MX2016011686A - Composiciones utiles en el tratamiento de deficiencia de la ornitina transcarbamilasa (otc). - Google Patents
Composiciones utiles en el tratamiento de deficiencia de la ornitina transcarbamilasa (otc).Info
- Publication number
- MX2016011686A MX2016011686A MX2016011686A MX2016011686A MX2016011686A MX 2016011686 A MX2016011686 A MX 2016011686A MX 2016011686 A MX2016011686 A MX 2016011686A MX 2016011686 A MX2016011686 A MX 2016011686A MX 2016011686 A MX2016011686 A MX 2016011686A
- Authority
- MX
- Mexico
- Prior art keywords
- treatment
- deficiency
- otc
- ornithine transcarbamylase
- compositions useful
- Prior art date
Links
- 101710198224 Ornithine carbamoyltransferase, mitochondrial Proteins 0.000 title abstract 2
- 230000007812 deficiency Effects 0.000 title abstract 2
- 102000007981 Ornithine carbamoyltransferase Human genes 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 102100028200 Ornithine transcarbamylase, mitochondrial Human genes 0.000 abstract 2
- 208000019425 cirrhosis of liver Diseases 0.000 abstract 2
- 206010016654 Fibrosis Diseases 0.000 abstract 1
- 101000986595 Homo sapiens Ornithine transcarbamylase, mitochondrial Proteins 0.000 abstract 1
- 206010020575 Hyperammonaemia Diseases 0.000 abstract 1
- 230000007882 cirrhosis Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 201000011278 ornithine carbamoyltransferase deficiency Diseases 0.000 abstract 1
- 208000024891 symptom Diseases 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 1
Classifications
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
-
- 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.)
- C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
- C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y201/00—Transferases transferring one-carbon groups (2.1)
- C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
- C12Y201/03003—Ornithine carbamoyltransferase (2.1.3.3)
-
- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10341—Use of virus, viral particle or viral elements as a vector
- C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14033—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14041—Use of virus, viral particle or viral elements as a vector
- C12N2750/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14071—Demonstrated in vivo effect
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Diabetes (AREA)
- Gastroenterology & Hepatology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Abstract
Se proveen vectores virales que comprenden secuencias de ADN y ARN de hOTC modificadas genéticamente que cuando se suministran a un sujeto en necesidad del mismo son útiles para el tratamiento de la hiperamonemia, deficiencia de ornitina transcarbamilasa transcarbamilasa y síntomas asociados con la misma; también se proveen métodos de uso de hOTC para el tratamiento de la cirrosis fibrosis hepática en pacientes mediante de OTCD mediante la administración de hOTC.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461950157P | 2014-03-09 | 2014-03-09 | |
| PCT/US2015/019513 WO2015138348A1 (en) | 2014-03-09 | 2015-03-09 | Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| MX2016011686A true MX2016011686A (es) | 2016-11-07 |
| MX377804B MX377804B (es) | 2025-03-11 |
Family
ID=52737408
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2016011686A MX377804B (es) | 2014-03-09 | 2015-03-09 | Composiciones utiles en el tratamiento de deficiencia de la ornitina transcarbamilasa (otc). |
Country Status (22)
| Country | Link |
|---|---|
| US (6) | US9890365B2 (es) |
| EP (2) | EP3116900B1 (es) |
| JP (2) | JP6822841B2 (es) |
