JP2022553375A5 - - Google Patents

Info

Publication number
JP2022553375A5
JP2022553375A5 JP2022523854A JP2022523854A JP2022553375A5 JP 2022553375 A5 JP2022553375 A5 JP 2022553375A5 JP 2022523854 A JP2022523854 A JP 2022523854A JP 2022523854 A JP2022523854 A JP 2022523854A JP 2022553375 A5 JP2022553375 A5 JP 2022553375A5
Authority
JP
Japan
Prior art keywords
polynucleotide construct
sequence
seq
composition
expression cassette
Prior art date
Legal status (The legal status 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 status listed.)
Granted
Application number
JP2022523854A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022553375A (ja
JP7826197B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/056890 external-priority patent/WO2021081225A1/en
Publication of JP2022553375A publication Critical patent/JP2022553375A/ja
Publication of JP2022553375A5 publication Critical patent/JP2022553375A5/ja
Application granted granted Critical
Publication of JP7826197B2 publication Critical patent/JP7826197B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2022523854A 2019-10-22 2020-10-22 オルニチントランスカルバミラーゼ(otc)構築物およびそれを使用する方法 Active JP7826197B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962924567P 2019-10-22 2019-10-22
US62/924,567 2019-10-22
PCT/US2020/056890 WO2021081225A1 (en) 2019-10-22 2020-10-22 Ornithine transcarbamylase (otc) constructs and methods of using the same

Publications (3)

Publication Number Publication Date
JP2022553375A JP2022553375A (ja) 2022-12-22
JP2022553375A5 true JP2022553375A5 (enExample) 2023-10-26
JP7826197B2 JP7826197B2 (ja) 2026-03-09

Family

ID=75620841

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022523854A Active JP7826197B2 (ja) 2019-10-22 2020-10-22 オルニチントランスカルバミラーゼ(otc)構築物およびそれを使用する方法

Country Status (9)

Country Link
US (1) US20230001021A1 (enExample)
EP (1) EP4048317A4 (enExample)
JP (1) JP7826197B2 (enExample)
KR (1) KR20240130161A (enExample)
CN (1) CN116096428A (enExample)
AU (1) AU2020371697A1 (enExample)
CA (1) CA3158626A1 (enExample)
IL (1) IL292420A (enExample)
WO (1) WO2021081225A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3864163T3 (da) 2018-10-09 2024-05-06 Univ British Columbia Sammensætninger og systemer omfattende transfektionskompetente vesikler fri for organiske opløsningsmidler og rengøringsmidler og fremgangsmåder forbundet dermed
WO2022229903A1 (en) * 2021-04-28 2022-11-03 Genevant Sciences Gmbh Mrna delivery constructs and methods of using the same
KR102971055B1 (ko) * 2021-12-03 2026-05-29 주식회사 녹십자 번역 효율이 향상된 5'-utr, 이를 포함하는 합성 핵산 분자 및 이를 포함하는 백신 또는 치료제 조성물
WO2024186940A1 (en) * 2023-03-06 2024-09-12 Braidwell Labs Llc Codon optimization and methods of use thereof

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8853377B2 (en) * 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
US9352042B2 (en) * 2012-02-24 2016-05-31 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
WO2015011633A1 (en) * 2013-07-23 2015-01-29 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
AU2014296278C1 (en) * 2013-07-30 2023-02-02 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
JP6420836B2 (ja) * 2013-08-23 2018-11-07 ダウ グローバル テクノロジーズ エルエルシー 発泡法及び膨張法の組み合わせを用いて作製される低密度ポリウレタンフォームが取り付けられた織物
EP3247328B1 (en) * 2015-01-21 2026-01-07 Genevant Sciences Gmbh Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
AU2017283479B2 (en) * 2016-06-13 2023-08-17 Translate Bio, Inc. Messenger RNA therapy for the treatment of ornithine transcarbamylase deficiency
US11938197B2 (en) * 2017-01-10 2024-03-26 The Sydney Children's Hospitals Network (Randwick And Westmead (Incorporating The Royal Alexandra Hospital For Children) Polynucleotides and vectors for the expression of transgenes
BR112020004351A2 (pt) * 2017-10-19 2020-09-08 Curevac Ag moléculas de ácido nucleico artificial
EP3714048B1 (en) * 2017-11-22 2025-04-09 ModernaTX, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders

Similar Documents

Publication Publication Date Title
JP2026041749A5 (enExample)
CN110914427B (zh) 调节性多核苷酸
EP3368687B1 (en) Compositions and methods for targeting cancer-specific sequence variations
AU780517B2 (en) Medicament for treatment of duchenne muscular dystrophy
CN112040988A (zh) 修饰的aav衣壳多肽用于治疗肌肉疾病
JP2022524434A (ja) Fviii治療薬を発現するための非ウイルス性dnaベクターおよびその使用
JP2023548838A (ja) ニワトリ貧血ウイルス(cav)に基づくベクター
CN116942700B (zh) 一种多靶点修饰的工程化m2巨噬细胞外囊泡及其制备方法和应用
TW202417632A (zh) 新穎指環病毒科(anelloviridae)載體組合物及方法
Lundstrom Gene therapy cargoes based on viral vector delivery
JP2021533801A5 (enExample)
JP3877148B2 (ja) 糖尿病性虚血性疾患遺伝子治療
JPWO2006101173A1 (ja) 核酸分子とコラーゲンの複合体微細粒子製剤
TW202302855A (zh) 用於治療法布瑞氏症之組成物及方法
JP2024501287A (ja) RNAを閉じ込めたアネロウイルス(Anellovirus)キャプシドのインビトロアセンブリ
JP2024501022A5 (enExample)
JP2024515317A5 (enExample)
JP2022552378A5 (enExample)
JP2025510946A5 (enExample)
JP2025538982A (ja) モヤモヤ病の予防または治療用組成物
CN115998905B (zh) 治疗心衰的mRNA药物制剂及其制备方法与应用
CN119818705B (zh) 一种mRNA组合物及其应用
JP3744583B2 (ja) 遺伝子導入用組成物及び該組成物を用いた遺伝子導入方法
JPWO2003103721A1 (ja) 脳血管障害遺伝子治療剤
EP1598420A2 (fr) Compositions pharmaceutiques et leur utilisation, notamment pour le traitement de maladies neurodégénératives