BR112016025263A2 - vetores de avv para terapia genética retiniana e do snc - Google Patents

vetores de avv para terapia genética retiniana e do snc

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Publication number
BR112016025263A2
BR112016025263A2 BR112016025263-2A BR112016025263A BR112016025263A2 BR 112016025263 A2 BR112016025263 A2 BR 112016025263A2 BR 112016025263 A BR112016025263 A BR 112016025263A BR 112016025263 A2 BR112016025263 A2 BR 112016025263A2
Authority
BR
Brazil
Prior art keywords
snc
disorders
gene therapy
enhanced
treating
Prior art date
Application number
BR112016025263-2A
Other languages
English (en)
Other versions
BR112016025263B1 (pt
Inventor
Scaria Abraham
Sullivan Jennifer
M. Stanek Lisa
Original Assignee
Genzyme Corporation
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=BR112016025263(A2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corporation filed Critical Genzyme Corporation
Publication of BR112016025263A2 publication Critical patent/BR112016025263A2/pt
Publication of BR112016025263B1 publication Critical patent/BR112016025263B1/pt

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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
    • 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
    • A61P35/00—Antineoplastic agents
    • 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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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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  • Health & Medical Sciences (AREA)
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Abstract

são fornecidos aqui raav aprimorados (por exemplo, raav2, raavrh8r, etc.) para terapia genética intensificada de transtornos oculares ou transtornos do snc, em que o raav compreende uma ou mais substituições de aminoácidos que interagem com proteoglicanos de sulfato de heparana. a invenção fornece métodos para aprimorar a transdução de células retinianas e métodos para o tratamento de doenças oculares com composições de partículas de raav aprimoradas. são ainda fornecidos aqui vírus adenoassociados recombinantes (raav) aprimorados (por exemplo, raav2, raavrh8r, etc.) para terapia genética intensificada de transtornos do snc. a invenção fornece métodos para distribuição de raav ao snc, métodos para o tratamento de transtornos do snc com composições aprimoradas de partículas de raav e kits para distribuição de raav ao snc e/ou tratamento de um transtorno do snc.
BR112016025263-2A 2014-05-02 2015-05-02 Usos: de uma partícula de vírus adenoassociado recombinante (raav), de um kit, e de uma composição compreendendo uma partícula de vírus adenoassociado recombinante (raav) BR112016025263B1 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461988131P 2014-05-02 2014-05-02
US61/988,131 2014-05-02
US201562114575P 2015-02-10 2015-02-10
US62/114,575 2015-02-10
PCT/US2015/028966 WO2015168666A2 (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy

Publications (2)

Publication Number Publication Date
BR112016025263A2 true BR112016025263A2 (pt) 2018-02-20
BR112016025263B1 BR112016025263B1 (pt) 2024-08-06

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MY188987A (en) 2022-01-17
JP2017518271A (ja) 2017-07-06
SG11201607991WA (en) 2016-10-28
IL248102B (en) 2022-07-01
MX2016014220A (es) 2017-02-06
US20210189430A1 (en) 2021-06-24
CR20160555A (es) 2017-01-27
SI3137497T1 (sl) 2021-08-31
EP3137497A2 (en) 2017-03-08
SG10201912977RA (en) 2020-02-27
AU2021200242B2 (en) 2023-09-21
IL292951B2 (en) 2024-10-01
CN114231563B (zh) 2024-08-16
JP2023011812A (ja) 2023-01-24
ZA201606763B (en) 2023-05-31
GT201600227A (es) 2018-01-22
US20170096683A1 (en) 2017-04-06
CA2946593A1 (en) 2015-11-05
AU2015252797B2 (en) 2020-10-15
JP2020164540A (ja) 2020-10-08
EA034355B1 (ru) 2020-01-30
AU2021200242A1 (en) 2021-03-18
US10982228B2 (en) 2021-04-20
CL2016002713A1 (es) 2017-08-11
HRP20211024T1 (hr) 2021-10-01
IL248102A0 (en) 2016-11-30
CN106661591B (zh) 2021-10-29
WO2015168666A2 (en) 2015-11-05
PH12016502046A1 (en) 2017-02-06
CL2018000170A1 (es) 2018-06-29
EP3913061A1 (en) 2021-11-24
JP6741591B2 (ja) 2020-08-19
AU2021200242B9 (en) 2023-10-05
PL3137497T3 (pl) 2021-10-25
CN114231563A (zh) 2022-03-25
MX2022012490A (es) 2022-10-27
ES2879636T3 (es) 2021-11-22
SG10201809739QA (en) 2018-12-28
CN106661591A (zh) 2017-05-10
WO2015168666A3 (en) 2015-12-23
JP7174011B2 (ja) 2022-11-17
TWI686476B (zh) 2020-03-01
UY36106A (es) 2015-11-30
IL292951A (en) 2022-07-01
DOP2016000280A (es) 2016-11-30
LT3137497T (lt) 2021-07-26
PL3137497T4 (pl) 2023-04-11
KR20240032156A (ko) 2024-03-08
EA201692206A1 (ru) 2017-03-31
TW201625792A (zh) 2016-07-16
RS62078B1 (sr) 2021-07-30
AU2015252797C1 (en) 2021-04-22
PT3137497T (pt) 2021-07-12
PE20170260A1 (es) 2017-04-12
IL292951B1 (en) 2024-06-01
EP3137497B1 (en) 2021-04-07
KR20160147974A (ko) 2016-12-23
DK3137497T3 (da) 2021-07-12
HUE054768T2 (hu) 2021-09-28
AU2023285817A1 (en) 2024-01-18
AU2015252797A1 (en) 2016-11-17

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