IN2014DN09744A - - Google Patents
Info
- Publication number
- IN2014DN09744A IN2014DN09744A IN9744DEN2014A IN2014DN09744A IN 2014DN09744 A IN2014DN09744 A IN 2014DN09744A IN 9744DEN2014 A IN9744DEN2014 A IN 9744DEN2014A IN 2014DN09744 A IN2014DN09744 A IN 2014DN09744A
- Authority
- IN
- India
- Prior art keywords
- mrna
- pei
- lung
- expressed
- combination
- Prior art date
Links
- 108020004999 messenger RNA Proteins 0.000 abstract 5
- 229920002873 Polyethylenimine Polymers 0.000 abstract 4
- 210000004072 lung Anatomy 0.000 abstract 2
- 210000005265 lung cell Anatomy 0.000 abstract 2
Classifications
-
- 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
- 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
- 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)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Genetics & Genomics (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Biomedical Technology (AREA)
- Pulmonology (AREA)
- Communicable Diseases (AREA)
- Oncology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (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 |
|---|---|
| IN2014DN09744A true IN2014DN09744A (enrdf_load_stackoverflow) | 2015-07-31 |
Family
ID=48782278
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IN9744DEN2014 IN2014DN09744A (enrdf_load_stackoverflow) | 2012-06-08 | 2014-11-18 |
Country Status (14)
Families Citing this family (84)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4115874A1 (en) | 2009-12-01 | 2023-01-11 | Translate Bio, Inc. | Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases |
| EP2338520A1 (de) | 2009-12-21 | 2011-06-29 | Ludwig Maximilians Universität | Konjugat mit Zielfindungsligand und dessen Verwendung |
| CA2807552A1 (en) | 2010-08-06 | 2012-02-09 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| LT3590949T (lt) | 2010-10-01 | 2022-07-25 | Modernatx, Inc. | Ribonukleorūgštys, kurių sudėtyje yra n1-metil-pseudouracilų, ir jų naudojimas |
| 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 |
| WO2012135805A2 (en) | 2011-03-31 | 2012-10-04 | modeRNA Therapeutics | Delivery and formulation of engineered nucleic acids |
| ES2740248T3 (es) | 2011-06-08 | 2020-02-05 | Translate Bio Inc | Composiciones de nanopartículas lipídicas y métodos para la administración ARNm |
| US9464124B2 (en) | 2011-09-12 | 2016-10-11 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| SMT202200229T1 (it) | 2011-10-03 | 2022-07-21 | Modernatx Inc | Nucleosidi, nucleotidi e acidi nucleici modificati e loro usi |
| MX2014007233A (es) | 2011-12-16 | 2015-02-04 | Moderna Therapeutics Inc | Composiciones de nucleosidos, nucleotidos y acidos nucleicos modificados. |
| US9572897B2 (en) | 2012-04-02 | 2017-02-21 | Modernatx, Inc. | Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins |
| US9254311B2 (en) | 2012-04-02 | 2016-02-09 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins |
| US9283287B2 (en) | 2012-04-02 | 2016-03-15 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of nuclear proteins |
| DE18200782T1 (de) | 2012-04-02 | 2021-10-21 | Modernatx, Inc. | Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen |
| WO2013185067A1 (en) | 2012-06-08 | 2013-12-12 | Shire Human Genetic Therapies, Inc. | Nuclease resistant polynucleotides and uses thereof |
| WO2014081507A1 (en) | 2012-11-26 | 2014-05-30 | Moderna Therapeutics, Inc. | Terminally modified rna |
| EA202190410A1 (ru) | 2013-03-14 | 2022-03-31 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ мРНК CFTR И СВЯЗАННЫЕ С НИМИ СПОСОБЫ И ВАРИАНТЫ ПРИМЕНЕНИЯ |
