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

    • 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
    • A61K38/45—Transferases (2)
    • 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
    • A61K48/0033—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 non-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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/08—Plasma substitutes; Perfusion solutions; Dialytics or haemodialytics; Drugs for electrolytic or acid-base disorders, e.g. hypovolemic shock
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y205/00—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5)
    • C12Y205/01—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5) transferring alkyl or aryl groups, other than methyl groups (2.5.1)
    • C12Y205/01061—Hydroxymethylbilane 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes 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)

Families Citing this family (61)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HRP20220070T1 (hr) 2014-04-23 2022-04-01 Modernatx, Inc. Cjepiva nukleinske kiseline
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
EP3328394A4 (fr) 2015-07-30 2019-03-13 ModernaTX, Inc. Arn épitope peptidiques concatémériques
WO2017031232A1 (fr) 2015-08-17 2017-02-23 Modernatx, Inc. Procédés de préparation de particules et compositions associées
EP3364949A4 (fr) 2015-10-22 2019-07-31 ModernaTX, Inc. Vaccins anticancéreux
EP3364981A4 (fr) 2015-10-22 2019-08-07 ModernaTX, Inc. Vaccin contre le cytomégalovirus humain
DE20164728T1 (de) 2015-10-22 2021-09-30 Modernatx, Inc. Impfstoffe gegen atemwegsvirus
SG11201803360UA (en) 2015-10-22 2018-05-30 Modernatx Inc Nucleic acid vaccines for varicella zoster virus (vzv)
WO2017070624A1 (fr) 2015-10-22 2017-04-27 Modernatx, Inc. Vaccins contre des maladies tropicales
DK3386484T3 (da) 2015-12-10 2022-07-04 Modernatx Inc Sammensætninger og fremgangsmåder til afgivelse af terapeutiske midler
WO2017106799A1 (fr) 2015-12-17 2017-06-22 Modernatx, Inc. Polynucléotides codant pour la méthylmalonyl-coa mutase
US11801227B2 (en) 2016-05-18 2023-10-31 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
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
US11001861B2 (en) 2016-05-18 2021-05-11 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
EP3458106A4 (fr) 2016-05-18 2020-03-18 Modernatx, Inc. Polynucléotides codant pour la lipoprotéine lipase destinés au traitement de l'hyperlipidémie
AU2017268396B2 (en) 2016-05-18 2023-05-18 Modernatx, Inc. Polynucleotides encoding citrin for the treatment of citrullinemia type 2
US12128113B2 (en) 2016-05-18 2024-10-29 Modernatx, Inc. Polynucleotides encoding JAGGED1 for the treatment of Alagille syndrome
SG11201901941YA (en) 2016-09-14 2019-04-29 Modernatx Inc High purity rna compositions and methods for preparation thereof
EP3528821A4 (fr) 2016-10-21 2020-07-01 ModernaTX, Inc. Vaccin contre le cytomégalovirus humain
MA46766A (fr) 2016-11-11 2019-09-18 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
WO2018170260A1 (fr) 2017-03-15 2018-09-20 Modernatx, Inc. Vaccin contre le virus respiratoire syncytial
US11576961B2 (en) 2017-03-15 2023-02-14 Modernatx, Inc. Broad spectrum influenza virus vaccine
WO2018170256A1 (fr) * 2017-03-15 2018-09-20 Modernatx, Inc. Vaccin contre le virus de l'herpès simplex
RS63953B1 (sr) * 2017-03-15 2023-02-28 Modernatx Inc Jedinjenje i kompozicije za intracelularnu isporuku terapeutskih sredstava
WO2018170270A1 (fr) 2017-03-15 2018-09-20 Modernatx, Inc. Vaccin contre le virus varicelle-zona
WO2018170347A1 (fr) 2017-03-17 2018-09-20 Modernatx, Inc. Vaccins à base d'arn contre des maladies zoonotiques
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
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
MA49421A (fr) 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
WO2019036685A1 (fr) 2017-08-18 2019-02-21 Modernatx, Inc. Procédés pour analyse par clhp
EP3668977A4 (fr) 2017-08-18 2021-04-21 Modernatx, Inc. Procédés analytiques par hplc
WO2019036682A1 (fr) 2017-08-18 2019-02-21 Modernatx, Inc. Variants d'arn polymérase
MX2020002348A (es) 2017-08-31 2020-10-08 Modernatx Inc Métodos de elaboración de nanopartículas lipídicas.
EP3681514A4 (fr) 2017-09-14 2021-07-14 ModernaTX, Inc. Vaccins à arn contre le virus zika
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
US11911453B2 (en) 2018-01-29 2024-02-27 Modernatx, Inc. RSV RNA vaccines
CN112996854B (zh) 2018-09-19 2024-08-30 摩登纳特斯有限公司 高纯度peg脂质和其用途
US12151029B2 (en) 2018-09-19 2024-11-26 Modernatx, Inc. PEG lipids and uses thereof
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
CN113939282A (zh) 2019-01-31 2022-01-14 摩登纳特斯有限公司 制备脂质纳米颗粒的方法
US11851694B1 (en) 2019-02-20 2023-12-26 Modernatx, Inc. High fidelity in vitro transcription
MX2021010075A (es) 2019-02-20 2021-12-10 Modernatx Inc Variantes de arn polimerasa para la formacion de casquetes cotranscripcionales.
MX2021011031A (es) 2019-03-11 2021-12-10 Modernatx Inc Proceso de transcripción in vitro en lotes alimentados.
MA55321A (fr) 2019-03-15 2022-01-19 Modernatx Inc Vaccins à base d'arn contre le vih
CN115667207B (zh) * 2020-04-01 2025-09-16 翻译生物公司 基于酚酸脂质的阳离子脂质
WO2021204179A1 (fr) 2020-04-09 2021-10-14 Suzhou Abogen Biosciences Co., Ltd. Vaccins à base d'acide nucléique pour coronavirus
TWI899203B (zh) 2020-04-09 2025-10-01 大陸商蘇州艾博生物科技有限公司 脂質奈米粒子組成物
CN118490818A (zh) 2020-04-22 2024-08-16 生物技术欧洲股份公司 冠状病毒疫苗
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
MX2023004371A (es) 2020-10-14 2023-07-26 George Mason Res Foundation Inc Lípidos ionizables, y métodos de fabricación y uso de los mismos.
WO2022150717A1 (fr) 2021-01-11 2022-07-14 Modernatx, Inc. Vaccins contre le virus de la grippe à arn saisonnière
EP4326227A1 (fr) 2021-04-23 2024-02-28 ModernaTX, Inc. Formulations stabilisées
US20220363937A1 (en) 2021-05-14 2022-11-17 Armstrong World Industries, Inc. Stabilization of antimicrobial coatings
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
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
TW202404568A (zh) * 2022-04-07 2024-02-01 美商Rna免疫公司 用於藥物遞送之化合物及組合物
US11878055B1 (en) 2022-06-26 2024-01-23 BioNTech SE Coronavirus vaccine
KR102921785B1 (ko) * 2022-12-19 2026-02-03 주식회사 녹십자 핵산 전달용 사이클로알칸계 지질 화합물 및 이를 포함하는 지질 나노입자

