BR112014029807A2 - administração pulmonar de rna mensageiro - Google Patents
administração pulmonar de rna mensageiroInfo
- Publication number
- BR112014029807A2 BR112014029807A2 BR112014029807A BR112014029807A BR112014029807A2 BR 112014029807 A2 BR112014029807 A2 BR 112014029807A2 BR 112014029807 A BR112014029807 A BR 112014029807A BR 112014029807 A BR112014029807 A BR 112014029807A BR 112014029807 A2 BR112014029807 A2 BR 112014029807A2
- Authority
- BR
- Brazil
- Prior art keywords
- rnam
- pulmonary administration
- messenger rna
- pei
- lung
- Prior art date
Links
- 230000002685 pulmonary effect Effects 0.000 title abstract 2
- 108020004999 messenger RNA Proteins 0.000 title 1
- 210000004072 lung Anatomy 0.000 abstract 2
- 210000005265 lung cell Anatomy 0.000 abstract 2
- 229920002873 Polyethylenimine Polymers 0.000 abstract 1
Classifications
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- 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
- A61K48/0041—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 polymeric
-
- 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
- A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
-
- 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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- 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
- A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/56—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
- A61K47/59—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
-
- 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
- A61P11/06—Antiasthmatics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/04—Antibacterial agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
- A61P31/16—Antivirals for RNA viruses for influenza or rhinoviruses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- 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
-
- 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
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/12—Antihypertensives
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Genetics & Genomics (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Biomedical Technology (AREA)
- Pulmonology (AREA)
- Communicable Diseases (AREA)
- Oncology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Cardiology (AREA)
- Diabetes (AREA)
- Heart & Thoracic Surgery (AREA)
- Hematology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261657344P | 2012-06-08 | 2012-06-08 | |
| PCT/EP2013/061811 WO2013182683A1 (en) | 2012-06-08 | 2013-06-07 | Pulmonary delivery of messenger rna |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112014029807A2 true BR112014029807A2 (pt) | 2017-06-27 |
Family
ID=48782278
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112014029807A BR112014029807A2 (pt) | 2012-06-08 | 2013-06-07 | administração pulmonar de rna mensageiro |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20150126589A1 (enExample) |
| EP (2) | EP3800254A1 (enExample) |
| JP (1) | JP6208750B2 (enExample) |
| KR (2) | KR102087643B1 (enExample) |
| CN (1) | CN104349794B (enExample) |
| AU (1) | AU2013273504B2 (enExample) |
| BR (1) | BR112014029807A2 (enExample) |
| CA (1) | CA2873274C (enExample) |
| DK (1) | DK2858677T3 (enExample) |
| EA (1) | EA037448B1 (enExample) |
| ES (1) | ES2826203T3 (enExample) |
| IN (1) | IN2014DN09744A (enExample) |
| WO (1) | WO2013182683A1 (enExample) |
| ZA (1) | ZA201408184B (enExample) |
Families Citing this family (87)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RS58405B1 (sr) | 2009-12-01 | 2019-04-30 | Translate Bio Inc | Stereoidni derivati za isporuku irnk u humanim genetskim oboljenjima |
| EP2338520A1 (de) | 2009-12-21 | 2011-06-29 | Ludwig Maximilians Universität | Konjugat mit Zielfindungsligand und dessen Verwendung |
