US6924128B2
(en)
|
1994-12-06 |
2005-08-02 |
Targeted Genetics Corporation |
Packaging cell lines for generation of high titers of recombinant AAV vectors
|
PT1944362E
(pt)
*
|
1997-09-05 |
2016-01-27 |
Genzyme Corp |
Métodos de produção de preparações de alto título de vetores aav recombinantes desprovidos de adjuvantes
|
US6989264B2
(en)
|
1997-09-05 |
2006-01-24 |
Targeted Genetics Corporation |
Methods for generating high titer helper-free preparations of released recombinant AAV vectors
|
US6566118B1
(en)
|
1997-09-05 |
2003-05-20 |
Targeted Genetics Corporation |
Methods for generating high titer helper-free preparations of released recombinant AAV vectors
|
US6346415B1
(en)
|
1997-10-21 |
2002-02-12 |
Targeted Genetics Corporation |
Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
|
EP2942393A1
(en)
|
1998-09-04 |
2015-11-11 |
Genzyme Corporation |
Methods for generating high titer helper-free preparations of released recombinant aav vectors
|
CA2342849C
(en)
*
|
1998-09-04 |
2013-01-29 |
Targeted Genetics Corporation |
Methods for generating high titer helper-free preparations of released recombinant aav vectors
|
US6893865B1
(en)
|
1999-04-28 |
2005-05-17 |
Targeted Genetics Corporation |
Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
|
JP2003501043A
(ja)
|
1999-05-28 |
2003-01-14 |
ターゲティッド ジェネティクス コーポレイション |
腫瘍壊死因子(tnf)関連障害において、tnfのレベルを低下させるための方法および組成物
|
EP1939300A1
(en)
|
1999-05-28 |
2008-07-02 |
Targeted Genetics Corporation |
Methods and compositions for lowering the level of tumor necrosis factor (TNF) in TNF-associated disorders
|
HUP0202295A3
(en)
|
1999-07-23 |
2005-01-28 |
Genentech Inc |
Method for rnase- and organic solvent-free plasmid dna purification using tangential flow filtration
|
EP1916258B1
(en)
|
1999-08-09 |
2014-04-23 |
Targeted Genetics Corporation |
Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
|
JP4827353B2
(ja)
|
1999-08-09 |
2011-11-30 |
ターゲティッド ジェネティクス コーポレイション |
鎖内塩基対を形成するような配列の設計による、組換えウイルスベクターからの一本鎖の異種ヌクレオチド配列の発現の増大
|
US7115391B1
(en)
|
1999-10-01 |
2006-10-03 |
Genovo, Inc. |
Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
|
CA2799545A1
(en)
*
|
2000-03-07 |
2001-09-13 |
Merck Sharp & Dohme Corp. |
Adenovirus formulations
|
FR2808804B1
(fr)
|
2000-05-09 |
2002-08-02 |
Nautilus Biotech |
Procede de determination du titre d'agents biologiques en temps reel dans des cellules cibles vivantes et ses applications
|
US6593123B1
(en)
*
|
2000-08-07 |
2003-07-15 |
Avigen, Inc. |
Large-scale recombinant adeno-associated virus (rAAV) production and purification
|
US6573092B1
(en)
|
2000-10-10 |
2003-06-03 |
Genvec, Inc. |
Method of preparing a eukaryotic viral vector
|
US7647184B2
(en)
|
2001-08-27 |
2010-01-12 |
Hanall Pharmaceuticals, Co. Ltd |
High throughput directed evolution by rational mutagenesis
|
AU2003263552A1
(en)
|
2002-09-09 |
2004-03-29 |
Nautilus Biotech |
Rational evolution of cytokines for higher stability, the cytokines and encoding nucleic acid molecules
|
JP4559429B2
(ja)
|
2003-05-21 |
2010-10-06 |
ジェンザイム・コーポレーション |
空キャプシドを実質的に含まない組換えaavビリオン調製物を生成するための方法
|
ES2400235T3
(es)
|
2006-04-28 |
2013-04-08 |
The Trustees Of The University Of Pennsylvania |
Método de producción escalable de AAV
|
CA2693178C
(en)
|
2006-11-29 |
2018-12-04 |
Nationwide Children's Hospital, Inc. |
Myostatin inhibition for enhancing muscle and/or improving muscle function
|
US9415121B2
(en)
|
2008-12-19 |
2016-08-16 |
Nationwide Children's Hospital |
Delivery of MECP2 polynucleotide using recombinant AAV9
|
US11219696B2
(en)
|
2008-12-19 |
2022-01-11 |
Nationwide Children's Hospital |
Delivery of polynucleotides using recombinant AAV9
|
ES2903127T3
(es)
|
2009-05-02 |
2022-03-31 |
Genzyme Corp |
Terapia génica para trastornos neurodegenerativos
|
ES2693194T3
(es)
|
2009-06-16 |
2018-12-10 |
Genzyme Corporation |
