US5436146A
(en)
*
|
1989-09-07 |
1995-07-25 |
The Trustees Of Princeton University |
Helper-free stocks of recombinant adeno-associated virus vectors
|
PT733103E
(pt)
*
|
1993-11-09 |
2004-07-30 |
Targeted Genetics Corp |
Criacao de elevados titulos de vectores de aav recombinantes
|
JPH10501686A
(ja)
|
1994-04-13 |
1998-02-17 |
ザ ロックフェラー ユニヴァーシティ |
神経系の細胞へのdnaのaav仲介送達
|
US5658785A
(en)
*
|
1994-06-06 |
1997-08-19 |
Children's Hospital, Inc. |
Adeno-associated virus materials and methods
|
US20020159979A1
(en)
|
1994-06-06 |
2002-10-31 |
Children's Hospital, Inc. |
Adeno-associated virus materials and methods
|
EP0785991A1
(de)
*
|
1994-10-13 |
1997-07-30 |
Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts |
Bereitstellung von rep-negativen aav-mutanten und hierfür verwendbare zellen
|
US5856152A
(en)
*
|
1994-10-28 |
1999-01-05 |
The Trustees Of The University Of Pennsylvania |
Hybrid adenovirus-AAV vector and methods of use therefor
|
US6342390B1
(en)
|
1994-11-23 |
2002-01-29 |
The United States Of America As Represented By The Secretary Of Health And Human Services |
Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy
|
US6326356B1
(en)
|
1996-10-18 |
2001-12-04 |
Board Of Regents, The University Of Texas System |
Suppression of neu overexpression using a mini-E1A gene
|
WO1996017947A1
(en)
*
|
1994-12-06 |
1996-06-13 |
Targeted Genetics Corporation |
Packaging cell lines for generation of high titers of recombinant aav vectors
|
US6924128B2
(en)
|
1994-12-06 |
2005-08-02 |
Targeted Genetics Corporation |
Packaging cell lines for generation of high titers of recombinant AAV vectors
|
US5843742A
(en)
*
|
1994-12-16 |
1998-12-01 |
Avigen Incorporated |
Adeno-associated derived vector systems for gene delivery and integration into target cells
|
US6752987B1
(en)
|
1995-02-28 |
2004-06-22 |
The Regents Of The University Of California |
Adenovirus encoding human adenylylcyclase (AC) VI
|
JP3961019B2
(ja)
|
1995-02-28 |
2007-08-15 |
ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア |
遺伝子転移媒介の血管形成療法
|
US5756283A
(en)
*
|
1995-06-05 |
1998-05-26 |
The Trustees Of The University Of Pennsylvania |
Method for improved production of recombinant adeno-associated viruses for gene therapy
|
US6281010B1
(en)
|
1995-06-05 |
2001-08-28 |
The Trustees Of The University Of Pennsylvania |
Adenovirus gene therapy vehicle and cell line
|
US6093570A
(en)
*
|
1995-06-07 |
2000-07-25 |
The University Of North Carolina At Chapel Hill |
Helper virus-free AAV production
|
EP0842287B1
(en)
*
|
1995-08-03 |
2004-02-04 |
Avigen, Inc. |
High efficiency helper system for aav vector production
|
US5622856A
(en)
*
|
1995-08-03 |
1997-04-22 |
Avigen |
High efficiency helper system for AAV vector production
|
US6001650A
(en)
|
1995-08-03 |
1999-12-14 |
Avigen, Inc. |
High-efficiency wild-type-free AAV helper functions
|
US6162796A
(en)
*
|
1995-09-27 |
2000-12-19 |
The Rockefeller University |
Method for transferring genes to the heart using AAV vectors
|
US6086913A
(en)
*
|
1995-11-01 |
2000-07-11 |
University Of British Columbia |