| US8980864B2 (en) | 2013-03-15 | 2015-03-17 | Moderna Therapeutics, Inc. | Compositions and methods of altering cholesterol levels |
| WO2015048744A2 (en) | 2013-09-30 | 2015-04-02 | Moderna Therapeutics, Inc. | Polynucleotides encoding immune modulating polypeptides |
| SG11201602503TA (en) | 2013-10-03 | 2016-04-28 | Moderna Therapeutics Inc | Polynucleotides encoding low density lipoprotein receptor |
| EP3060258A1 (en) | 2013-10-22 | 2016-08-31 | Shire Human Genetic Therapies, Inc. | Mrna therapy for phenylketonuria |
| US11224642B2 (en) | 2013-10-22 | 2022-01-18 | Translate Bio, Inc. | MRNA therapy for argininosuccinate synthetase deficiency |
| HRP20201544T1 (hr) | 2014-03-09 | 2021-02-19 | The Trustees Of The University Of Pennsylvania | Pripravci korisni u liječenju nedostatka ornitinske transkarbamilaze (otc) |
| KR20250005450A (ko) | 2014-04-25 | 2025-01-09 | 샤이어 휴먼 지네틱 테라피즈 인크. | 메신저 rna 의 정제 방법 |
| WO2016090262A1 (en) | 2014-12-05 | 2016-06-09 | Shire Human Genetic Therapies, Inc. | Messenger rna therapy for treatment of articular disease |
| US10676726B2 (en) | 2015-02-09 | 2020-06-09 | Duke University | Compositions and methods for epigenome editing |
| JP6851319B2 (ja) | 2015-04-27 | 2021-03-31 | 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 | ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系 |
| GB2560250A (en) * | 2015-06-30 | 2018-09-05 | Ethris Gmbh | ATP-Binding cassette family coding polyribonucleotides and formulations thereof |
| ES2667644T3 (es) | 2015-06-30 | 2018-05-14 | Ethris Gmbh | UTR que aumentan la eficacia de la traducción de las moléculas de ARN |
| EP3325018B1 (en) | 2015-07-22 | 2025-01-15 | Duke University | High-throughput screening of regulatory element function with epigenome editing technologies |
| EP4177346A3 (en) | 2015-08-25 | 2023-07-26 | Duke University | Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases |
| EP3362571A4 (en) | 2015-10-13 | 2019-07-10 | Duke University | GENOM ENGINEERING WITH TYPE I CRISPR SYSTEMS IN EUKARYOTIC CELLS |
| DE20164728T1 (de) | 2015-10-22 | 2021-09-30 | Modernatx, Inc. | Impfstoffe gegen atemwegsvirus |
| CA3009813A1 (en) * | 2015-12-30 | 2017-07-06 | Visiongate, Inc. | System and method for automated detection and monitoring of dysplasia and administration of chemoprevention |
| MA45053A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique |
| WO2017205767A1 (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 |
| EP3548005A4 (en) | 2016-11-29 | 2020-06-17 | Puretech Health LLC | EXOSOMES FOR THE ADMINISTRATION OF THERAPEUTIC AGENTS |
| EP3551193A4 (en) | 2016-12-08 | 2020-08-19 | Modernatx, Inc. | NUCLEIC ACID VACCINES AGAINST RESPIRATORY VIRUSES |
| 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 |
| SG11201907714UA (en) | 2017-02-28 | 2019-09-27 | Univ Pennsylvania | Adeno-associated virus (aav) clade f vector and uses therefor |
| JP7211960B2 (ja) | 2017-03-01 | 2023-01-24 | 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 | 眼疾患の遺伝子治療 |
| EP3634986A4 (en) | 2017-04-24 | 2021-09-08 | The Trustees of The University of Pennsylvania | GENE THERAPY FOR EYE DISORDERS |
| SG10201912401QA (en) | 2017-05-11 | 2020-02-27 | Univ Pennsylvania | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2018213476A1 (en) | 2017-05-16 | 2018-11-22 | Translate Bio, Inc. | Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr |
| EP3630986A4 (en) | 2017-05-31 | 2021-08-11 | The Trustees of The University of Pennsylvania | GENE THERAPY FOR THE TREATMENT OF PEROXYSOMA DISORDERS |