Métodos mejorados para la purificación de vectores de AAV recombinantes
|
CN103476456B
(zh)
|
2010-11-05 |
2017-10-03 |
斯坦福大学托管董事会 |
奖赏相关行为的光遗传学控制
|
WO2012061679A2
(en)
|
2010-11-05 |
2012-05-10 |
The Board Of Trustees Of The Leland Stanford Junior University |
Light-activated chimeric opsins and methods of using the same
|
EP2643045A4
(en)
|
2010-11-23 |
2016-01-13 |
Presage Biosciences Inc |
THERAPEUTIC PROCESSES AND COMPOSITIONS FOR SOLID FORM DELIVERY
|
WO2012142526A1
(en)
|
2011-04-14 |
2012-10-18 |
Modiano Jaime |
Use of tumor fas expression to determine response to anti-cancer therapy
|
EP2700399B1
(en)
|
2011-04-18 |
2017-05-31 |
National Center of Neurology and Psychiatry |
Drug delivery particles and method for producing same
|
WO2012145509A2
(en)
|
2011-04-19 |
2012-10-26 |
The Research Foundation Of State University Of New York |
Adeno-associated-virus rep sequences, vectors, and viruses
|
US10196636B2
(en)
|
2011-04-21 |
2019-02-05 |
Nationwide Children's Hospital, Inc. |
Recombinant virus products and methods for inhibition of expression of myotilin
|
WO2012145597A2
(en)
|
2011-04-21 |
2012-10-26 |
Nationwide Children's Hospital, Inc. |
Recombinant virus products and methods for inhibition of expression of myotilin
|
US20130039888A1
(en)
|
2011-06-08 |
2013-02-14 |
Nationwide Children's Hospital Inc. |
Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
|
WO2013016352A1
(en)
|
2011-07-25 |
2013-01-31 |
Nationwide Children's Hospital, Inc. |
Recombinant virus products and methods for inhibition of expression of dux4
|
US9434928B2
(en)
|
2011-11-23 |
2016-09-06 |
Nationwide Children's Hospital, Inc. |
Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
|
CN104093833B
(zh)
|
2011-12-16 |
2017-11-07 |
斯坦福大学托管董事会 |
视蛋白多肽及其使用方法
|
ES2628006T3
(es)
|
2012-02-21 |
2017-08-01 |
Circuit Therapeutics, Inc. |
Composiciones para el tratamiento de trastornos neurogénicos del suelo pélvico
|
DK3415167T3
(da)
|
2012-08-01 |
2024-09-02 |
Nationwide Childrens Hospital |
Intratekal administration af rekombinant adenoassocieret virus 9
|
EP2895606A4
(en)
|
2012-09-17 |
2016-07-06 |
Res Inst Nationwide Childrens Hospital |
COMPOSITIONS AND METHOD FOR THE TREATMENT OF AMYOTROPHER LATERAL SCLEROSIS (ALS)
|
WO2014081449A1
(en)
|
2012-11-21 |
2014-05-30 |
Circuit Therapeutics, Inc. |
System and method for optogenetic therapy
|
EP2968997B1
(en)
|
2013-03-15 |
2019-06-26 |
The Board of Trustees of the Leland Stanford Junior University |
Optogenetic control of behavioral state
|
CN105324392A
(zh)
|
2013-04-20 |
2016-02-10 |
全国儿童医院研究所 |
外显子2靶向U7snRNA多核苷酸构建体的重组腺相关病毒递送
|
AU2014260101B2
(en)
|
2013-04-29 |
2018-07-26 |
Humboldt-Universitat Zu Berlin |
Devices, systems and methods for optogenetic modulation of action potentials in target cells
|
CA2917490A1
(en)
|
2013-07-09 |
2015-01-15 |
Takeda Pharmaceutical Company Limited |
Heterocyclic compound
|
US10307609B2
(en)
|
2013-08-14 |
2019-06-04 |
The Board Of Trustees Of The Leland Stanford Junior University |
Compositions and methods for controlling pain
|
RS60434B1
(sr)
|
2013-08-27 |
2020-07-31 |
Res Institute At Nationwide Children's Hospital |
Proizvodi i postupci za tretman amiotrofične lateralne skleroze
|
CA2923833C
(en)
*
|
2013-10-22 |
2020-02-25 |
F. Hoffmann-La Roche Ag |
Methods for measuring cell-free virus particles from dried blood spots
|
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
|
US10047130B2
(en)
|
2014-03-18 |
2018-08-14 |
Washington University |
Methods and compositions for red-shifted chromophore substitution for optogenetic applications
|
US10052383B2
(en)
|
2014-03-28 |
2018-08-21 |
The Board Of Trustees Of The Leland Stanford Junior University |
Engineered light-activated anion channel proteins and methods of use 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
|
