CL2021002754A1 - Composiciones útiles para el tratamiento de la enfermedad de pompe. - Google Patents
Composiciones útiles para el tratamiento de la enfermedad de pompe.Info
- Publication number
- CL2021002754A1 CL2021002754A1 CL2021002754A CL2021002754A CL2021002754A1 CL 2021002754 A1 CL2021002754 A1 CL 2021002754A1 CL 2021002754 A CL2021002754 A CL 2021002754A CL 2021002754 A CL2021002754 A CL 2021002754A CL 2021002754 A1 CL2021002754 A1 CL 2021002754A1
- Authority
- CL
- Chile
- Prior art keywords
- disease
- raav
- pompe
- protein
- treatment
- 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
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- 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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/575—Hormones
- C07K14/65—Insulin-like growth factors (Somatomedins), e.g. IGF-1, IGF-2
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- 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
- C12N15/62—DNA sequences coding for fusion proteins
- C12N15/625—DNA sequences coding for fusion proteins containing a sequence coding for 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
- 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)
-
- 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)
- C12N9/2405—Glucanases
- C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
-
- 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)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- 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
Abstract
Se proporciona un virus adenoasociado recombinante (rAAV) útil para tratar la enfermedad por almacenamiento de glucógeno tipo II (enfermedad de Pompe). El rAAV comprende una cápside de AAV que se dirige a células de al menos uno de los músculos, el corazón, el riñón y el sistema nervioso central y que tiene empaquetado allí un genoma de vector que comprende una secuencia de ácido nucleico que codifica una proteína de fusión quimérica que comprende un péptido señal y un péptido de vIGF2 fusionado a una proteína hGAA780I de ácido-α-glucosidasa humana bajo el control de secuencias reguladoras que dirigen su expresión. También se proporcionan métodos para hacer y usar este rAAV.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962840911P | 2019-04-30 | 2019-04-30 | |
US201962913401P | 2019-10-10 | 2019-10-10 |
Publications (1)
Publication Number | Publication Date |
---|---|
CL2021002754A1 true CL2021002754A1 (es) | 2022-05-27 |
Family
ID=73029176
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2021002755A CL2021002755A1 (es) | 2019-04-30 | 2021-10-20 | Composiciones útiles para el tratamiento de la enfermedad de pompe. |
CL2021002754A CL2021002754A1 (es) | 2019-04-30 | 2021-10-20 | Composiciones útiles para el tratamiento de la enfermedad de pompe. |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2021002755A CL2021002755A1 (es) | 2019-04-30 | 2021-10-20 | Composiciones útiles para el tratamiento de la enfermedad de pompe. |
Country Status (15)
Country | Link |
---|---|
US (2) | US20220193261A1 (es) |
EP (2) | EP3963063A4 (es) |
JP (2) | JP2022530824A (es) |
KR (2) | KR20220004696A (es) |
CN (2) | CN114072515A (es) |
AU (2) | AU2020266552A1 (es) |
BR (2) | BR112021021720A2 (es) |
CA (2) | CA3134523A1 (es) |
CL (2) | CL2021002755A1 (es) |
CO (2) | CO2021016200A2 (es) |
IL (2) | IL287522A (es) |
MX (2) | MX2021013365A (es) |
SG (2) | SG11202111380VA (es) |
TW (1) | TW202100541A (es) |
WO (2) | WO2020223362A1 (es) |
Families Citing this family (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA3177954A1 (en) * | 2020-05-14 | 2021-11-18 | James M. Wilson | Compositions useful for treatment of pompe disease |
US20230220069A1 (en) | 2020-06-17 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of gene therapy patients |
WO2023086928A2 (en) * | 2021-11-12 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treatment of mucopolysaccharidosis iiia |
NL2031676B1 (en) * | 2022-04-22 | 2023-11-07 | Univ Erasmus Med Ct Rotterdam | Gene therapy for Pompe Disease |
WO2024003687A1 (en) * | 2022-06-28 | 2024-01-04 | Pfizer Inc. | Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy |
CN116693633B (zh) * | 2023-02-21 | 2023-12-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
Family Cites Families (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP1587923B1 (en) * | 2003-01-22 | 2011-08-24 | Duke University | Improved constructs for expressing lysosomal polypeptides |
