HU230406B1
(hu)
|
2001-11-13 |
2016-04-28 |
The Trustees Of The University Of Pennsylvania |
Eljárás adeno-asszociált vírus(AAV)szekvenciák detektálására és/vagy azonosítására és az azzal azonosított új szekvenciák izolálására
|
US9217155B2
(en)
|
2008-05-28 |
2015-12-22 |
University Of Massachusetts |
Isolation of novel AAV'S and uses thereof
|
US8734809B2
(en)
|
2009-05-28 |
2014-05-27 |
University Of Massachusetts |
AAV's and uses thereof
|
EP3540055A1
(en)
|
2010-04-23 |
2019-09-18 |
University of Massachusetts |
Cns targeting aav vectors and methods of use thereof
|
WO2011133874A1
(en)
|
2010-04-23 |
2011-10-27 |
University Of Massachusetts |
Multicistronic expression constructs
|
WO2012149265A2
(en)
|
2011-04-29 |
2012-11-01 |
Selecta Biosciences, Inc. |
Tolerogenic synthetic nanocarriers to reduce cytotoxic t lymphocyte responses
|
AU2012340567B2
(en)
|
2011-11-22 |
2017-11-23 |
The Children's Hospital Of Philadelphia |
Virus vectors for highly efficient transgene delivery
|
US9255154B2
(en)
|
2012-05-08 |
2016-02-09 |
Alderbio Holdings, Llc |
Anti-PCSK9 antibodies and use thereof
|
WO2014043696A2
(en)
|
2012-09-17 |
2014-03-20 |
The Research Institute At Nationwide Children's Hospital |
Compositions and methods for treating amyotrophic lateral sclerosis
|
JP2016517278A
(ja)
*
|
2013-03-15 |
2016-06-16 |
ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia |
スタッファー/フィラーポリヌクレオチド配列を含むベクターおよびその使用方法
|
US10357482B2
(en)
|
2013-05-03 |
2019-07-23 |
Selecta Biosciences, Inc. |
Methods providing a therapeutic macromolecule and synthetic nanocarriers comprising immunosuppressant locally and concomitantly to reduce both type I and type IV hypersensitivity
|
HUE047996T2
(hu)
|
2013-07-22 |
2020-05-28 |
Childrens Hospital Philadelphia |
AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
|
KR20160067219A
(ko)
|
2013-10-03 |
2016-06-13 |
모더나 세라퓨틱스, 인코포레이티드 |
저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
|
CA2929669A1
(en)
*
|
2013-11-05 |
2015-05-14 |
The Research Institute At Nationwide Children's Hospital |
Compositions and methods for inhibiting nf-.kappa.b and sod-1 to treat amyotrophic lateral sclerosis
|
WO2015127128A2
(en)
|
2014-02-19 |
2015-08-27 |
University Of Massachusetts |
Recombinant aavs having useful transcytosis properties
|
ES2856403T3
(es)
|
2014-03-18 |
2021-09-27 |
Univ Massachusetts |
Composiciones a base de rAAV y procedimientos para el tratamiento de la esclerosis lateral amiotrofica
|
EP4345165A2
(en)
*
|
2014-03-21 |
2024-04-03 |
Genzyme Corporation |
Gene therapy for retinitis pigmentosa
|
US10975391B2
(en)
|
2014-04-25 |
2021-04-13 |
University Of Massachusetts |
Recombinant AAV vectors useful for reducing immunity against transgene products
|
ES2876409T3
(es)
|
2014-04-25 |
2021-11-12 |
Univ Pennsylvania |
Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol
|
US10689653B2
(en)
|
2014-06-03 |
2020-06-23 |
University Of Massachusetts |
Compositions and methods for modulating dysferlin expression
|
WO2015191508A1
(en)
|
2014-06-09 |
2015-12-17 |
Voyager Therapeutics, Inc. |
Chimeric capsids
|
CN105396143B
(zh)
*
|
2014-09-01 |
2020-06-26 |
江苏命码生物科技有限公司 |
埃博拉病毒特异性的miRNA以及通过miRNA抑制埃博拉病毒的方法