US10842886B2
(en)
|
2014-10-10 |
2020-11-24 |
Research Institute At Nationwide Children's Hospital |
Guided injections for AAV gene transfer to muscle
|
EP3690024A1
(en)
|
2014-11-05 |
2020-08-05 |
The Research Institute at Nationwide Children's Hospital |
Methods and materials for producing recombinant viruses in eukaryotic microalgae
|
MA41451A
(fr)
|
2015-02-04 |
2017-12-12 |
Univ Washington |
Constructions anti-tau
|
EP3262172A2
(en)
|
2015-02-23 |
2018-01-03 |
Crispr Therapeutics AG |
Materials and methods for treatment of hemoglobinopathies
|
DK3313987T3
(da)
*
|
2015-06-23 |
2020-02-03 |
Deutsches Krebsforsch |
Fremgangsmåde til produktion i stor målestok samt oprensning af parvovirus
|
US10017832B2
(en)
|
2015-08-25 |
2018-07-10 |
Washington University |
Compositions and methods for site specific recombination at asymmetric sites
|
CA2998636A1
(en)
|
2015-09-17 |
2017-03-23 |
Research Institute At Nationwide Children's Hospital |
Methods and materials for galgt2 gene therapy
|
EP3353297A1
(en)
|
2015-09-24 |
2018-08-01 |
Crispr Therapeutics AG |
Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
|
US11369692B2
(en)
|
2015-10-28 |
2022-06-28 |
Vertex Pharmaceuticals Incorporated |
Materials and methods for treatment of Duchenne Muscular Dystrophy
|
GB201519303D0
(en)
*
|
2015-11-02 |
2015-12-16 |
Imp Innovations Ltd |
Phagemid vector
|
BR112018008971A2
(pt)
|
2015-11-06 |
2018-11-27 |
Crispr Therapeutics Ag |
materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a
|
CN108463211B
(zh)
|
2015-11-16 |
2022-09-27 |
全国儿童医院研究所 |
用于治疗肌联蛋白类肌病和其它肌联蛋白病变的材料和方法
|
AU2016364667A1
(en)
|
2015-12-01 |
2018-06-21 |
Crispr Therapeutics Ag |
Materials and methods for treatment of Alpha-1 antitrypsin deficiency
|
CA3006309A1
(en)
*
|
2015-12-01 |
2017-06-08 |
Spark Therapeutics, Inc. |
Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use
|
ES2918998T3
(es)
|
2015-12-11 |
2022-07-21 |
Univ Pennsylvania |
Método de purificación escalable para AAVrh10
|
WO2017160360A2
(en)
|
2015-12-11 |
2017-09-21 |
The Trustees Of The University Of Pennsylvania |
Scalable purification method for aav9
|
US11015173B2
(en)
|
2015-12-11 |
2021-05-25 |
The Trustees Of The University Of Pennsylvania |
Scalable purification method for AAV1
|
EP3387117B1
(en)
|
2015-12-11 |
2022-11-23 |
The Trustees Of The University Of Pennsylvania |
Scalable purification method for aav8
|
EP3394260B1
(en)
|
2015-12-23 |
2021-02-17 |
CRISPR Therapeutics AG |
Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
|
US11938193B2
(en)
|
2016-01-08 |
2024-03-26 |
Washington University |
Compositions comprising chemerin and methods of use thereof
|
US20190038771A1
(en)
|
2016-02-02 |
2019-02-07 |
Crispr Therapeutics Ag |
Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
|
US20190112353A1
(en)
|
2016-02-18 |
2019-04-18 |
Crispr Therapeutics Ag |
Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
|
WO2017147509A1
(en)
|
2016-02-25 |
2017-08-31 |
Marco Colonna |
Compositions comprising trem2 and methods of use thereof
|
EP3420109A4
(en)
|
2016-02-26 |
2019-11-06 |
Research Institute at Nationwide Children's Hospital |
RECOMBINANT VIRUS PRODUCTS AND METHOD OF INDUCING DUX4 EXON SKIPPING
|
EP3429632B1
(en)
|
2016-03-16 |
2023-01-04 |
CRISPR Therapeutics AG |
Materials and methods for treatment of hereditary haemochromatosis
|
AU2017240721B2
(en)
*
|
2016-03-31 |
2023-10-12 |
Spark Therapeutics, Inc. |
Column-based fully scalable rAAV manufacturing process
|
RU2768289C2
(ru)
|
2016-04-02 |
2022-03-23 |
Рисерч Инститьют Эт Нэшнуайд Чилдрен'С Хоспитал |
Модифицированная промоторная система u6 для тканеспецифической экспрессии
|
DK3442600T5
(da)
|
2016-04-15 |
2024-09-16 |
Res Inst Nationwide Childrens Hospital |
ADENOASSOCIERET VIRUSVEKTORFREMFØRING AF ß-SARCOGLYCAN OG MIKRORNA-29 OG BEHANDLING AF MUSKELDYSTROFI