| KR (1) | KR102390075B1 (es) |
| AU (2) | AU2015229750A1 (es) |
| BR (2) | BR122023023004A2 (es) |
| CA (1) | CA2939950C (es) |
| CL (2) | CL2016002235A1 (es) |
| CY (1) | CY1123378T1 (es) |
| DK (1) | DK3116900T3 (es) |
| ES (1) | ES2821938T3 (es) |
| HR (1) | HRP20201544T1 (es) |
| HU (1) | HUE051311T2 (es) |
| IL (2) | IL247329B (es) |
| LT (1) | LT3116900T (es) |
| MX (1) | MX377804B (es) |
| PL (1) | PL3116900T3 (es) |
| PT (1) | PT3116900T (es) |
| RS (1) | RS60902B1 (es) |
| SI (1) | SI3116900T1 (es) |
| SM (1) | SMT202000531T1 (es) |
| WO (2) | WO2015138357A2 (es) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX373641B (es) | 2011-04-29 | 2020-05-04 | Selecta Biosciences Inc | Nanoportadores sintéticos tolerogénicos para la terapia contra alergias. |
| CN105283175A (zh) | 2013-05-03 | 2016-01-27 | 西莱克塔生物科技公司 | 用于降低的或增强的药效学作用的致耐受性合成纳米载体和治疗性大分子 |
| AU2014296278C1 (en) | 2013-07-30 | 2023-02-02 | Genevant Sciences Gmbh | Block copolymers and their conjugates or complexes with oligonucleotides |
| CA2939950C (en) | 2014-03-09 | 2023-08-22 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency |
| KR20170045344A (ko) | 2014-09-07 | 2017-04-26 | 셀렉타 바이오사이언시즈, 인크. | 항-바이러스 전달 벡터 면역 반응을 약화시키기 위한 방법 및 조성물 |
| RU2716991C2 (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| WO2016118697A1 (en) | 2015-01-21 | 2016-07-28 | Phaserx, Inc. | Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells |
| WO2017059902A1 (en) * | 2015-10-07 | 2017-04-13 | Biontech Rna Pharmaceuticals Gmbh | 3' utr sequences for stabilization of rna |
| EP3452101A2 (en) | 2016-05-04 | 2019-03-13 | CureVac AG | Rna encoding a therapeutic protein |
| MX2019000962A (es) | 2016-07-26 | 2019-08-01 | Biomarin Pharm Inc | Novedosas proteinas de la capside del virus adenoasociado. |
| JP2019524162A (ja) | 2016-08-18 | 2019-09-05 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | モジュラーＡＡＶ送達システムによるＣＲＩＳＰＲ−Ｃａｓゲノム編集 |
| JP7560946B2 (ja) * | 2016-11-10 | 2024-10-03 | トランスレイト バイオ， インコーポレイテッド | メッセンジャーｒｎａの皮下送達 |
| CN110325199A (zh) * | 2016-12-30 | 2019-10-11 | 宾夕法尼亚州立大学托管会 | 用于治疗苯丙酮尿症的基因疗法 |
| US11684584B2 (en) | 2016-12-30 | 2023-06-27 | Genevant Sciences Gmbh | Branched peg molecules and related compositions and methods |
| US11104887B2 (en) | 2017-01-03 | 2021-08-31 | Ethris Gmbh | Ornithine transcarbamylase coding polyribonucleotides and formulations thereof |
| WO2018129586A1 (en) | 2017-01-10 | 2018-07-19 | Children's Medical Research Institute | Polynucleotides and vectors for the expression of transgenes |
| JP7523909B2 (ja) | 2017-03-11 | 2024-07-29 | セレクタ バイオサイエンシーズ インコーポレーテッド | 抗炎症剤および免疫抑制剤を含む合成ナノキャリアによる組み合わせ処置に関連する方法および組成物 |
| KR20240010098A (ko) | 2017-05-31 | 2024-01-23 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 최적화된 인간 응고 인자 ix 유전자 발현 카세트 및 이의 사용 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| MY203184A (en) | 2017-08-09 | 2024-06-13 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof |
| MA50803A (fr) | 2017-11-22 | 2020-09-30 | Modernatx Inc | Polynucléotides codant pour l'ornithine transcarbamylase pour le traitement de troubles du cycle de l'urée |
| WO2019217513A2 (en) | 2018-05-09 | 2019-11-14 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| AU2019271132A1 (en) * | 2018-05-15 | 2020-11-12 | Translate Bio, Inc. | Subcutaneous delivery of messenger RNA |
| BR112020023082A2 (pt) | 2018-05-15 | 2021-02-09 | Voyager Therapeutics, Inc. | composições e métodos para o tratamento de doença de parkinson |
| WO2020018583A1 (en) | 2018-07-16 | 2020-01-23 | Selecta Biosciences, Inc. | Methods and compositions of otc constructs and vectors |
