PE20212072A1 - Composiciones utiles en el tratamiento de la enfermedad de krabbe - Google Patents
Composiciones utiles en el tratamiento de la enfermedad de krabbeInfo
- Publication number
- PE20212072A1 PE20212072A1 PE2021001391A PE2021001391A PE20212072A1 PE 20212072 A1 PE20212072 A1 PE 20212072A1 PE 2021001391 A PE2021001391 A PE 2021001391A PE 2021001391 A PE2021001391 A PE 2021001391A PE 20212072 A1 PE20212072 A1 PE 20212072A1
- Authority
- PE
- Peru
- Prior art keywords
- vector genome
- krabbe
- disease
- aav capsid
- galactosylceramidase
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 3
- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 title abstract 2
- 208000028226 Krabbe disease Diseases 0.000 title abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 5
- 210000000234 capsid Anatomy 0.000 abstract 3
- 102100028496 Galactocerebrosidase Human genes 0.000 abstract 2
- 108010042681 Galactosylceramidase Proteins 0.000 abstract 2
- 108091026890 Coding region Proteins 0.000 abstract 1
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 1
- 210000003169 central nervous system Anatomy 0.000 abstract 1
- 230000004064 dysfunction Effects 0.000 abstract 1
- 210000000578 peripheral nerve Anatomy 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- 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
-
- 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
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- 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
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
- C12N9/2468—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on beta-galactose-glycoside bonds, e.g. carrageenases (3.2.1.83; 3.2.1.157); beta-agarase (3.2.1.81)
- C12N9/2471—Beta-galactosidase (3.2.1.23), i.e. exo-(1-->4)-beta-D-galactanase
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
-
- 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
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/30—Vector systems having a special element relevant for transcription being an enhancer not forming part of the promoter region
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01046—Galactosylceramidase (3.2.1.46)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962810708P | 2019-02-26 | 2019-02-26 | |
| US201962817482P | 2019-03-12 | 2019-03-12 | |
| US201962877707P | 2019-07-23 | 2019-07-23 | |
| US201962916652P | 2019-10-17 | 2019-10-17 | |
| PCT/US2020/019794 WO2020176562A1 (en) | 2019-02-26 | 2020-02-26 | Compositions useful in treatment of krabbe disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PE20212072A1 true PE20212072A1 (es) | 2021-10-26 |
Family
ID=69941503
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2021001391A PE20212072A1 (es) | 2019-02-26 | 2020-02-26 | Composiciones utiles en el tratamiento de la enfermedad de krabbe |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20220118108A1 (enExample) |
| EP (1) | EP3931337A1 (enExample) |
| JP (2) | JP2022525848A (enExample) |
| KR (1) | KR20210132095A (enExample) |
| CN (1) | CN114040980A (enExample) |
| AU (1) | AU2020229772A1 (enExample) |
| BR (1) | BR112021016566A2 (enExample) |
| CA (1) | CA3130055A1 (enExample) |
| CL (1) | CL2021002248A1 (enExample) |
| CO (1) | CO2021011090A2 (enExample) |
| IL (1) | IL285776A (enExample) |
| MX (1) | MX2021010266A (enExample) |
| PE (1) | PE20212072A1 (enExample) |
| SG (1) | SG11202108480UA (enExample) |
| TW (1) | TW202045728A (enExample) |
| WO (1) | WO2020176562A1 (enExample) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2021271635A1 (en) * | 2020-05-12 | 2023-01-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of krabbe disease |
| IL307633A (en) * | 2021-04-12 | 2023-12-01 | Univ Pennsylvania | Useful preparations in the treatment of spinal muscular atrophy (SBMA) |
| CN120917141A (zh) * | 2023-04-10 | 2025-11-07 | M6P医疗公司 | 包含修饰的人半乳糖脑苷脂酶的组合物 |
Family Cites Families (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| 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 |