|
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
|
FI3445388T3
(fi)
|
2016-04-18 |
2024-05-15 |
Vertex Pharma |
Materiaaleja ja menetelmiä hemoglobinopatioiden hoitamiseksi
|
WO2017191503A1
(en)
|
2016-05-05 |
2017-11-09 |
Crispr Therapeutics Ag |
Materials and methods for treatment of hemoglobinopathies
|
US11427809B2
(en)
|
2016-05-25 |
2022-08-30 |
Lonza Houston, Inc. |
Methods for isolating adeno-associated virus using a polydialkylammonium salt
|
WO2018002762A1
(en)
|
2016-06-29 |
2018-01-04 |
Crispr Therapeutics Ag |
Materials and methods for treatment of amyotrophic lateral sclerosis (als) and other related disorders
|
WO2018002812A1
(en)
|
2016-06-29 |
2018-01-04 |
Crispr Therapeutics Ag |
Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders
|
EP3478828B1
(en)
|
2016-06-29 |
2024-09-04 |
CRISPR Therapeutics AG |
Materials and methods for treatment of friedreich ataxia and other related disorders
|
CN119074961A
(zh)
|
2016-07-06 |
2024-12-06 |
沃泰克斯药物股份有限公司 |
用于治疗疼痛相关病症的材料和方法
|
CA3029141A1
(en)
|
2016-07-06 |
2018-01-11 |
Crispr Therapeutics Ag |
Materials and methods for treatment of pain related disorders
|
WO2018007871A1
(en)
|
2016-07-08 |
2018-01-11 |
Crispr Therapeutics Ag |
Materials and methods for treatment of transthyretin amyloidosis
|
EP3484493A4
(en)
*
|
2016-07-12 |
2019-12-25 |
Ultragenyx Pharmaceutical Inc. |
USE OF GLUCOCORTICOID ANALOGS TO INCREASE THE PERFORMANCE OF RECOMBINANT ADENO-ASSOCIATED VIRUSES
|
WO2018020323A2
(en)
|
2016-07-25 |
2018-02-01 |
Crispr Therapeutics Ag |
Materials and methods for treatment of fatty acid disorders
|
EP3541429A4
(en)
|
2016-11-17 |
2020-08-12 |
Nationwide Children's Hospital, Inc. |
INTRATHECAL ADMINISTRATION OF RECOMBINANT ADENO-ASSOCIATED VIRUS CODING FOR METHYL-CPG-BINDING PROTEIN 2
|
JP7206214B2
(ja)
|
2016-12-13 |
2023-01-17 |
シアトル チルドレンズ ホスピタル (ディービーエイ シアトル チルドレンズ リサーチ インスティテュート) |
インビトロ及びインビボで操作された細胞において発現された化学誘導シグナル伝達複合体の外因性薬物活性化の方法
|
WO2018154439A1
(en)
|
2017-02-22 |
2018-08-30 |
Crispr Therapeutics Ag |
Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders
|
WO2018154418A1
(en)
|
2017-02-22 |
2018-08-30 |
Crispr Therapeutics Ag |
Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
|
WO2018154459A1
(en)
|
2017-02-22 |
2018-08-30 |
Crispr Therapeutics Ag |
Materials and methods for treatment of primary hyperoxaluria type 1 (ph1) and other alanine-glyoxylate aminotransferase (agxt) gene related conditions or disorders
|
US20200216857A1
(en)
|
2017-02-22 |
2020-07-09 |
Crispr Therapeutics Ag |
Materials and methods for treatment of spinocerebellar ataxia type 2 (sca2) and other spinocerebellar ataxia type 2 protein (atxn2) gene related conditions or disorders
|
AU2018224387B2
(en)
|
2017-02-22 |
2024-08-08 |
Crispr Therapeutics Ag |
Compositions and methods for gene editing
|
ES2948233T3
(es)
|
2017-03-17 |
2023-09-06 |
Res Inst Nationwide Childrens Hospital |
Administración mediante vector de virus adenoasociado de microdistrofina específica de músculo para tratar la distrofia muscular
|
JP7162021B2
(ja)
|
2017-03-17 |
2022-10-27 |
ニューカッスル ユニバーシティ |
筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
|
CN110914289B
(zh)
|
2017-05-12 |
2024-05-14 |
克里斯珀医疗股份公司 |
用于工程化细胞的材料和方法及其在免疫肿瘤学中的用途
|
CN111032176A
(zh)
*
|
2017-06-30 |
2020-04-17 |
星火治疗有限公司 |
Aav载体柱纯化方法
|
CA3068906A1
(en)
|
2017-07-08 |
2019-01-17 |
Genethon |
Treatment of spinal muscular atrophy
|
WO2019070741A1
(en)
|
2017-10-02 |
2019-04-11 |
Research Institute At Nationwide Children's Hospital |
MIARN DECIBLATION SYSTEM FOR SPECIFIC INTERFERENCE OF A FABRIC
|
US12123027B2
(en)
|
2017-10-12 |
2024-10-22 |
Ascend Advanced Therapies Limited |
Life-cycle-defective adenovirus helper viruses, their production and use for producing rAAV
|
WO2019079527A1
(en)
|
2017-10-17 |
2019-04-25 |
