|
NZ532635A
(en)
|
2001-11-13 |
2007-05-31 |
Univ Pennsylvania |
A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
|
|
US9217155B2
(en)
|
2008-05-28 |
2015-12-22 |
University Of Massachusetts |
Isolation of novel AAV'S and uses thereof
|
|
WO2010138263A2
(en)
|
2009-05-28 |
2010-12-02 |
University Of Massachusetts |
Novel aav 's and uses thereof
|
|
WO2011133874A1
(en)
|
2010-04-23 |
2011-10-27 |
University Of Massachusetts |
Multicistronic expression constructs
|
|
EP2826860B1
(en)
|
2010-04-23 |
2018-08-22 |
University of Massachusetts |
CNS targeting AAV vectors and methods of use thereof
|
|
KR20140041505A
(ko)
|
2011-04-29 |
2014-04-04 |
셀렉타 바이오사이언시즈, 인크. |
조절 b 세포 유도를 위한 관용원성 합성 나노운반체
|
|
JP6348064B2
(ja)
|
2011-11-22 |
2018-06-27 |
ザ チルドレンズ ホスピタル オブ フィラデルフィア |
効率の高いトランスジーン送達のためのウイルスベクター
|
|
US9255154B2
(en)
|
2012-05-08 |
2016-02-09 |
Alderbio Holdings, Llc |
Anti-PCSK9 antibodies and use thereof
|
|
AU2013315007A1
(en)
|
2012-09-17 |
2015-04-09 |
The Research Institute At Nationwide Children's Hospital |
Compositions and methods for treating amyotrophic lateral sclerosis
|
|
MX384795B
(es)
|
2013-03-15 |
2025-03-14 |
Childrens Hospital Philadelphia |
Vectores que comprenden secuencias de polinucleotidos rellenadoras/de relleno y metodos de uso.
|
|
HUE052599T2
(hu)
|
2013-05-03 |
2021-05-28 |
Selecta Biosciences Inc |
CD4+ szabályozó T-sejtek fokozására szolgáló eljárások és készítmények
|
|
KR20240090694A
(ko)
*
|
2013-07-22 |
2024-06-21 |
더 칠드런스 호스피탈 오브 필라델피아 |
변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
|
|
JP2016538829A
(ja)
|
2013-10-03 |
2016-12-15 |
モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. |
低密度リポタンパク質受容体をコードするポリヌクレオチド
|
|
WO2015069647A1
(en)
*
|
2013-11-05 |
2015-05-14 |
The Research Institute At Nationwide Children's Hospital |
COMPOSITIONS AND METHODS FOR INHIBITING NF- kB AND SOD-1 TO TREAT AMYOTROPHIC LATERAL SCLEROSIS
|
|
US10072251B2
(en)
|
2014-02-19 |
2018-09-11 |
University Of Massachusetts |
Recombinant AAVS having useful transcytosis properties
|
|
EP4410805A3
(en)
|
2014-03-18 |
2024-11-27 |
University of Massachusetts |
Raav-based compositions and methods for treating amyotrophic lateral sclerosis
|
|
FI3628334T3
(fi)
|
2014-03-21 |
2023-09-15 |
Genzyme Corp |
Geenihoito verkkokalvon pigmenttirappeuma
|
|
ES2876409T3
(es)
|
2014-04-25 |
2021-11-12 |
Univ Pennsylvania |
Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol
|
|
EP3134522B1
(en)
*
|
2014-04-25 |
2021-10-06 |
University of Massachusetts |
Recombinant aav vectors useful for reducing immunity against transgene products
|
|
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
|
|
AU2015311704B2
(en)
*
|
2014-09-07 |
2021-12-09 |
Selecta Biosciences, Inc. |
Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses
|
|
US10711270B2
(en)
|
2014-10-03 |
2020-07-14 |
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
|
|
AU2015335923B2
(en)
|
2014-10-21 |
2021-04-29 |