| US8822663B2 (en) | 2010-08-06 | 2014-09-02 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| CA2821992A1 (en) | 2010-10-01 | 2012-04-05 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| WO2012116715A1 (en) * | 2011-03-02 | 2012-09-07 | Curevac Gmbh | Vaccination in newborns and infants |
| WO2012116714A1 (en) | 2011-03-02 | 2012-09-07 | Curevac Gmbh | Vaccination in elderly patients |
| EP2680881B1 (en) * | 2011-03-02 | 2017-04-05 | CureVac AG | Vaccination in elderly patients |
| DE12722942T1 (de) | 2011-03-31 | 2021-09-30 | Modernatx, Inc. | Freisetzung und formulierung von manipulierten nukleinsäuren |
| JP6184945B2 (ja) | 2011-06-08 | 2017-08-23 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | ｍＲＮＡ送達のための脂質ナノ粒子組成物および方法 |
| US9464124B2 (en) | 2011-09-12 | 2016-10-11 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| HRP20220250T1 (hr) | 2011-10-03 | 2022-04-29 | Modernatx, Inc. | Modificirani nukleozidi, nukleotidi i nukleinske kiseline, te njihove uporabe |
| SI2791160T1 (sl) | 2011-12-16 | 2022-07-29 | Modernatx, Inc. | Sestave modificirane MRNA |
| US9283287B2 (en) | 2012-04-02 | 2016-03-15 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of nuclear proteins |
| US10501512B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides |
| US9572897B2 (en) | 2012-04-02 | 2017-02-21 | Modernatx, Inc. | Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins |
| AU2013243952A1 (en) | 2012-04-02 | 2014-10-30 | Modernatx, Inc. | Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins |
| US20150267192A1 (en) | 2012-06-08 | 2015-09-24 | Shire Human Genetic Therapies, Inc. | Nuclease resistant polynucleotides and uses thereof |
| SMT202200337T1 (it) | 2012-11-26 | 2022-09-14 | Modernatx Inc | Rna modificato al livello del terminale |
| WO2014153052A2 (en) | 2013-03-14 | 2014-09-25 | Shire Human Genetic Therapies, Inc. | Cftr mrna compositions and related methods and uses |
| US8980864B2 (en) | 2013-03-15 | 2015-03-17 | Moderna Therapeutics, Inc. | Compositions and methods of altering cholesterol levels |
| US10023626B2 (en) | 2013-09-30 | 2018-07-17 | Modernatx, Inc. | Polynucleotides encoding immune modulating polypeptides |
| US10323076B2 (en) | 2013-10-03 | 2019-06-18 | Modernatx, Inc. | Polynucleotides encoding low density lipoprotein receptor |
| MX2016005239A (es) | 2013-10-22 | 2016-08-12 | Shire Human Genetic Therapies | Tratamiento con acido ribonucleico mensajero para la fenilcetonuria. |
| BR112016009014B1 (pt) | 2013-10-22 | 2024-02-06 | Translate Bio, Inc | USO DE COMPOSIÇÃO COMPREENDENDO mRNA PARA DEFICIÊNCIA DE ARGININOSSUCINATO SINTETASE |
| SI3116900T1 (sl) | 2014-03-09 | 2021-02-26 | The Trustees Of The University Of Pennsylvania | Sestavki uporabni pri zdravljenju pomanjkanja ornitin transkarbamilaze(OTC) |
| SG11201608725YA (en) * | 2014-04-25 | 2016-11-29 | Shire Human Genetic Therapies | Methods for purification of messenger rna |
| US9943595B2 (en) * | 2014-12-05 | 2018-04-17 | Translate Bio, Inc. | Messenger RNA therapy for treatment of articular disease |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| US20180110877A1 (en) | 2015-04-27 | 2018-04-26 | The Trustees Of The University Of Pennsylvania | DUAL AAV VECTOR SYSTEM FOR CRISPR/Cas9 MEDIATED CORRECTION OF HUMAN DISEASE |
| ES2667644T3 (es) | 2015-06-30 | 2018-05-14 | Ethris Gmbh | UTR que aumentan la eficacia de la traducción de las moléculas de ARN |
| CA2990883A1 (en) * | 2015-06-30 | 2017-01-05 | Ethris Gmbh | Atp-binding cassette family coding polyribonucleotides and formulations thereof |
| WO2017015637A1 (en) | 2015-07-22 | 2017-01-26 | Duke University | High-throughput screening of regulatory element function with epigenome editing technologies |
| KR102699944B1 (ko) | 2015-08-25 | 2024-09-13 | 듀크 유니버시티 | Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법 |