|
KR20230053709A
(ko)
*
|
2014-09-07 |
2023-04-21 |
셀렉타 바이오사이언시즈, 인크. |
항-바이러스 전달 벡터 면역 반응을 약화시키기 위한 방법 및 조성물
|
EP3200830B1
(en)
|
2014-10-03 |
2020-09-09 |
University of Massachusetts |
High efficiency library-identified aav vectors
|
WO2016054554A1
(en)
|
2014-10-03 |
2016-04-07 |
University Of Massachusetts |
Heterologous targeting peptide grafted aavs
|
WO2016065001A1
(en)
|
2014-10-21 |
2016-04-28 |
University Of Massachusetts |
Recombinant aav variants and uses thereof
|
SG11201703148TA
(en)
|
2014-11-05 |
2017-05-30 |
Voyager Therapeutics Inc |
Aadc polynucleotides for the treatment of parkinson's disease
|
GB201420139D0
(en)
|
2014-11-12 |
2014-12-24 |
Ucl Business Plc |
Factor IX gene therapy
|
KR102584655B1
(ko)
|
2014-11-14 |
2023-10-06 |
보이저 테라퓨틱스, 인크. |
조절성 폴리뉴클레오티드
|
CN114717264A
(zh)
|
2014-11-14 |
2022-07-08 |
沃雅戈治疗公司 |
治疗肌萎缩性侧索硬化(als)的组合物和方法
|
EP3230441A4
(en)
|
2014-12-12 |
2018-10-03 |
Voyager Therapeutics, Inc. |
Compositions and methods for the production of scaav
|
EP3256170B1
(en)
|
2015-02-13 |
2020-09-23 |
University of Massachusetts |
Compositions and methods for transient delivery of nucleases
|
US11046955B2
(en)
|
2015-04-24 |
2021-06-29 |
University Of Massachusetts |
Modified AAV constructs and uses thereof
|
US11701410B2
(en)
|
2015-05-22 |
2023-07-18 |
Cls Therapeutics Limited |
Extracellular DNA as a therapeutic target in neurodegeneration
|
EP3313991A4
(en)
|
2015-06-23 |
2018-12-05 |
The Children's Hospital of Philadelphia |
Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
|
WO2017053677A1
(en)
|
2015-09-24 |
2017-03-30 |
Biomarin Pharmaceutical Inc. |
Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
|
CN108431216B
(zh)
*
|
2015-10-21 |
2022-08-05 |
美国政府(由卫生和人类服务部的部长所代表) |
经密码子优化的尺寸减小的atp7a cdna及用于治疗铜转运障碍的用途
|
EP3364997B1
(en)
|
2015-10-22 |
2024-01-17 |
University of Massachusetts |
Aspartoacylase gene therapy in the treatment of canavan disease
|
US11426469B2
(en)
|
2015-10-22 |
2022-08-30 |
University Of Massachusetts |
Prostate-targeting adeno-associated virus serotype vectors
|
EP3374387A1
(en)
|
2015-11-13 |
2018-09-19 |
Baxalta Incorporated |
Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
|
EA036944B1
(ru)
|
2015-11-13 |
2021-01-19 |
Баксалта Инкорпорейтед |
Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
|
RU2766583C2
(ru)
*
|
2015-12-01 |
2022-03-15 |
Спарк Терапьютикс, Инк. |
Масштабируемые способы получения рекомбинантного вектора на основе аденоассоциированного вируса (aav) в системе бессывороточной суспензионной культуры клеток, подходящего для клинического применения
|
WO2017096162A1
(en)
|
2015-12-02 |
2017-06-08 |
Voyager Therapeutics, Inc. |
Assays for the detection of aav neutralizing antibodies
|
IL259850B2
(en)
*
|
2015-12-11 |
2023-11-01 |
Univ Pennsylvania |
Gene therapy for the treatment of familial hypercholesterolemia
|
EP3411059A4
(en)
|
2016-02-02 |
2019-10-16 |
University Of Massachusetts |
METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM
|
WO2017139643A1
(en)
|
2016-02-12 |
2017-08-17 |
University Of Massachusetts |
Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
|
JP2019509748A
(ja)
*
|
2016-03-30 |