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
|
|
CA3193811A1
(en)
|
2014-11-14 |
2016-05-19 |
Voyager Therapeutics, Inc. |
Compositions and methods of treating amyotrophic lateral sclerosis (als)
|
|
CN119876138A
(zh)
|
2014-11-14 |
2025-04-25 |
沃雅戈治疗公司 |
调节性多核苷酸
|
|
HK1245326A1
(zh)
|
2014-12-12 |
2018-08-24 |
Voyager Therapeutics, Inc. |
用於生产scaav的组合物和方法
|
|
WO2016131009A1
(en)
|
2015-02-13 |
2016-08-18 |
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
|
|
WO2016190780A1
(en)
|
2015-05-22 |
2016-12-01 |
Dmitry Dmitrievich Genkin |
Extracellular dna as a therapeutic target in neurodegeneration
|
|
WO2016210170A1
(en)
|
2015-06-23 |
2016-12-29 |
The Children's Hospital Of Philadelphia |
Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
|
|
WO2017011352A1
(en)
*
|
2015-07-10 |
2017-01-19 |
Duke University |
Methods for treating pain
|
|
EA201890543A1
(ru)
|
2015-09-17 |
2018-08-31 |
Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл |
Способы и материалы для генной терапии galgt2
|
|
RU2742352C2
(ru)
|
2015-09-24 |
2021-02-05 |
Байомарин Фармасьютикал Инк. |
Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции
|
|
DK3365438T3
(da)
*
|
2015-10-21 |
2021-07-12 |
Us Health |
Codon-optimeret reduceret-størrelse atp7a cdna og anvendelser ved behandling af kobbertransportlidelser
|
|
CA3002980A1
(en)
|
2015-10-22 |
2017-04-27 |
University Of Massachusetts |
Prostate-targeting adeno-associated virus serotype vectors
|
|
WO2017070525A1
(en)
|
2015-10-22 |
2017-04-27 |
University Of Massachusetts |
Methods and compositions for treating metabolic imbalance in neurodegenerative disease
|
|
EA202092049A1
(ru)
|
2015-11-13 |
2020-11-10 |
Баксалта Инкорпорейтед |
Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
|
|
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
|
|
CA3005633C
(en)
|
2015-11-16 |
2023-11-21 |
Research Institute Of Nationwide Children's Hospital |
Materials and methods for treatment of titin-based myopathies and other titinopathies
|
|
AU2016362317B2
(en)
*
|
2015-12-01 |
2023-03-16 |
Spark Therapeutics, Inc. |
Scalable methods for producing recombinant Adeno-Associated Viral (AAV) vector in serum-free suspension cell culture system suitable for clinical use
|
|
WO2017096162A1
(en)
|
2015-12-02 |
2017-06-08 |
Voyager Therapeutics, Inc. |
Assays for the detection of aav neutralizing antibodies
|
|
IL303850A
(en)
*
|
2015-12-11 |
2023-08-01 |
Univ Pennsylvania |
Gene therapy for treating familial hypercholesterolemia
|
|
WO2017136536A1
(en)
|
2016-02-02 |
2017-08-10 |
University Of Massachusetts |
Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
|
|
US11060088B2
(en)
|
2016-02-12 |
2021-07-13 |
University Of Massachusetts |
Anti-angiogenic miRNA therapeutics for inhibiting corneal neovascularization
|
|
MX2018011928A
(es)
*
|
2016-03-30 |
2019-03-28 |
Spark Therapeutics Inc |
Linea celular para produccion de proteina recombinante y/o vector viral.
