JP2018536420A5 - - Google Patents

Download PDF

Info

Publication number
JP2018536420A5
JP2018536420A5 JP2018529927A JP2018529927A JP2018536420A5 JP 2018536420 A5 JP2018536420 A5 JP 2018536420A5 JP 2018529927 A JP2018529927 A JP 2018529927A JP 2018529927 A JP2018529927 A JP 2018529927A JP 2018536420 A5 JP2018536420 A5 JP 2018536420A5
Authority
JP
Japan
Prior art keywords
composition
promoter
transgene
cells
hair cells
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
JP2018529927A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018536420A (ja
JP6990182B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2016/066225 external-priority patent/WO2017100791A1/en
Publication of JP2018536420A publication Critical patent/JP2018536420A/ja
Publication of JP2018536420A5 publication Critical patent/JP2018536420A5/ja
Priority to JP2021196596A priority Critical patent/JP7309827B2/ja
Application granted granted Critical
Publication of JP6990182B2 publication Critical patent/JP6990182B2/ja
Priority to JP2023110371A priority patent/JP7745594B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2018529927A 2015-12-11 2016-12-12 蝸牛および前庭細胞に核酸を送達するための材料および方法 Active JP6990182B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2021196596A JP7309827B2 (ja) 2015-12-11 2021-12-03 蝸牛および前庭細胞に核酸を送達するための材料および方法
JP2023110371A JP7745594B2 (ja) 2015-12-11 2023-07-05 蝸牛および前庭細胞に核酸を送達するための材料および方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562266462P 2015-12-11 2015-12-11
US201562266477P 2015-12-11 2015-12-11
US62/266,477 2015-12-11
US62/266,462 2015-12-11
PCT/US2016/066225 WO2017100791A1 (en) 2015-12-11 2016-12-12 Materials and methods for delivering nucleic acids to cochlear and vestibular cells

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2021196596A Division JP7309827B2 (ja) 2015-12-11 2021-12-03 蝸牛および前庭細胞に核酸を送達するための材料および方法

Publications (3)

Publication Number Publication Date
JP2018536420A JP2018536420A (ja) 2018-12-13
JP2018536420A5 true JP2018536420A5 (https=) 2019-12-05
JP6990182B2 JP6990182B2 (ja) 2022-02-15

Family

ID=59013436

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2018529927A Active JP6990182B2 (ja) 2015-12-11 2016-12-12 蝸牛および前庭細胞に核酸を送達するための材料および方法
JP2021196596A Active JP7309827B2 (ja) 2015-12-11 2021-12-03 蝸牛および前庭細胞に核酸を送達するための材料および方法
JP2023110371A Active JP7745594B2 (ja) 2015-12-11 2023-07-05 蝸牛および前庭細胞に核酸を送達するための材料および方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2021196596A Active JP7309827B2 (ja) 2015-12-11 2021-12-03 蝸牛および前庭細胞に核酸を送達するための材料および方法
JP2023110371A Active JP7745594B2 (ja) 2015-12-11 2023-07-05 蝸牛および前庭細胞に核酸を送達するための材料および方法

Country Status (10)

Country Link
US (2) US11167042B2 (https=)
EP (2) EP3386537B1 (https=)
JP (3) JP6990182B2 (https=)
KR (1) KR102794870B1 (https=)
CN (2) CN109310745B (https=)
AU (1) AU2016366846B2 (https=)
CA (1) CA3007476A1 (https=)
ES (1) ES2903000T3 (https=)
SG (2) SG10201913266UA (https=)
WO (1) WO2017100791A1 (https=)