2019-04-11 |
スパーク セラピューティクス インコーポレイテッドSpark Therapeutics, Inc. |
組換えタンパク質及び/又はウイルスベクター製造のための細胞株
|
EP3440210A4
(en)
|
2016-04-05 |
2019-11-27 |
University of Massachusetts |
COMPOSITIONS AND METHODS FOR SELECTIVE INHIBITION OF EXPRESSION OF GRAINHEAD PROTEIN
|
BR112018071167A2
(pt)
*
|
2016-04-15 |
2019-02-05 |
Research Institute At Nationwide Children's Hospital |
entrega de beta-sarcoglicano e microrna-29 por vírus adenoassociado e tratamento de distrofia muscular
|
US11413356B2
(en)
|
2016-04-15 |
2022-08-16 |
University Of Massachusetts |
Methods and compositions for treating metabolic imbalance
|
WO2017189964A2
(en)
|
2016-04-29 |
2017-11-02 |
Voyager Therapeutics, Inc. |
Compositions for the treatment of disease
|
WO2017189959A1
(en)
|
2016-04-29 |
2017-11-02 |
Voyager Therapeutics, Inc. |
Compositions for the treatment of disease
|
FI3445773T3
(fi)
|
2016-05-13 |
2023-03-30 |
4D Molecular Therapeutics Inc |
Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä
|
US11951121B2
(en)
|
2016-05-18 |
2024-04-09 |
Voyager Therapeutics, Inc. |
Compositions and methods for treating Huntington's disease
|
BR112018073384A2
(pt)
|
2016-05-18 |
2019-03-06 |
Voyager Therapeutics, Inc. |
polinucleotídeos moduladores
|
US11882815B2
(en)
|
2016-06-15 |
2024-01-30 |
University Of Massachusetts |
Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
|
WO2018022511A1
(en)
|
2016-07-25 |
2018-02-01 |
The Trustees Of The University Of Pennsylvania |
Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
|
KR20230161535A
(ko)
|
2016-07-26 |
2023-11-27 |
바이오마린 파머수티컬 인크. |
신규 아데노-관련 바이러스 캡시드 단백질
|
CN110225975A
(zh)
|
2016-08-23 |
2019-09-10 |
阿库斯股份有限公司 |
用于治疗人受试者中非年龄相关的听力损害的组合物和方法
|
CN106344932B
(zh)
*
|
2016-08-26 |
2019-12-27 |
首都医科大学附属北京同仁医院 |
一种用于提高视网膜退行性疾病基因治疗效果的组合物及其应用
|
WO2018044933A1
(en)
|
2016-08-30 |
2018-03-08 |
The Regents Of The University Of California |
Methods for biomedical targeting and delivery and devices and systems for practicing the same
|
US10457940B2
(en)
|
2016-09-22 |
2019-10-29 |
University Of Massachusetts |
AAV treatment of Huntington's disease
|
CA3040483A1
(en)
|
2016-10-13 |
2018-04-19 |
University Of Massachusetts |
Aav capsid designs
|
WO2018082093A1
(en)
*
|
2016-11-07 |
2018-05-11 |
Wuhan Institute Of Virology, Chinese Academy Of Sciences |
Anterograde monosynaptic transneuronal tracer system
|
WO2018131551A1
(ja)
|
2017-01-13 |
2018-07-19 |
学校法人自治医科大学 |
肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのaavベクター
|
EP3595688A4
(en)
|
2017-02-20 |
2020-12-30 |
The Trustees Of The University Of Pennsylvania |
GENE THERAPY TO TREAT FAMILY HYPERCHOLESTERINEMIA
|
AU2018236123B2
(en)
|
2017-03-11 |
2024-04-18 |
Selecta Biosciences, Inc. |
Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
|
WO2018170408A1
(en)
*
|
2017-03-17 |
2018-09-20 |
Research Institute At Nationwide Children's Hospital, Inc. |
Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
|
EP3607073B1
(en)
|
2017-04-03 |
2024-06-05 |
Encoded Therapeutics, Inc. |
Tissue selective transgene expression
|
MX2019013172A
(es)
|
2017-05-05 |
2020-09-07 |
Voyager Therapeutics Inc |
Composiciones y metodos para tratar la enfermedad de huntington.