|
|
US11207426B2
(en)
|
2016-04-05 |
2021-12-28 |
University Of Massachusetts |
Compositions and methods for selective inhibition of grainyhead-like protein expression
|
|
PT3442600T
(pt)
*
|
2016-04-15 |
2024-05-09 |
Res Inst Nationwide Childrens Hospital |
Distribuição de vetor de vírus adeno-associado de bsarcoglicano e microrna-29 e o tratamento da distrofia muscular
|
|
US11413356B2
(en)
|
2016-04-15 |
2022-08-16 |
University Of Massachusetts |
Methods and compositions for treating metabolic imbalance
|
|
EP3448874A4
(en)
|
2016-04-29 |
2020-04-22 |
Voyager Therapeutics, Inc. |
COMPOSITIONS FOR TREATING A DISEASE
|
|
EP3448987A4
(en)
|
2016-04-29 |
2020-05-27 |
Voyager Therapeutics, Inc. |
COMPOSITIONS FOR TREATING A DISEASE
|
|
FI3445773T3
(fi)
|
2016-05-13 |
2023-03-30 |
4D Molecular Therapeutics Inc |
Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä
|
|
CA3024448C
(en)
|
2016-05-18 |
2025-09-09 |
Voyager Therapeutics, Inc. |
MODULATING POLYNUCLEOTIDES
|
|
CA3024449A1
(en)
|
2016-05-18 |
2017-11-23 |
Voyager Therapeutics, Inc. |
Compositions and methods of treating huntington's disease
|
|
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
|
|
PE20190401A1
(es)
|
2016-07-26 |
2019-03-13 |
Biomarin Pharm Inc |
Novedosas proteinas de la capside del virus adenoasociado
|
|
JP2019530737A
(ja)
|
2016-08-23 |
2019-10-24 |
アコーオス インコーポレイテッド |
ヒト対象において非加齢性聴力障害を治療するための組成物および方法
|
|
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
|
|
EP3526333A4
(en)
|
2016-10-13 |
2020-07-29 |
University of Massachusetts |
AAV CAPSIDE DESIGNS
|
|
WO2018082093A1
(en)
*
|
2016-11-07 |
2018-05-11 |
Wuhan Institute Of Virology, Chinese Academy Of Sciences |
Anterograde monosynaptic transneuronal tracer system
|
|
MX2019007873A
(es)
|
2016-12-30 |
2019-11-18 |
Univ Pennsylvania |
Terapia génica para tratar la enfermedad de wilson.
|
|
US20230190958A1
(en)
|
2017-01-13 |
2023-06-22 |
Jichi Medical University |
AAV Vector for Disrupting Coagulation Factor-Related Gene on Liver Genome
|
|
US11554147B2
(en)
|
2017-02-20 |
2023-01-17 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for treating familial hypercholesterolemia
|
|
US20200054711A1
(en)
*
|
2017-02-28 |
2020-02-20 |
The Penn State Research Foundation |
Regenerating functional neurons for treatment of neural injury caused by disruption of blood flow
|
|
BR112019017327A2
(pt)
*
|
2017-03-01 |
2020-04-14 |
Univ Pennsylvania |
terapia gênica para distúrbios oculares
|
|
WO2018169811A1
(en)
|
2017-03-11 |
2018-09-20 |
Selecta Biosciences, Inc. |
Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
|
|
MX2019011046A
(es)
*
|
2017-03-17 |
2019-10-17 |
Res Inst Nationwide Childrens Hospital |
Administracion de vectores del virus adenoasociado de microdistrofina especifica del musculo para tratar la distrofia muscular.
|
|
WO2019012336A2
(en)
|
2017-03-17 |
2019-01-17 |
Newcastle University |
ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY
|
|
EA201992358A1
(ru)
|
2017-04-03 |
2020-03-24 |
Инкоудид Терапьютикс, Инк. |
Тканеселективная экспрессия трансгена
|
|
JP2020518259A
(ja)
|
2017-05-05 |
2020-06-25 |
ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. |
ハンチントン病治療組成物および方法
|
|
EP3618839A4
(en)
|
2017-05-05 |
2021-06-09 |
Voyager Therapeutics, Inc. |
COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
|
|
WO2018208972A1
(en)
|
2017-05-09 |
2018-11-15 |
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
|
|
CN110945127B
(zh)
|
2017-05-22 |
2024-07-12 |
武田药品工业株式会社 |
用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体
|
|
JOP20190269A1
(ar)
|
2017-06-15 |
2019-11-20 |
Voyager Therapeutics Inc |
بولي نوكليوتيدات aadc لعلاج مرض باركنسون
|
|
JP7229989B2
(ja)
|
2017-07-17 |
2023-02-28 |
ボイジャー セラピューティクス インコーポレイテッド |
軌道アレイガイドシステム
|
|
MX2020000713A
(es)
*
|
2017-07-17 |
2020-08-31 |
Spark Therapeutics Inc |
Metodos de aferesis y sus usos.
