (fr)
JP (2) JP2019519601A (fr)
MA (1) MA45032A (fr)

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US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
WO2017031232A1 (fr) 2015-08-17 2017-02-23 Modernatx, Inc. Procédés de préparation de particules et compositions associées
WO2017070624A1 (fr) 2015-10-22 2017-04-27 Modernatx, Inc. Vaccins contre des maladies tropicales
JP6921833B2 (ja) 2015-10-22 2021-08-18 モデルナティーエックス, インコーポレイテッド ヒトサイトメガロウイルスワクチン
JP6925688B2 (ja) 2015-10-22 2021-08-25 モデルナティーエックス, インコーポレイテッド 水痘帯状疱疹ウイルス(vzv)のための核酸ワクチン
MD3718565T2 (ro) 2015-10-22 2022-09-30 Modernatx Inc Vaccinuri împotriva virusului respirator
MD3386484T2 (ro) 2015-12-10 2022-11-30 Modernatx Inc Compoziții și metode de livrare a unor agenți terapeutici
CA3007108A1 (fr) 2015-12-17 2017-06-22 Modernatx, Inc. Polynucleotides codant pour la methylmalonyl-coa mutase
MA45036A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2
US11801227B2 (en) 2016-05-18 2023-10-31 Modernatx, Inc. Polynucleotides encoding 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
CA3036831A1 (fr) 2016-09-14 2018-03-22 Modernatx, Inc. Compositions d'arn de haute purete et procedes pour leur preparation
WO2018075980A1 (fr) 2016-10-21 2018-04-26 Modernatx, Inc. Vaccin contre le cytomégalovirus humain
US10925958B2 (en) 2016-11-11 2021-02-23 Modernatx, Inc. Influenza vaccine
WO2018107088A2 (fr) 2016-12-08 2018-06-14 Modernatx, Inc. Vaccins à acide nucléique contre des virus respiratoires
US11384352B2 (en) 2016-12-13 2022-07-12 Modernatx, Inc. RNA affinity purification
WO2018170245A1 (fr) 2017-03-15 2018-09-20 Modernatx, Inc. Vaccin à large spectre contre le virus de la grippe
US11752206B2 (en) * 2017-03-15 2023-09-12 Modernatx, Inc. Herpes simplex virus vaccine
US11045540B2 (en) 2017-03-15 2021-06-29 Modernatx, Inc. Varicella zoster virus (VZV) vaccine
US11464848B2 (en) 2017-03-15 2022-10-11 Modernatx, Inc. Respiratory syncytial virus vaccine
EP3595676A4 (fr) 2017-03-17 2021-05-05 Modernatx, Inc. Vaccins à base d'arn contre des maladies zoonotiques
EP3607074A4 (fr) 2017-04-05 2021-07-07 Modernatx, Inc. Réduction ou élimination de réponses immunitaires à des protéines thérapeutiques administrées par voie non intraveineuse, par exemple par voie sous-cutanée
WO2018206125A1 (fr) * 2017-05-09 2018-11-15 Fundacion Para La Investigacion Medica Aplicada Protéines dérivées de la porphobilinogène désaminase humaine et polynucléotides et leurs utilisations
WO2018232357A1 (fr) 2017-06-15 2018-12-20 Modernatx, Inc. Formulations d'arn
WO2019036685A1 (fr) 2017-08-18 2019-02-21 Modernatx, Inc. Procédés pour analyse par clhp
CN111212905A (zh) 2017-08-18 2020-05-29 摩登纳特斯有限公司 Rna聚合酶变体
US11866696B2 (en) 2017-08-18 2024-01-09 Modernatx, Inc. Analytical HPLC methods
WO2019046809A1 (fr) 2017-08-31 2019-03-07 Modernatx, Inc. Procédés de fabrication de nanoparticules lipidiques
EP3681514A4 (fr) 2017-09-14 2021-07-14 ModernaTX, Inc. Vaccins à arn contre le virus zika
MA54676A (fr) 2018-01-29 2021-11-17 Modernatx Inc Vaccins à base d'arn contre le vrs
US11851694B1 (en) 2019-02-20 2023-12-26 Modernatx, Inc. High fidelity in vitro transcription
WO2021204179A1 (fr) 2020-04-09 2021-10-14 Suzhou Abogen Biosciences Co., Ltd. Vaccins à base d'acide nucléique pour coronavirus
TW202204309A (zh) 2020-04-09 2022-02-01 大陸商蘇州艾博生物科技有限公司 脂質奈米粒子組成物
WO2021213924A1 (fr) 2020-04-22 2021-10-28 BioNTech SE Vaccin contre un coronavirus
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
CN114469996B (zh) * 2021-12-23 2023-10-20 中国医学科学院医学生物学研究所 一种包含miR-135b-5p的外泌体及在抗轮状病毒感染中的应用
WO2023196615A1 (fr) * 2022-04-07 2023-10-12 RNAimmune, Inc. Composés et compositions pour l'administration de médicaments
WO2024002985A1 (fr) 2022-06-26 2024-01-04 BioNTech SE Vaccin contre le coronavirus

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2507540T3 (es) * 2008-09-29 2014-10-15 Uniqure Ip B.V. Terapia génica de porfobilinógeno deaminasa
JP2013095755A (ja) * 2011-11-02 2013-05-20 Kyowa Hakko Kirin Co Ltd カチオン性脂質
WO2013151667A1 (fr) * 2012-04-02 2013-10-10 modeRNA Therapeutics Polynucléotides modifiés

Also Published As

Publication number Publication date
JP2023062097A (ja) 2023-05-02
US20200085916A1 (en) 2020-03-19
US20230112986A1 (en) 2023-04-13
JP2019519601A (ja) 2019-07-11

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