|
CN110913866A
(zh)
|
2017-05-05 |
2020-03-24 |
沃雅戈治疗公司 |
治疗肌萎缩性侧索硬化(als)的组合物和方法
|
AU2018264996A1
(en)
|
2017-05-09 |
2019-12-05 |
University Of Massachusetts |
Methods of treating Amyotrophic Lateral Sclerosis (ALS)
|
CA3063464A1
(en)
*
|
2017-05-19 |
2018-11-22 |
Encoded Therapeutics, Inc. |
High activity regulatory elements
|
US10842853B2
(en)
|
2017-05-22 |
2020-11-24 |
Baxalta Incorporated |
Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia B
|
JOP20190269A1
(ar)
|
2017-06-15 |
2019-11-20 |
Voyager Therapeutics Inc |
بولي نوكليوتيدات aadc لعلاج مرض باركنسون
|
CA3070087A1
(en)
|
2017-07-17 |
2019-01-24 |
Voyager Therapeutics, Inc. |
Trajectory array guide system
|
BR112020000995A2
(pt)
*
|
2017-07-17 |
2020-09-08 |
Spark Therapeutics, Inc. |
métodos e usos de aférese
|
EP3662060A2
(en)
|
2017-08-03 |
2020-06-10 |
Voyager Therapeutics, Inc. |
Compositions and methods for delivery of aav
|
WO2019051424A2
(en)
*
|
2017-09-08 |
2019-03-14 |
Poseida Therapeutics, Inc. |
COMPOSITIONS AND METHODS FOR CONDITIONAL GENE EXPRESSION MEDIATED BY A CHIMERIC LIGAND RECEPTOR (CLR)
|
CR20200165A
(es)
|
2017-09-20 |
2020-06-05 |
4D Molecular Therapeutics Inc |
Variantes de cápsides de virus adenoasociados y métodos de uso de estas
|
JP7397488B2
(ja)
|
2017-09-22 |
2023-12-13 |
ユニバーシティ オブ マサチューセッツ |
Sod1二重発現ベクターおよびその使用
|
EP3697908A1
(en)
|
2017-10-16 |
2020-08-26 |
Voyager Therapeutics, Inc. |
Treatment of amyotrophic lateral sclerosis (als)
|
CN111479924B
(zh)
|
2017-10-16 |
2024-06-14 |
沃雅戈治疗公司 |
肌萎缩性侧索硬化症(als)的治疗
|
CN107760719B
(zh)
*
|
2017-10-24 |
2020-09-22 |
北京领柯生物科技有限公司 |
柯萨奇病毒在过继免疫基因递送系统中的应用
|
UA127831C2
(uk)
|
2017-11-27 |
2024-01-17 |
4Д Молекьюлар Терапьютикс Інк. |
Капсид аденоасоційованого вірусу та його застосування для інгібування ангіогенезу
|
CA3085945A1
(en)
*
|
2017-12-19 |
2019-06-27 |
The University Of North Carolina At Chapel Hill |
Methods and compositions for delivery of viral vectors across the blood-brain barrier
|
CN111542613B
(zh)
*
|
2017-12-29 |
2023-11-21 |
赫利世弥斯株式会社 |
导入有杂交的肝细胞生长因子的腺相关病毒载体
|
KR20200122320A
(ko)
*
|
2018-01-16 |
2020-10-27 |
씨엘에스 테라퓨틱스 리미티드 |
데옥시리보뉴클레아제 (dnase) 활성을 갖는 효소의 간 발현에 의한 질환의 치료
|
US10610606B2
(en)
|
2018-02-01 |
2020-04-07 |
Homology Medicines, Inc. |
Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