|
|
JP7221275B2
(ja)
|
2017-08-03 |
2023-02-13 |
ボイジャー セラピューティクス インコーポレイテッド |
Aavを送達するための組成物および方法
|
|
TW202545984A
(zh)
|
2017-08-09 |
2025-12-01 |
美商生物化學醫療公司 |
核酸分子及其用途
|
|
AU2018329741B2
(en)
*
|
2017-09-08 |
2025-02-20 |
Poseida Therapeutics, Inc. |
Compositions and methods for chimeric ligand receptor (CLR)-mediated conditional gene expression
|
|
SG11202002276VA
(en)
|
2017-09-20 |
2020-04-29 |
4D Molecular Therapeutics Inc |
Adeno-associated virus variant capsids and methods of use thereof
|
|
CA3075643A1
(en)
|
2017-09-22 |
2019-03-28 |
University Of Massachusetts |
Sod1 dual expression vectors and uses thereof
|
|
US20200263199A1
(en)
|
2017-09-29 |
2020-08-20 |
Voyager Therapeutics, Inc. |
Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
|
|
WO2019079242A1
(en)
|
2017-10-16 |
2019-04-25 |
Voyager Therapeutics, Inc. |
TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
|
|
TW202413649A
(zh)
|
2017-10-16 |
2024-04-01 |
美商航海家醫療公司 |
肌萎縮性脊髓側索硬化症(als)之治療
|
|
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
|
|
DK3717636T5
(da)
|
2017-11-27 |
2024-08-26 |
4D Molecular Therapeutics Inc |
Adeno-associeret-virus-variantcapsider og anvendelse til inhibering af angiogenese
|
|
IL274926B2
(en)
|
2017-12-01 |
2026-04-01 |
Encoded Therapeutics Inc |
Pyrazolopyrimidines with activity against respiratory syncytial virus
|
|
US11905533B2
(en)
|
2017-12-19 |
2024-02-20 |
The University Of North Carolina At Chapel Hill |
Methods and compositions for delivery of viral vectors across the blood-brain barrier
|
|
CA3086046C
(en)
*
|
2017-12-29 |
2023-02-21 |
Helixmith Co., Ltd. |
Adeno-associated virus (aav) vector having hybrid hgf gene introduced thereto
|
|
KR20200122320A
(ko)
|
2018-01-16 |
2020-10-27 |
씨엘에스 테라퓨틱스 리미티드 |
데옥시리보뉴클레아제 (dnase) 활성을 갖는 효소의 간 발현에 의한 질환의 치료
|
|
MX2020007876A
(es)
|
2018-01-31 |
2020-12-03 |
Res Inst Nationwide Childrens Hospital |
Terapia genica para la distrofia muscular de cinturas tipo 2c.
|
|
US10610606B2
(en)
|
2018-02-01 |
2020-04-07 |
Homology Medicines, Inc. |
Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
|
|
CN111902164A
(zh)
|
2018-02-01 |
2020-11-06 |
同源药物公司 |
用于恢复pah基因功能的腺相关病毒组合物及其使用方法
|
|
CN112041437A
(zh)
|
2018-02-19 |
2020-12-04 |
同源药物公司 |
用于恢复f8基因功能的腺相关病毒组合物和其使用方法
|
|
EP3775173A1
(en)
|
2018-04-05 |
2021-02-17 |
Genethon |
Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism
|
|
CA3096293A1
(en)
|
2018-04-27 |
2019-10-31 |
Rocket Pharmaceuticals, Ltd. |
Gene therapy for cns degeneration
|
|
BR112020022722A8
(pt)
|
2018-05-09 |
2022-01-18 |
Biomarin Pharm Inc |
Genoma e seu uso, vetor, partícula, molécula de ácido nucleico isolada, ácido nucleico, composição, tratamento, método
|
|
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
|
|
SG11202011296VA
(en)
|
2018-05-15 |
2020-12-30 |
Voyager Therapeutics Inc |
Compositions and methods for the treatment of parkinson's disease
|
|
US12163129B2
(en)
|
2018-06-08 |
2024-12-10 |
University Of Massachusetts |
Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
|
|
CN120393053A
(zh)
|
2018-06-18 |
2025-08-01 |
全国儿童医院研究所 |
用于治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送
|
|
MX2020014119A
(es)
|
2018-06-29 |
2021-06-18 |
Res Inst Nationwide Childrens Hospital |
Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a.