| EP3717652A4 (en) | 2017-11-30 | 2021-11-24 | The Trustees of the University of Pennsylvania | GENE THERAPY FOR MUCOPOLYSACCHARIDOSIS TYPE IIIB |
| BR112020010735A2 (pt) | 2017-11-30 | 2020-11-10 | The Trustees Of The University Of Pennsylvania | terapia genética para mucopolissacaridose iii a |
| AU2019258679B2 (en) | 2018-04-25 | 2024-08-15 | Ethris Gmbh | Cryoprotective agents for particulate formulations |
| CA3120647A1 (en) | 2018-11-21 | 2020-05-28 | 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 |
| WO2020165352A1 (en) * | 2019-02-14 | 2020-08-20 | Ethris Gmbh | Treatment of ciliopathies |
| 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 |
| BR112022022212A2 (pt) | 2020-05-12 | 2022-12-13 | Univ Pennsylvania | Composições para redução específica de drg de expressão de transgene |
| IL299167A (en) | 2020-06-17 | 2023-02-01 | Univ Pennsylvania | Compositions and methods for treating patients with gene therapy |
| TW202216244A (zh) | 2020-07-13 | 2022-05-01 | 賓州大學委員會 | 有用於治療夏馬杜三氏病之組成物 |
| KR20230066000A (ko) * | 2020-08-12 | 2023-05-12 | 액팀 테라퓨틱스, 인코퍼레이티드 | 면역자극성 박테리아-기초 백신, 치료제, 및 rna 전달 플랫폼 |
| WO2022076582A1 (en) | 2020-10-07 | 2022-04-14 | 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 |
| EP4256065A2 (en) | 2020-12-01 | 2023-10-11 | The Trustees of The University of Pennsylvania | Novel compositions with tissue-specific targeting motifs and compositions containing same |
| IL303239A (en) | 2020-12-01 | 2023-07-01 | Univ Pennsylvania | Compositions and their uses for the treatment of Engelmann syndrome |
| MX2023008826A (es) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| MX2023012052A (es) | 2021-04-12 | 2024-03-15 | Univ Pennsylvania | Composiciones útiles para tratar la atrofia muscular espinal y bulbar (sbma). |
| US20240207452A1 (en) | 2021-04-23 | 2024-06-27 | The Trustees Of The University Of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20240408157A1 (en) | 2021-12-02 | 2024-12-12 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| WO2023133574A1 (en) | 2022-01-10 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful for treatment of c9orf72-mediated disorders |
| WO2023183300A1 (en) * | 2022-03-22 | 2023-09-28 | The Children's Medical Center Corporation | Compositions and methods for prevention and treatment of genetic disease |
| IL317874A (en) | 2022-06-24 | 2025-02-01 | Tune Therapeutics Inc | Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression |
| WO2024121160A1 (en) | 2022-12-05 | 2024-06-13 | Ethris Gmbh | Regulator(s) of energy homeostasis-encoding rna molecule(s) with increased translation efficiency |
| 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 |
| TW202440939A (zh) | 2022-12-17 | 2024-10-16 | 賓州大學委員會 | 具心臟及骨骼肌特異性靶向模體的重組aav突變載體及含有其之組成物 |
| 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 |
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 |
| NZ514415A (en) * | 1999-04-02 | 2005-03-24 | Res Dev Foundation | Polyethyleneimine:DNA formulations for aerosol delivery |
| WO2002000870A2 (en) * | 2000-06-26 | 2002-01-03 | Christian Plank | Method for transfecting cells using a magnetic field |
| US20020086842A1 (en) * | 2000-06-26 | 2002-07-04 | Christian Plank | Method for transfecting cells using a magnetic field |
| US20030157030A1 (en) * | 2001-11-02 | 2003-08-21 | Insert Therapeutics, Inc. | Methods and compositions for therapeutic use of rna interference |
| EP1592412A1 (en) * | 2003-01-31 | 2005-11-09 | Novartis AG | Down-regulation of target-gene with pei/single-stranded oligoribonucleotide complexes |
| GB0313259D0 (en) * | 2003-06-09 | 2003-07-16 | Consejo Superior Investigacion | Magnetic nanoparticles |
