AU4645697A
(en)
*
|
1996-09-11 |
1998-04-02 |
Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The |
Aav4 vector and uses thereof
|
JP4060531B2
(ja)
*
|
1998-05-28 |
2008-03-12 |
アメリカ合衆国 |
Aav5ベクターおよびその使用
|
JP2004538005A
(ja)
|
2001-08-08 |
2004-12-24 |
ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア |
シアル酸に結合するタンパク質を有するウイルスベクターの精製法
|
AU2013202568B2
(en)
*
|
2001-11-13 |
2015-09-17 |
The Trustees Of The University Of Pennsylvania |
Adeno-associated virus rh.8 (AAVrh.8) sequences and recombinant AAVs comprising same
|
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
|
PT1453547T
(pt)
|
2001-12-17 |
2016-12-28 |
Univ Pennsylvania |
Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
|
JP4769417B2
(ja)
*
|
2001-12-17 |
2011-09-07 |
ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア |
アデノ随伴ウイルス(aav)血清型9の配列、それを含むベクターおよびその使用
|
US7419817B2
(en)
*
|
2002-05-17 |
2008-09-02 |
The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. |
Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
|
AU2003304396A1
(en)
*
|
2002-06-28 |
2005-02-25 |
University Of Florida Research Foundation, Inc. |
Raav compositions and methods for delivery of human factor vii polypeptides and treatment of hemophilia a
|
WO2004108922A2
(en)
*
|
2003-04-25 |
2004-12-16 |
The Trustees Of The University Of Pennsylvania |
Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
|
WO2005017101A2
(en)
|
2003-05-19 |
2005-02-24 |
THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH |
Avian adenoassociated virus (aaav) and uses thereof
|
WO2004110483A1
(en)
*
|
2003-05-31 |
2004-12-23 |
Yiyou Chen |
Method and composition of a novel vaccine design for the prevention and treatment of sars
|
US7186699B2
(en)
*
|
2003-06-03 |
2007-03-06 |
Cell Genesys, Inc. |
Method for treating cancer by vector-mediated delivery of one or more anti-angiogenic or pro-apoptotic genes
|
ES2648241T3
(es)
|
2003-09-30 |
2017-12-29 |
The Trustees Of The University Of Pennsylvania |
Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
|
US7655467B2
(en)
|
2003-11-14 |
2010-02-02 |
The University Of Washington |
Compositions and methods for systemic nucleic acid sequence delivery
|
WO2005056807A2
(en)
*
|
2003-12-04 |
2005-06-23 |
The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health |
Bovine adeno-associated viral (baav) vector and uses thereof
|
WO2005077333A2
(en)
*
|
2004-02-10 |
2005-08-25 |
University Of Florida Research Foundation, Inc. |
Gel-based delivery of recombinant adeno-associated virus vectors
|
EP1742657B1
(en)
|
2004-04-28 |
2013-11-06 |
The Trustees of The University of Pennsylvania |
Immunization regimen with e4-deleted adenovirus prime and e1-deleted adenovirus boost
|
ES2361000T3
(es)
|
2004-04-28 |
2011-06-13 |
The Trustees Of The University Of Pennsylvania |
Suministro secuencial de moléculas inmunogénicas mediante administraciones de un adenovirus y de un virus adeno-asociado.
|
US7964196B2
(en)
*
|
2004-05-25 |
2011-06-21 |
Chimeros, Inc. |
Self-assembling nanoparticle drug delivery system
|
US20080188431A1
(en)
*
|
2004-09-08 |
2008-08-07 |
Chiorini John A |
Transcytosis of Adeno-Associated Viruses
|
WO2006119432A2
(en)
*
|
2005-04-29 |
2006-11-09 |
The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services |
Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
|
ATE525092T1
(de)
|
2005-05-02 |
2011-10-15 |
Genzyme Corp |
Gentherapie für neurometabolische erkrankungen
|
CN105770909A
(zh)
*
|
2005-05-02 |
2016-07-20 |
建新公司 |
脊髓失调症的基因治疗
|
WO2007120542A2
(en)
|
2006-03-30 |
2007-10-25 |
The Board Of Trustees Of The Leland Stanford Junior University |
Aav capsid library and aav capsid proteins
|
CA2648581A1
(en)
*
|
2006-04-07 |
2008-09-12 |
Chimeros, Inc. |
Compositions and methods for treating b- cell malignancies
|
ES2400235T3
(es)
|
2006-04-28 |
2013-04-08 |
The Trustees Of The University Of Pennsylvania |
Método de producción escalable de AAV
|
CN101495624A
(zh)
*
|
2006-04-28 |
2009-07-29 |
宾夕法尼亚州立大学托管会 |
衣壳免疫原性降低的经修饰aav载体及其用途
|
US20090226525A1
(en)
*
|
2007-04-09 |
2009-09-10 |
Chimeros Inc. |
Self-assembling nanoparticle drug delivery system
|
US9217155B2
(en)
*
|
2008-05-28 |
2015-12-22 |
University Of Massachusetts |
Isolation of novel AAV'S and uses thereof
|
US8263601B2
(en)
|
2009-02-27 |
2012-09-11 |
Concert Pharmaceuticals, Inc. |
Deuterium substituted xanthine derivatives
|
WO2010120874A2
(en)
|
2009-04-14 |
2010-10-21 |
Chimeros, Inc. |
Chimeric therapeutics, compositions, and methods for using same
|
US8734809B2
(en)
|
2009-05-28 |
2014-05-27 |
University Of Massachusetts |
AAV's and uses thereof
|
WO2011032100A1
(en)
|
2009-09-11 |
2011-03-17 |
Government Of The U.S.A., As Represented By The Secretary, Department Of Health And Human Services |
Inhibitors of kshv vil6 and human il6
|
WO2011041502A1
(en)
|
2009-10-01 |
2011-04-07 |
The Trustees Of The University Of Pennsylvania |
Aav vectors expressing sec1o for treating kidney damage
|
CA2793633A1
(en)
|
2010-03-29 |
2011-10-13 |
The Trustees Of The University Of Pennsylvania |
Pharmacologically induced transgene ablation system
|
US9315825B2
(en)
|
2010-03-29 |
2016-04-19 |
The Trustees Of The University Of Pennsylvania |
Pharmacologically induced transgene ablation system
|
WO2011133874A1
(en)
|
2010-04-23 |
2011-10-27 |
University Of Massachusetts |
Multicistronic expression constructs
|
WO2011133901A2
(en)
|
2010-04-23 |
2011-10-27 |
University Of Massachusetts |
Aav-based treatment of cholesterol-related disorders
|
DK2826860T3
(en)
|
2010-04-23 |
2018-12-03 |
Univ Massachusetts |
CNS targeting AAV vectors and methods for their use
|
US9309534B2
(en)
|
2010-07-12 |
2016-04-12 |
Universidad Autonoma De Barcelona |
Gene therapy composition for use in diabetes treatment
|
WO2012075040A2
(en)
*
|
2010-11-30 |
2012-06-07 |
Shire Human Genetic Therapies, Inc. |
mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
|
US10392632B2
(en)
|
2011-02-14 |
2019-08-27 |
The Children's Hospital Of Philadelphia |
AAV8 vector with enhanced functional activity and methods of use thereof
|
US20140031418A1
(en)
|
2011-04-20 |
2014-01-30 |
The Trustees Of The University Of Pennsylvania |
Regimens and Compositions for AAV-Mediated Passive Immunization of Airborne Pathogens
|
EA027410B1
(ru)
|
2011-04-29 |
2017-07-31 |
Селекта Байосайенсиз, Инк. |
Наноносители, вызывающие иммунную толерантность, для снижения ответной реакции цитотоксических t-лимфоцитов
|
US8865188B2
(en)
|
2011-09-09 |
2014-10-21 |
Biomed Realty, L.P. |
Methods and compositions for controlling assembly of viral proteins
|
EP2797613B1
(en)
|
2011-10-27 |
2019-12-04 |
Wellstat Ophthalmics Corporation |
Vectors encoding rod-derived cone viability factor
|
US9434928B2
(en)
|
2011-11-23 |
2016-09-06 |
Nationwide Children's Hospital, Inc. |
Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
|
EP3311830A1
(en)
*
|
2012-02-14 |
2018-04-25 |
The Regents of The University of California |
Systemic delivery and regulated expression of paracrine genes for cardiovascular diseases and other conditions
|
JP5926580B2
(ja)
|
2012-03-01 |
2016-05-25 |
ユー・ディー・シー アイルランド リミテッド |
有機電界発光素子、有機電界発光素子用材料、並びに、該素子を用いた発光装置、表示装置、照明装置及び該素子に用いられる化合物
|
WO2014011210A1
(en)
|
2012-07-11 |
2014-01-16 |
The Trustees Of The University Of Pennsylvania |
Aav-mediated gene therapy for rpgr x-linked retinal degeneration
|
EP2692868A1
(en)
|
2012-08-02 |
2014-02-05 |
Universitat Autònoma De Barcelona |
Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
|
CN104797593B
(zh)