|
|
EP3823985A1
(en)
|
2018-07-16 |
2021-05-26 |
Baxalta Incorporated |
Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
|
|
WO2020033863A1
(en)
|
2018-08-09 |
2020-02-13 |
Bioverativ Therapeutics Inc. |
Nucleic acid molecules and uses thereof for non-viral gene therapy
|
|
AU2019336793B2
(en)
*
|
2018-09-06 |
2024-11-21 |
The Regents Of The University Of California |
RNA and DNA base editing via engineered ADAR recruitment
|
|
US12281321B2
(en)
|
2018-09-28 |
2025-04-22 |
Voyager Therapeutics, Inc. |
Frataxin expression constructs having engineered promoters and methods of use thereof
|
|
US20220089670A1
(en)
*
|
2018-12-28 |
2022-03-24 |
University Of Rochester |
Gene Therapy for BEST1 Dominant Mutations
|
|
KR20210112339A
(ko)
*
|
2019-01-04 |
2021-09-14 |
울트라제닉스 파마수티컬 인코포레이티드 |
윌슨병을 치료하기 위한 유전자 요법 구축물
|
|
TWI851647B
(zh)
|
2019-01-16 |
2024-08-11 |
日商武田藥品工業股份有限公司 |
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
|
|
AU2020221842A1
(en)
*
|
2019-02-12 |
2021-08-12 |
Spacecraft Seven, Llc |
Gene therapy vectors for treatment of Danon Disease
|
|
JP2022520875A
(ja)
|
2019-02-25 |
2022-04-01 |
ノバルティス アーゲー |
Biettiクリスタリン網膜症を治療するための組成物及び方法
|
|
AU2020229085A1
(en)
|
2019-02-25 |
2021-08-19 |
Friedrich Miescher Institute For Biomedical Research |
Compositions and methods to treat bietti crystalline dystrophy
|
|
MX2021010356A
(es)
|
2019-02-26 |
2022-03-04 |
Res Inst Nationwide Childrens Hospital |
SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR.
|
|
MA55087A
(fr)
|
2019-02-28 |
2022-01-05 |
Encoded Therapeutics Inc |
Compositions et procédés de traitement de laminopathies
|
|
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
|
|
AU2020277683A1
(en)
*
|
2019-05-20 |
2021-11-04 |
BioNTech SE |
Therapeutic RNA for ovarian cancer
|
|
WO2020243651A1
(en)
|
2019-05-29 |
2020-12-03 |
Encoded Therapeutics, Inc. |
Compositions and methods for selective gene regulation
|
|
CN114207440A
(zh)
|
2019-06-04 |
2022-03-18 |
西莱克塔生物科技公司 |
聚乙二醇化尿酸酶的制剂和剂量
|
|
WO2020251887A1
(en)
*
|
2019-06-10 |
2020-12-17 |
The Children's Medical Center Corporation |
Compositions and methods for the treatment of dba using gata1 gene therapy
|
|
PT4017871T
(pt)
|
2019-08-21 |
2024-04-22 |
Res Inst Nationwide Childrens Hospital |
Distribuição de vetor de vírus adeno-associado de alfasarcoglicano e o tratamento da distrofia muscular
|
|
BR112022003505A2
(pt)
|
2019-08-27 |
2022-05-24 |
Vertex Pharma |
Composições e métodos para tratamento de distúrbios associados a dna repetitivo
|
|
KR20220106742A