Liposomal delivery of AAV vectors
|
US6004797A
(en)
*
|
1995-11-09 |
1999-12-21 |
Avigen, Inc. |
Adenovirus helper-free recombinant AAV Virion production
|
US20020193580A1
(en)
*
|
1995-12-15 |
2002-12-19 |
Mitchell Lloyd G. |
Methods and compositions for use in spliceosome mediated RNA trans-splicing
|
US20060088938A1
(en)
*
|
1995-12-15 |
2006-04-27 |
Mitchell Lloyd G |
Methods and compositions for use in spliceosome mediated RNA trans-splicing in plants
|
US20030027250A1
(en)
*
|
1995-12-15 |
2003-02-06 |
Mitchell Lloyd G. |
Methods and compositions for use in spliceosome mediated RNA trans-splicing
|
US5952221A
(en)
*
|
1996-03-06 |
1999-09-14 |
Avigen, Inc. |
Adeno-associated virus vectors comprising a first and second nucleic acid sequence
|
DE19608751B4
(de)
|
1996-03-06 |
2006-05-18 |
Medigene Ag |
Verwendung eines Adeno-assoziierten Virus-Vektors zur Steigerung der Immunogenität von Zellen
|
US6294370B1
(en)
|
1997-06-24 |
2001-09-25 |
Medigene Ag |
System for the production of AAV vectors
|
US6541012B2
(en)
|
1996-06-24 |
2003-04-01 |
Christoph Bogedain |
System for the production of AAV vectors
|
DE19625188A1
(de)
*
|
1996-06-24 |
1998-01-08 |
Medigene Gmbh |
System zur Herstellung von AAV-Vektoren
|
CA2264499A1
(en)
|
1996-09-06 |
1998-03-12 |
The Trustees Of The University Of Pennsylvania |
Methods using cre-lox for production of recombinant adeno-associated viruses
|
US5866552A
(en)
*
|
1996-09-06 |
1999-02-02 |
The Trustees Of The University Of Pennsylvania |
Method for expressing a gene in the absence of an immune response
|
AU722624B2
(en)
*
|
1996-09-06 |
2000-08-10 |
Trustees Of The University Of Pennsylvania, The |
An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
|
AU742696B2
(en)
*
|
1996-11-18 |
2002-01-10 |
Mcgill University |
Post-mitotic neurons containing adenovirus vectors that modulate apoptosis and growth
|
US6541258B2
(en)
|
1996-12-18 |
2003-04-01 |
Targeted Genetics Corporation |
AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
|
ES2224375T3
(es)
*
|
1997-04-14 |
2005-03-01 |
Cell Genesys, Inc. |
Metodos para aumentar la eficacia del producto de aav recombinante.
|
US6251677B1
(en)
|
1997-08-25 |
2001-06-26 |
The Trustees Of The University Of Pennsylvania |
Hybrid adenovirus-AAV virus and methods of use thereof
|
US6566118B1
(en)
|
1997-09-05 |
2003-05-20 |
Targeted Genetics Corporation |
Methods for generating high titer helper-free preparations of released recombinant AAV vectors
|
AU758708B2
(en)
|
1997-09-05 |
2003-03-27 |
Targeted Genetics Corporation |
Methods for generating high titer helper-free preparations of recombinant AAV vectors
|
US6989264B2
(en)
|
1997-09-05 |
2006-01-24 |
Targeted Genetics Corporation |
Methods for generating high titer helper-free preparations of released recombinant AAV vectors
|
US6642051B1
(en)
*
|
1997-10-21 |
2003-11-04 |
Targeted Genetics Corporation |
Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of 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
|
IT1297074B1
(it)
|
1997-11-21 |
1999-08-03 |
Angeletti P Ist Richerche Bio |
Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le
|
US6953690B1
(en)
|
1998-03-20 |
2005-10-11 |
The Trustees Of The University Of Pennsylvania |
Compositions and methods for helper-free production of recombinant adeno-associated viruses
|
DK1930418T3
(da)
|
1998-09-04 |
2015-07-13 |
Genzyme Corp |
Fremgangsmåder til frembringelse af hjælperfri præparater med høj titer af frigjorte rekombinante AAV-vektorer
|
EP1939215A1
(en)
|
1998-10-22 |
2008-07-02 |
The University of Montana |
Omp85 proteins of neisseria gonorrhoeae and neisseria meningitidis, compositions containing same and methods of use thereof
|
US6303362B1
(en)
*
|
1998-11-19 |
2001-10-16 |
The Board Of Trustees Of The Leland Stanford Junior University |
Adenoviral vector and methods for making and using the same
|
US6387368B1
(en)
|
1999-02-08 |
2002-05-14 |
The Trustees Of The University Of Pennsylvania |
Hybrid adenovirus-AAV virus and methods of use thereof
|
US6893865B1
(en)
*
|
1999-04-28 |
2005-05-17 |
Targeted Genetics Corporation |
Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
|
US6793926B1
(en)
*
|
1999-05-27 |
2004-09-21 |
Genovo, Inc. |
Methods for production of a recombinant adeno-associated virus
|
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
|
AU781958C
(en)
|
1999-08-09 |
2006-03-30 |
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
|
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
|
US6627617B1
(en)
|
1999-10-01 |
2003-09-30 |
University Of North Carolina At Chapel Hill |
Temperature-sensitive regulation of viral vector production
|
US7115391B1
(en)
|
1999-10-01 |
2006-10-03 |
Genovo, Inc. |
Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
|
AU7879000A
(en)
*
|
1999-10-12 |
2001-04-23 |
University Of North Carolina At Chapel Hill, The |
Adeno-associated virus vectors encoding factor viii and methods of using the same
|
AU2460001A
(en)
|
1999-12-27 |
2001-07-09 |
Regents Of The University Of California, The |
Gene therapy for congestive heart failure
|
US6916635B2
(en)
*
|
2000-10-02 |
2005-07-12 |
The Research Foundation Of State University Of New York |
Hybrid adenovirus/adeno-associated virus vectors and methods of use thereof
|
US20040126774A1
(en)
*
|
2001-01-08 |
2004-07-01 |
Mitchell Lioyd G. |
Correction of factor VIII genetic defects using spliceosome mediated RNA trans splicing
|
US6844192B2
(en)
|
2001-06-29 |
2005-01-18 |
Wake Forest University |
Adenovirus E4 protein variants for virus production
|
EP1427816A4
(en)
|
2001-09-18 |
2004-12-01 |
Clontech Lab Inc |
METHOD FOR PRODUCING ADENOVIRAL VECTORS BASED ON A SITE SPECIFIC RECOMBINASE
|
US7399753B2
(en)
*
|
2002-02-25 |
2008-07-15 |
Virxsys Corporation |
Trans-splicing mediated photodynamic therapy
|
US7332276B2
(en)
|
2002-03-01 |
2008-02-19 |
Celltech R&D, Inc. |
Methods to increase or decrease bone density
|
EP1504016A4
(en)
*
|
2002-05-08 |
2006-11-22 |
Intronn Inc |
USE OF SPLICEOSOM-MEDIATED RNA-TRANS-SPLICING TO GIVE ADENOVIRES A CELLSELECTIVE REPLICATION
|
AU2003285009B2
(en)
*
|
2002-10-23 |
2010-10-07 |
Virxsys Corporation |