Casebia Therapeutics Limited Liability Partnership |
COMPOSITIONS AND METHODS FOR GENETIC EDITION FOR HEMOPHILIA A
|
US11534501B2
(en)
|
2017-10-18 |
2022-12-27 |
Research Institute At Nationwide Children's Hospital |
Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
|
BR112020007790A2
(pt)
|
2017-10-20 |
2020-10-20 |
Research Institute At Nationwide Children's Hospital |
métodos e materiais para terapia genética com nt-3
|
MA50849A
(fr)
|
2017-10-26 |
2020-09-02 |
Vertex Pharma |
Substances et procédés pour le traitement d'hémoglobinopathies
|
CA3082136A1
(en)
|
2017-11-08 |
2019-05-16 |
Avexis, Inc. |
Means and method for preparing viral vectors and uses of same
|
MA50579A
(fr)
|
2017-11-09 |
2020-09-16 |
Crispr Therapeutics Ag |
Systèmes crispr/cas ou crispr/cpf1 à auto-inactivation (sin) et leurs utilisations
|
CN111727251B
(zh)
|
2017-11-21 |
2024-09-20 |
克里斯珀医疗股份公司 |
用于治疗常染色体显性色素性视网膜炎的材料和方法
|
CA3082907A1
(en)
|
2017-11-27 |
2019-05-31 |
Coda Biotherapeutics, Inc. |
Compositions and methods for neurological diseases
|
CA3084825A1
(en)
|
2017-12-14 |
2019-06-20 |
Crispr Therapeutics Ag |
Novel rna-programmable endonuclease systems and their use in genome editing and other applications
|
EP3728595A1
(en)
|
2017-12-21 |
2020-10-28 |
CRISPR Therapeutics AG |
Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
|
CA3084632A1
(en)
|
2017-12-21 |
2019-06-27 |
Crispr Therapeutics Ag |
Materials and methods for treatment of usher syndrome type 2a
|
EP3737762A1
(en)
|
2018-01-12 |
2020-11-18 |
CRISPR Therapeutics AG |
Compositions and methods for gene editing by targeting transferrin
|
WO2019150196A1
(en)
|
2018-02-05 |
2019-08-08 |
Crispr Therapeutics Ag |
Materials and methods for treatment of hemoglobinopathies
|
EP3749768A1
(en)
|
2018-02-05 |
2020-12-16 |
Vertex Pharmaceuticals Incorporated |
Materials and methods for treatment of hemoglobinopathies
|
US20210130824A1
(en)
|
2018-02-16 |
2021-05-06 |
Crispr Therapeutics Ag |
Compositions and methods for gene editing by targeting fibrinogen-alpha
|
JP7550648B2
(ja)
|
2018-03-19 |
2024-09-13 |
クリスパー セラピューティクス アーゲー |
新規rnaプログラム可能エンドヌクレアーゼ系およびその使用
|
WO2019204668A1
(en)
|
2018-04-18 |
2019-10-24 |
Casebia Therapeutics Limited Liability Partnership |
Compositions and methods for knockdown of apo(a) by gene editing for treatment of cardiovascular disease
|
SG11202007874XA
(en)
|
2018-04-27 |
2020-09-29 |
Seattle Childrens Hospital Dba Seattle Childrens Res Inst |
Rapamycin resistant cells
|
KR20210018919A
(ko)
|
2018-06-08 |
2021-02-18 |
노파르티스 아게 |
약물 산물 효능을 측정하기 위한 세포-기반 분석
|
GB201809588D0
(en)
|
2018-06-12 |
2018-07-25 |
Univ Bristol |
Materials and methods for modulating intraocular and intracranial pressure
|
SG11202012450QA
(en)
|
2018-06-18 |
2021-01-28 |
Res Inst Nationwide Childrens Hospital |
Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
|
US20240254181A1
(en)
|
2018-06-18 |
2024-08-01 |
Research Institute At Nationwide Children's Hospital |
Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
|
CA3105216A1
(en)
|
2018-06-29 |
2020-01-02 |
Research Institute At Nationwide Children's Hospital |
Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
|
AU2019328270A1
(en)
|
2018-08-29 |
2021-03-25 |
Research Institute At Nationwide Children's Hospital |
Products and methods for inhibition of expression of mutant GARS protein
|
CA3116885A1
(en)
|
2018-10-17 |
2020-04-23 |
Crispr Therapeutics Ag |
Compositions and methods for delivering transgenes
|
TWI844587B
(zh)
|
2018-11-30 |
2024-06-11 |
瑞士商諾華公司 |
Aav病毒載體及其用途
|
JP2022516010A
(ja)
|
2018-12-21 |
2022-02-24 |
ジェネトン |
遺伝子療法ベクターのための発現カセット
|
AU2019419494A1
(en)
|
2018-12-31 |
2021-07-15 |
Research Institute At Nationwide Children's Hospital |
DUX4 RNA silencing using RNA targeting CRISPR-Cas13b
|
WO2020163300A1
(en)
|
2019-02-04 |
2020-08-13 |