(ko)
*
|
2019-09-25 |
2022-07-29 |
유니버시티 오브 유타 리서치 파운데이션 |
Vmd2 프로모터로부터 구성적으로 활성인 rap1a의 발현을 위한 방법 및 조성물
|
|
KR20220071248A
(ko)
*
|
2019-10-02 |
2022-05-31 |
상가모 테라퓨틱스, 인코포레이티드 |
알파-시누클레인 발현을 억제시키기 위한 아연 핑거 단백질 전사 인자
|
|
CA3159549A1
(en)
*
|
2019-10-30 |
2021-05-06 |
Decibel Therapeutics, Inc. |
Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
|
|
WO2021091950A1
(en)
*
|
2019-11-04 |
2021-05-14 |
Decibel Therapeutics, Inc. |
Vestibular supporting cell promoters and uses thereof
|
|
WO2021096927A1
(en)
*
|
2019-11-13 |
2021-05-20 |
University Of Pittsburgh-Of The Commonwealth System Of Higher Education |
Compositions and uses thereof for treating, prognosing and diagnosing pulmonary hypertension
|
|
CN114829391A
(zh)
|
2019-11-14 |
2022-07-29 |
生物马林药物股份有限公司 |
用肝特异性基因疗法载体治疗遗传性血管性水肿
|
|
US20220409745A1
(en)
*
|
2019-11-19 |
2022-12-29 |
The Regents Of The University Of California |
Compositions and methods of using engineered fusion proteins that bind g4c2 human repeats
|
|
CN114929268A
(zh)
*
|
2019-11-20 |
2022-08-19 |
巴法里安诺迪克有限公司 |
4-1bbl辅助的重组修饰的安卡拉痘苗病毒(mva)的医疗用途
|
|
WO2021102250A1
(en)
*
|
2019-11-21 |
2021-05-27 |
Luppino Francesco |
Growth factor restoration
|
|
CN120114618A
(zh)
*
|
2019-12-05 |
2025-06-10 |
纽约市哥伦比亚大学理事会 |
用于治疗阿尔茨海默病和其他神经退行性病症的逆转运复合体的稳定化
|
|
BR112022011339A2
(pt)
*
|
2019-12-11 |
2022-10-04 |
Myeloid Therapeutics Inc |
Composições de células terapêuticas e métodos para fabricação e usos das mesmas
|
|
CN115176005A
(zh)
*
|
2019-12-18 |
2022-10-11 |
诺华股份有限公司 |
用于治疗血红蛋白病的组合物和方法
|
|
GB201918879D0
(en)
*
|
2019-12-19 |
2020-02-05 |
Ucl Business Ltd |
Treatment of chronic pain
|
|
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
|
|
JP7703543B2
(ja)
*
|
2020-01-17 |
2025-07-07 |
ザ ユナイテッド ステイツ オブ アメリカ アズ リプリゼンテッド バイ ザ セクレタリー、デパートメント オブ ヘルス アンド ヒューマン サービシーズ |
Crx常染色体優性網膜症の処置のための遺伝子治療
|
|
JP7807380B2
(ja)
|
2020-02-21 |
2026-01-27 |
アコーオス インコーポレイテッド |
ヒト対象において非加齢関連聴力障害を処置するための組成物および方法
|
|
EP4114952A4
(en)
*
|
2020-03-05 |
2024-05-08 |
Board of Regents of the University of Nebraska |
Crispr/cas9 system for multistrain hiv-1 treatment
|
|
US20230374085A1
(en)
*
|
2020-03-11 |
2023-11-23 |
City Of Hope |
Dual-targeting chimeric antigen receptor modified t cells comprising il-13 and chlorotoxin for cancer treatment
|
|
AR122409A1
(es)
|
2020-04-03 |
2022-09-07 |
Biomarin Pharm Inc |
Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas
|
|
TW202208632A
(zh)
|
2020-05-27 |
2022-03-01 |
美商同源醫藥公司 |
用於恢復pah基因功能的腺相關病毒組成物及其使用方法
|
|
JP7785023B2
(ja)
*
|