| WO2020016655A2 (en) | 2018-07-17 | 2020-01-23 | Helixmith Co., Ltd. | Treatment of neuropathy with dna constructs expressing igf-1 isoforms |
| MX2021001599A (es) | 2018-08-09 | 2021-07-02 | Bioverativ Therapeutics Inc | Moleculas de acido nucleico y sus usos para la terapia genica no viral. |
| CA3114621A1 (en) | 2018-09-28 | 2020-04-02 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| AU2019394996B2 (en) * | 2018-12-06 | 2025-11-06 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
| CN119390787A (zh) | 2019-01-31 | 2025-02-07 | 俄勒冈健康与科学大学 | 用于aav衣壳的使用转录依赖性定向进化的方法 |
| US12351834B2 (en) | 2020-03-03 | 2025-07-08 | Arcturus Therapeutics, Inc. | Compositions and methods for the treatment of ornithine transcarbamylase deficiency |
| EP4114469A1 (en) * | 2020-03-04 | 2023-01-11 | Poseida Therapeutics, Inc. | Compositions and methods for the treatment of metabolic liver disorders |
| WO2021188892A1 (en) * | 2020-03-19 | 2021-09-23 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav |
| KR20230010670A (ko) | 2020-05-12 | 2023-01-19 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 이식유전자 발현의 drg-특이적 감소를 위한 조성물 |
| CA3209779A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| US20250339480A1 (en) * | 2021-04-21 | 2025-11-06 | Jichi Medical University | Adeno-associated virus virion for treating ornithine transcarbamylase deficiency |
| US20230091932A1 (en) * | 2021-08-23 | 2023-03-23 | Bioverativ Therapeutics Inc. | Closed-end dna production with inverted terminal repeat sequences |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| AU2022435116A1 (en) * | 2022-01-21 | 2024-07-11 | The Trustees Of The University Of Pennsylvania | Methods for treatment of ornithine transcarbamylase (otc) deficiency |
| AU2023360709A1 (en) * | 2022-10-11 | 2025-05-22 | Pacific Northwest Research Institute | Constructs relating to genetic disorders |
Family Cites Families (81)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US1016745A (en) | 1911-04-11 | 1912-02-06 | Edwin C Henrikson | Table-caster. |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| US5652224A (en) | 1995-02-24 | 1997-07-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US5866552A (en) | 1996-09-06 | 1999-02-02 | The Trustees Of The University Of Pennsylvania | Method for expressing a gene in the absence of an immune response |
| CN1233291A (zh) | 1996-09-06 | 1999-10-27 | 宾西法尼亚大学托管会 | 重组腺伴随病毒定向基因治疗的方法 |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| US20020037867A1 (en) | 1999-02-26 | 2002-03-28 | James M. Wilson | Method for recombinant adeno-associated virus-directed gene therapy |
| CA2264499A1 (en) | 1996-09-06 | 1998-03-12 | The Trustees Of The University Of Pennsylvania | Methods using cre-lox for production of recombinant adeno-associated viruses |
| CA2265460A1 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| NZ336185A (en) | 1996-12-05 | 2001-02-23 | Introgene Bv | Genetic modification of primate hemopoietic repopulating stem cells |
| DE69837913T2 (de) | 1997-04-01 | 2008-02-07 | Solexa Ltd., Saffron Walden | Verfahren zur vervielfältigung von nukleinsäure |
| DE69824859T2 (de) | 1997-04-14 | 2005-08-04 | Cell Genesys, Inc., Foster City | Methoden zur erhöhung der effizienz rekombinanter aav produkte |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6251677B1 (en) | 1997-08-25 | 2001-06-26 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| WO1999015677A1 (en) | 1997-09-19 | 1999-04-01 | The Trustees Of The University Of Pennsylvania | Method for gene transfer using bcl2 and compositions useful therein |
| JP2001517454A (ja) | 1997-09-19 | 2001-10-09 | 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 | 組換えアデノ随伴ウイルスの産生に有用な方法および細胞株 |