Families Citing this family (74)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10834922B2 (en) * 2014-11-26 2020-11-17 Microban Products Company Surface disinfectant with residual biocidal property
SG10201913266UA (en) 2015-12-11 2020-02-27 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
WO2018145111A1 (en) * 2017-02-06 2018-08-09 Children's Medical Center Corporation Materials and methods for delivering nucleic acids to cochlear and vestibular cells
JP2020509786A (ja) * 2017-03-17 2020-04-02 レスキュー ヒヤリング インコーポレイテッド 聴覚損失の治療のための遺伝子治療構成物および方法
US12391936B2 (en) 2017-03-17 2025-08-19 Rescue Hearing, Inc. Gene therapy systems and related methods for treatment of hearing loss
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
US20210154326A1 (en) * 2017-06-26 2021-05-27 Arizona Board Of Regents On Behalf Of Arizona State University CRISPR-Based Synthetic Gene Circuits as Next Generation Gene Therapy of Inner Ear
EP3661523A4 (en) * 2017-08-03 2021-08-04 University of Iowa Research Foundation TREATMENT METHODS FOR GENETIC HEARING LOSS
EP3727468A4 (en) 2017-12-19 2021-09-22 Akouos, Inc. AAV-MEDIATED THERAPEUTIC ANTIBODIES DELIVERY TO THE INTERNAL EAR
DE102018100619A1 (de) 2018-01-12 2019-07-18 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
AU2019209770B2 (en) 2018-01-16 2025-07-31 Cls Therapeutics Limited Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity
DE102018103924A1 (de) 2018-02-21 2019-08-22 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
EP3762040A4 (en) * 2018-03-05 2022-01-05 Children's Medical Center Corporation COMPOSITIONS AND METHODS OF ADMINISTRATION OF NUCLEIC ACIDS TO COCHLEAR AND VESTIBULAR CELLS
BR112020019079A2 (pt) 2018-03-23 2020-12-29 Massachusetts Eye And Ear Infirmary Abordagem de salto de éxon mediada por crispr/cas9 para síndrome de usher associada a ush2a
WO2019200016A1 (en) * 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN112703016B (zh) * 2018-04-27 2024-11-22 分贝治疗公司 肌球蛋白15启动子及其用途
CA3100006A1 (en) * 2018-05-11 2019-11-14 Massachusetts Eye And Ear Infirmary Altering tissue tropism of adeno-associated viruses
US20210277417A1 (en) * 2018-06-25 2021-09-09 Akouos, Inc. Methods of treating clrn1-associated hearing loss and/or vision loss
WO2020014625A1 (en) * 2018-07-13 2020-01-16 Akouos, Inc. Methods of treating non-syndromic sensorineural hearing loss
US20210290727A1 (en) * 2018-08-01 2021-09-23 University Of Maryland, Baltimore MODULATION OF mTORCI ACTIVITY AND AUTOPHAGY VIA CIB2-RHEB INTERACTION
EP3856227A4 (en) * 2018-09-27 2022-07-06 Akouos, Inc. METHODS OF TREATMENT OF AUDITORY NEUROPATHY SPECTRUM DISEASE
WO2020077295A1 (en) * 2018-10-11 2020-04-16 Decibel Therapeutics, Inc. Aav1 vectors and uses thereof for treatment of otic indications
EP3880827A4 (en) * 2018-11-07 2023-04-26 Akouos, Inc. COMPOSITIONS AND METHODS FOR INDUCING DIFFERENTIATION OF A HAIR CELL
EP4063510A1 (en) * 2018-11-07 2022-09-28 Akouos, Inc. Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear
EP4295913A3 (en) 2019-01-18 2024-03-06 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
CA3129422A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
EP3921032A4 (en) * 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS
US20220202957A1 (en) * 2019-04-09 2022-06-30 The Regents Of The University Of California Long-lasting analgesia via targeted in vivo epigenetic repression
CN110129368B (zh) * 2019-04-22 2021-03-09 中国科学院脑科学与智能技术卓越创新中心 一种感染支持细胞和毛细胞的aav载体
US11278013B2 (en) 2019-04-26 2022-03-22 Usher 2020 Foundation Large animal model for developing therapeutic agents to treat impaired ophthalmic function in usher syndrome
EP3959324B1 (en) 2019-04-26 2025-09-17 President and Fellows of Harvard College Aav vectors encoding mini-pcdh15 and uses thereof
WO2020223933A1 (zh) * 2019-05-08 2020-11-12 中国科学院脑科学与智能技术卓越创新中心 高效感染支持细胞的aav突变体
EP3999647A1 (en) * 2019-07-18 2022-05-25 Lysogene Compositions and methods for the treatment of sanfilippo disease and other disorders
CN110423778B (zh) * 2019-07-26 2023-04-21 上海科技大学 一种逆行性重组2型腺相关病毒在感染螺旋神经元中的应用
CA3153972A1 (en) 2019-09-09 2021-03-18 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
WO2021067448A1 (en) 2019-09-30 2021-04-08 Applied Genetic Technologies Corporation Adeno-associated virus (aav) systems for treatment of genetic hearing loss
AU2020366531A1 (en) * 2019-10-18 2022-05-26 Research Institute At Nationwide Children's Hospital Gene therapy targeting cochlear cells
KR20220130093A (ko) 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
EP4055175A4 (en) 2019-11-04 2023-11-29 Decibel Therapeutics, Inc. Cochlear inner hair cell promoters and uses thereof
TWI902721B (zh) * 2019-11-04 2025-11-01 美商迪賽博治療公司 前庭支持細胞啟動子及其用途
WO2021108512A1 (en) * 2019-11-27 2021-06-03 Icahn School Of Medicine At Mount Sinai Gene therapy for the regeneration of auditory hair cells
US20230047254A1 (en) * 2019-12-29 2023-02-16 Gloriana Therapeutics Mammalian Cells Secreting GDNF and Their Therapeutic Use
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
CN115666658A (zh) 2020-04-01 2023-01-31 佛罗里达州大学研究基金会 具有提高的治疗ush1b的安全性的双aav-myo7a载体
WO2021222682A1 (en) * 2020-04-30 2021-11-04 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Application of aav44.9 vector in gene therapy for the inner ear
IL298128A (en) * 2020-05-13 2023-01-01 Akouos Inc Compositions and methods for treating slc26a4-associated hearing loss
WO2021231538A2 (en) * 2020-05-13 2021-11-18 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
WO2021231808A2 (en) * 2020-05-13 2021-11-18 Akouos, Inc. Compositions and methods for treating gjb2-associated hearing loss
KR102326582B1 (ko) * 2020-05-15 2021-11-16 사회복지법인 삼성생명공익재단 청각장애의 진단용 마커 및 그의 용도
WO2022032151A1 (en) * 2020-08-07 2022-02-10 University Of Miami Treatment of sensorineural deafness
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
EP4262882A1 (en) * 2020-12-18 2023-10-25 Institut Pasteur Gene therapy for treating usher syndrome
CA3216712A1 (en) * 2021-04-14 2022-10-20 Research Institute At Nationwide Children's Hospital Materials and methods for neurofibromin 2/merlin (nf2) gene therapy
AU2022271238A1 (en) * 2021-05-04 2023-12-14 Decibel Therapeutics, Inc. Vestibular supporting cell promoters and uses thereof
CN113604469B (zh) * 2021-08-11 2023-01-24 复旦大学附属眼耳鼻喉科医院 基于CRISPR/CasRx的基因编辑方法及其应用
EP4409009A4 (en) * 2021-09-30 2025-12-17 Akouos Inc Compositions for administering gene therapy and methods for treating hearing loss
US20250073354A1 (en) * 2021-12-23 2025-03-06 University Of Rochester Compositions and methods for delivery of agents to inner ear
WO2023147383A1 (en) 2022-01-25 2023-08-03 Bridgebio Services Inc. Gene therapy for genetic hearing loss
GB202214972D0 (en) * 2022-10-11 2022-11-23 Ucl Business Ltd Products and methods for use in treating NDP-related diseases
WO2024151703A2 (en) * 2023-01-12 2024-07-18 Ohio State Innovation Foundation Gene therapy for treating hearing loss disorders
CN121311586A (zh) * 2023-05-12 2026-01-09 苏州星奥拓维生物技术有限公司 耳蜗內毛细胞特异性启动子及其应用
CN121816425A (zh) 2023-09-11 2026-04-07 学校法人顺天堂 能够感染内耳的aav变体
WO2025085845A1 (en) * 2023-10-19 2025-04-24 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Gene therapy for hearing loss and vestibular disorders
US20250276095A1 (en) 2024-03-04 2025-09-04 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
WO2026011008A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Expression control by skeletal muscle-expressed mirnas
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy
WO2026030244A1 (en) 2024-07-30 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of limb girdle muscular dystrophy 2a
WO2026050402A1 (en) 2024-08-29 2026-03-05 Kate Therapeutics, Inc. Liver de-targeted muscle tropic capsids