Screening methods for identification of efficient pre-trans-splicing molecules
|
ATE521701T1
(de)
|
2003-01-22 |
2011-09-15 |
Univ Duke |
Verbesserte konstrukte zur expression lysosomaler polypeptide
|
WO2004075861A2
(en)
*
|
2003-02-26 |
2004-09-10 |
Children's Hospital, Inc. |
Recombinant adeno-associated virus production
|
US20060177933A1
(en)
*
|
2004-01-23 |
2006-08-10 |
Madaiah Puttaraju |
Expression of apoA-1 and variants thereof using spliceosome mediated RNA trans-splicing
|
US8053232B2
(en)
*
|
2004-01-23 |
2011-11-08 |
Virxsys Corporation |
Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing
|
US7968334B2
(en)
*
|
2004-01-23 |
2011-06-28 |
Virxsys Corporation |
Expression of apoAI and variants thereof using spliceosome mediated RNA trans-splicing
|
EP1723227A4
(en)
|
2004-02-10 |
2007-09-19 |
Dartmouth College |
NICOTINAMIDRIBOSIDE KINASE COMPOSITIONS AND METHOD FOR THEIR USE
|
US20060094110A1
(en)
*
|
2004-07-30 |
2006-05-04 |
Mcgarrity Gerard J |
Use of spliceosome mediated RNA trans-splicing for immunotherapy
|
US20060134658A1
(en)
*
|
2004-08-09 |
2006-06-22 |
Garcia-Blanco Mariano A |
Use of RNA trans-splicing for generation of interfering RNA molecules
|
US7166963B2
(en)
*
|
2004-09-10 |
2007-01-23 |
Axcelis Technologies, Inc. |
Electrodeless lamp for emitting ultraviolet and/or vacuum ultraviolet radiation
|
DE102004047492B4
(de)
*
|
2004-09-23 |
2006-07-20 |
Jost-Werke Gmbh & Co. Kg |
Verfahren zum Übertragen von elektrischer, pneumatischer oder hydraulischer Energie sowie ein Energieübertragungssystem
|
US7871795B2
(en)
|
2004-10-08 |
2011-01-18 |
Virxsys Corporation |
Targeted trans-splicing of highly abundant transcripts for in vivo production of recombinant proteins
|
AU2005326784B2
(en)
*
|
2004-10-08 |
2012-03-15 |
Virxsys Corporation |
Use of RNA trans-splicing for antibody gene transfer and antibody polypeptide production
|
ES2569365T3
(es)
|
2006-11-29 |
2016-05-10 |
Nationwide Children's Hospital |
Inhibición de miostatina para la potenciación de músculo y/o la mejora de la función muscular
|
US11219696B2
(en)
|
2008-12-19 |
2022-01-11 |
Nationwide Children's Hospital |
Delivery of polynucleotides using recombinant AAV9
|
US9415121B2
(en)
|
2008-12-19 |
2016-08-16 |
Nationwide Children's Hospital |
Delivery of MECP2 polynucleotide using recombinant AAV9
|
JP2012521750A
(ja)
|
2009-03-27 |
2012-09-20 |
プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ |
肝硬変及び肝線維症の治療のための方法及び組成物
|
SI3421603T1
(sl)
|
2009-05-02 |
2022-02-28 |
Genzyme Corporation |
Genska terapija za nevrodegenerativne motnje
|
AU2010316996A1
(en)
|
2009-11-05 |
2012-06-14 |
Proyecto De Biomedicina Cima, S.L. |
Regulated expression systems
|
JP2013544600A
(ja)
|
2010-11-23 |
2013-12-19 |
プレサージュ バイオサイエンシズ,インコーポレイテッド |
固体デリバリーのための治療方法および組成物
|
US20140155469A1
(en)
|
2011-04-19 |
2014-06-05 |
The Research Foundation Of State University Of New York |
Adeno-associated-virus rep sequences, vectors and viruses
|
WO2012145597A2
(en)
|
2011-04-21 |
2012-10-26 |
Nationwide Children's Hospital, Inc. |
Recombinant virus products and methods for inhibition of expression of myotilin