DE602005020745D1 (de) * | 2004-02-10 | 2010-06-02 | Zystor Therapeutics Inc | Saure alpha-glukosidase und fragmente davon |
CA2669347A1 (en) * | 2006-11-13 | 2008-05-29 | Zystor Therapeutics, Inc. | Methods for treating pompe disease |
AU2008306327B2 (en) * | 2007-10-04 | 2014-05-15 | Roche Innovation Center Copenhagen A/S | Micromirs |
WO2010096369A1 (en) * | 2009-02-18 | 2010-08-26 | Amicus Therapeutics, Inc. | Mouse model for pompe disease and methods of use thereof |
WO2012070014A2 (en) * | 2010-11-26 | 2012-05-31 | Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. | Identification of novel cell surface markers for pancreatic progenitor cells and definite endodermal cells |
US9545450B2 (en) * | 2011-05-27 | 2017-01-17 | Amicus Therapeutics Inc. | Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases |
WO2013013019A2 (en) * | 2011-07-21 | 2013-01-24 | Alnylam Pharmaceuticals, Inc. | Lysosomal polypeptides, methods of making and using |
WO2017100671A1 (en) * | 2015-12-11 | 2017-06-15 | California Institute Of Technology | TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) |
EP3510149A1 (en) * | 2016-09-12 | 2019-07-17 | Genethon | Acid-alpha glucosidase variants and uses thereof |
EP3293260A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
EP3293259A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
AU2018241847A1 (en) * | 2017-03-27 | 2019-10-17 | Vrije Universiteit Brussel | Diaphragm-specific nucleic acid regulatory elements and methods and use thereof |
-
2020
- 2020-04-29 KR KR1020217038231A patent/KR20220004696A/ko unknown
- 2020-04-29 WO PCT/US2020/030493 patent/WO2020223362A1/en unknown
- 2020-04-29 AU AU2020266552A patent/AU2020266552A1/en active Pending
- 2020-04-29 CA CA3134523A patent/CA3134523A1/en active Pending
- 2020-04-29 EP EP20799541.6A patent/EP3963063A4/en active Pending
- 2020-04-29 KR KR1020217038223A patent/KR20220008280A/ko unknown
- 2020-04-29 US US17/606,414 patent/US20220193261A1/en active Pending
- 2020-04-29 AU AU2020266829A patent/AU2020266829A1/en active Pending
- 2020-04-29 TW TW109114314A patent/TW202100541A/zh unknown
- 2020-04-29 BR BR112021021720A patent/BR112021021720A2/pt unknown
- 2020-04-29 BR BR112021021792A patent/BR112021021792A2/pt unknown
- 2020-04-29 MX MX2021013365A patent/MX2021013365A/es unknown
- 2020-04-29 WO PCT/US2020/030484 patent/WO2020223356A1/en unknown
- 2020-04-29 CN CN202080049015.0A patent/CN114072515A/zh active Pending
- 2020-04-29 CA CA3134485A patent/CA3134485A1/en active Pending
- 2020-04-29 EP EP20798851.0A patent/EP3980548A4/en active Pending
- 2020-04-29 SG SG11202111380VA patent/SG11202111380VA/en unknown
- 2020-04-29 CN CN202080048355.1A patent/CN114127275A/zh active Pending
- 2020-04-29 MX MX2021013364A patent/MX2021013364A/es unknown
- 2020-04-29 SG SG11202111400TA patent/SG11202111400TA/en unknown
- 2020-04-29 JP JP2021564730A patent/JP2022530824A/ja active Pending
- 2020-04-29 JP JP2021565001A patent/JP2022530833A/ja active Pending
- 2020-04-29 US US17/606,410 patent/US20220193207A1/en active Pending
-
2021
- 2021-10-20 CL CL2021002755A patent/CL2021002755A1/es unknown
- 2021-10-20 CL CL2021002754A patent/CL2021002754A1/es unknown
- 2021-10-24 IL IL287522A patent/IL287522A/en unknown
- 2021-10-24 IL IL287523A patent/IL287523A/en unknown
- 2021-11-29 CO CONC2021/0016200A patent/CO2021016200A2/es unknown
- 2021-11-29 CO CONC2021/0016198A patent/CO2021016198A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
CN114072515A (zh) | 2022-02-18 |
CL2021002755A1 (es) | 2022-05-27 |
TW202100541A (zh) | 2021-01-01 |
WO2020223362A1 (en) | 2020-11-05 |
CO2021016198A2 (es) | 2022-01-17 |
JP2022530833A (ja) | 2022-07-01 |
SG11202111400TA (en) | 2021-11-29 |
CA3134523A1 (en) | 2020-11-05 |
EP3980548A1 (en) | 2022-04-13 |
WO2020223362A8 (en) | 2021-01-14 |
MX2021013364A (es) | 2022-01-26 |
JP2022530824A (ja) | 2022-07-01 |
WO2020223356A1 (en) | 2020-11-05 |
CO2021016200A2 (es) | 2022-01-17 |
EP3963063A1 (en) | 2022-03-09 |
EP3963063A4 (en) | 2023-09-27 |
IL287523A (en) | 2021-12-01 |
CN114127275A (zh) | 2022-03-01 |
AU2020266829A1 (en) | 2021-11-11 |
KR20220004696A (ko) | 2022-01-11 |
US20220193261A1 (en) | 2022-06-23 |
BR112021021720A2 (pt) | 2021-12-28 |
KR20220008280A (ko) | 2022-01-20 |
SG11202111380VA (en) | 2021-11-29 |
IL287522A (en) | 2021-12-01 |
BR112021021792A2 (pt) | 2022-01-04 |
MX2021013365A (es) | 2022-01-26 |
US20220193207A1 (en) | 2022-06-23 |
EP3980548A4 (en) | 2023-09-06 |
CA3134485A1 (en) | 2020-11-05 |
AU2020266552A1 (en) | 2021-11-11 |
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