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5037384B2 (en) 1988-01-12 1995-12-26 Cornell Res Foundation Inc Method and apparatus for the treatment of complicated retinal detachments
US20040115621A1 (en) 2000-02-18 2004-06-17 Allen Rodrigo Ancestral viruses and vaccines
US6692526B1 (en) 2000-03-15 2004-02-17 Michael E. Snyder Ophthalmological surgery colorant and delivery system
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
EP1755400A2 (en) * 2004-06-18 2007-02-28 The University Of Montana Aav mediated gene delivery to cochlear cells
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
US8388668B2 (en) 2005-08-05 2013-03-05 Gholam A. Peyman Methods to regulate polarization of excitable cells
WO2011075838A1 (en) 2009-12-21 2011-06-30 Audigen Inc. Method of treating or preventing hearing loss
US20150079110A1 (en) * 2012-03-05 2015-03-19 Wake Forest University Health Sciences Regeneration of inner ear cells
CN106232618A (zh) * 2013-10-11 2016-12-14 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
JP7103750B2 (ja) * 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのCRISPR-Cas系及び組成物の送達、使用及び治療適用
EP3258955A1 (en) * 2015-02-20 2017-12-27 Institut Pasteur Prevention and/or treatment of hearing loss or impairment
SG10201913266UA (en) 2015-12-11 2020-02-27 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
EP3510161A4 (en) * 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON

Similar Documents

Publication Publication Date Title
JP2018536420A5 (https=)
CN113121652B (zh) 视网膜和肌肉高亲和性腺相关病毒衣壳蛋白及相关应用
CN114072179A (zh) 肌球蛋白15启动子及其用途
CN113121655B (zh) 眼部和肌肉特异靶向型腺相关病毒载体及其应用
CN113564187B (zh) 基于aav的抗血管生成基因递送系统及其用途
JP2021502978A5 (https=)
CN118679182A (zh) 用于治疗前庭神经鞘瘤相关症状的抗vegf抗体构建体和相关方法
CN116925239A (zh) 双载体系统表达Otof基因的组合物和方法
WO2023198050A1 (zh) 一种融合型腺相关病毒及其应用
CN113584043B (zh) 用于治疗视网膜疾病和癌症的转基因表达盒
CN113563430B (zh) 用于治疗眼部疾病的基因递送系统及其应用
CN113480615B (zh) 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
CN113121653B (zh) 肌肉和视网膜特异性的新型腺相关病毒衣壳蛋白
JP2023551533A (ja) 前庭神経鞘腫関連症状を治療するための抗vegf抗体構築物及び関連する方法
CN116218849B (zh) 针对utrophin激活治疗杜氏肌营养不良的sgRNA和应用
WO2024083095A1 (zh) 环状rna及载体和载体的应用
CN113121654A (zh) 新型腺相关病毒衣壳蛋白及包含其的新型腺相关病毒载体
CN112961220B (zh) 腺相关病毒衣壳蛋白及包含其的腺相关病毒载体
CN114765991A (zh) 非色素睫状上皮细胞特异性启动子
KR20230015891A (ko) 인간 핵 인자 e2-관련 인자 2의 발현 벡터 및 발현 벡터의 적용
CN119220539A (zh) Gjb2启动子序列及其载体和应用
WO2025057943A1 (ja) 内耳に感染性を有するaav変異体
CN121311586A (zh) 耳蜗內毛细胞特异性启动子及其应用
WO2023005906A1 (zh) 新型腺相关病毒血清型介导的血管生成抑制剂及其应用
KR20260003656A (ko) 합성 회로 및 이의 용도