MX2016014220A - Vectores de aav para la terapia genica de la retina y el snc. - Google Patents

Vectores de aav para la terapia genica de la retina y el snc.

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Publication number
MX2016014220A
MX2016014220A MX2016014220A MX2016014220A MX2016014220A MX 2016014220 A MX2016014220 A MX 2016014220A MX 2016014220 A MX2016014220 A MX 2016014220A MX 2016014220 A MX2016014220 A MX 2016014220A MX 2016014220 A MX2016014220 A MX 2016014220A
Authority
MX
Mexico
Prior art keywords
cns
raav
gene therapy
improved
disorders
Prior art date
Application number
MX2016014220A
Other languages
English (en)
Inventor
Scaria Abraham
Sullivan Jennifer
M Stanek Lisa
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=53189202&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=MX2016014220(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of MX2016014220A publication Critical patent/MX2016014220A/es

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    • A—HUMAN NECESSITIES
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    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
    • A61P9/00—Drugs for disorders of the cardiovascular system
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
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    • C07—ORGANIC CHEMISTRY
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    • 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
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    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Abstract

En la presente memoria se proporcionan rAAV mejorados (p.ej., rAAV2, rAAVrh8R, etc.) para la potenciación de la terapia génica de trastornos oculares o trastornos de SNC en donde el rAAV comprende una o más sustituciones de aminoácidos que interactúan con el proteoglicano heparán sulfato. La invención proporciona método para mejorar la transducción de células retinianas y método para tratar enfermedades oculares con composiciones mejoradas de partículas de rAAV. En la presente memoria se proporcionan adicionalmente virus adeno-asociados recombinantes (rAAV) (p.ej., rAAV2, rAAVrh8R, etc.) para potenciar la terapia génica de trastornos del SNC. La invención proporciona métodos para suministrar el rAAV al SNC, métodos para tratar trastornos del SNC con composiciones mejoradas de partículas de rAAV, y kits para suministrar la rAAV al SNC y/o tratar un trastorno del SNC.
MX2016014220A 2014-05-02 2015-05-02 Vectores de aav para la terapia genica de la retina y el snc. MX2016014220A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461988131P 2014-05-02 2014-05-02
US201562114575P 2015-02-10 2015-02-10
PCT/US2015/028966 WO2015168666A2 (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy

Publications (1)

Publication Number Publication Date
MX2016014220A true MX2016014220A (es) 2017-02-06

Family

ID=53189202

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2016014220A MX2016014220A (es) 2014-05-02 2015-05-02 Vectores de aav para la terapia genica de la retina y el snc.
MX2022012490A MX2022012490A (es) 2014-05-02 2016-10-28 Vectores de aav para la terapia genica de la retina y el snc.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2022012490A MX2022012490A (es) 2014-05-02 2016-10-28 Vectores de aav para la terapia genica de la retina y el snc.

Country Status (32)

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US (2) US10982228B2 (es)
EP (2) EP3913061A1 (es)
JP (3) JP6741591B2 (es)
KR (2) KR20160147974A (es)
CN (2) CN114231563A (es)
AU (3) AU2015252797C1 (es)
BR (1) BR112016025263A2 (es)
CA (1) CA2946593A1 (es)
CL (2) CL2016002713A1 (es)
CR (1) CR20160555A (es)
DK (1) DK3137497T3 (es)
DO (1) DOP2016000280A (es)
EA (1) EA034355B1 (es)
ES (1) ES2879636T3 (es)
GT (1) GT201600227A (es)
HR (1) HRP20211024T1 (es)
HU (1) HUE054768T2 (es)
IL (2) IL248102B (es)
LT (1) LT3137497T (es)
MX (2) MX2016014220A (es)
MY (1) MY188987A (es)
PE (1) PE20170260A1 (es)
PH (1) PH12016502046A1 (es)
PL (1) PL3137497T4 (es)
PT (1) PT3137497T (es)
RS (1) RS62078B1 (es)
SG (3) SG11201607991WA (es)
SI (1) SI3137497T1 (es)
TW (1) TWI686476B (es)
UY (1) UY36106A (es)
WO (1) WO2015168666A2 (es)
ZA (1) ZA201606763B (es)

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