BR112021024534A2 - Métodos para o tratamento da deficiência de alfa-1 antitripsina (aatd) - Google Patents
Métodos para o tratamento da deficiência de alfa-1 antitripsina (aatd)Info
- Publication number
- BR112021024534A2 BR112021024534A2 BR112021024534A BR112021024534A BR112021024534A2 BR 112021024534 A2 BR112021024534 A2 BR 112021024534A2 BR 112021024534 A BR112021024534 A BR 112021024534A BR 112021024534 A BR112021024534 A BR 112021024534A BR 112021024534 A2 BR112021024534 A2 BR 112021024534A2
- Authority
- BR
- Brazil
- Prior art keywords
- aatd
- treatment
- methods
- alpha
- deficiency
- Prior art date
Links
Classifications
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
- A61K47/02—Inorganic compounds
-
- 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
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- 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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Gastroenterology & Hepatology (AREA)
- Dermatology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Pulmonology (AREA)
- Inorganic Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
métodos para o tratamento da deficiência de alfa-1 antitripsina (aatd). são descritos métodos para o tratamento da deficiência de alfa-1 antitripsina (aatd) em um paciente humano em necessidade de tratamento, usando composições farmacêuticas que incluem agentes aat rnai. as composições farmacêuticas divulgadas neste documento que incluem agentes aat rnai, quando administradas a um paciente humano em necessidade do mesmo, tratam doenças hepáticas associadas à deficiência de aat, como hepatite crônica, cirrose, risco aumentado de carcinoma hepatocelular, transaminite, colestase, fibrose, insuficiência hepática fulminante e outras doenças relacionadas com o fígado.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962858059P | 2019-06-06 | 2019-06-06 | |
PCT/US2020/036359 WO2020247774A1 (en) | 2019-06-06 | 2020-06-05 | Methods for the treatment of alpha-1 antitrypsin deficiency (aatd) |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112021024534A2 true BR112021024534A2 (pt) | 2022-02-08 |
Family
ID=73652297
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112021024534A BR112021024534A2 (pt) | 2019-06-06 | 2020-06-05 | Métodos para o tratamento da deficiência de alfa-1 antitripsina (aatd) |
Country Status (12)
Country | Link |
---|---|
US (1) | US20220305046A1 (pt) |
EP (1) | EP3980540A4 (pt) |
JP (1) | JP2022535127A (pt) |
KR (1) | KR20220019255A (pt) |
CN (1) | CN114222820A (pt) |
AU (1) | AU2020286462A1 (pt) |
BR (1) | BR112021024534A2 (pt) |
CA (1) | CA3141762A1 (pt) |
CO (1) | CO2022000016A2 (pt) |
MX (1) | MX2021015003A (pt) |
TW (1) | TW202113081A (pt) |
WO (1) | WO2020247774A1 (pt) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
KR20190098748A (ko) | 2017-01-10 | 2019-08-22 | 애로우헤드 파마슈티컬스 인코포레이티드 | 알파-1 항트립신 (AAT) RNAi 작용제, AAT RNAi 작용제를 포함하는 조성물, 및 사용 방법 |
MX2023003066A (es) * | 2020-09-15 | 2023-04-10 | Arrowhead Pharmaceuticals Inc | Metodos para la reduccion de los niveles de proteina alfa-1 antitripsina z (z-aat). |
US20230107967A1 (en) * | 2021-08-25 | 2023-04-06 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting alpha-1 antitrypsin expression |
WO2024044542A2 (en) * | 2022-08-22 | 2024-02-29 | Arrowhead Pharmaceuticals, Inc. | Methods for the treatment of angptl3-related diseases and disorders |
Family Cites Families (10)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
NL9201440A (nl) | 1992-08-11 | 1994-03-01 | Univ Leiden | Triantennaire clusterglycosiden, hun bereiding en toepassing. |
US20050137153A1 (en) * | 2002-02-20 | 2005-06-23 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA) |
US8765700B2 (en) * | 2004-10-20 | 2014-07-01 | Antisense Therapeutics Ltd. | Topical administrations of antisense compounds to VLA-4 for the treatment of respiratory conditions |
TW201620526A (zh) * | 2014-06-17 | 2016-06-16 | 愛羅海德研究公司 | 用於抑制α-1抗胰蛋白酶基因表現之組合物及方法 |
WO2017139616A1 (en) * | 2016-02-10 | 2017-08-17 | Arrowhead Pharmaceuticals, Inc. | Methods of treatment for alpha-1 antitrypsin deficiency |
UY37146A (es) | 2016-03-07 | 2017-09-29 | Arrowhead Pharmaceuticals Inc | Ligandos de direccionamiento para compuestos terapéuticos |