|
US10196636B2
(en)
|
2011-04-21 |
2019-02-05 |
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
|
CA2842798C
(en)
|
2011-07-25 |
2021-02-23 |
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
|
US9163259B2
(en)
|
2012-05-04 |
2015-10-20 |
Novartis Ag |
Viral vectors for the treatment of retinal dystrophy
|
BR112015002168A2
(pt)
|
2012-08-01 |
2017-11-07 |
Nationwide Childrens Hospital |
liberação intratecal de vírus 9 adeno-associado recombinante
|
US9539307B2
(en)
|
2012-09-17 |
2017-01-10 |
The Research Institute At Nationwide Children's Hospital |
Compositions and methods for treating amyotrophic lateral sclerosis
|
JP6576904B2
(ja)
|
2013-04-04 |
2019-09-18 |
トラスティーズ・オブ・ダートマス・カレッジ |
HIV−1プロウイルスDNAのinvivo切除のための組成物及び方法
|
CA2909085C
(en)
|
2013-04-08 |
2023-08-29 |
University Of Iowa Research Foundation |
Chimeric adeno-associated virus/ bocavirus parvovirus vector
|
EA032706B1
(ru)
|
2013-04-20 |
2019-07-31 |
Рисёрч Инститъют Эт Нэйшнвайд Чилдрен'С Хоспитал |
ДОСТАВКА НАЦЕЛЕННЫХ НА ЭКЗОН 2 ПОЛИНУКЛЕОТИДНЫХ КОНСТРУКЦИЙ U7snRNA ПРИ ПОМОЩИ РЕКОМБИНАНТНОГО АДЕНОАССОЦИИРОВАННОГО ВИРУСА
|
SG11201509419QA
(en)
|
2013-05-15 |
2015-12-30 |
Univ Minnesota |
Adeno-associated virus mediated gene transfer to the central nervous system
|
EP3039146B1
(en)
|
2013-08-27 |
2020-05-27 |
Research Institute at Nationwide Children's Hospital |
Products and methods for treatment of amyotrophic lateral sclerosis
|
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
|
JP6986444B2
(ja)
|
2014-08-09 |
2021-12-22 |
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 |
Dmd遺伝子のエクソン5内の内部リボソーム進入部位を活性化するための方法及び材料
|
WO2016057975A2
(en)
|
2014-10-10 |
2016-04-14 |
Research Institute At Nationwide Children's Hospital |
Guided injections for aav gene transfer to muscle
|
AU2015342997B2
(en)
|
2014-11-05 |
2021-11-18 |
Research Institute At Nationwide Children's Hospital |
Methods and materials for producing recombinant viruses in eukaryotic microalgae
|
WO2016122791A1
(en)
|
2015-01-30 |
2016-08-04 |
The Regents Of The University Of California |
Spinal subpial gene delivery system
|
MA41451A
(fr)
|
2015-02-04 |
2017-12-12 |
Univ Washington |
Constructions anti-tau
|
WO2016135558A2
(en)
|
2015-02-23 |
2016-09-01 |
Crispr Therapeutics Ag |
Materials and methods for treatment of hemoglobinopathies
|
KR102406615B1
(ko)
|
2015-05-15 |
2022-06-08 |
리젠츠 오브 더 유니버시티 오브 미네소타 |
중추 신경계로의 아데노-연관 치료적 전달
|
US10017832B2
(en)
|
2015-08-25 |
2018-07-10 |
Washington University |
Compositions and methods for site specific recombination at asymmetric sites
|
CN108136049A
(zh)
|
2015-09-17 |
2018-06-08 |
全国儿童医院研究所 |
Galgt2基因治疗的方法和材料
|
US20190048340A1
(en)
|
2015-09-24 |
2019-02-14 |
Crispr Therapeutics Ag |
Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
|
AU2016344609B2
(en)
|
2015-10-28 |
2022-05-12 |
Vertex Pharmaceuticals Incorporated |
Materials and methods for treatment of duchenne muscular dystrophy
|
BR112018008971A2
(pt)
|
2015-11-06 |
2018-11-27 |
Crispr Therapeutics Ag |
materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a