2020-06-15 |
2025-12-12 |
サレプタ セラピューティクス, インコーポレイテッド |
アデノ関連ウイルス抗体及びそのフラグメント
|
|
US20230256117A1
(en)
*
|
2020-06-19 |
2023-08-17 |
Genethon |
Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
|
|
KR20230031329A
(ko)
*
|
2020-06-30 |
2023-03-07 |
칠드런스 내셔널 메디컬 센터 |
골격 근섬유 복구를 개선하기 위한 재조합 인간 산성 스핑고미엘리나제의 용도
|
|
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
|
|
CN116333995A
(zh)
*
|
2020-07-14 |
2023-06-27 |
广州泛恩生物科技有限公司 |
基于cxcr6阳性t细胞的用于杀伤肿瘤的tcr-t细胞及其制备方法和应用
|
|
CN116121274B
(zh)
*
|
2020-07-29 |
2024-11-19 |
北京三诺佳邑生物技术有限责任公司 |
一组肝靶向新型腺相关病毒的获得及其应用
|
|
JP2023538519A
(ja)
|
2020-08-07 |
2023-09-08 |
スペースクラフト セブン リミテッド ライアビリティ カンパニー |
Aavベクターを使用したプラコフィリン-2(pkp2)遺伝子治療
|
|
US20220096606A1
(en)
|
2020-09-09 |
2022-03-31 |
Vertex Pharmaceuticals Incorporated |
Compositions and Methods for Treatment of Duchenne Muscular Dystrophy
|
|
JP2023548746A
(ja)
|
2020-10-13 |
2023-11-21 |
クリヤ セラピューティクス インコーポレイテッド |
サイトカインをコードする核酸を送達するためのウイルスベクターコンストラクトおよびがんを処置するためのその使用
|
|
US20220110970A1
(en)
*
|
2020-10-14 |
2022-04-14 |
The Texas A&M University System |
Engineered Extracellular Vesicle Delivery Systems and Uses Thereof
|
|
EP4240854A1
(en)
|
2020-11-06 |
2023-09-13 |
Vertex Pharmaceuticals Incorporated |
Compositions and methods for treatment of dm1 with slucas9 and sacas9
|
|
KR102803305B1
(ko)
*
|
2020-11-25 |
2025-05-07 |
연세대학교 산학협력단 |
트랜스-스플라이싱 아데노-연관 바이러스 벡터를 포함하는 프라임에디팅용 조성물
|
|
US20220238230A1
(en)
*
|
2021-01-15 |
2022-07-28 |
Trustees Of Tufts College |
Methods for modulating the function of biological regulatory networks in health and disease by exploiting their memory properties
|
|
EP4298221A1
(en)
|
2021-02-26 |
2024-01-03 |
Vertex Pharmaceuticals Incorporated |
Compositions and methods for treatment of myotonic dystrophy type 1 with crispr/slucas9
|
|
TW202302848A
(zh)
|
2021-02-26 |
2023-01-16 |
美商維泰克斯製藥公司 |
以crispr/sacas9治療第1型肌強直性營養不良之組合物及方法
|
|
EP4314295A1
(en)
|
2021-03-26 |
2024-02-07 |
The Board Of Regents Of The University Of Texas System |
Nucleotide editing to reframe dmd transcripts by base editing and prime editing
|
|
WO2022229851A1
(en)
|
2021-04-26 |
2022-11-03 |
Crispr Therapeutics Ag |
Compositions and methods for using slucas9 scaffold sequences
|
|
WO2022234519A1
(en)
|
2021-05-05 |
2022-11-10 |
Crispr Therapeutics Ag |
Compositions and methods for using sacas9 scaffold sequences
|
|
MX2023014041A
(es)
|
2021-05-28 |
2023-12-15 |
Shanghai Regenelead Therapies Co Ltd |
Virus adenoasociado recombinante que tiene una capsida variante y su aplicacion.