*
|
2012-09-28 |
2020-05-08 |
北卡罗来纳-查佩尔山大学 |
靶向少突胶质细胞的aav载体
|
US10266845B2
(en)
|
2013-02-08 |
2019-04-23 |
The Trustees Of The University Of Pennsylvania |
Enhanced AAV-mediated gene transfer for retinal therapies
|
WO2014124282A1
(en)
|
2013-02-08 |
2014-08-14 |
The Trustees Of The University Of Pennsylvania |
Enhanced aav-mediated gene transfer for retinal therapies
|
SI3889173T1
(sl)
|
2013-02-15 |
2023-11-30 |
Bioverativ Therapeutics Inc. |
Optimiran gen dejavnika VIII
|
US8957044B2
(en)
|
2013-03-01 |
2015-02-17 |
Wake Forest University Health Sciences |
Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
|
JP6591956B2
(ja)
|
2013-03-15 |
2019-10-16 |
ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア |
Mps1を治療するための組成物および方法
|
KR102268473B1
(ko)
|
2013-04-20 |
2021-06-25 |
더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 |
엑손 2-표적 U7snRNA 폴리뉴클레오티드 작제물의 재조합형 아데노 부속 바이러스 전달
|
WO2015012924A2
(en)
|
2013-04-29 |
2015-01-29 |
The Trustees Of The University Of Pennsylvania |
Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
|
WO2015013313A2
(en)
*
|
2013-07-22 |
2015-01-29 |
The Children's Hospital Of Philadelphia |
Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
|
CN106164271B
(zh)
|
2014-02-11 |
2020-06-02 |
科罗拉多州立大学董事会(法人团体) |
Crispr支持的多路基因组工程化
|
GB201403684D0
(en)
|
2014-03-03 |
2014-04-16 |
King S College London |
Vector
|
SI3116900T1
(sl)
|
2014-03-09 |
2021-02-26 |
The Trustees Of The University Of Pennsylvania |
Sestavki uporabni pri zdravljenju pomanjkanja ornitin transkarbamilaze(OTC)
|
EP2933335A1
(en)
|
2014-04-18 |
2015-10-21 |
Genethon |
A method of treating peripheral neuropathies and motor neuron diseases
|
WO2015164723A1
(en)
|
2014-04-25 |
2015-10-29 |
The Trustees Of The University Of Pennsylvania |
Methods and compositions for treating metastatic breast cancer and other cancers in the brain
|
ES2876409T3
(es)
|
2014-04-25 |
2021-11-12 |
Univ Pennsylvania |
Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol
|
MY186389A
(en)
|
2014-05-13 |
2021-07-22 |
Univ Pennsylvania |
Compositions comprising aav expressing dual antibody constructs and uses thereof
|
WO2015191508A1
(en)
|
2014-06-09 |
2015-12-17 |
Voyager Therapeutics, Inc. |
Chimeric capsids
|
EP2960336A1
(en)
|
2014-06-27 |
2015-12-30 |
Genethon |
Efficient systemic treatment of dystrophic muscle pathologies
|
WO2016004113A1
(en)
|
2014-06-30 |
2016-01-07 |
Biogen Ma Inc. |
Optimized factor ix gene
|
EP2975116B1
(en)
*
|
2014-07-16 |
2019-08-21 |
Max-Delbrück-Centrum für Molekulare Medizin |
Tgif2-induced reprogramming of hepatic cells to pancreatic progenitor cells and medical uses thereof
|
US11053494B2
(en)
|
2014-08-09 |
2021-07-06 |
Research Institute At Nationwide Children's Hospital |
Methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene
|
CA2957737A1
(en)
|
2014-09-07 |
2016-03-10 |
Selecta Biosciences, Inc. |
Methods and compositions for attenuating gene expression modulating anti-viral transfer vector immune responses
|
US10973931B2
(en)
|
2014-09-16 |
2021-04-13 |
Universitat Autònoma De Barcelona |
Adeno-associated viral vectors for the gene therapy of metabolic diseases
|
JP6842410B2
(ja)
*
|
2014-10-03 |
2021-03-17 |
ユニバーシティ オブ マサチューセッツ |
新規の高効率ライブラリーにより同定されるaavベクター
|
WO2016054554A1
(en)
|
2014-10-03 |
2016-04-07 |
University Of Massachusetts |
Heterologous targeting peptide grafted aavs
|
CN107073051B
(zh)
|
2014-10-21 |
2021-08-24 |
马萨诸塞大学 |
重组aav变体及其用途
|
MX2017005834A
(es)
|
2014-11-05 |
2017-11-17 |
Voyager Therapeutics Inc |
Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
|
CN112375760A
(zh)
|
2014-11-14 |
2021-02-19 |
沃雅戈治疗公司 |
调节性多核苷酸
|
AU2015346162B2
(en)
|
2014-11-14 |
2022-02-10 |
Voyager Therapeutics, Inc. |
Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
|
WO2016094783A1
(en)
|
2014-12-12 |
2016-06-16 |
Voyager Therapeutics, Inc. |
Compositions and methods for the production of scaav
|
EP3233131A1
(en)
|
2014-12-16 |
2017-10-25 |
Board of Regents of the University of Nebraska |
Gene therapy for juvenile batten disease
|
WO2016110518A1
(en)
|
2015-01-07 |
2016-07-14 |
Universitat Autònoma De Barcelona |
Single-vector gene construct comprising insulin and glucokinase genes
|
EP3245221A4
(en)
|
2015-01-16 |
2018-06-13 |
Voyager Therapeutics, Inc. |
Central nervous system targeting polynucleotides
|
EP3256487A4
(en)
|
2015-02-09 |
2018-07-18 |
Duke University |
Compositions and methods for epigenome editing
|
EP3256170B1
(en)
|
2015-02-13 |
2020-09-23 |
University of Massachusetts |
Compositions and methods for transient delivery of nucleases
|
CN115074366A
(zh)
|
2015-04-16 |
2022-09-20 |
埃默里大学 |
用于肝脏中蛋白质表达的重组启动子和载体及其用途
|
US11046955B2
(en)
|
2015-04-24 |
2021-06-29 |
University Of Massachusetts |
Modified AAV constructs and uses thereof
|
JP6851319B2
(ja)
|
2015-04-27 |
2021-03-31 |
ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア |
ヒト疾患のCRISPR/Cas9媒介性の修正のためのデュアルAAVベクター系
|
GB201508026D0
(en)
|
2015-05-11 |
2015-06-24 |
Ucl Business Plc |
Capsid
|
WO2016200543A2
(en)
|
2015-05-13 |
2016-12-15 |
The Trustees Of The University Of Pennsylvania |
Aav-mediated expression of anti-inluenza antibodies and methods of use thereof
|
KR102415896B1
(ko)
|
2015-06-23 |
2022-06-30 |
더 칠드런스 호스피탈 오브 필라델피아 |
변형된 인자 ix, 및 세포, 기관 및 조직으로 유전자를 전달하기 위한 조성물, 방법 및 용도
|
WO2017015637A1
(en)
|
2015-07-22 |
2017-01-26 |
Duke University |
High-throughput screening of regulatory element function with epigenome editing technologies
|
ES2929110T3
(es)
|
2015-08-25 |
2022-11-24 |
Univ Duke |
Composiciones y métodos para mejorar la especificidad en ingeniería genética usando endonucleasas guiadas por ARN
|
CA2995733A1
(en)
|
2015-08-31 |
2017-03-09 |
The Trustees Of The University Of Pennsylvania |
Aav-epo for treating companion animals
|
RU2727411C2
(ru)
|
2015-09-24 |
2020-07-21 |
Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания |
Композиция и способ для лечения заболевания, опосредованного комплементом
|
PL3356390T3
(pl)
|
2015-09-28 |
2021-07-05 |
The University Of North Carolina At Chapel Hill |
Sposoby i kompozycje dla wektorów wirusowych unikających przeciwciał
|
WO2017062750A1
(en)
|
2015-10-09 |
2017-04-13 |
The Trustees Of The University Of Pennsylvania |
Compositions and methods useful in treating stargardt's disease and other ocular disorders
|
US11970710B2
(en)
|
2015-10-13 |
2024-04-30 |
Duke University |
Genome engineering with Type I CRISPR systems in eukaryotic cells
|
WO2017066764A2
(en)
*
|
2015-10-16 |
2017-04-20 |
William Marsh Rice University |
Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
|
DK3364997T3
(da)
|
2015-10-22 |
2024-04-22 |
Univ Massachusetts |
Aspartoacylase genterapi til behandling af canavans sygdom
|
CA3002980A1
(en)
|
2015-10-22 |
2017-04-27 |
University Of Massachusetts |
Prostate-targeting adeno-associated virus serotype vectors
|
US20180230489A1
(en)
|
2015-10-28 |
2018-08-16 |
Voyager Therapeutics, Inc. |
Regulatable expression using adeno-associated virus (aav)
|
EP4316512A3
(en)
|
2015-10-28 |
2024-04-24 |
The Trustees of The University of Pennsylvania |
Intrathecal administration of adeno-associated-viral vectors for gene therapy
|
BR112018009732A8
(pt)
|
2015-11-13 |
2019-02-26 |
Baxalta GmbH |
?polinucleotídeo, vetor de vírus adeno-associado, partícula de vírus adeno-associado, célula hospedeira, métodos para produzir uma partícula de vírus adeno-associado, para tratar hemofilia a, para transduzir uma célula hospedeira, e, uso de uma partícula de vírus adeno-associado?