Research Institute At Nationwide Children's Hospital |
Adeno-associated virus delivery of cln3 polynucleotide
|
JP2022519597A
(ja)
|
2019-02-04 |
2022-03-24 |
リサーチ・インスティチュート・アット・ネーションワイド・チルドレンズ・ホスピタル |
Cln6ポリヌクレオチドのアデノ随伴ウイルス送達
|
EP3923992A1
(en)
|
2019-02-15 |
2021-12-22 |
CRISPR Therapeutics AG |
Gene editing for hemophilia a with improved factor viii expression
|
CN113766935A
(zh)
|
2019-02-26 |
2021-12-07 |
全国儿童医院研究所 |
β-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗
|
AU2020239225A1
(en)
|
2019-03-12 |
2021-09-30 |
Bayer Healthcare Llc |
Novel high fidelity RNA-programmable endonuclease systems and uses thereof
|
AU2020270960A1
(en)
*
|
2019-04-12 |
2021-11-04 |
Ultragenyx Pharmaceutical Inc. |
Engineered producer cell lines and methods of making and using the same
|
US20220193259A1
(en)
|
2019-04-15 |
2022-06-23 |
Sanford Research |
Gene therapy for treating or preventing visual effects in batten disease
|
US20210047649A1
(en)
|
2019-05-08 |
2021-02-18 |
Vertex Pharmaceuticals Incorporated |
Crispr/cas all-in-two vector systems for treatment of dmd
|
US20220226507A1
(en)
|
2019-05-17 |
2022-07-21 |
Research Institute At Nationwide Children's Hospital |
Optimized gene therapy targeting retinal cells
|
WO2020264254A1
(en)
|
2019-06-28 |
2020-12-30 |
Crispr Therapeutics Ag |
Materials and methods for controlling gene editing
|
JP2022541070A
(ja)
|
2019-07-25 |
2022-09-21 |
ノバルティス アーゲー |
調節可能な発現系
|
DK4017871T5
(da)
|
2019-08-21 |
2024-08-05 |
Res Inst Nationwide Childrens Hospital |
Adenoassocieret virusvektoradministration af alfa-sarcoglycan og behandling af muskeldystrofi
|
CA3151920A1
(en)
|
2019-08-21 |
2021-02-25 |
Coda Biotherapeutics, Inc. |
Compositions and methods for neurological diseases
|
JP2022552014A
(ja)
|
2019-10-18 |
2022-12-14 |
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 |
Irf2bpl遺伝子の変異に関連する障害の治療のための材料および方法
|
CA3158286A1
(en)
|
2019-10-18 |
2021-04-22 |
Research Institute At Nationwide Children's Hospital |
Gene therapy targeting cochlear cells
|
JP2023502474A
(ja)
|
2019-11-22 |
2023-01-24 |
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 |
Ighmbp2遺伝子に関連する障害の治療のための材料および方法
|
IL294072A
(en)
|
2019-12-20 |
2022-08-01 |
Res Inst Nationwide Childrens Hospital |
Optimized gene therapy for targeting muscle in muscle diseases
|
JP2023513932A
(ja)
|
2020-02-18 |
2023-04-04 |
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 |
X連鎖障害の治療におけるmiRNAのAAV媒介標的化
|
GB202004498D0
(en)
|
2020-03-27 |
2020-05-13 |
Ucl Business Ltd |
Activity-dependent gene therapy for neurological disorders
|
JP2023523573A
(ja)
|
2020-04-14 |
2023-06-06 |
ジェネトン |
酸性セラミダーゼ欠乏症を処置するためのベクター
|
US20230111672A1
(en)
*
|
2020-04-27 |
2023-04-13 |
The Regents Of The University Of California |
Compositions and methods for production of recombinant adeno-associated virus
|
TW202208630A
(zh)
|
2020-06-15 |
2022-03-01 |
美國全美兒童醫院之研究學會 |
針對肌肉營養不良症的腺相關病毒載體遞送
|
GB202010981D0
(en)
|
2020-07-16 |
2020-09-02 |
Ucl Business Ltd |
Gene therapy for neuromuscular and neuromotor disorders
|
WO2022018638A1
(en)
|
2020-07-21 |
2022-01-27 |
Crispr Therapeutics Ag |
Genome-editing compositions and methods to modulate faah for treatment of neurological disorders
|
TW202227634A
(zh)
|
2020-09-08 |
2022-07-16 |
美商薩羅塔治療公司 |
表現γ—肌聚醣之腺相關病毒載體之全身性遞送及肌肉失養症之治療
|
US20230357795A1
(en)
|
2020-09-15 |
2023-11-09 |
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
|
AU2021349277A1
(en)
|
2020-09-28 |
2023-05-11 |
Research Institute At Nationwide Children's Hospital |
Products and methods for treating muscular dystrophy
|
US20230392134A1
(en)
|
2020-09-30 |
2023-12-07 |
Crispr Therapeutics Ag |
Materials and methods for treatment of amyotrophic lateral sclerosis
|
JP2023551279A
(ja)
|
2020-11-30 |
2023-12-07 |
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 |
顔面肩甲上腕筋ジストロフィー(fshd)を治療するための組成物及び方法
