MY188987A - Aav vectors for retinal and cns gene therapy - Google Patents

Aav vectors for retinal and cns gene therapy

Info

Publication number
MY188987A
MY188987A MYPI2016703542A MYPI2016703542A MY188987A MY 188987 A MY188987 A MY 188987A MY PI2016703542 A MYPI2016703542 A MY PI2016703542A MY PI2016703542 A MYPI2016703542 A MY PI2016703542A MY 188987 A MY188987 A MY 188987A
Authority
MY
Malaysia
Prior art keywords
cns
raav
gene therapy
improved
disorders
Prior art date
Application number
MYPI2016703542A
Other languages
English (en)
Inventor
Abraham Scaria
Jennifer Sullivan
Lisa M Stanek
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.)
Filing date
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=MY188987(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 MY188987A publication Critical patent/MY188987A/en

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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
    • C12N15/63—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
    • 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
    • A61K48/0075—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
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    • 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
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    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • A—HUMAN NECESSITIES
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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
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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
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • 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

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MYPI2016703542A 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy MY188987A (en)

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
MY188987A true MY188987A (en) 2022-01-17

Family

ID=53189202

Family Applications (1)

Application Number Title Priority Date Filing Date
MYPI2016703542A MY188987A (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy

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US (3) US10982228B2 (enExample)
EP (3) EP4600255A3 (enExample)
JP (4) JP6741591B2 (enExample)
KR (3) KR102863726B1 (enExample)
CN (2) CN114231563B (enExample)
AU (3) AU2015252797C1 (enExample)
CA (1) CA2946593A1 (enExample)
CL (2) CL2016002713A1 (enExample)
CR (1) CR20160555A (enExample)
DK (2) DK3137497T4 (enExample)
DO (1) DOP2016000280A (enExample)
EA (1) EA034355B1 (enExample)
ES (1) ES2879636T5 (enExample)
FI (2) FI3913061T3 (enExample)
GT (1) GT201600227A (enExample)
HR (1) HRP20211024T4 (enExample)
HU (1) HUE054768T2 (enExample)
IL (2) IL248102B (enExample)
LT (2) LT3137497T (enExample)
MX (2) MX2016014220A (enExample)
MY (1) MY188987A (enExample)
PE (1) PE20170260A1 (enExample)
PH (1) PH12016502046A1 (enExample)
PL (1) PL3137497T5 (enExample)
PT (2) PT3913061T (enExample)
RS (2) RS62078B2 (enExample)
SG (3) SG11201607991WA (enExample)
SI (1) SI3137497T2 (enExample)
TW (1) TWI686476B (enExample)
UY (1) UY36106A (enExample)
WO (1) WO2015168666A2 (enExample)
ZA (1) ZA201606763B (enExample)

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JP2016523835A (ja) 2013-05-15 2016-08-12 ăƒȘăƒŒă‚žă‚§ăƒłăƒ„ ă‚Șブ ザ ăƒŠăƒ‹ăƒăƒŒă‚·ăƒ†ă‚Ł ă‚Șブ ăƒŸăƒă‚œă‚ż äž­æžąç„žç”Œçł»ăžăźă‚ąăƒ‡ăƒŽéšäŒŽă‚Šă‚€ăƒ«ă‚čă‚’ä»‹ă—ăŸéșäŒć­ć°Žć…„
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
ES2822551T3 (es) 2014-03-17 2021-05-04 Adverum Biotechnologies Inc Composiciones y métodos para la expresión génica mejorada en células de los conos
JP6756700B2 (ja) 2014-03-18 2020-09-16 ăƒŠăƒ‹ăƒăƒŒă‚·ăƒ†ă‚Ł ă‚Șブ ăƒžă‚”ăƒăƒ„ăƒŒă‚»ăƒƒăƒ„ ç­‹èŽçžźæ€§ćŽçŽąçĄŹćŒ–ç—‡ă‚’ć‡Šçœźă™ă‚‹ăŸă‚ăźïœ’ïŒĄïŒĄïŒ¶ăƒ™ăƒŒă‚čăźç”„æˆç‰©ăŠă‚ˆăłæ–čæł•
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genƳ terapijai
SG11201609314XA (en) * 2014-05-14 2016-12-29 Esteve Labor Dr Adenoassociated virus vectors for the treatment of lysosomal storage disorders
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
WO2016054557A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Novel high efficiency library-identified aav vectors
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
EA201791803A1 (ru) * 2015-02-10 2017-11-30 Đ”Đ¶Đ”ĐœĐ·ĐžĐŒ ĐšĐŸŃ€ĐżĐŸŃ€Đ”ĐčŃˆĐœ ĐŁĐ»ŃƒŃ‡ŃˆĐ”ĐœĐœĐ°Ń ĐŽĐŸŃŃ‚Đ°ĐČĐșĐ° ĐČĐžŃ€ŃƒŃĐœŃ‹Ń… частоц ĐČ ĐżĐŸĐ»ĐŸŃĐ°Ń‚ĐŸĐ” Ń‚Đ”Đ»ĐŸ Đž ĐșĐŸŃ€Ńƒ Đ±ĐŸĐ»ŃŒŃˆĐžŃ… ĐżĐŸĐ»ŃƒŃˆĐ°Ń€ĐžĐč
CA3255657A1 (en) 2015-02-10 2025-12-01 Genzyme Corporation VARIANT RNAi
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
FI3265178T3 (fi) 2015-03-02 2026-08-03 Adverum Biotechnologies Inc Koostumuksia ja menetelmiÀ polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin
CA2979229A1 (en) * 2015-03-11 2016-09-15 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Rp2 vectors for treating x-linked retinitis pigmentosa
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