| WO2017066497A2 (en) | 2015-10-13 | 2017-04-20 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| EP4349405A3 (en) | 2015-10-22 | 2024-06-19 | ModernaTX, Inc. | Respiratory virus vaccines |
| ES2987312T3 (es) * | 2015-12-30 | 2024-11-14 | Visiongate Inc | Sistema y procedimiento para la detección y el seguimiento automatizados de displasia |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| EP3458108A4 (en) | 2016-05-18 | 2020-04-22 | ModernaTX, Inc. | POLYNUCLEOTIDES ENCODING A CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR FOR THE TREATMENT OF CYSTIC FIBROSIS |
| CA3025626A1 (en) * | 2016-05-27 | 2017-11-30 | Transcriptx, Inc. | Treatment of primary ciliary dyskinesia with synthetic messenger rna |
| US10927383B2 (en) | 2016-06-30 | 2021-02-23 | Ethris Gmbh | Cas9 mRNAs |
| WO2018010815A1 (en) * | 2016-07-15 | 2018-01-18 | Biontech Rna Pharmaceuticals Gmbh | Formulation for administration of rna |
| EP4275747A3 (en) | 2016-07-19 | 2024-01-24 | Duke University | Therapeutic applications of cpf1-based genome editing |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| MA50335A (fr) | 2016-12-08 | 2020-08-19 | Modernatx Inc | Vaccins à acide nucléique contre des virus respiratoires |
| EP3565605A1 (en) | 2017-01-03 | 2019-11-13 | ethris GmbH | Ornithine transcarbamylase coding polyribonucleotides and formulations thereof |
| AU2018224326B2 (en) | 2017-02-27 | 2024-01-04 | Translate Bio, Inc. | Novel codon-optimized CFTR mRNA |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| US11827906B2 (en) | 2017-02-28 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clade f vector and uses therefor |
| KR20230093072A (ko) | 2017-03-01 | 2023-06-26 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 안구 장애에 대한 유전자 치료 |
| EP3634986A4 (en) | 2017-04-24 | 2021-09-08 | The Trustees of The University of Pennsylvania | GENE THERAPY FOR EYE DISORDERS |
| CA3061655A1 (en) | 2017-05-11 | 2018-11-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for neuronal ceroid lipofuscinoses |
| IL270631B2 (en) | 2017-05-16 | 2024-03-01 | Translate Bio Inc | Treatment of cystic fibrosis through the administration of mRNA with an optimal codon encoding ctfr |
| EP3630986A4 (en) | 2017-05-31 | 2021-08-11 | The Trustees of The University of Pennsylvania | GENE THERAPY FOR TREATMENT OF PEROXISOMAL DISEASES |
| KR20200104864A (ko) | 2017-11-30 | 2020-09-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 뮤코다당류증 iiib형에 대한 유전자 요법 |
| JP7389744B2 (ja) | 2017-11-30 | 2023-11-30 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ムコ多糖症ｉｉｉａ型のための遺伝子療法 |
| ES2960939T3 (es) | 2018-04-25 | 2024-03-07 | Ethris Gmbh | Agentes crioprotectores para formulaciones particuladas |
| US20200157157A1 (en) | 2018-11-21 | 2020-05-21 | Translate Bio, Inc. | TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR |
| US11351242B1 (en) | 2019-02-12 | 2022-06-07 | Modernatx, Inc. | HMPV/hPIV3 mRNA vaccine composition |
| CN113423418B (zh) * | 2019-02-14 | 2025-01-10 | 埃泽瑞斯公司 | 纤毛疾病的治疗 |
| US11065260B1 (en) * | 2020-04-13 | 2021-07-20 | Alan C Nelson | Method of treatment for reducing pulmonary inflammation in a patient with pathogen infection using oral prostacyclin analog drugs |
| CN115803064A (zh) | 2020-05-12 | 2023-03-14 | 宾夕法尼亚州大学信托人 | 用于drg特异性降低转基因表达的组合物 |
| US20230220069A1 (en) | 2020-06-17 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of gene therapy patients |
| EP4179097A1 (en) | 2020-07-13 | 2023-05-17 | The Trustees of The University of Pennsylvania | Compositions useful for treatment of charcot-marie-tooth disease |
| AU2021324883A1 (en) * | 2020-08-12 | 2023-03-23 | Actym Therapeutics, Inc. | Immunostimulatory bacteria-based vaccines, therapeutics, and rna delivery platforms |