|
SG10202106307UA
(en)
|
2015-11-13 |
2021-07-29 |
Takeda Pharmaceuticals Co |
Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
|
US10385320B2
(en)
|
2015-12-02 |
2019-08-20 |
The Board Of Trustees Of The Leland Stanford Junior University |
Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
|
CA3006569A1
(en)
|
2015-12-02 |
2017-06-08 |
Voyager Therapeutics, Inc. |
Assays for the detection of aav neutralizing antibodies
|
US10406244B2
(en)
|
2015-12-02 |
2019-09-10 |
The Board Of Trustees Of The Leland Stanford Junior University |
AAV vectors with expanded packaging capacity
|
FR3044926B1
(fr)
*
|
2015-12-09 |
2020-01-31 |
Genethon |
Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
|
US11015173B2
(en)
|
2015-12-11 |
2021-05-25 |
The Trustees Of The University Of Pennsylvania |
Scalable purification method for AAV1
|
US11015174B2
(en)
|
2015-12-11 |
2021-05-25 |
The Trustees Of The University Of Pennsylvania |
Scalable purification method for AAV8
|
EP3992283A1
(en)
|
2015-12-11 |
2022-05-04 |
The Trustees Of The University Of Pennsylvania |
Scalable purification method for aavrh10
|
CA3008142A1
(en)
*
|
2015-12-11 |
2017-06-15 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for treating familial hypercholesterolemia
|
US11098286B2
(en)
|
2015-12-11 |
2021-08-24 |
The Trustees Of The University Of Pennsylvania |
Scalable purification method for AAV9
|
WO2017106202A2
(en)
|
2015-12-14 |
2017-06-22 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for ocular disorders
|
CA3007330A1
(en)
*
|
2015-12-14 |
2017-06-22 |
The Trustees Of The University Of Pennsylvania |
Composition for treatment of crigler-najjar syndrome
|
JP7217630B2
(ja)
|
2016-02-01 |
2023-02-03 |
バイオベラティブ セラピューティクス インコーポレイテッド |
最適化第viii因子遺伝子
|
US11826433B2
(en)
|
2016-02-02 |
2023-11-28 |
University Of Massachusetts |
Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
|
CA3012195A1
(en)
|
2016-02-03 |
2017-08-10 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for treating mucopolysaccharidosis type i
|
EP3417055B1
(en)
|
2016-02-16 |
2021-10-13 |
The Board of Trustees of the Leland Stanford Junior University |
Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
|
WO2017173411A1
(en)
*
|
2016-04-02 |
2017-10-05 |
Research Institute At Nation Wide Children's Hospital |
Modified u6 promoter system for tissue specific expression
|
JP7171439B2
(ja)
|
2016-04-15 |
2022-11-15 |
ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア |
ムコ多糖症ii型を処置するための遺伝子療法
|
SG11201808812RA
(en)
|
2016-04-15 |
2018-11-29 |
Univ Pennsylvania |
Novel aav8 mutant capsids and compositions containing same
|
EP3452103A1
(en)
|
2016-04-15 |
2019-03-13 |
The Trustees Of The University Of Pennsylvania |
Compositions for treatment of wet age-related macular degeneration
|
RU2762257C2
(ru)
|
2016-04-15 |
2021-12-17 |
Зе Трастис Оф Зе Юниверсити Оф Пенсильвания |
Генная терапия для лечения гемофилии a
|
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
|
US11413356B2
(en)
|
2016-04-15 |
2022-08-16 |
University Of Massachusetts |
Methods and compositions for treating metabolic imbalance
|
WO2017184463A1
(en)
|
2016-04-17 |
2017-10-26 |
The Trustees Of The University Of Pennsylvania |
Compositions and methods useful for prophylaxis of organophosphates
|
WO2017189959A1
(en)
|
2016-04-29 |
2017-11-02 |
Voyager Therapeutics, Inc. |
Compositions for the treatment of disease
|
EP3448987A4
(en)
|
2016-04-29 |
2020-05-27 |
Voyager Therapeutics, Inc. |
COMPOSITIONS FOR THE TREATMENT OF DISEASES
|
CN115925999A
(zh)
|
2016-05-13 |
2023-04-07 |
4D分子治疗有限公司 |
腺相关病毒变体衣壳和其使用方法
|
SG11201809699XA
(en)
|
2016-05-18 |
2018-12-28 |
Voyager Therapeutics Inc |
Modulatory polynucleotides
|
RU2764587C2
(ru)
|
2016-05-18 |
2022-01-18 |
Вояджер Терапьютикс, Инк. |
Способы и композиции для лечения хореи гентингтона
|
US11882815B2
(en)
|
2016-06-15 |
2024-01-30 |
University Of Massachusetts |
Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
|
WO2018005445A1
(en)
*
|
2016-06-27 |
2018-01-04 |
The Broad Institute, Inc. |
Compositions and methods for detecting and treating diabetes
|
KR20230062878A
(ko)
|
2016-07-08 |
2023-05-09 |
더 트러스티스 오브 더 유니버시티 오브 펜실베니아 |
Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물
|
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
|
US11298041B2
(en)
|
2016-08-30 |
2022-04-12 |
The Regents Of The University Of California |
Methods for biomedical targeting and delivery and devices and systems for practicing the same
|
US11578340B2
(en)
|
2016-10-13 |
2023-02-14 |
University Of Massachusetts |
AAV capsid designs
|
EP3548065B1
(en)
|
2016-12-01 |
2022-11-09 |
INSERM - Institut National de la Santé et de la Recherche Médicale |
Pharmaceutical compositions for the treatment of retinal degenerative diseases
|
KR102604096B1
(ko)
|
2016-12-30 |
2023-11-23 |
더 트러스티스 오브 더 유니버시티 오브 펜실베니아 |
윌슨병을 치료하기 위한 유전자 치료
|
BR112019013576A2
(pt)
*
|
2016-12-30 |
2020-02-04 |
Univ Pennsylvania |
terapia genica para o tratamento da fenilcetonuria
|
KR20190104194A
(ko)
|
2017-01-07 |
2019-09-06 |
셀렉타 바이오사이언시즈, 인크. |
합성 나노담체에 커플링된 면역억제제의 패턴화된 투여
|
EP3576760A2
(en)
|
2017-02-01 |
2019-12-11 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for treating citrullenemia
|
WO2018152485A1
(en)
|
2017-02-20 |
2018-08-23 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for treating familial hypercholesterolemia
|
JOP20190200A1
(ar)
|
2017-02-28 |
2019-08-27 |
Univ Pennsylvania |
تركيبات نافعة في معالجة ضمور العضل النخاعي
|
RS65241B1
(sr)
|
2017-02-28 |
2024-03-29 |
Univ Pennsylvania |
Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe
|
SG11201907611WA
(en)
|
2017-02-28 |
2019-09-27 |
Univ Pennsylvania |
Influenza vaccines based on aav vectors
|
CA3054136A1
(en)
|
2017-03-01 |
2018-09-07 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for ocular disorders
|
EP3596112A2
(en)
|
2017-03-17 |
2020-01-22 |
Newcastle University |
Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
|
SG11201908575SA
(en)
|
2017-03-17 |
2019-10-30 |
Res Inst Nationwide Childrens Hospital |
Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
|
EP3601367A4
(en)
|
2017-03-30 |
2020-09-16 |
The University of Queensland |
CHEMERICAL MOLECULES AND ASSOCIATED USES
|
WO2018189208A1
(en)
|
2017-04-10 |
2018-10-18 |
Genethon |
Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
|
KR20190139951A
(ko)
|
2017-04-14 |
2019-12-18 |
리젠엑스바이오 인크. |
인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료
|
CA3060112A1
(en)
|
2017-04-21 |
2018-10-25 |
Precision Biosciences, Inc. |
Engineered meganucleases specific for recognition sequences in the pcsk9 gene
|
US11879133B2
(en)
|
2017-04-24 |
2024-01-23 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for ocular disorders
|
SG11201909868YA
(en)
|
2017-05-05 |
2019-11-28 |
Voyager Therapeutics Inc |
Compositions and methods of treating huntington's disease
|
JP2020518258A