|
WO2022133246A1
(en)
|
2020-12-17 |
2022-06-23 |
Vertex Pharmaceuticals Incorporated |
Compositions and methods for editing beta-globin for treatment of hemaglobinopathies
|
GB202100311D0
(en)
|
2021-01-11 |
2021-02-24 |
Univ Court Of The Univ Of Aberdeen |
Treatment for Lipodystrophy
|
WO2022164860A1
(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)
|
EP4288539A1
(en)
|
2021-02-03 |
2023-12-13 |
Research Institute at Nationwide Children's Hospital |
Compositions and methods for treating disease associated with dux4 overexpression
|
WO2022170038A1
(en)
|
2021-02-05 |
2022-08-11 |
Amicus Therapeutics, Inc. |
Adeno-associated virus delivery of cln3 polynucleotide
|
AU2022229489A1
(en)
|
2021-03-04 |
2023-08-31 |
Research Institute At Nationwide Children's Hospital |
Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications
|
EP4305157A1
(en)
|
2021-03-09 |
2024-01-17 |
Huidagene Therapeutics (Singapore) Pte. Ltd. |
Engineered crispr/cas13 system and uses thereof
|
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
|
US20240200066A1
(en)
|
2021-04-23 |
2024-06-20 |
Research Institute At Nationwide Children's Hospital |
Products and methods for treating muscular dystrophy
|
US20250018059A1
(en)
|
2021-05-17 |
2025-01-16 |
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
|
BR112023023768A2
(pt)
|
2021-06-11 |
2024-02-27 |
Bayer Ag |
Sistemas de endonucleases programáveis por rna tipo v
|
EP4101928A1
(en)
|
2021-06-11 |
2022-12-14 |
Bayer AG |
Type v rna programmable endonuclease systems
|
WO2023283962A1
(en)
|
2021-07-16 |
2023-01-19 |
Huigene Therapeutics Co., Ltd. |
Modified aav capsid for gene therapy and methods thereof
|
IL310725A
(en)
|
2021-08-11 |
2024-04-01 |
Solid Biosciences Inc |
Treatment of muscular dystrophy
|
EP4144841A1
(en)
|
2021-09-07 |
2023-03-08 |
Bayer AG |
Novel small rna programmable endonuclease systems with impoved pam specificity and uses thereof
|
WO2023042104A1
(en)
|
2021-09-16 |
2023-03-23 |
Novartis Ag |
Novel transcription factors
|
CA3234702A1
(en)
|
2021-10-07 |
2023-04-13 |
Research Institute At Nationwide Children's Hospital |
Products and methods for myelin protein zero silencing and treating cmt1b disease
|
US20240424137A1
(en)
|
2021-10-08 |
2024-12-26 |
Amicus Therapeutics, Inc. |
Biomarkers For Lysosomal Storage Diseases
|
US20230139985A1
(en)
|
2021-10-15 |
2023-05-04 |
Research Institute At Nationwide Children's Hospital |
Self-Complementary Adeno-Associated Virus Vector and its Use in Treatment of Muscular Dystrophy
|
US20230279431A1
(en)
|
2021-11-30 |
2023-09-07 |
Research Institute At Nationwide Children's Hospital |
Self-Complementary Adeno-Associated Virus Vector and its Use in Treatment of Muscular Dystrophy
|
US20230323395A1
(en)
*
|
2021-12-15 |
2023-10-12 |
Homology Medicines, Inc. |
Methods and compositions for the production of adeno-associated virus
|
EP4198134A1
(en)
|
2021-12-16 |
2023-06-21 |
Genethon |
Gamma-sarcoglycan gene transfer increase using modified itr sequences
|
EP4198046A1
(en)
|
2021-12-16 |
2023-06-21 |
Genethon |
Alpha-sarcoglycan gene transfer increase using modified itr sequences
|
EP4198048A1
(en)
|
2021-12-16 |
2023-06-21 |
Genethon |
Calpain-3 gene transfer increase using modified itr sequences
|
EP4198047A1
(en)
|
2021-12-16 |
2023-06-21 |
Genethon |
Fukutin related protein gene transfer increase using modified itr sequences
|
EP4453013A1
(en)
|
2021-12-21 |
2024-10-30 |
Research Institute at Nationwide Children's Hospital |
Materials and methods for the treatment of limb girdle muscular dystrophy
|
US20250084391A1
(en)
|
2021-12-23 |
2025-03-13 |
Bayer Aktiengesellschaft |
Novel small type v rna programmable endonuclease systems
|
WO2023131682A1
(en)
|
2022-01-06 |
2023-07-13 |
Ucl Business Ltd |
Endogenous gene regulation to treat neurological disorders and diseases