|
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
|
EP3384024B1
(en)
|
2015-12-01 |
2022-02-02 |
CRISPR Therapeutics AG |
Materials and methods for treatment of alpha-1 antitrypsin deficiency
|
CN109312339B
(zh)
|
2015-12-23 |
2022-01-28 |
克里斯珀医疗股份公司 |
用于治疗肌萎缩侧索硬化症和/或额颞叶变性的材料和方法
|
WO2017120589A1
(en)
|
2016-01-08 |
2017-07-13 |
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
|
WO2017139381A1
(en)
|
2016-02-08 |
2017-08-17 |
University Of Iowa Research Foundation |
Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
|
US20190112353A1
(en)
|
2016-02-18 |
2019-04-18 |
Crispr Therapeutics Ag |
Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
|
US11066456B2
(en)
|
2016-02-25 |
2021-07-20 |
Washington University |
Compositions comprising TREM2 and methods of use thereof
|
CA3015633A1
(en)
|
2016-02-26 |
2017-08-31 |
Research Institute At Nationwide Children's Hospital |
Recombinant virus products and methods for inducing dux4 exon skipping
|
WO2017158422A1
(en)
|
2016-03-16 |
2017-09-21 |
Crispr Therapeutics Ag |
Materials and methods for treatment of hereditary haemochromatosis
|
EP3436427B1
(en)
|
2016-03-28 |
2021-12-08 |
The Regents of the University of California |
Method and composition for treating neuronal hyper-excitability
|
BR112018070249A2
(pt)
|
2016-04-02 |
2019-01-29 |
Research Institute At Nationwide Children's Hospital |
sistema promotor de u6 modificada para expressão específica de tecido
|
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
|
MA44681A
(fr)
|
2016-04-15 |
2019-02-20 |
Res Inst Nationwide Childrens Hospital |
Administration de b-sarcoglycane et de microarn-29 à l'aide d'un vecteur de virus adéno-associé, et traitement de la dystrophie musculaire
|
JP6974349B2
(ja)
|
2016-04-18 |
2021-12-01 |
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 |
ヘモグロビン異常症の処置のための材料及び方法
|
WO2017191503A1
(en)
|
2016-05-05 |
2017-11-09 |
Crispr Therapeutics Ag |
Materials and methods for treatment of hemoglobinopathies
|
US11174469B2
(en)
|
2016-06-29 |
2021-11-16 |
Crispr Therapeutics Ag |
Materials and methods for treatment of Amyotrophic Lateral Sclerosis (ALS) and other related disorders
|
EP3478829A1
(en)
|
2016-06-29 |
2019-05-08 |
Crispr Therapeutics AG |
Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders
|
AU2017290614C1
(en)
|
2016-06-29 |
2024-01-18 |
Crispr Therapeutics Ag |
Materials and methods for treatment of friedreich ataxia and other related disorders
|
JP7305534B2
(ja)
|
2016-07-06 |
2023-07-10 |
バーテックス ファーマシューティカルズ インコーポレイテッド |
疼痛関連障害を処置するための物質及び方法
|
WO2018007980A1
(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
|
WO2018020323A2
(en)
|
2016-07-25 |
2018-02-01 |
Crispr Therapeutics Ag |
Materials and methods for treatment of fatty acid disorders
|
KR20190086503A
(ko)
|
2016-11-15 |
2019-07-22 |
리젠츠 오브 더 유니버시티 오브 미네소타 |
Mpsi 및 mpsii에서의 신경계 기능 및 다른 신경계 장애를 개선하는 방법
|
JP7106534B2
(ja)
|
2016-11-17 |
2022-07-26 |
ネイションワイド チルドレンズ ホスピタル, インコーポレイテッド |
メチル-cpg結合タンパク質2をコードする組換えアデノ随伴ウイルスの髄腔内送達
|
RU2019119272A
(ru)
|
2016-11-23 |
2020-12-24 |