(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. |
筋萎縮性側索硬化症(als)治療組成物および方法
|
US11633504B2
(en)
|
2017-05-09 |
2023-04-25 |
Emory University |
Nucleic acids encoding clotting factor variants and their use
|
WO2018209205A1
(en)
|
2017-05-11 |
2018-11-15 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for neuronal ceroid lipofuscinoses
|
JP7235676B2
(ja)
|
2017-05-17 |
2023-03-08 |
ザ ジェネラル ホスピタル コーポレイション |
結節性硬化症の遺伝子治療
|
EP3630974A1
(en)
|
2017-05-22 |
2020-04-08 |
Baxalta Incorporated |
Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia b
|
CA3098592A1
(en)
|
2017-05-31 |
2018-12-06 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for treating peroxisomal disorders
|
EP3638316A4
(en)
|
2017-06-14 |
2021-03-24 |
The Trustees Of The University Of Pennsylvania |
GENE THERAPY FOR EYE DISORDERS
|
JOP20190269A1
(ar)
|
2017-06-15 |
2019-11-20 |
Voyager Therapeutics Inc |
بولي نوكليوتيدات aadc لعلاج مرض باركنسون
|
AU2018298133A1
(en)
|
2017-07-06 |
2020-01-23 |
The Trustees Of The University Of Pennsylvania |
AAV9-mediated gene therapy for treating mucopolysaccharidosis type I
|
CA3069045A1
(en)
|
2017-07-07 |
2019-01-10 |
Genethon |
Novel polynucleotides encoding a human fkrp protein
|
CN111132626B
(zh)
|
2017-07-17 |
2024-01-30 |
沃雅戈治疗公司 |
轨迹阵列引导系统
|
KR20200044793A
(ko)
|
2017-08-03 |
2020-04-29 |
보이저 테라퓨틱스, 인크. |
Aav의 전달을 위한 조성물 및 방법
|
WO2019060454A2
(en)
|
2017-09-20 |
2019-03-28 |
4D Molecular Therapeutics Inc. |
CAPSID VARIANT ADENO-ASSOCIATED VIRUSES AND METHODS OF USE
|
US11819539B2
(en)
|
2017-09-22 |
2023-11-21 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for treating Mucopolysaccharidosis type II
|
JP7427584B2
(ja)
|
2017-10-13 |
2024-02-05 |
セレクタ バイオサイエンシーズ インコーポレーテッド |
抗ウイルス導入ベクターigm応答を減弱化するための方法および組成物
|
JP2021502060A
(ja)
|
2017-10-16 |
2021-01-28 |
ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. |
筋萎縮性側索硬化症(als)の治療
|
WO2019079242A1
(en)
|
2017-10-16 |
2019-04-25 |
Voyager Therapeutics, Inc. |
TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
|
MX2020003945A
(es)
|
2017-10-18 |
2020-11-09 |
Regenxbio Inc |
Tratamiento de enfermedades oculares y cancer de colon metastasico con vegf-trap humano modificado post-traduccionalmente .
|
WO2019078916A1
(en)
|
2017-10-18 |
2019-04-25 |
Research Institute At Nationwide Children's Hospital |
ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MUSCLE-SPECIFIC MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY
|
KR20200067195A
(ko)
|
2017-10-18 |
2020-06-11 |
리젠엑스바이오 인크. |
완전-인간 번역후 변형된 항체 치료제
|
EP3697916A4
(en)
|
2017-10-20 |
2021-08-11 |
Research Institute at Nationwide Children's Hospital |
METHODS AND MATERIALS FOR NT-3 GENE THERAPY
|
ES2946747T3
(es)
|
2017-11-27 |
2023-07-25 |
4D Molecular Therapeutics Inc |
Variantes de cápsides de virus adenoasociado y su utilización para inhibir la angiogénesis
|
US11723989B2
(en)
|
2017-11-30 |
2023-08-15 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for mucopolysaccharidosis IIIB
|
WO2019108857A1
(en)
|
2017-11-30 |
2019-06-06 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for mucopolysaccharidosis iiia
|
BR112020012336A2
(pt)
|
2017-12-19 |
2021-03-30 |
Akouos, Inc. |
Administração de anticorpos terapêuticos mediada por aav para o ouvido interno
|
BR112020014404A2
(pt)
|
2018-01-17 |
2020-12-29 |
Adrenas Therapeutics, Inc. |
Terapia gênica de vírus adenoassociado para deficiência de 21-hidroxilase
|
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
|
US10610606B2
(en)
|
2018-02-01 |
2020-04-07 |
Homology Medicines, Inc. |
Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
|
WO2019161365A1
(en)
|
2018-02-19 |
2019-08-22 |
Homology Medicines, Inc. |
Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
|
WO2019165436A1
(en)
|
2018-02-26 |
2019-08-29 |
Antolrx |
Tolerogenic liposomes and methods of use thereof
|
JP2021519065A
(ja)
*
|
2018-03-23 |
2021-08-10 |
ユニバーシティ オブ マサチューセッツ |
骨障害を処置するための遺伝子治療法
|
US11608510B2
(en)
|
2018-03-30 |
2023-03-21 |
The Board Of Trustees Of The Leland Stanford Junior University |
Recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
|
JP7406677B2
(ja)
*
|
2018-04-03 |
2023-12-28 |
ギンコ バイオワークス インコーポレイテッド |
抗体を回避するウイルスベクター
|
CA3098565A1
(en)
|
2018-04-29 |
2019-11-07 |
Claire G. ZHANG |
Scalable clarification process for recombinant aav production
|
WO2019212922A1
(en)
|
2018-04-29 |
2019-11-07 |
Regenxbio Inc. |
Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
|
SG11202011975WA
(en)
|
2018-06-05 |
2020-12-30 |
Lifeedit Inc |
Rna-guided nucleases and active fragments and variants thereof and methods of use
|
EP3807404A1
(en)
|
2018-06-13 |
2021-04-21 |
Voyager Therapeutics, Inc. |
Engineered 5' untranslated regions (5' utr) for aav production
|
AU2019285186A1
(en)
|
2018-06-14 |
2021-01-07 |
Regenxbio Inc. |
Anion exchange chromatography for recombinant AAV production
|
BR112020025995A2
(pt)
|
2018-06-18 |
2021-03-23 |
Research Institute At Nationwide Children's Hospital |
administração de microdistrofina músculo-específica por vetor de vírus adeno-associado para tratar a distrofia muscular
|
GB201811368D0
(en)
|
2018-07-11 |
2018-08-29 |
Ucb Biopharma Sprl |
Antibody
|
JP2021530571A
(ja)
|
2018-07-16 |
2021-11-11 |
セレクタ バイオサイエンシーズ インコーポレーテッドSelecta Biosciences, Inc. |
Mmaコンストラクトおよびベクターの方法および組成物
|
CA3106639A1
(en)
|
2018-07-16 |
2020-01-23 |
Selecta Biosciences, Inc. |
Methods and compositions of otc constructs and vectors
|
US20210355454A1
(en)
|
2018-07-24 |
2021-11-18 |
Voyager Therapeutics, Inc. |
Systems and methods for producing gene therapy formulations
|
CN112567027A
(zh)
|
2018-08-10 |
2021-03-26 |
再生生物股份有限公司 |
用于重组aav生产的可扩展方法
|
US20210324417A1
(en)
|
2018-08-29 |
2021-10-21 |
Research Institute At Nationwide Children's Hospital |
Products and methods for inhibition of expression of mutant gars protein
|
BR112021003897A2
(pt)
|
2018-08-30 |
2021-05-25 |
Tenaya Therapeutics, Inc. |
reprogramação de células cardíacas com miocarina e asci1
|
US20210348242A1
(en)
|
2018-10-04 |
2021-11-11 |
Voyager Therapeutics, Inc. |
Methods for measuring the titer and potency of viral vector particles
|
TW202028458A
(zh)
|
2018-10-05 |
2020-08-01 |
美商航海家醫療公司 |
編碼腺相關病毒(aav)生產蛋白之經基因工程化核酸構築體
|
EP3867389A1
(en)
|
2018-10-15 |
2021-08-25 |
Voyager Therapeutics, Inc. |
Expression vectors for large-scale production of raav in the baculovirus/sf9 system
|
UY38407A
(es)
|
2018-10-15 |
2020-05-29 |
Novartis Ag |
Anticuerpos estabilizadores de trem2
|
WO2020081415A1
(en)
|
2018-10-15 |
2020-04-23 |
Regenxbio Inc. |
Method for measuring the infectivity of replication defective viral vectors and viruses
|
US20210363509A1
(en)
|
2018-10-22 |
2021-11-25 |
University Of Rochester |
Genome Editing by Directed Non-Homologous DNA Insertion Using a Retroviral Integrase-Cas9 Fusion Protein
|
MX2021007835A
(es)
|
2018-12-27 |
2021-08-11 |
Lifeedit Therapeutics Inc |
Polipeptidos utiles para edicion de genes y metodos de uso.