|
US11306329B2
(en)
|
2018-02-19 |
2022-04-19 |
City Of Hope |
Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
|
KR20210015770A
(ko)
|
2018-04-05 |
2021-02-10 |
제네똥 |
감소된 간 향성을 갖는 AAV9과 AAVrh74의 하이브리드 재조합 아데노-관련 바이러스 세로타입
|
TW202016314A
(zh)
|
2018-05-09 |
2020-05-01 |
美商拜奧馬林製藥公司 |
治療苯丙酮尿症之方法
|
TW202005978A
(zh)
|
2018-05-14 |
2020-02-01 |
美商拜奧馬林製藥公司 |
新穎肝靶向腺相關病毒載體
|
TW202039546A
(zh)
|
2019-01-16 |
2020-11-01 |
美商巴克斯歐塔公司 |
用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
|
KR20210125999A
(ko)
*
|
2019-02-12 |
2021-10-19 |
스페이스크래프트 세븐, 엘엘씨 |
다논병 치료를 위한 유전자 요법 벡터
|
WO2020174369A2
(en)
|
2019-02-25 |
2020-09-03 |
Novartis Ag |
Compositions and methods to treat bietti crystalline dystrophy
|
KR20210132684A
(ko)
|
2019-02-25 |
2021-11-04 |
노파르티스 아게 |
비에띠 결정 이상증을 치료하기 위한 조성물 및 방법
|
CN109999182B
(zh)
*
|
2019-03-29 |
2023-03-07 |
四川大学华西医院 |
凝血因子在制备抗肿瘤药物中的应用
|
WO2021041546A1
(en)
|
2019-08-27 |
2021-03-04 |
Vertex Pharmaceuticals Incorporated |
Compositions and methods for treatment of disorders associated with repetitive dna
|
TW202128733A
(zh)
|
2019-11-14 |
2021-08-01 |
美商拜奧馬林製藥公司 |
使用肝特異性基因療法載體之遺傳性血管性水腫的治療
|
CN113025618B
(zh)
*
|
2019-12-24 |
2024-02-06 |
朗信启昇(苏州)生物制药有限公司 |
一种x连锁遗传性视网膜劈裂症的基因治疗方案与应用
|
TW202140791A
(zh)
|
2020-01-13 |
2021-11-01 |
美商霍蒙拉奇醫藥公司 |
治療苯酮尿症之方法
|
MX2022010050A
(es)
|
2020-02-21 |
2022-09-05 |
Akouos Inc |
Composiciones y metodos para tratar deficiencia auditiva no asociada con la edad en un sujeto humano.
|
AR122409A1
(es)
|
2020-04-03 |
2022-09-07 |
Biomarin Pharm Inc |
Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas
|
WO2021257497A1
(en)
*
|
2020-06-15 |
2021-12-23 |
Sarepta Therapeutis, Inc. |
Adeno-associated virus antibodies and fragments thereof
|
CN111500635B
(zh)
*
|
2020-07-02 |
2020-10-09 |
北京大学第三医院(北京大学第三临床医学院) |
一种包含携带核酸分子的载体的试剂盒
|
CN111876432B
(zh)
*
|
2020-07-29 |
2022-10-28 |
舒泰神(北京)生物制药股份有限公司 |
一组肝靶向新型腺相关病毒的获得及其应用
|
MX2023001615A
(es)
|
2020-08-07 |
2023-03-08 |
Spacecraft Seven Llc |
Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav.
|
WO2022056000A1
(en)
|
2020-09-09 |
2022-03-17 |
Vertex Pharmaceuticals Incorporated |
Compositions and methods for treatment of duchenne muscular dystrophy
|
US20230414648A1
(en)
|
2020-11-06 |
2023-12-28 |
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