| WO1999014354A1 (en) | 1997-09-19 | 1999-03-25 | The Trustees Of The University Of The Pennsylvania | Methods and vector constructs useful for production of recombinant aav |
| WO1999047691A1 (en) | 1998-03-20 | 1999-09-23 | Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US6953690B1 (en) | 1998-03-20 | 2005-10-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
| ES2313784T3 (es) | 1998-05-28 | 2009-03-01 | The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services | Vector aav5 y usos del mismo. |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| DE69941905D1 (de) | 1998-11-10 | 2010-02-25 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| US6387368B1 (en) | 1999-02-08 | 2002-05-14 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| JP4693244B2 (ja) | 1999-03-18 | 2011-06-01 | 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 | 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法 |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| EP1183380A1 (en) | 1999-06-02 | 2002-03-06 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful for production of recombinant viruses which require helper viruses |
| ES2308989T3 (es) | 1999-08-09 | 2008-12-16 | Targeted Genetics Corporation | Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios. |
| WO2001023001A2 (en) | 1999-09-29 | 2001-04-05 | The Trustees Of The University Of Pennsylvania | Rapid peg-modification |
| US6365394B1 (en) | 1999-09-29 | 2002-04-02 | The Trustees Of The University Of Pennsylvania | Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus |
| US7115391B1 (en) | 1999-10-01 | 2006-10-03 | Genovo, Inc. | Production of recombinant AAV using adenovirus comprising AAV rep/cap genes |
| JP2003511037A (ja) | 1999-10-01 | 2003-03-25 | ジェノボ， インコーポレイテッド | ＡＡＶｒｅｐ／ｃａｐ遺伝子を含むアデノウイルスを使用する組換えＡＡＶの産生 |
| AU1796801A (en) | 1999-12-03 | 2001-06-12 | Trustees Of The University Of Pennsylvania, The | Compositions and methods for increasing packaging and yields of recombinant adenoviruses using multiple packaging signals |
| US6821512B1 (en) | 1999-12-03 | 2004-11-23 | The Trustees Of The University Of Pennsylvania | Compositions and methods for increasing packaging and yield of recombinant adenoviruses using multiple packaging signals |
| US6468524B1 (en) | 2000-03-22 | 2002-10-22 | The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services | AAV4 vector and uses thereof |
| US6855314B1 (en) | 2000-03-22 | 2005-02-15 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector for transducing brain cells and lung cells |
| AU2001255575B2 (en) | 2000-04-28 | 2006-08-31 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| AU2001265187A1 (en) * | 2000-05-30 | 2001-12-11 | Baylor College Of Medicine | Chimeric viral vectors for gene therapy |
| EP1286703B1 (en) | 2000-06-01 | 2009-08-05 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| CA2421078A1 (en) | 2000-08-30 | 2002-03-07 | Haplogen, Llc | Method for determining alleles |
| CA2447240C (en) | 2001-05-18 | 2013-02-19 | Wisconsin Alumni Research Foundation | Method for the synthesis of dna sequences |
| WO2003016475A2 (en) | 2001-08-14 | 2003-02-27 | The General Hospital Corporation | Nucleic acid and amino acid sequences involved in pain |
| WO2003029433A2 (en) * | 2001-10-04 | 2003-04-10 | Aguilar-Cordova Carlos Estuard | Chimeric viral vectors for gene therapy |
| NZ618298A (en) | 2001-11-13 | 2015-04-24 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| PT2359869T (pt) | 2001-12-17 | 2019-04-16 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| DK2573170T3 (en) | 2001-12-17 | 2018-04-09 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use |