|
WO2020140007A1
(en)
|
2018-12-28 |
2020-07-02 |
University Of Rochester |
Gene therapy for best1 dominant mutations
|
KR20210110345A
(ko)
|
2018-12-31 |
2021-09-07 |
더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 |
RNA 표적화 CRISPR-Cas13b를 사용한 DUX4 RNA 침묵화
|
CN113518628A
(zh)
|
2019-01-04 |
2021-10-19 |
阿尔特拉吉尼克斯制药公司 |
治疗威尔逊病的基因疗法构建体
|
MX2021008487A
(es)
|
2019-01-14 |
2021-11-12 |
Univ Rochester |
Escisión y poliadenilación del arn nuclear dirigido con crispr-cas.
|
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
|
CN109628490A
(zh)
*
|
2019-01-26 |
2019-04-16 |
青岛农业大学 |
一种预防犬瘟热的shRNA重组腺相关病毒
|
KR20210130158A
(ko)
|
2019-01-31 |
2021-10-29 |
오레곤 헬스 앤드 사이언스 유니버시티 |
Aav 캡시드의 전사 의존적 유도 진화를 사용하는 방법
|
CR20210444A
(es)
|
2019-02-25 |
2021-11-02 |
Novartis Ag |
Composiciones y métodos para tratar distrofia cristalina de bietti
|
WO2020174369A2
(en)
|
2019-02-25 |
2020-09-03 |
Novartis Ag |
Compositions and methods to treat bietti crystalline dystrophy
|
JP2022526526A
(ja)
|
2019-03-25 |
2022-05-25 |
ジェネトン |
オーバーラップaavベクターを使用する大きいサイズのクアシジストロフィンの産生
|
EP3946467A1
(en)
|
2019-04-03 |
2022-02-09 |
REGENXBIO Inc. |
Gene therapy for eye pathologies
|
TW202102526A
(zh)
|
2019-04-04 |
2021-01-16 |
美商銳進科斯生物股份有限公司 |
重組腺相關病毒及其用途
|
US20220275358A1
(en)
|
2019-04-11 |
2022-09-01 |
Regenxbio Inc. |
Methods of size exclusion chromatography for the characterization of recombinant adeno-associated virus compositions
|
JP2022529002A
(ja)
|
2019-04-19 |
2022-06-16 |
レジェンクスバイオ インコーポレーテッド |
アデノ随伴ウイルスベクター製剤及び方法
|
CN114144197A
(zh)
|
2019-04-24 |
2022-03-04 |
再生生物股份有限公司 |
完全人类翻译后修饰的抗体治疗剂
|
CA3138525A1
(en)
|
2019-04-28 |
2020-11-05 |
Selecta Biosciences, Inc. |
Methods for treatment of subjects with preexisting immunity to viral transfer vectors
|
US20200390718A1
(en)
|
2019-05-28 |
2020-12-17 |
Selecta Biosciences, Inc. |
Methods and compositions for attenuated anti-viral transfer vector immune response
|
JP2022538497A
(ja)
|
2019-07-02 |
2022-09-02 |
エム6ピー セラピューティクス (スイッツァランド) ゲーエムベーハー |
ライソゾーム蓄積症を処置するためのベクター組成物およびそれを使用する方法
|
US20220251567A1
(en)
|
2019-07-10 |
2022-08-11 |
Inserm (Institut National De La Santè Et De La Recherche Médicale) |
Methods for the treatment of epilepsy
|
EP3997226A1
(en)
|
2019-07-11 |
2022-05-18 |
Tenaya Therapeutics, Inc. |
Cardiac cell reprogramming with micrornas and other factors
|
US10557149B1
(en)
*
|
2019-07-15 |
2020-02-11 |
Vigene Biosciences, Inc. |
Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
|
US10801042B1
(en)
|
2019-07-15 |
2020-10-13 |
Vigene Biosciences, Inc. |
Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
|
US10653731B1
(en)
|
2019-07-15 |
2020-05-19 |
Vigene Biosciences Inc. |
Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
|
EP4004206A1
(en)
|
2019-07-23 |
2022-06-01 |
University of Rochester |
Targeted rna cleavage with crispr-cas
|
EP4004214A1
(en)
|
2019-07-26 |
2022-06-01 |
RegenxBio Inc. |
Engineered nucleic acid regulatory element and methods of uses thereof
|
EP4007814A1
(en)
|
2019-07-26 |
2022-06-08 |
Akouos, Inc. |
Methods of treating hearing loss using a secreted target protein
|
RU2751592C2
(ru)
*
|
2019-08-22 |
2021-07-15 |
Общество С Ограниченной Ответственностью "Анабион" |
Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
|
WO2021041373A1
(en)
|
2019-08-26 |
2021-03-04 |
Regenxbio Inc. |
Treatment of diabetic retinopathy with fully-human post-translationally modified anti-vegf fab
|
CA3149449A1
(en)
|
2019-09-19 |
2021-03-25 |
Evelyne GICQUEL |
Gene therapy expression system alleviating cardiac toxicity of fkrp
|
WO2021067598A1
(en)
|
2019-10-04 |
2021-04-08 |
Ultragenyx Pharmaceutical Inc. |
Methods for improved therapeutic use of recombinant aav
|
CA3156984A1
(en)
|
2019-10-07 |
2021-04-15 |
Regenxbio Inc. |
Adeno-associated virus vector pharmaceutical composition and methods
|
WO2021072129A2
(en)
|
2019-10-08 |
2021-04-15 |
Trustees Of Boston College |
Proteins containing multiple, different unnatural amino acids and methods of making and using such proteins
|
CA3157700A1
(en)
|
2019-10-17 |
2021-04-22 |
Stridebio, Inc. |
Adeno-associated viral vectors for treatment of niemann-pick disease type c
|
AU2020366242A1
(en)
|
2019-10-18 |
2022-05-26 |
Research Institute At Nationwide Children's Hospital |
Materials and methods for the treatment of disorders associated mutations in the IRF2BPL gene
|
EP4055030A1
(en)
*
|
2019-11-08 |
2022-09-14 |
President And Fellows Of Harvard College |
Viral capsid polypeptides
|
US20230016983A1
(en)
|
2019-11-19 |
2023-01-19 |
lNSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) |
Antisense oligonucleotides and thier use for the treatment of cancer
|
IL293210A
(en)
|
2019-11-22 |
2022-07-01 |
Res Inst Nationwide Childrens Hospital |
Materials and methods for treating disorders associated with the ighmbp2 gene
|
JP2023503455A
(ja)
*
|
2019-11-22 |
2023-01-30 |
ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア |
アデノ随伴ウイルスベクター変種
|
US20230270886A1
(en)
|
2019-11-28 |
2023-08-31 |
Regenxbio Inc. |
Microdystrophin gene therapy constructs and uses thereof
|
CA3164189A1
(en)
|
2019-12-10 |
2021-06-17 |
Takeda Pharmaceutical Company Limited |
Adeno associated virus vectors for the treatment of hunter disease
|
CN115516100A
(zh)
|
2019-12-20 |
2022-12-23 |
全国儿童医院研究所 |
靶向肌肉疾病中肌肉的优化基因疗法
|
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
|
BR112022014563A2
(pt)
|
2020-01-22 |
2022-09-13 |
Regenxbio Inc |
Método para tratamento de um sujeito humano diagnosticado com mucopolissacaridose i (mps i)
|
AU2021214174A1
(en)
|
2020-01-29 |
2022-08-18 |
Regenxbio Inc. |
Treatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS) produced by human neural or glial cells
|
KR20220133900A
(ko)
|
2020-01-29 |
2022-10-05 |
리젠엑스바이오 인크. |
점액다당류증 iva의 치료
|
WO2021158982A2
(en)
|
2020-02-07 |
2021-08-12 |
University Of Rochester |
Targeted translation of rna with crispr-cas13 to enhance protein synthesis
|
KR20220141829A
(ko)
|
2020-02-07 |
2022-10-20 |
유니버시티 오브 로체스터 |
리보자임-매개 rna 조립 및 발현
|
BR112022015921A2
(pt)
|
2020-02-14 |
2022-10-04 |
Ultragenyx Pharmaceutical Inc |
Terapia gênica para tratar o transtorno de deficiência de cdkl5
|
EP4114421A1
(en)
|
2020-03-02 |
2023-01-11 |
Tenaya Therapeutics, Inc. |
Gene vector control by cardiomyocyte-expressed micrornas