JP2019518028A (ja) * | 2016-06-10 | 2019-06-27 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | 補体成分C5iRNA組成物及び発作性夜間血色素尿症(PNH)を処置するためのその使用方法 |
KR20230115344A (ko) | 2016-09-02 | 2023-08-02 | 애로우헤드 파마슈티컬스 인코포레이티드 | 표적화 리간드 |
KR20190098748A (ko) * | 2017-01-10 | 2019-08-22 | 애로우헤드 파마슈티컬스 인코포레이티드 | 알파-1 항트립신 (AAT) RNAi 작용제, AAT RNAi 작용제를 포함하는 조성물, 및 사용 방법 |
SE1750774A1 (en) * | 2017-06-19 | 2018-12-11 | Hepgene Medical Ab | Novel nucleic acid molecules and their use in therapy |
-
2020
- 2020-06-05 AU AU2020286462A patent/AU2020286462A1/en active Pending
- 2020-06-05 TW TW109119068A patent/TW202113081A/zh unknown
- 2020-06-05 WO PCT/US2020/036359 patent/WO2020247774A1/en active Application Filing
- 2020-06-05 CA CA3141762A patent/CA3141762A1/en active Pending
- 2020-06-05 BR BR112021024534A patent/BR112021024534A2/pt unknown
- 2020-06-05 CN CN202080041338.5A patent/CN114222820A/zh active Pending
- 2020-06-05 US US17/616,292 patent/US20220305046A1/en active Pending
- 2020-06-05 KR KR1020227000043A patent/KR20220019255A/ko unknown
- 2020-06-05 EP EP20818171.9A patent/EP3980540A4/en active Pending
- 2020-06-05 MX MX2021015003A patent/MX2021015003A/es unknown
- 2020-06-05 JP JP2021572103A patent/JP2022535127A/ja active Pending
-
2022
- 2022-01-04 CO CONC2022/0000016A patent/CO2022000016A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
CA3141762A1 (en) | 2020-12-10 |
JP2022535127A (ja) | 2022-08-04 |
KR20220019255A (ko) | 2022-02-16 |
TW202113081A (zh) | 2021-04-01 |
CO2022000016A2 (es) | 2022-04-08 |
WO2020247774A1 (en) | 2020-12-10 |
MX2021015003A (es) | 2022-01-24 |
US20220305046A1 (en) | 2022-09-29 |
CN114222820A (zh) | 2022-03-22 |
AU2020286462A1 (en) | 2022-01-27 |
EP3980540A4 (en) | 2023-11-01 |
EP3980540A1 (en) | 2022-04-13 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
BR112021024534A2 (pt) | Métodos para o tratamento da deficiência de alfa-1 antitripsina (aatd) | |
JOP20180003B1 (ar) | عوامل RNAi ألفا-1 مضادة للتريبسين (AAT)، وتركيبات تتضمن عوامل AAT RNAi، وطرق الاستخدام | |
BR112018002046A2 (pt) | métodos de tratamento da síndrome de lennox-gastaut usando fenfluramina | |
BR112018000639A2 (pt) | sistema de cateter funcionalmente integrável | |
BR112018016001A2 (pt) | composição farmacêutica, métodos de tratamento e usos da mesma | |
PH12020551425A1 (en) | Rimegepant for cgrp related disorders | |
BR112018000641A2 (pt) | ?sistema de cateter funcionalmente integrável? | |
PA8744101A1 (es) | Metodo de terapia combinada para el tratamiento de la infeccion por el virus de la hepatitis c y composiciones farmaceuticas para usar en dicha terapia | |
CL2018001548A1 (es) | Tratamiento de colestasis intrahepática y enfermedades del hígado relacionadas | |
MX2021010145A (es) | Derivados de amida utiles en el tratamiento de una infeccion por vhb o de enfermedades inducidas por vhb. | |
MX2023004188A (es) | Compuestos fosfolipidos y usos de estos. | |
BR112021024845A2 (pt) | Combinação para o tratamento de infecções causadas por mycoplasma genitalium | |
NO20064753L (no) | Kombinasjonsterapi | |
BR112019004791A2 (pt) | tratamento de esclerose múltipla com chs-131 | |
BR112016029437A2 (pt) | métodos para tratamento e prevenção de doenças de instabilidade vascular | |
BR112019002945A2 (pt) | terapia de combinação para o tratamento de câncer pancreático | |
MX2021004207A (es) | Uso de reboxetina para el tratamiento de narcolepsia. | |
WO2016090107A3 (en) | Treatment of hepatitis delta virus infection | |
MX2023007826A (es) | Metodos que comprenden una dosificacion intermitente fija de cediranib. | |
MX2022001261A (es) | Derivados de dihidropirimidina y usos de los mismos en el tratamiento de infeccion por vhb o de enfermedades inducidas por vhb. | |
BR112023004756A2 (pt) | Métodos para a redução de níveis de proteína z-aat | |
BR112022003687A2 (pt) | Método para tratar tinnitus, composição farmacêutica para administração intranasal, e, dose unitária pulverizável de ocitocina | |
WO2016122288A3 (ko) | 베르베논 유도체 및 재관류 치료제를 포함하는 뇌혈관 질환, 동맥경화증 또는 심혈관 질환 치료 또는 예방용 병용 제제 | |
BR112022008639A2 (pt) | Tratamento de distúrbios do fígado | |
MX2019013014A (es) | Tratamiento de carcinoma hepatocelular. |