| EP1007637B1 (en) | 1997-04-14 | 2004-06-30 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| EP1135468B1 (en) | 1998-11-10 | 2010-01-06 | University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
| US6596535B1 (en) | 1999-08-09 | 2003-07-22 | Targeted Genetics Corporation | Metabolically activated recombinant viral vectors and methods for the preparation and use |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| WO2005012877A2 (en) | 2003-08-01 | 2005-02-10 | Dna Twopointo Inc. | Systems and methods for antibody engineering |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| DE23193606T1 (de) | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| 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 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| PL2158322T3 (pl) * | 2007-06-06 | 2017-10-31 | Genzyme Corp | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| US9986722B2 (en) * | 2012-08-07 | 2018-06-05 | Chiesi Farmaceutici S.P.A. | Animal model of Krabbe's disease |
| US9719106B2 (en) * | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| WO2016049230A1 (en) | 2014-09-24 | 2016-03-31 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| US10195746B2 (en) | 2014-09-26 | 2019-02-05 | Teradyne, Inc. | Grasping gripper |
| KR20220082100A (ko) * | 2015-05-15 | 2022-06-16 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 치료적 전달 |
| US11135313B2 (en) * | 2015-10-28 | 2021-10-05 | The Trustees Of The University Of Pennsylvania | Intrathecal administration of adeno-associated-viral vectors for gene therapy |
| JP2020505317A (ja) * | 2017-01-20 | 2020-02-20 | ユニバーシティ オブ ピッツバーグ − オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション | 臍帯血移植（ｕｃｂｔ）および増加したガラクトセレブロシダーゼの発現によるクラッベ病の処置 |
| RS65241B1 (sr) | 2017-02-28 | 2024-03-29 | Univ Pennsylvania | Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe |
| WO2018160852A1 (en) | 2017-03-03 | 2018-09-07 | Cook Medical Technologies, LLC | Rapid exchange multiple catheter system |
| EP3692158A4 (en) * | 2017-10-03 | 2021-08-04 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
-
2020
- 2020-02-26 EP EP20713474.3A patent/EP3931337A1/en active Pending
- 2020-02-26 CA CA3130055A patent/CA3130055A1/en active Pending
- 2020-02-26 BR BR112021016566A patent/BR112021016566A2/pt unknown
- 2020-02-26 AU AU2020229772A patent/AU2020229772A1/en not_active Abandoned
- 2020-02-26 CN CN202080017113.6A patent/CN114040980A/zh active Pending
- 2020-02-26 WO PCT/US2020/019794 patent/WO2020176562A1/en not_active Ceased
- 2020-02-26 TW TW109106249A patent/TW202045728A/zh unknown
- 2020-02-26 PE PE2021001391A patent/PE20212072A1/es unknown
- 2020-02-26 KR KR1020217029570A patent/KR20210132095A/ko active Pending
- 2020-02-26 SG SG11202108480UA patent/SG11202108480UA/en unknown
- 2020-02-26 MX MX2021010266A patent/MX2021010266A/es unknown
- 2020-02-26 US US17/433,195 patent/US20220118108A1/en active Pending
- 2020-02-26 JP JP2021549986A patent/JP2022525848A/ja active Pending
-
2021
- 2021-08-22 IL IL285776A patent/IL285776A/en unknown
- 2021-08-24 CO CONC2021/0011090A patent/CO2021011090A2/es unknown
- 2021-08-25 CL CL2021002248A patent/CL2021002248A1/es unknown
-
2024
- 2024-11-29 JP JP2024208739A patent/JP2025032175A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CO2021011090A2 (es) | 2021-09-09 |
| KR20210132095A (ko) | 2021-11-03 |
| WO2020176562A1 (en) | 2020-09-03 |
| US20220118108A1 (en) | 2022-04-21 |
| SG11202108480UA (en) | 2021-09-29 |
| CL2021002248A1 (es) | 2022-03-04 |
| EP3931337A1 (en) | 2022-01-05 |
| BR112021016566A2 (pt) | 2021-11-03 |
| IL285776A (en) | 2021-10-31 |
| CA3130055A1 (en) | 2020-09-03 |
| AU2020229772A1 (en) | 2021-08-26 |
| JP2022525848A (ja) | 2022-05-20 |
| TW202045728A (zh) | 2020-12-16 |
| JP2025032175A (ja) | 2025-03-11 |
| MX2021010266A (es) | 2021-09-23 |
| CN114040980A (zh) | 2022-02-11 |
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