|
KR20220150353A
(ko)
|
2020-03-05 |
2022-11-10 |
네오티엑스 테라퓨틱스 엘티디. |
면역 세포를 사용하여 암을 치료하기 위한 방법 및 조성물
|
JP2023518415A
(ja)
|
2020-03-19 |
2023-05-01 |
ウルトラジェニックス ファーマシューティカル インコーポレイテッド |
組換えaavにおいてcapおよびrep配列の逆パッケージングを低減するための組成物および方法
|
CN115667298A
(zh)
|
2020-03-27 |
2023-01-31 |
Ucb生物制药有限责任公司 |
自主凸起结构域肽
|
EP4127170A1
(en)
|
2020-03-27 |
2023-02-08 |
University of Rochester |
Crispr-cas13 crrna arrays
|
WO2021195519A1
(en)
|
2020-03-27 |
2021-09-30 |
University Of Rochester |
Targeted destruction of viral rna by crispr-cas13
|
EP4127189A1
(en)
|
2020-03-31 |
2023-02-08 |
Ultragenyx Pharmaceutical Inc. |
Gene therapy for treating propionic acidemia
|
EP4132955A2
(en)
|
2020-04-10 |
2023-02-15 |
SOLA Biosciences LLC |
Compositions and methods for the treatment of protein aggregation disorders
|
TW202208626A
(zh)
|
2020-04-24 |
2022-03-01 |
美商生命編輯公司 |
Rna引導核酸酶及其活性片段與變體,以及使用方法
|
US20230190956A1
(en)
|
2020-04-24 |
2023-06-22 |
INSERM (Institut National de la Santé et de la Recherche Médicale) |
Methods for preventing induction of immune responses to the transduced cells expressing a transgene product after ocular gene therapy
|
EP4143215A2
(en)
|
2020-04-28 |
2023-03-08 |
SOLA Biosciences LLC |
Compositions and methods for the treatment of tdp-43 proteinopathies
|
CN115485291A
(zh)
|
2020-04-29 |
2022-12-16 |
百时美施贵宝公司 |
具血影蛋白融合结构域的微型化抗肌萎缩蛋白及其用途
|
US20230304034A1
(en)
|
2020-05-12 |
2023-09-28 |
The Trustees Of The University Of Pennsylvania |
Compositions for drg-specific reduction of transgene expression
|
CA3183171A1
(en)
|
2020-05-13 |
2021-11-18 |
Akouos, Inc. |
Compositions and methods for treating slc26a4-associated hearing loss
|
EP4165194A2
(en)
|
2020-05-13 |
2023-04-19 |
Akouos, Inc. |
Compositions and methods for treating kcnq4-associated hearing loss
|
WO2021236908A2
(en)
|
2020-05-20 |
2021-11-25 |
Biomarin Pharmaceutical Inc. |
Use of regulatory proteins for the production of adeno-associated virus
|
CA3183251A1
(en)
|
2020-06-05 |
2021-12-09 |
Sola Biosciences Llc |
Compositions and methods for the treatment of synucleinopathies
|
CN116348149A
(zh)
|
2020-06-15 |
2023-06-27 |
全国儿童医院研究所 |
用于肌营养不良症的腺相关病毒载体递送
|
US20230220069A1
(en)
|
2020-06-17 |
2023-07-13 |
The Trustees Of The University Of Pennsylvania |
Compositions and methods for treatment of gene therapy patients
|
CN115996758A
(zh)
|
2020-06-19 |
2023-04-21 |
吉尼松公司 |
使sgcg在肌肉和心脏中充分表达的基因治疗表达系统
|
US20240018524A1
(en)
|
2020-07-10 |
2024-01-18 |
INSERM (Institut National de la Santé et de la Recherche Médicale) |
Methods and compositions for treating epilepsy
|
BR112023000305A2
(pt)
|
2020-07-10 |
2023-03-21 |
Genethon |
Promotor, sistema de expressão e composição farmacêutica
|
US20230270884A1
(en)
|
2020-07-13 |
2023-08-31 |
The Trustees Of The University Of Pennsylvania |
Compositions useful for treatment of charcot-marie-tooth disease
|
JP2023534464A
(ja)
|
2020-07-15 |
2023-08-09 |
ユニバーシティ オブ ロチェスター |
dCas13-RNase融合タンパク質による標的RNAの切断
|
AU2021319208A1
(en)
|
2020-07-30 |
2023-03-02 |
Shape Therapeutics Inc. |
Stable cell lines for inducible production of rAAV virions
|
JP2023537625A
(ja)
|
2020-08-14 |
2023-09-04 |
ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア |
新規aavカプシド及びそれを含む組成物
|
CA3190399A1
(en)
|
2020-08-24 |
2022-03-03 |
James M. Wilson |
Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
|
WO2022060915A1
(en)
|
2020-09-15 |
2022-03-24 |
Regenxbio Inc. |
Vectorized lanadelumab and administration thereof
|
EP4214317A2
(en)
|
2020-09-15 |
2023-07-26 |
Research Institute at Nationwide Children's Hospital |
Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy
|
EP4214242A1
(en)
|
2020-09-15 |
2023-07-26 |
RegenxBio Inc. |
Vectorized antibodies for anti-viral therapy
|
US20230383278A1
(en)
|
2020-09-18 |
2023-11-30 |
The United States Of America,As Represented By The Secretary,Department Of Health And Human Services |
Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency
|
JP2023543029A
(ja)
|
2020-09-28 |
2023-10-12 |
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 |
筋ジストロフィーの治療のための製品及び方法
|
WO2022073041A2
(en)
|
2020-09-30 |
2022-04-07 |
NGGT, Inc. |
Dual functional expression vectors and methods of use thereof
|
AU2021358048A1
(en)
|
2020-10-07 |
2023-05-25 |
Regenxbio Inc. |
Formulations for suprachoroidal administration such as high viscosity formulations
|
WO2022076750A2
(en)
|
2020-10-07 |
2022-04-14 |
Regenxbio Inc. |
Recombinant adeno-associated viruses for cns or muscle delivery
|
WO2022076591A1
(en)
|
2020-10-07 |
2022-04-14 |
Regenxbio Inc. |
Formulations for suprachoroidal administration such as formulations with aggregate formation
|
IL301643A
(en)
|
2020-10-07 |
2023-05-01 |
Regenxbio Inc |
Gene therapy for ocular manifestations of CLN2 disease
|
TW202228646A
(zh)
|
2020-10-07 |
2022-08-01 |
美商銳進科斯生物股份有限公司 |
諸如凝膠調配物之用於脈絡膜上投與之調配物
|
EP4225777A2
(en)
|
2020-10-07 |
2023-08-16 |
RegenxBio Inc. |
Adeno-associated viruses for ocular delivery of gene therapy
|
US11781156B2
(en)
|
2020-10-09 |
2023-10-10 |
Tenaya Therapeutics, Inc. |
Plakophillin-2 gene therapy methods and compositions
|
WO2022076803A1
(en)
|
2020-10-09 |
2022-04-14 |
The Trustees Of The University Of Pennsylvania |
Compositions and methods for treatment of fabry disease
|
WO2022094157A1
(en)
|
2020-10-28 |
2022-05-05 |
Regenxbio Inc. |
Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
|
CN116528892A
(zh)
|
2020-10-28 |
2023-08-01 |
再生生物股份有限公司 |
用于眼部适应症的载体化抗TNF-α抗体
|
IL302282A
(en)
|
2020-10-29 |
2023-06-01 |
Regenxbio Inc |
Vectored TNF-alpha antagonists for ocular indications
|
EP4236974A2
(en)
|
2020-10-29 |
2023-09-06 |
RegenxBio Inc. |
Vectorized factor xii antibodies and administration thereof
|
EP4236975A1
(en)
|
2020-10-29 |
2023-09-06 |
The Trustees of The University of Pennsylvania |
Aav capsids and compositions containing same
|
AU2021372262A1
(en)
|
2020-11-02 |
2023-06-01 |
Biomarin Pharmaceutical Inc. |
Process for enriching adeno-associated virus
|
WO2022115745A1
(en)
|
2020-11-30 |
2022-06-02 |
Research Institute At Nationwide Children's Hospital |
Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)
|
MX2023006445A
(es)
|
2020-12-01 |
2023-08-10 |
Univ Pennsylvania |
Composiciones y usos de estas para el tratamiento del síndrome de angelman.