| MXPA05013983A (es) * | 2003-06-30 | 2006-03-02 | Canji Inc | Encapsulacion polimerica de adenovirus. |
| 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 |
| PL2578685T3 (pl) | 2005-08-23 | 2020-01-31 | The Trustees Of The University Of Pennsylvania | Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania |
| 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 |
| WO2011113065A2 (en) * | 2010-03-12 | 2011-09-15 | Intezyne Technologies, Incorporated | Pegylated polyplexes for polynucleotide delivery |
-
2013
- 2013-06-07 WO PCT/EP2013/061811 patent/WO2013182683A1/en active Application Filing
- 2013-06-07 CA CA2873274A patent/CA2873274C/en active Active
- 2013-06-07 ES ES13735212T patent/ES2826203T3/es active Active
- 2013-06-07 KR KR20147033746A patent/KR20150020288A/ko not_active Ceased
- 2013-06-07 AU AU2013273504A patent/AU2013273504B2/en active Active
- 2013-06-07 EP EP20189217.1A patent/EP3800254A1/en active Pending
- 2013-06-07 JP JP2015515538A patent/JP6208750B2/ja active Active
- 2013-06-07 DK DK13735212.6T patent/DK2858677T3/da active
- 2013-06-07 US US14/406,112 patent/US20150126589A1/en not_active Abandoned
- 2013-06-07 BR BR112014029807A patent/BR112014029807A2/pt not_active Application Discontinuation
- 2013-06-07 KR KR1020197011630A patent/KR102087643B1/ko active Active
- 2013-06-07 EP EP13735212.6A patent/EP2858677B1/en active Active
- 2013-06-07 EA EA201401337A patent/EA037448B1/ru unknown
- 2013-06-07 CN CN201380027591.5A patent/CN104349794B/zh active Active
-
2014
- 2014-11-10 ZA ZA2014/08184A patent/ZA201408184B/en unknown
- 2014-11-18 IN IN9744DEN2014 patent/IN2014DN09744A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP3800254A1 (en) | 2021-04-07 |
| EA201401337A1 (ru) | 2015-05-29 |
| JP2015518874A (ja) | 2015-07-06 |
| WO2013182683A1 (en) | 2013-12-12 |
| ZA201408184B (en) | 2015-10-28 |
| BR112014029807A2 (pt) | 2017-06-27 |
| JP6208750B2 (ja) | 2017-10-04 |
| AU2013273504A1 (en) | 2014-11-27 |
| CA2873274C (en) | 2021-06-01 |
| HK1209340A1 (en) | 2016-04-01 |
| KR102087643B1 (ko) | 2020-03-11 |
| KR20190045403A (ko) | 2019-05-02 |
| AU2013273504B2 (en) | 2017-12-07 |
| CA2873274A1 (en) | 2013-12-12 |
| CN104349794B (zh) | 2019-01-04 |
| ES2826203T3 (es) | 2021-05-17 |
| EP2858677A1 (en) | 2015-04-15 |
| DK2858677T3 (da) | 2020-08-31 |
| KR20150020288A (ko) | 2015-02-25 |
| CN104349794A (zh) | 2015-02-11 |
| US20150126589A1 (en) | 2015-05-07 |
| EP2858677B1 (en) | 2020-08-05 |
| EA037448B1 (ru) | 2021-03-30 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IN2014DN09744A (enrdf_load_stackoverflow) | ||
| ZA201309343B (en) | Centrifugal blower system and fuel cell incorporating same | |
| AU344238S (en) | Connector | |
| AU344219S (en) | Connector | |
| IL226064A0 (en) | Social circles in social networks | |
| AU337705S (en) | Storage system | |
| EP3010528A4 (en) | In vivo targeting of cells with ligand-conjugated particles | |
| AU342351S (en) | Automobile | |
| AU344708S (en) | Bottle | |
| AU337590S (en) | Electric vehicle | |
| AU334107S (en) | Connector plug | |
| AU334573S (en) | Assembly for dispensing cable | |
| AU334129S (en) | Connector plug | |
| AU340629S (en) | Tray | |
| CA143874S (en) | Electric connector | |
| AU344737S (en) | Pallet | |
| AU341483S (en) | Electrical connection module | |
| AU336078S (en) | Container | |
| AU335941S (en) | Container | |
| AU335855S (en) | Container | |
| AU335854S (en) | Container | |
| AU336648S (en) | Door lever | |
| AU344236S (en) | Connector | |
| AU344235S (en) | Connector | |
| AU344237S (en) | Connector |