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
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
- A61K48/005—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)
Abstract
Esta referido a una composicion que comprende un virus adenoasociado recombinante (rAAV) que comprende una capside de AAV que se dirige a celulas en el sistema nervioso central y un genoma del vector que comprende (i) una secuencia codificante de galactosilceramidasa que codifica una proteina galactosilceramidasa madura que tiene la secuencia de aminoacidos en SEQ ID NO: 10 bajo el control de secuencias reguladoras que dirigen la expresion de la proteina, y (ii) repeticiones terminales invertidas de AAV necesarias para empaquetar el genoma del vector en una capside de AAV, en donde el genoma del vector esta empaquetado en la capside de AAV. Esta composicion es util para tratar la disfuncion de los nervios perifericos y/o para tratar la enfermedad de Krabbe
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 (15)
Country | Link |
---|---|
US (1) | US20220118108A1 (es) |
EP (1) | EP3931337A1 (es) |
JP (1) | JP2022525848A (es) |
KR (1) | KR20210132095A (es) |
CN (1) | CN114040980A (es) |
AU (1) | AU2020229772A1 (es) |
CA (1) | CA3130055A1 (es) |
CL (1) | CL2021002248A1 (es) |
CO (1) | CO2021011090A2 (es) |
IL (1) | IL285776A (es) |
MX (1) | MX2021010266A (es) |
PE (1) | PE20212072A1 (es) |
SG (1) | SG11202108480UA (es) |
TW (1) | TW202045728A (es) |
WO (1) | WO2020176562A1 (es) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA3177924A1 (en) * | 2020-05-12 | 2021-11-18 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of krabbe disease |
Family Cites Families (32)
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 |
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 |
AU728220B2 (en) | 1997-04-14 | 2001-01-04 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
CA2348382C (en) | 1998-11-10 | 2013-09-17 | The University Of North Carolina At Chapel Hill | Chimeric parvovirus vectors and methods of making and administering the same |
CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
WO2001091803A2 (en) | 2000-06-01 | 2001-12-06 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
NZ578982A (en) | 2001-11-13 | 2011-03-31 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby |
AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
WO2005013090A2 (en) | 2003-08-01 | 2005-02-10 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
EP3910063A1 (en) | 2003-09-30 | 2021-11-17 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
EP2359865B1 (en) | 2005-04-07 | 2013-10-02 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
MX342858B (es) | 2010-03-29 | 2016-10-13 | The Trustees Of The Univ Of Pennsylvania * | Sistema de ablacion transgenica inducida farmacologicamente. |
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 |
CN104735976B (zh) * | 2012-08-07 | 2017-03-01 | 意大利凯西制药公司 | 克拉伯病的动物模型 |
WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
DK3198018T3 (da) | 2014-09-24 | 2021-03-01 | Hope City | Vektorvarianter af adenoassocieret virus til højeffektiv genomredigering og fremgangsmåder dertil |
DE112015003537T5 (de) | 2014-09-26 | 2017-07-13 | Teradyne, Inc. | Greifzange |
WO2018136710A1 (en) * | 2017-01-20 | 2018-07-26 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Treatment of krabbe disease with umbilical cord blood transplantion (ucbt) and increased galactocerebrosidase (galc) expression |
AU2018227440A1 (en) | 2017-02-28 | 2019-08-29 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clade f vector and uses therefor |
WO2018160852A1 (en) | 2017-03-03 | 2018-09-07 | Cook Medical Technologies, LLC | Rapid exchange multiple catheter system |
KR20220015499A (ko) * | 2017-10-03 | 2022-02-08 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
-
2020
- 2020-02-26 CN CN202080017113.6A patent/CN114040980A/zh active Pending
- 2020-02-26 US US17/433,195 patent/US20220118108A1/en active Pending
- 2020-02-26 PE PE2021001391A patent/PE20212072A1/es unknown
- 2020-02-26 JP JP2021549986A patent/JP2022525848A/ja active Pending
- 2020-02-26 SG SG11202108480UA patent/SG11202108480UA/en unknown
- 2020-02-26 AU AU2020229772A patent/AU2020229772A1/en active Pending
- 2020-02-26 CA CA3130055A patent/CA3130055A1/en active Pending
- 2020-02-26 TW TW109106249A patent/TW202045728A/zh unknown
- 2020-02-26 MX MX2021010266A patent/MX2021010266A/es unknown
- 2020-02-26 KR KR1020217029570A patent/KR20210132095A/ko unknown
- 2020-02-26 WO PCT/US2020/019794 patent/WO2020176562A1/en active Application Filing
- 2020-02-26 EP EP20713474.3A patent/EP3931337A1/en 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
Also Published As
Publication number | Publication date |
---|---|
KR20210132095A (ko) | 2021-11-03 |
EP3931337A1 (en) | 2022-01-05 |
CN114040980A (zh) | 2022-02-11 |
MX2021010266A (es) | 2021-09-23 |
SG11202108480UA (en) | 2021-09-29 |
CO2021011090A2 (es) | 2021-09-09 |
JP2022525848A (ja) | 2022-05-20 |
US20220118108A1 (en) | 2022-04-21 |
AU2020229772A1 (en) | 2021-08-26 |
CL2021002248A1 (es) | 2022-03-04 |
CA3130055A1 (en) | 2020-09-03 |
TW202045728A (zh) | 2020-12-16 |
WO2020176562A1 (en) | 2020-09-03 |
IL285776A (en) | 2021-10-31 |
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