PL3442600T3 - Dostarczanie za pośrednictwem wirusa związanego z adenowirusem wektora b- sarkoglikanu i mikroRNA-29 oraz leczenie dystrofii mięśniowej - Google Patents

Dostarczanie za pośrednictwem wirusa związanego z adenowirusem wektora b- sarkoglikanu i mikroRNA-29 oraz leczenie dystrofii mięśniowej

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Publication number
PL3442600T3
PL3442600T3 PL17783207.8T PL17783207T PL3442600T3 PL 3442600 T3 PL3442600 T3 PL 3442600T3 PL 17783207 T PL17783207 T PL 17783207T PL 3442600 T3 PL3442600 T3 PL 3442600T3
Authority
PL
Poland
Prior art keywords
sarcoglycan
microrna
adeno
treatment
associated virus
Prior art date
Application number
PL17783207.8T
Other languages
English (en)
Inventor
Louise RODINO-KLAPAC
Jerry R. MENDELL
Original Assignee
Research Institute At Nationwide Children's Hospital
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Filing date
Publication date
Application filed by Research Institute At Nationwide Children's Hospital filed Critical Research Institute At Nationwide Children's Hospital
Publication of PL3442600T3 publication Critical patent/PL3442600T3/pl

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    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
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    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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PL17783207.8T 2016-04-15 2017-04-14 Dostarczanie za pośrednictwem wirusa związanego z adenowirusem wektora b- sarkoglikanu i mikroRNA-29 oraz leczenie dystrofii mięśniowej PL3442600T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662323333P 2016-04-15 2016-04-15
US201662433548P 2016-12-13 2016-12-13
PCT/US2017/027583 WO2017180976A1 (en) 2016-04-15 2017-04-14 Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy

Publications (1)

Publication Number Publication Date
PL3442600T3 true PL3442600T3 (pl) 2024-07-08

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PL17783207.8T PL3442600T3 (pl) 2016-04-15 2017-04-14 Dostarczanie za pośrednictwem wirusa związanego z adenowirusem wektora b- sarkoglikanu i mikroRNA-29 oraz leczenie dystrofii mięśniowej

Country Status (28)

Country Link
US (3) US11358993B2 (pl)
EP (2) EP4407035A3 (pl)
JP (2) JP7079733B2 (pl)
KR (1) KR102502091B1 (pl)
CN (2) CN109121395B (pl)
AU (2) AU2017250756B2 (pl)
BR (1) BR112018071167A2 (pl)
CA (1) CA3021057A1 (pl)
CO (1) CO2018012082A2 (pl)
DK (1) DK3442600T5 (pl)
ES (1) ES2979174T3 (pl)
FI (1) FI3442600T3 (pl)
HR (1) HRP20240580T1 (pl)
HU (1) HUE066442T2 (pl)
IL (1) IL262262B2 (pl)
LT (1) LT3442600T (pl)
MA (1) MA44681A (pl)
MX (2) MX391208B (pl)
MY (1) MY195439A (pl)
PL (1) PL3442600T3 (pl)
PT (1) PT3442600T (pl)
RS (1) RS65490B1 (pl)
SA (1) SA518400245B1 (pl)
SG (1) SG11201809029RA (pl)
SI (1) SI3442600T1 (pl)
SM (1) SMT202400169T1 (pl)
WO (1) WO2017180976A1 (pl)
ZA (1) ZA201806863B (pl)

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US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
PL3442600T3 (pl) * 2016-04-15 2024-07-08 Research Institute At Nationwide Children's Hospital Dostarczanie za pośrednictwem wirusa związanego z adenowirusem wektora b- sarkoglikanu i mikroRNA-29 oraz leczenie dystrofii mięśniowej
LT3442602T (lt) 2016-04-15 2025-06-10 Research Institute At Nationwide Children's Hospital Mikrodistrofino adeno-asocijuoto viruso vektoriaus pateikimas raumenų distrofijos gydymui
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
KR20200083495A (ko) * 2017-10-20 2020-07-08 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Nt-3 유전자 치료를 위한 방법 및 물질
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
CN119770682A (zh) 2018-01-31 2025-04-08 国家儿童医院研究所 2c型肢带型肌营养不良的基因疗法
DK3807413T3 (da) 2018-06-18 2025-08-25 Res Inst Nationwide Childrens Hospital Adenoassocieret virusvektorfremføring af muskelspecifik mikrodystrofin til behandling af muskeldystrofi
CN112543810A (zh) 2018-06-29 2021-03-23 全国儿童医院研究所 治疗肢带型肌营养不良2a型的重组腺相关病毒产品和方法
EP3598978B1 (en) 2018-07-26 2024-05-29 EXOFIX S.r.l. Fibroadipogenic progenitor-derived exosomes for regeneration of dystrophic muscles
US20220119489A1 (en) * 2019-01-25 2022-04-21 Children's Medical Center Corporation Compositions and methods for treating muscular dystrophy and related disorders
CN113710809A (zh) * 2019-02-02 2021-11-26 联邦高等教育系统-匹兹堡大学 通过基因置换和抗炎的针对杜氏肌营养不良的一步基因疗法
AU2020229340A1 (en) * 2019-02-26 2021-09-16 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy
KR20220092489A (ko) * 2019-08-21 2022-07-01 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 알파-사코글리칸의 아데노 관련 바이러스 벡터 전달 및 근위축증 치료
EP4117783A4 (en) * 2019-12-16 2024-08-21 Research Institute at Nationwide Children's Hospital COMPOSITIONS AND METHODS FOR RESTORING AND MAINTAINING DYSTROPHIN-ASSOCIATED PROTEIN COMPLEX (DAPC)
WO2021255245A2 (en) * 2020-06-19 2021-12-23 Genethon Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
US12285497B2 (en) * 2021-10-15 2025-04-29 Research Institute At Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
KR20250044484A (ko) 2022-06-24 2025-03-31 튠 쎄라퓨틱스, 인코포레이티드 표적 유전자 억제를 통해 저밀도 지단백질을 감소시키기 위한 조성물, 시스템, 및 방법
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