|
PE20240115A1
(es)
|
2020-12-01 |
2024-01-22 |
Akouos Inc |
Construcciones de anticuerpos anti-vegf y metodos relacionados para el tratamiento de los sintomas asociados al schwannoma vestibular
|
AR124216A1
(es)
|
2020-12-01 |
2023-03-01 |
Univ Pennsylvania |
Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen
|
WO2022122733A1
(en)
|
2020-12-08 |
2022-06-16 |
Genethon |
New gene therapy for the treatment of duchenne muscular dystrophy
|
AU2021403076A1
(en)
|
2020-12-16 |
2023-06-29 |
Regenxbio Inc. |
Method of producing a recombinant adeno-associated virus particle
|
EP4271419A1
(en)
|
2020-12-29 |
2023-11-08 |
Akouos, Inc. |
Compositions and methods for treating clrn1-associated hearing loss and/or vision loss
|
TW202241943A
(zh)
|
2020-12-29 |
2022-11-01 |
美商銳進科斯生物股份有限公司 |
Tau特異性抗體基因療法組合物、方法及其用途
|
AU2022206197A1
(en)
|
2021-01-05 |
2023-07-13 |
Selecta Biosciences, Inc. |
Viral vector dosing protocols
|
CA3205209A1
(en)
|
2021-01-21 |
2022-07-28 |
Regenxbio Inc. |
Improved production of recombinant polypeptides and viruses
|
CA3209471A1
(en)
|
2021-01-27 |
2022-08-04 |
Research Institute At Nationwide Children's Hospital |
Materials and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
|
US20240091380A1
(en)
|
2021-02-01 |
2024-03-21 |
Regenxbio Inc. |
Gene therapy for neuronal ceroid lipofuscinoses
|
CA3210662A1
(en)
|
2021-02-03 |
2022-08-11 |
Research Institute At Nationwide Children's Hospital |
Compositions and methods for treating disease associated with dux4 overexpression
|
WO2022173605A2
(en)
|
2021-02-10 |
2022-08-18 |
Regenxbio Inc. |
Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
|
BR112023016983A2
(pt)
|
2021-02-26 |
2023-11-07 |
Takeda Pharmaceuticals Co |
Vetor de vírus adeno-associado recombinante, método para tratar doença de fabry, composição farmacêutica, célula, e, método de expressão da enzima ¿-gal em uma célula
|
EP4301462A1
(en)
|
2021-03-04 |
2024-01-10 |
Research Institute at Nationwide Children's Hospital |
Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications
|
BR112023018430A2
(pt)
|
2021-03-19 |
2023-11-28 |
Adrenas Therapeutics Inc |
Terapias gênicas para deficiência de 21-hidroxilase
|
EP4323520A1
(en)
|
2021-04-12 |
2024-02-21 |
The Trustees of The University of Pennsylvania |
Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
|
WO2022221424A1
(en)
|
2021-04-13 |
2022-10-20 |
Research Institute At Nationwide Children's Hospital |
Recombinant adeno-associated virus encoding methyl-cpg binding protein 2 for treating pitt hopkins syndrome via intrathecal delivery
|
KR20240000580A
(ko)
|
2021-04-23 |
2024-01-02 |
유니버시티 오브 로체스터 |
레트로바이러스 인테그라제-Cas 융합 단백질을 이용한 직접 비상동 DNA 삽입에 의한 게놈 편집 및 치료 방법
|
WO2022226263A1
(en)
|
2021-04-23 |
2022-10-27 |
The Trustees Of The University Of Pennsylvania |
Novel compositions with brain-specific targeting motifs and compositions containing same
|
WO2022226334A1
(en)
|
2021-04-23 |
2022-10-27 |
Research Institute At Nationwide Children's Hospital |
Products and methods for treating muscular dystrophy
|
EP4329821A1
(en)
|
2021-04-26 |
2024-03-06 |
RegenxBio Inc. |
Microdystrophin gene therapy administration for treatment of dystrophinopathies
|
AR125467A1
(es)
|
2021-04-27 |
2023-07-19 |
Univ Pennsylvania |
Cápsides de virus adenoasociados derivados de porcinos y usos de los estos
|
EP4330270A2
(en)
|
2021-04-30 |
2024-03-06 |
Takeda Pharmaceutical Company Limited |
Aav8 capsid variants with enhanced liver targeting
|
WO2022229703A2
(en)
|
2021-04-30 |
2022-11-03 |
Takeda Pharmaceutical Company, Ltd. |
New aav8 based immune escaping variants
|
WO2022235614A2
(en)
|
2021-05-04 |
2022-11-10 |
Regenxbio Inc. |
Novel aav vectors and methods and uses thereof
|
WO2022241030A1
(en)
|
2021-05-11 |
2022-11-17 |
Regenxbio Inc. |
Treatment of duchenne muscular dystrophy and combinations thereof
|
CA3218350A1
(en)
|
2021-05-17 |
2022-11-24 |
Sarepta Therapeutics, Inc. |
Production of recombinant aav vectors for treating muscular dystrophy
|
EP4108263A3
(en)
|
2021-06-02 |
2023-03-22 |
Research Institute at Nationwide Children's Hospital |
Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
|
WO2022272297A1
(en)
*
|
2021-06-25 |
2022-12-29 |
Oxford Biomedica Solutions Llc |
Adeno-associated virus packaging systems
|
EP4366757A1
(en)
|
2021-07-07 |
2024-05-15 |
INSERM (Institut National de la Santé et de la Recherche Médicale) |
Synergistic combination of rdcfv2 and rdcvf2l for the treatment of tauopathies
|
KR20240032971A
(ko)
|
2021-07-08 |
2024-03-12 |
테나야 테라퓨틱스, 인코포레이티드 |
유전자 요법을 위한 최적화된 발현 카세트
|
WO2023004331A1
(en)
|
2021-07-19 |
2023-01-26 |
New York University |
Auf1 combination therapies for treatment of muscle degenerative disease
|
CA3228365A1
(en)
|
2021-08-11 |
2023-02-16 |
Solid Biosciences Inc. |
Treatment of muscular dystrophy
|
AR126839A1
(es)
*
|
2021-08-20 |
2023-11-22 |
Llc «Anabion» |
Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
|
AR126840A1
(es)
*
|
2021-08-20 |
2023-11-22 |
Llc «Anabion» |
Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en esta
|
WO2023056329A1
(en)
|
2021-09-30 |
2023-04-06 |
Akouos, Inc. |
Compositions and methods for treating kcnq4-associated hearing loss
|
WO2023056399A1
(en)
|
2021-10-02 |
2023-04-06 |
The Trustees Of The University Of Pennsylvania |
Novel aav capsids and compositions containing same
|
WO2023060113A1
(en)
|
2021-10-05 |
2023-04-13 |
Regenxbio Inc. |
Compositions and methods for recombinant aav production
|
WO2023060269A1
(en)
|
2021-10-07 |
2023-04-13 |
Regenxbio Inc. |
Recombinant adeno-associated viruses for targeted delivery
|
AU2022360382A1
(en)
|
2021-10-07 |
2024-05-09 |
Research Institute At Nationwide Children's Hospital |
Products and methods for myelin protein zero silencing and treating cmt1b disease
|
WO2023060272A2
(en)
|
2021-10-07 |
2023-04-13 |
Regenxbio Inc. |
Recombinant adeno-associated viruses for cns tropic delivery
|
WO2023060248A1
(en)
|
2021-10-08 |
2023-04-13 |
Sola Biosciences Llc |
Compositions and methods for the treatment of p53-mediated cancers
|
WO2023060221A2
(en)
|
2021-10-08 |
2023-04-13 |
Sola Biosciences Llc |
Compositions and methods for the treatment of proteopathies
|
CA3235269A1
(en)
|
2021-10-12 |
2023-04-20 |