| IL301643A (en) | 2020-10-07 | 2023-05-01 | Regenxbio Inc | Gene therapy for ocular manifestations of CLN2 disease |
| KR20230118075A (ko) | 2020-10-09 | 2023-08-10 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 파브리병 치료를 위한 조성물 및 방법 |
| IL303239A (en) | 2020-12-01 | 2023-07-01 | Univ Pennsylvania | Compositions and their uses for the treatment of Engelmann syndrome |
| MX2023006444A (es) | 2020-12-01 | 2023-08-10 | Univ Pennsylvania | Composiciones novedosas con motivos dirigidos a tejido específico y composiciones que los contienen. |
| AU2022214429A1 (en) | 2021-02-01 | 2023-09-14 | Tern Therapeutics, Llc | Gene therapy for neuronal ceroid lipofuscinoses |
| KR20230170022A (ko) | 2021-04-12 | 2023-12-18 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물 |
| AR125406A1 (es) | 2021-04-23 | 2023-07-12 | Univ Pennsylvania | Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2023102517A1 (en) | 2021-12-02 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| TW202340467A (zh) | 2022-01-10 | 2023-10-16 | 賓州大學委員會 | 有用於治療c9orf72介導之病症之組成物及方法 |
| WO2023183300A1 (en) * | 2022-03-22 | 2023-09-28 | The Children's Medical Center Corporation | Compositions and methods for prevention and treatment of genetic disease |
| CN119731321A (zh) | 2022-06-24 | 2025-03-28 | 图恩疗法股份有限公司 | 通过靶向基因阻遏减少低密度脂蛋白的组合物、系统和方法 |
| WO2024121160A1 (en) | 2022-12-05 | 2024-06-13 | Ethris Gmbh | Regulator(s) of energy homeostasis-encoding rna molecule(s) with increased translation efficiency |
| EP4634394A2 (en) | 2022-12-17 | 2025-10-22 | The Trustees of The University of Pennsylvania | Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same |
| WO2024130070A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof |
| WO2024258961A1 (en) | 2023-06-12 | 2024-12-19 | The Trustees Of The University Of Pennsylvania | Aav gene therapy for mucopolysaccharidosis iiib |
| WO2025007046A1 (en) | 2023-06-29 | 2025-01-02 | The Trustees Of The University Of Pennsylvania | Mutant aav with central nervous system targeting motifs and compositions containing same |
| WO2025035143A1 (en) | 2023-08-10 | 2025-02-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of spinal muscular atrophy |
| WO2025102034A1 (en) | 2023-11-10 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for barth syndrome |
| WO2025106661A1 (en) | 2023-11-14 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof |
| WO2025129157A1 (en) | 2023-12-15 | 2025-06-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treatment of canavan disease |
| WO2025184170A1 (en) | 2024-02-27 | 2025-09-04 | Overt Bio, Inc. | Methods and compositions for improving t cell function in an immunosuppressive environment |
| WO2025184172A1 (en) | 2024-02-27 | 2025-09-04 | Overt Bio, Inc. | Cancer reactive t cell receptors |
Family Cites Families (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5756353A (en) * | 1991-12-17 | 1998-05-26 | The Regents Of The University Of California | Expression of cloned genes in the lung by aerosol-and liposome-based delivery |
| FR2722506B1 (fr) * | 1994-07-13 | 1996-08-14 | Rhone Poulenc Rorer Sa | Composition contenant des acides nucleiques, preparation et utilisations |
| AU4680699A (en) * | 1998-06-12 | 1999-12-30 | Aradigm Corporation | Methods of delivering aerosolized polynucleotides to the respiratory tract |
| IL145592A0 (en) | 1999-04-02 | 2002-06-30 | Res Dev Foundation | Polyethyleneimine: dna formulations for aerosol delivery |
| US20020086842A1 (en) * | 2000-06-26 | 2002-07-04 | Christian Plank | Method for transfecting cells using a magnetic field |
| EP1297169B1 (en) * | 2000-06-26 | 2012-08-08 | Ethris Gmbh | Method for transfecting cells using a magnetic field |
| EP2325193A3 (en) * | 2001-11-02 | 2012-05-02 | Insert Therapeutics, Inc. | Methods and compositions for therapeutic use of RNA interference |