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE217799T1 (de) 1998-01-27 2002-06-15 Hemebiotech As Behandlung von akuter intermittierender porphyria oder anderen porphyrischen krankheiten
AU774603B2 (en) 1999-07-27 2004-07-01 Zymenex A/S Production of rhPBGD and new therapeutic methods for treating patients with acute intermittent porphyria (AIP) and other porphyric diseases
KR20110086553A (ko) * 2008-09-29 2011-07-28 암스테르담 몰레큘러 테라퓨틱스 비. 브이. 포르포빌리노겐 디아미나아제 유전자 치료
SI2506857T1 (en) 2009-12-01 2018-08-31 Translate Bio, Inc. Delivery of mRNA for the enrichment of proteins and enzymes in human genetic diseases
JP2013095755A (ja) 2011-11-02 2013-05-20 Kyowa Hakko Kirin Co Ltd カチオン性脂質
US20140308304A1 (en) 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
WO2013151664A1 (fr) 2012-04-02 2013-10-10 modeRNA Therapeutics Polynucléotides modifiés pour la production de protéines
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
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US20140275229A1 (en) 2012-04-02 2014-09-18 Moderna Therapeutics, Inc. Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1
US8980864B2 (en) * 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
CA2953341C (fr) 2014-06-25 2023-01-24 Acuitas Therapeutics Inc. Lipides et formulations de nanoparticules de lipides pour l'administration d'acides nucleiques
HRP20220156T1 (hr) 2015-09-17 2022-04-15 Modernatx, Inc. Spojevi i pripravci za unutarstaničnu isporuku terapeutskih sredstava
CN108368028B (zh) * 2015-10-28 2021-09-03 爱康泰生治疗公司 用于递送核酸的新型脂质和脂质纳米颗粒制剂
WO2017106799A1 (fr) 2015-12-17 2017-06-22 Modernatx, Inc. Polynucléotides codant pour la méthylmalonyl-coa mutase
EP3458106A4 (fr) 2016-05-18 2020-03-18 Modernatx, Inc. Polynucléotides codant pour la lipoprotéine lipase destinés au traitement de l'hyperlipidémie
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
AU2017268396B2 (en) 2016-05-18 2023-05-18 Modernatx, Inc. Polynucleotides encoding citrin for the treatment of citrullinemia type 2
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
WO2017201332A1 (fr) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucléotides codant pour l'acyl-coa déshydrogénase, à très longue chaîne pour le traitement de l'insuffisance en acyl-coa déshydrogénase à très longue chaîne