|
|
WO2023280175A1
(en)
*
|
2021-07-06 |
2023-01-12 |
National Institute Of Biological Sciences, Beijing |
Methods for treating complex i deficiencies or cancers by modulating gro3p biosynthesis
|
|
WO2023018637A1
(en)
|
2021-08-09 |
2023-02-16 |
Vertex Pharmaceuticals Incorporated |
Gene editing of regulatory elements
|
|
EP4399302A2
(en)
|
2021-09-08 |
2024-07-17 |
Vertex Pharmaceuticals Incorporated |
Precise excisions of portions of exon 51 for treatment of duchenne muscular dystrophy
|
|
AR127217A1
(es)
|
2021-10-01 |
2023-12-27 |
Biomarin Pharm Inc |
Tratamiento de angioedema hereditario con vectores de genoterapia de aav y formulaciones terapéuticas
|
|
JP2023059858A
(ja)
|
2021-10-15 |
2023-04-27 |
リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル |
自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
|
|
IL311786A
(en)
|
2021-10-21 |
2024-05-01 |
Vertex Pharma |
hypoimmune cells
|
|
CA3238867A1
(en)
|
2021-11-29 |
2023-06-01 |
Shanghai Regenelead Therapies Co., Ltd. |
Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease
|
|
CN118660913A
(zh)
|
2022-02-21 |
2024-09-17 |
上海瑞宏迪医药有限公司 |
Vegf结合分子及其医药用途
|
|
GB202202628D0
(en)
*
|
2022-02-25 |
2022-04-13 |
Babraham Inst |
Novel use and method comprising IL-2 and a tissue- or organic-specific targeting moiety
|
|
EP4490291A1
(en)
|
2022-03-08 |
2025-01-15 |
Vertex Pharmaceuticals Incorporated |
Precise excisions of portions of exons for treatment of duchenne muscular dystrophy
|
|
TW202345911A
(zh)
|
2022-03-08 |
2023-12-01 |
美商維泰克斯製藥公司 |
用於治療杜興氏肌肉失養症(duchenne muscular dystrophy)之部分外顯子44、50及53之精確切除
|
|
CA3249261A1
(en)
|
2022-04-18 |
2023-10-26 |
Vertex Pharmaceuticals Incorporated |
COMPOSITIONS AND PROCESSES TO IMPROVE ADENE-ASSOCIATED VIRUS (AAV) THERAPY AND REDUCE AAV TROPISM TO THE LIVER
|
|
WO2023202469A1
(zh)
*
|
2022-04-19 |
2023-10-26 |
康霖生物科技(杭州)有限公司 |
一种用于遗传性凝血因子缺乏病治疗的核酸构建体及其用途
|
|
EP4558634A1
(en)
|
2022-07-18 |
2025-05-28 |
Vertex Pharmaceuticals Incorporated |
Tandem guide rnas (tg-rnas) and their use in genome editing
|
|
KR20250108100A
(ko)
|
2022-09-22 |
2025-07-15 |
디나코 아게 |
Aav 유전자 치료법 벡터를 이용한 심근병증의 치료
|
|
WO2024243563A1
(en)
|
2023-05-25 |
2024-11-28 |
Juno Therapeutics, Inc. |
Aav purification method
|
|
WO2025040166A1
(zh)
|
2023-08-23 |
2025-02-27 |
上海瑞宏迪医药有限公司 |
药物组合物及其用途
|
|
WO2025082618A1
(en)
|
2023-10-20 |
2025-04-24 |
Fuse Vectors Aps |
In vitro parvovirus capsid manufacturing
|
|
WO2025106374A1
(en)
|
2023-11-13 |
2025-05-22 |
Juno Therapeutics, Inc. |
Aav production method
|
|
WO2025214477A1
(en)
|
2024-04-12 |
2025-10-16 |
Skyline Therapeutics (Shanghai) Co., Ltd. |
Treatment of genetic cardiomyopathies with aav gene therapy vectors
|