|
GB202201744D0
(en)
|
2022-02-10 |
2022-03-30 |
Ucl Business Ltd |
Treatment of acquired focal epilepsy
|
JP2025507751A
(ja)
|
2022-03-01 |
2025-03-21 |
クリスパー・セラピューティクス・アクチェンゲゼルシャフト |
アンジオポエチン様3(angptl3)関連状態を処置するための方法および組成物
|
EP4486900A2
(en)
|
2022-03-03 |
2025-01-08 |
Research Institute at Nationwide Children's Hospital |
Materials and methods for the treatment of eif2b5 mutations and diseases resulting therefrom
|
AU2023250649A1
(en)
|
2022-04-04 |
2024-11-14 |
The Regents Of The University Of California |
Genetic complementation compositions and methods
|
GB202205514D0
(en)
*
|
2022-04-13 |
2022-05-25 |
Freeline Therapeutics Ltd |
Mechanical lysis
|
CN118974284A
(zh)
|
2022-05-06 |
2024-11-15 |
诺华股份有限公司 |
新型重组aav vp2融合多肽
|
EP4526335A1
(en)
|
2022-05-17 |
2025-03-26 |
Centre Hospitalier Universitaire Vaudois |
Designed biosensors for enhanced t cell therapy
|
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
|
KR20250022020A
(ko)
|
2022-06-10 |
2025-02-14 |
바이엘 악티엔게젤샤프트 |
신규 소형 유형 v rna 프로그램가능한 엔도뉴클레아제 시스템
|
AU2023303015A1
(en)
|
2022-07-06 |
2025-01-23 |
Research Institute At Nationwide Children's Hospital |
Adeno-associated virus delivery of cln1 polynucleotide
|
WO2024035782A1
(en)
|
2022-08-10 |
2024-02-15 |
Aav Gene Therapeutics, Inc. |
Aav-mediated intramuscular delivery of insulin
|
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)
|
WO2024092126A1
(en)
|
2022-10-27 |
2024-05-02 |
Cargo Therapeutics, Inc. |
Compositions and methods for improved immunotherapies
|
WO2024129743A2
(en)
|
2022-12-13 |
2024-06-20 |
Bluerock Therapeutics Lp |
Engineered type v rna programmable endonucleases and their uses
|
WO2024151982A1
(en)
|
2023-01-13 |
2024-07-18 |
Amicus Therapeutics, Inc. |
Gene therapy constructs for the treatment of pompe disease
|
WO2024163560A1
(en)
|
2023-02-01 |
2024-08-08 |
Sarepta Therapeutics, Inc. |
Raav production methods
|
WO2024168276A2
(en)
|
2023-02-09 |
2024-08-15 |
Cargo Therapeutics, Inc. |
Compositions and methods for immunotherapies
|
WO2024194280A1
(en)
|
2023-03-21 |
2024-09-26 |
F. Hoffmann-La Roche Ag |
Method for the production of recombinant aav particle preparations
|
WO2024220592A2
(en)
|
2023-04-18 |
2024-10-24 |
Research Institute At Nationwide Children's Hospital, Inc. |
Gene therapy for treating limb girdle muscular dystrophy r9 and congenital muscular dystrophy 1c
|
WO2024229115A1
(en)
|
2023-05-02 |
2024-11-07 |
Research Institute At Nationwide Children's Hospital |
Treatment of multiple sclerosis using nt-3 gene therapy
|
WO2024229211A2
(en)
|
2023-05-02 |
2024-11-07 |
Research Institute At Nationwide Children's Hospital |
A modular system to convert therapeutic microrna expression cassettes from polymerase iii-based to polymerase ii-based promoters
|
WO2024229259A1
(en)
|
2023-05-02 |
2024-11-07 |
Research Institute At Nationwide Children's Hospital |
Gene therapy for treatment of protein misfolding diseases
|
WO2024254319A1
(en)
|
2023-06-07 |
2024-12-12 |
Research Institute At Nationwide Children's Hospital |
Gene therapy for lysosomal acid lipase deficiency (lal-d)
|
WO2024259064A1
(en)
|
2023-06-13 |
2024-12-19 |
Research Institute At Nationwide Children's Hospital |
Materials and methods for the treatment of neurofibromin 1 mutations and diseases resulting therefrom
|
US20250027087A1
(en)
|
2023-07-21 |
2025-01-23 |
Crispr Therapeutics Ag |
Modulating expression of alas1 (5'-aminolevulinate synthase 1) gene
|
EP4512403A1
(en)
|
2023-08-22 |
2025-02-26 |
Friedrich-Schiller-Universität Jena |
Neuropeptide b and w-receptor as a target for treating mood disorders and/or chronic stress
|
WO2025047758A1
(ja)
*
|
2023-08-28 |
2025-03-06 |
味の素株式会社 |
ウイルス産生細胞培養用培地
|
WO2025049993A1
(en)
|
2023-08-31 |
2025-03-06 |
Dyno Therapeutics, Inc. |
Capsid polypeptides and methods of use thereof
|