Cartesian Therapeutics, Inc. |
Viral vector dosing protocols
|
TW202333776A
(zh)
|
2021-10-12 |
2023-09-01 |
美商阿斯帕醫療公司 |
用於治療腦白質失養症的方法及組合物
|
WO2023064367A1
(en)
|
2021-10-12 |
2023-04-20 |
Selecta Biosciences, Inc. |
Methods and compositions for attenuating anti-viral transfer vector igm responses
|
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
|
WO2023077092A1
(en)
|
2021-10-28 |
2023-05-04 |
Regenxbio Inc. |
Engineered nucleic acid regulatory elements and methods and uses thereof
|
WO2023086615A1
(en)
|
2021-11-14 |
2023-05-19 |
Selecta Biosciences, Inc. |
Multiple dosing with viral vectors
|
WO2023087019A2
(en)
|
2021-11-15 |
2023-05-19 |
The Trustees Of The University Of Pennsylvania |
Compositions for drg-specific reduction of transgene expression
|
JP2023081369A
(ja)
|
2021-11-30 |
2023-06-09 |
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
|
WO2023102517A1
(en)
|
2021-12-02 |
2023-06-08 |
The Trustees Of The University Of Pennsylvania |
Compositions and methods for treatment of fabry disease
|
WO2023122669A1
(en)
|
2021-12-21 |
2023-06-29 |
Research Institute At Nationwide Children's Hospital |
Materials and methods for the treatment of limb girdle muscular dystrophy
|
AR128239A1
(es)
|
2022-01-10 |
2024-04-10 |
Univ Pennsylvania |
Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
|
EP4215614A1
(en)
|
2022-01-24 |
2023-07-26 |
Dynacure |
Combination therapy for dystrophin-related diseases
|
WO2023139557A1
(en)
|
2022-01-24 |
2023-07-27 |
LifeEDIT Therapeutics, Inc. |
Rna-guided nucleases and active fragments and variants thereof and methods of use
|
WO2023150142A1
(en)
|
2022-02-02 |
2023-08-10 |
Akouos, Inc. |
Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
|
WO2023168400A2
(en)
|
2022-03-03 |
2023-09-07 |
Research Institute At Nationwide Children's Hospital |
Materials and methods for the treatment of eif2b5 mutations and diseases resulting therefrom
|
US20230357437A1
(en)
|
2022-03-09 |
2023-11-09 |
Selecta Biosciences, Inc. |
Immunosuppressants in combination with anti-igm agents and related dosing
|
TW202346590A
(zh)
|
2022-03-13 |
2023-12-01 |
美商銳進科斯生物股份有限公司 |
經修飾之肌肉特異性啟動子
|
WO2023178220A1
(en)
|
2022-03-16 |
2023-09-21 |
Regenxbio Inc. |
Compositions and methods for recombinant aav production
|
WO2023183623A1
(en)
|
2022-03-25 |
2023-09-28 |
Regenxbio Inc. |
Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
|
WO2023182476A1
(ja)
*
|
2022-03-25 |
2023-09-28 |
学校法人自治医科大学 |
アデノ随伴ウイルス(aav)ベクターの効率の良い産生システム
|
WO2023187728A1
(en)
|
2022-04-01 |
2023-10-05 |
Takeda Pharmaceutical Company Limited |
Gene therapy for diseases with cns manifestations
|
TW202345913A
(zh)
|
2022-04-06 |
2023-12-01 |
美商銳進科斯生物股份有限公司 |
用於脈絡膜上投與之調配物諸如凝膠調配物
|
TW202404651A
(zh)
|
2022-04-06 |
2024-02-01 |
美商銳進科斯生物股份有限公司 |
用於脈絡膜上投與之調配物諸如形成聚集體之調配物
|
WO2023196873A1
(en)
|
2022-04-06 |
2023-10-12 |
Regenxbio Inc. |
Pharmaceutical composition comprising a recombinant adeno-associated virus vector with an expression cassette encoding a transgene forsuprachoidal administration
|
WO2023196892A1
(en)
|
2022-04-06 |
2023-10-12 |
The Trustees Of The University Of Pennsylvania |
Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
|
WO2023201207A1
(en)
|
2022-04-11 |
2023-10-19 |
Tenaya Therapeutics, Inc. |
Adeno-associated virus with engineered capsid
|
WO2023201277A1
(en)
|
2022-04-14 |
2023-10-19 |
Regenxbio Inc. |
Recombinant adeno-associated viruses for cns tropic delivery
|
WO2023201308A1
(en)
|
2022-04-14 |
2023-10-19 |
Regenxbio Inc. |
Gene therapy for treating an ocular disease
|
WO2023205610A2
(en)
|
2022-04-18 |
2023-10-26 |
Regenxbio Inc. |
Hybrid aav capsids
|
WO2023215807A1
(en)
|
2022-05-03 |
2023-11-09 |
Regenxbio Inc. |
VECTORIZED ANTI-TNF-α INHIBITORS FOR OCULAR INDICATIONS
|
WO2023215806A2
(en)
|
2022-05-03 |
2023-11-09 |
Regenxbio Inc. |
Vectorized anti-complement antibodies and complement agents and administration thereof
|
WO2023214346A1
(en)
|
2022-05-06 |
2023-11-09 |
Novartis Ag |
Novel recombinant aav vp2 fusion polypeptides
|
WO2023239627A2
(en)
|
2022-06-08 |
2023-12-14 |
Regenxbio Inc. |
Methods for recombinant aav production
|
WO2023240177A1
(en)
|
2022-06-08 |
2023-12-14 |
Research Instiitute At Nationwide Children's Hospital |
Products and methods for treating diseases or conditions associated with mutant or pathogenic kcnq3 expression
|
WO2024015966A2
(en)
|
2022-07-15 |
2024-01-18 |
The Trustees Of The University Of Pennsylvania |
Recombinant aav having aav clade d and clade e capsids and compositions containing same
|
WO2024017990A1
(en)
|
2022-07-21 |
2024-01-25 |
Institut National de la Santé et de la Recherche Médicale |
Methods and compositions for treating chronic pain disorders
|
WO2024036324A1
(en)
|
2022-08-11 |
2024-02-15 |
Selecta Biosciences, Inc. |
Compositions and methods related to immunoglobulin proteases and fusions thereof
|
WO2024033901A1
(en)
|
2022-08-12 |
2024-02-15 |
LifeEDIT Therapeutics, Inc. |
Rna-guided nucleases and active fragments and variants thereof and methods of use
|
WO2024044725A2
(en)
|
2022-08-24 |
2024-02-29 |
Regenxbio Inc. |
Recombinant adeno-associated viruses and uses thereof
|
WO2024042485A1
(en)
|
2022-08-25 |
2024-02-29 |
Takeda Pharmaceutical Company Limited |
Composition for use in the treatment of fabry disease
|
WO2024064863A2
(en)
|
2022-09-22 |
2024-03-28 |
Biomarin Pharmaceutical Inc. |
Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors
|
WO2024064856A1
(en)
|
2022-09-22 |
2024-03-28 |
Biomarin Pharmaceutical Inc. |
Treatment of cardiomyopathy with aav gene therapy vectors
|
WO2024064913A1
(en)
|
2022-09-23 |
2024-03-28 |
Sarepta Therapeutics, Inc. |
Recombinant aav vectors for treating muscular dystrophy
|
WO2024073669A1
(en)
|
2022-09-30 |
2024-04-04 |
Regenxbio Inc. |
Treatment of ocular diseases with recombinant viral vectors encoding anti-vegf fab
|
WO2024081706A1
(en)
|
2022-10-11 |
2024-04-18 |
Research Institute At Nationwide Children's Hospital |
Adeno-associated virus delivery to treat spinal muscular atrophy with respiratory distress type 1 (smard1) and charcot-marie-tooth type 2s (cmt2s)
|
WO2024081746A2
(en)
|
2022-10-11 |
2024-04-18 |
Regenxbio Inc. |
Engineered nucleic acid regulatory elements and methods and uses thereof
|