| WO2003104392A2 (en) | 2001-12-18 | 2003-12-18 | University Of North Carolina At Chapel Hill | Improved reagents and methods for producing parvoviruses |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP2292780B1 (en) | 2003-09-30 | 2017-08-23 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| EP2359865B1 (en) | 2005-04-07 | 2013-10-02 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| DK2548438T3 (en) | 2006-11-08 | 2015-10-19 | Veritas Bio LLC | IN VIVO SUBMITTING DOUBLE-STRENGTH RNA TO A CELL |
| EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| WO2009130208A1 (en) | 2008-04-22 | 2009-10-29 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
| CN103342789B (zh) | 2008-05-13 | 2016-11-23 | 华盛顿大学 | 用于向细胞内递送的二嵌段共聚物和其多核苷酸复合物 |
| US8822213B2 (en) | 2008-11-06 | 2014-09-02 | University Of Washington | Bispecific intracellular delivery vehicles |
| MX2011004242A (es) | 2008-11-06 | 2011-07-20 | Univ Washington | Copolimeros multibloque. |
| JP6087504B2 (ja) | 2008-11-07 | 2017-03-01 | マサチューセッツ インスティテュート オブ テクノロジー | アミノアルコールリピドイドおよびその使用 |
| TR201819229T4 (tr) | 2009-07-24 | 2019-01-21 | Immune Design Corp | Entegre olmayan lentiviral vektörler. |
| EP2281579A1 (en) | 2009-08-05 | 2011-02-09 | BioNTech AG | Vaccine composition comprising 5'-Cap modified RNA |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| SG183929A1 (en) | 2010-03-29 | 2012-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2708660B1 (en) | 2011-05-12 | 2017-09-20 | Hitachi Construction Machinery Co., Ltd. | Construction machine |
| CA3107288A1 (en) | 2011-06-08 | 2012-12-13 | Translate Bio, Inc. | Lipid nanoparticle compositions and methods for mrna delivery |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| EP4074694A1 (en) | 2011-10-27 | 2022-10-19 | Massachusetts Institute Of Technology | Amino acid-, peptide- an polypeptide-lipids, isomers, compositions, an uses thereof |
| WO2013143555A1 (en) | 2012-03-26 | 2013-10-03 | Biontech Ag | Rna formulation for immunotherapy |
| HK1206601A1 (en) | 2012-04-02 | 2016-01-15 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of biologics and proteins associated with human disease |
| 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 |
| BR112014029807A2 (pt) | 2012-06-08 | 2017-06-27 | Ethris Gmbh | administração pulmonar de rna mensageiro |
| AU2014296278C1 (en) * | 2013-07-30 | 2023-02-02 | Genevant Sciences Gmbh | Block copolymers and their conjugates or complexes with oligonucleotides |
| CA2939950C (en) | 2014-03-09 | 2023-08-22 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency |
-
2015
- 2015-03-09 CA CA2939950A patent/CA2939950C/en active Active
- 2015-03-09 AU AU2015229750A patent/AU2015229750A1/en not_active Abandoned
- 2015-03-09 WO PCT/US2015/019536 patent/WO2015138357A2/en not_active Ceased
- 2015-03-09 PL PL15712226T patent/PL3116900T3/pl unknown
- 2015-03-09 KR KR1020167027891A patent/KR102390075B1/ko active Active
- 2015-03-09 EP EP15712226.8A patent/EP3116900B1/en active Active
- 2015-03-09 SM SM20200531T patent/SMT202000531T1/it unknown
- 2015-03-09 US US15/124,620 patent/US9890365B2/en active Active
- 2015-03-09 ES ES15712226T patent/ES2821938T3/es active Active
- 2015-03-09 LT LTEP15712226.8T patent/LT3116900T/lt unknown
- 2015-03-09 HR HRP20201544TT patent/HRP20201544T1/hr unknown
- 2015-03-09 MX MX2016011686A patent/MX377804B/es active IP Right Grant
- 2015-03-09 JP JP2016556768A patent/JP6822841B2/ja active Active
- 2015-03-09 SI SI201531366T patent/SI3116900T1/sl unknown
- 2015-03-09 EP EP20184346.3A patent/EP3778627A1/en not_active Withdrawn