Also Published As

Publication number Publication date
US20200085916A1 (en) 2020-03-19
JP2023062097A (ja) 2023-05-02
US12377136B2 (en) 2025-08-05
JP2019519601A (ja) 2019-07-11
US20230112986A1 (en) 2023-04-13

Similar Documents

Publication Publication Date Title
MA45036A (fr) Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2
EP3458106A4 (fr) Polynucléotides codant pour la lipoprotéine lipase destinés au traitement de l'hyperlipidémie
MA45041A (fr) Polynucléotides codant pour la galactose-1-phosphate uridylyltransférase destinés au traitement de la galactosémie de type 1
MA47719A (fr) Esketamine pour le traitement de la dépression
EP3349751A4 (fr) Polythérapie faisant appel au cénicriviroc pour le traitement de la fibrose
EP3370721A4 (fr) Traitement de l'ostéoarthrite
MA45192A (fr) Traitement d'association
EP3362065A4 (fr) Polythérapie pour le traitement de malignités
EP3452060A4 (fr) Polythérapie pour le traitement du cancer
MA41449A (fr) Polythérapies pour le traitement de cancers
EP3351189A4 (fr) Instrument de traitement pour endoscope
EP3400011A4 (fr) Combinaisons anti-cd20 pour le traitement des tumeurs
EP3400010A4 (fr) Combinaisons anti-her2 pour le traitement des tumeurs
MA50801A (fr) Polynucléotides codant pour la phénylalanine hydroxylase pour le traitement de la phénylcétonurie
EP3380200A4 (fr) Compositions pour le traitement des cheveux
EP3335041A4 (fr) Biomarqueurs pour le traitement de la pelade
MA43000A (fr) Polythérapie pour le traitement de tumeurs malignes
MA46524A (fr) Composés de biphényl-sulfonamide destinés au traitement de néphropathies
EP3389645A4 (fr) Combinaisons pour le traitement du cancer
EP3328374A4 (fr) Antagonistes de la glutamine pour le traitement de déficits cognitifs
EP3380063A4 (fr) Compositions pour le traitement des cheveux
EP3490561A4 (fr) Combinaisons pour le traitement du cancer
EP3454793A4 (fr) Bobines de traitement d'anévrisme
EP3345002A4 (fr) Sélection de patients pour une polythérapie
EP3386967A4 (fr) Combinaisons pour le traitement de calculs rénaux