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
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
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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    • A61K48/0075—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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    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
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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

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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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WO2017180915A2 (en) 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
MA45477A (fr) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
HUE066442T2 (hu) * 2016-04-15 2024-08-28 Res Institute At Nationwide ß-szarkoglikán és mikroRNS-29 adenoasszociáltvírus-vektorral történõ bejuttatása és izomdisztrófia kezelése
EP3487523B1 (en) 2016-07-19 2023-09-06 Duke University Therapeutic applications of cpf1-based genome editing
JP7162021B2 (ja) 2017-03-17 2022-10-27 ニューカッスル ユニバーシティ 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
EP3697916A4 (en) * 2017-10-20 2021-08-11 Research Institute at Nationwide Children's Hospital METHODS AND MATERIALS FOR NT-3 GENE THERAPY
SG11202006722RA (en) 2018-01-31 2020-08-28 Res Inst Nationwide Childrens Hospital Gene therapy for limb-girdle muscular dystrophy type 2c
MY208145A (en) 2018-06-18 2025-04-18 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
KR20210028162A (ko) * 2018-06-29 2021-03-11 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
EP3598978B1 (en) 2018-07-26 2024-05-29 EXOFIX S.r.l. Fibroadipogenic progenitor-derived exosomes for regeneration of dystrophic muscles
WO2020154034A1 (en) * 2019-01-25 2020-07-30 Children's Medical Center Corporation Compositions and methods for treating muscular dystrophy and related disorders
CA3146364A1 (en) * 2019-02-02 2020-08-06 University Of Pittsburgh-Of The Commonwealth System Of Higher Education One-step gene therapy for duchenne muscular dystrophy via gene replacement and anti-inflammation
CA3131390A1 (en) * 2019-02-26 2020-09-03 Research Institute Of Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
HRP20240505T1 (hr) 2019-08-21 2024-07-05 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana vektorom povezanim s adenovirusom i liječenje mišićne distrofije
US20230040544A1 (en) * 2019-12-16 2023-02-09 Research Institute At Nationwide Children's Hospital Compositions and methods for restoring and maintaining the 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
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
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