| BRPI0407107A (pt) * | 2003-01-31 | 2006-01-24 | Novartis Ag | Infra-regulação de gene alvo com complexos de oligoribonucleotìdeo de filamento único e polìmero de polietilenoimina (pei) |
| GB0313259D0 (en) * | 2003-06-09 | 2003-07-16 | Consejo Superior Investigacion | Magnetic nanoparticles |
| EP1646684A4 (en) * | 2003-06-30 | 2010-09-29 | Canji Inc | POLYMER ENCAPSULATION OF ADENOVIRES |
| GB0411537D0 (en) * | 2004-05-24 | 2004-06-23 | Midatech Ltd | Nanoparticles comprising rna ligands |
| GB0508110D0 (en) * | 2005-04-22 | 2005-06-01 | Univ Keele | Gene delivery |
| ES2937245T3 (es) * | 2005-08-23 | 2023-03-27 | Univ Pennsylvania | ARN que contiene nucleósidos modificados y métodos de uso del mismo |
| DE102005042768A1 (de) * | 2005-09-08 | 2007-03-15 | Ludwig-Maximilian-Universität | Magnetfeld-gesteuerter Wirkstofftransfer für die Aerosoltherapie |
| WO2009127230A1 (en) * | 2008-04-16 | 2009-10-22 | Curevac Gmbh | MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION |
| WO2010037408A1 (en) * | 2008-09-30 | 2010-04-08 | Curevac Gmbh | Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof |
| JPWO2010110314A1 (ja) * | 2009-03-27 | 2012-10-04 | 協和発酵キリン株式会社 | 核酸を含有する肺高血圧症治療剤 |
| EP2338520A1 (de) | 2009-12-21 | 2011-06-29 | Ludwig Maximilians Universität | Konjugat mit Zielfindungsligand und dessen Verwendung |
| US20110229528A1 (en) * | 2010-03-12 | 2011-09-22 | Intezyne Technologies, Incorporated | Pegylated polyplexes for polynucleotide delivery |
-
2013
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- 2013-06-07 WO PCT/EP2013/061811 patent/WO2013182683A1/en not_active Ceased
- 2013-06-07 CN CN201380027591.5A patent/CN104349794B/zh active Active
- 2013-06-07 EP EP13735212.6A patent/EP2858677B1/en active Active
- 2013-06-07 KR KR1020197011630A patent/KR102087643B1/ko active Active
- 2013-06-07 JP JP2015515538A patent/JP6208750B2/ja active Active
- 2013-06-07 EA EA201401337A patent/EA037448B1/ru unknown
- 2013-06-07 US US14/406,112 patent/US20150126589A1/en not_active Abandoned
- 2013-06-07 CA CA2873274A patent/CA2873274C/en active Active
- 2013-06-07 KR KR20147033746A patent/KR20150020288A/ko not_active Ceased
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- 2013-06-07 ES ES13735212T patent/ES2826203T3/es active Active
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2014
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- 2014-11-18 IN IN9744DEN2014 patent/IN2014DN09744A/en unknown
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| Publication number | Publication date |
|---|---|
| JP2015518874A (ja) | 2015-07-06 |
| CN104349794B (zh) | 2019-01-04 |
| EP2858677B1 (en) | 2020-08-05 |
| JP6208750B2 (ja) | 2017-10-04 |
| AU2013273504A1 (en) | 2014-11-27 |
| EP2858677A1 (en) | 2015-04-15 |
| EA037448B1 (ru) | 2021-03-30 |
| KR20150020288A (ko) | 2015-02-25 |
| KR20190045403A (ko) | 2019-05-02 |
| WO2013182683A1 (en) | 2013-12-12 |
| CA2873274A1 (en) | 2013-12-12 |
| CN104349794A (zh) | 2015-02-11 |
| AU2013273504B2 (en) | 2017-12-07 |
| ES2826203T3 (es) | 2021-05-17 |
| ZA201408184B (en) | 2015-10-28 |
| KR102087643B1 (ko) | 2020-03-11 |
| DK2858677T3 (da) | 2020-08-31 |
| EA201401337A1 (ru) | 2015-05-29 |
| IN2014DN09744A (enExample) | 2015-07-31 |
| US20150126589A1 (en) | 2015-05-07 |
| HK1209340A1 (en) | 2016-04-01 |
| EP3800254A1 (en) | 2021-04-07 |
| CA2873274C (en) | 2021-06-01 |
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Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI NO 10196/2001, QUE MODIFICOU A LEI NO 9279/96, A CONCESSAO DA PATENTE ESTA CONDICIONADA A ANUENCIA PREVIA DA ANVISA. CONSIDERANDO A APROVACAO DOS TERMOS DO PARECER NO 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL NO 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVIDENCIAS CABIVEIS. |
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