- 2015-03-09 BR BR122023023004-3A patent/BR122023023004A2/pt not_active Application Discontinuation
- 2015-03-09 RS RS20201154A patent/RS60902B1/sr unknown
- 2015-03-09 DK DK15712226.8T patent/DK3116900T3/da active
- 2015-03-09 HU HUE15712226A patent/HUE051311T2/hu unknown
- 2015-03-09 US US15/122,853 patent/US10167454B2/en active Active
- 2015-03-09 BR BR112016020688-6A patent/BR112016020688B1/pt active IP Right Grant
- 2015-03-09 PT PT157122268T patent/PT3116900T/pt unknown
- 2015-03-09 WO PCT/US2015/019513 patent/WO2015138348A1/en not_active Ceased
-
2016
- 2016-08-17 IL IL247329A patent/IL247329B/en active IP Right Grant
- 2016-09-06 CL CL2016002235A patent/CL2016002235A1/es unknown
-
2018
- 2018-01-03 US US15/861,427 patent/US10626382B2/en active Active
- 2018-11-15 US US16/191,709 patent/US10781430B2/en active Active
-
2019
- 2019-08-12 CL CL2019002280A patent/CL2019002280A1/es unknown
-
2020
- 2020-02-19 AU AU2020201190A patent/AU2020201190B2/en active Active
- 2020-03-25 JP JP2020054236A patent/JP6920500B2/ja active Active
- 2020-07-01 IL IL275799A patent/IL275799B2/en unknown
- 2020-08-17 US US16/995,572 patent/US11732246B2/en active Active
- 2020-09-24 CY CY20201100902T patent/CY1123378T1/el unknown
-
2023
- 2023-06-22 US US18/339,457 patent/US20230416700A1/en active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| MX2016011686A (es) | Composiciones utiles en el tratamiento de deficiencia de la ornitina transcarbamilasa (otc). | |
| MX2022006652A (es) | Proteinas de capside modificadas para un suministro mejorado de vectores de parvovirus. | |
| CO2019006541A2 (es) | Administración viral de neoantígenos | |
| AR105470A1 (es) | Métodos para inducir una respuesta inmune | |
| MX2017008817A (es) | Composiciones y metodos para la glicosilacion de proteinas. | |
| MX2017006695A (es) | Variantes de subtilasa y polinucleotidos que las codifican. | |
| BR112018012873A2 (pt) | vacina de vírus zika | |
| EP4219744A3 (en) | Methods of preparing nucleic acids for sequencing | |
| MX384795B (es) | Vectores que comprenden secuencias de polinucleotidos rellenadoras/de relleno y metodos de uso. | |
| PE20150163A1 (es) | Composicion y metodos para una transferencia de genes altamente eficiente usando variantes capside aav | |
| EA201790505A1 (ru) | Соединение, нацеленое на ил-23а и фно-альфа, и его применение | |
| WO2016019134A8 (en) | Flagellin-based agents and uses including effective vaccination | |
| ZA202403393B (en) | Synthetic chimeric poxviruses | |
| PH12017501539A1 (en) | Disacetoxytubulysin h and analogs thereof | |
| MX377258B (es) | Composiciones de vectores poxvirus y su uso para inducir una respuesta inmunitaria. | |
| EP3939604A3 (en) | Influenza hemagglutinin protein vaccines | |
| MX2020001223A (es) | Proteinas de fusion tatk-cdkl5, composiciones, formulaciones y uso de estas. | |
| MX381617B (es) | Conjugados de peptido-curcumina y formulaciones de los mismos. | |
| DOP2016000202A (es) | Proteínas de fusión uti | |
| CO2018001339A2 (es) | Sistema vector de adenovirus aviar 9 (fadv-9) y métodos asociados | |
| EA201692518A2 (ru) | Способы улучшения производительности миокарда у пациентов после операции фонтена с применением композиций уденафила | |
| WO2016141320A3 (en) | Non-neuroinvasive viruses and uses thereof | |
| MX2015012905A (es) | Formulaciones de factor viii recombinantes. | |
| CL2016001745A1 (es) | Ácido (s)-3'-metil-abscísico y ésteres del mismo | |
| EA201791437A1 (ru) | Композиции микросфер c инкапсулированным в липиде газом и соответствующие способы |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FG | Grant or registration |