Гритстоун Онколоджи, Инк. |
Вирусная доставка неоантигенов
|
CN118546256A
(zh)
|
2016-12-13 |
2024-08-27 |
西雅图儿童医院(Dba西雅图儿童研究所) |
在体外和体内对工程化的细胞中表达的化学物质诱导的信号传导复合物进行外源性药物激活的方法
|
US11142775B2
(en)
|
2017-01-13 |
2021-10-12 |
University Of Iowa Research Foundation |
Bocaparvovirus small noncoding RNA and uses thereof
|
WO2018150271A1
(en)
|
2017-02-17 |
2018-08-23 |
Lonza Ltd. |
Mammalian cells for producing adeno-associated viruses
|
EP3585899A1
(en)
|
2017-02-22 |
2020-01-01 |
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
|
EP3585898A1
(en)
|
2017-02-22 |
2020-01-01 |
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
|
EP3585807A1
(en)
|
2017-02-22 |
2020-01-01 |
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
|
CA3054031A1
(en)
|
2017-02-22 |
2018-08-30 |
Crispr Therapeutics Ag |
Compositions and methods for gene editing
|
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
|
KR20240112964A
(ko)
|
2017-03-17 |
2024-07-19 |
더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 |
근이영양증을 치료하기 위한 근육 특이적 마이크로-디스트로핀의 아데노-관련 바이러스 벡터 전달
|
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
|
WO2018208856A1
(en)
|
2017-05-08 |
2018-11-15 |
Gritstone Oncology, Inc. |
Alphavirus neoantigen vectors
|
US11622977B2
(en)
|
2017-05-12 |
2023-04-11 |
Crispr Therapeutics Ag |
Materials and methods for engineering cells and uses thereof in immuno-oncology
|
CN111065741A
(zh)
|
2017-07-08 |
2020-04-24 |
吉尼松公司 |
脊髓性肌萎缩症的治疗
|
CA3070654C
(en)
|
2017-07-25 |
2021-07-27 |
Oxford Genetics Limited |
Adenoviral vectors
|
EP3697907A1
(en)
|
2017-10-17 |
2020-08-26 |
CRISPR Therapeutics AG |
Compositions and methods for gene editing 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
|
CN111433367A
(zh)
|
2017-10-20 |
2020-07-17 |
全国儿童医院研究所 |
Nt-3基因疗法的方法和材料
|
US20210180091A1
(en)
|
2017-10-26 |
2021-06-17 |
Vertex Pharmaceuticals Incorporated |
Materials and methods for treatment of hemoglobinopathies
|
WO2019094253A1
(en)
|
2017-11-08 |
2019-05-16 |
Avexis Inc. |
Means and method for preparing viral vectors and uses of same
|
WO2019092505A1
(en)
|
2017-11-09 |
2019-05-16 |
Casebia Therapeutics Llp |
Self-inactivating (sin) crispr/cas or crispr/cpf1 systems and uses thereof
|
AU2018372763A1
(en)
|
2017-11-21 |
2020-05-28 |
Bayer Healthcare Llc |
Materials and methods for treatment of autosomal dominant Retinitis Pigmentosa
|
AU2018386301A1
(en)
|
2017-12-14 |
2020-06-18 |
Bayer Healthcare Llc |
Novel RNA-programmable endonuclease systems and their use in genome editing and other applications
|
JP7402163B2
(ja)
|
2017-12-21 |
2023-12-20 |
クリスパー セラピューティクス アーゲー |
2a型アッシャー症候群の処置のための材料および方法
|
CA3084633A1
(en)
|
2017-12-21 |
2019-06-27 |
Crispr Therapeutics Ag |
Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
|
JP2021509823A
(ja)
|
2018-01-04 |
2021-04-08 |
アイコニック セラピューティクス インコーポレイテッド |
抗組織因子抗体、抗体薬物コンジュゲート、及び関連する方法
|
MA51637A
(fr)
|
2018-01-12 |
2020-11-18 |
Bayer Healthcare Llc |
Compositions et méthodes pour l'édition génique par ciblage de la transferrine
|
MA51788A
(fr)
|
2018-02-05 |
2020-12-16 |
Vertex Pharma |
Substances et méthodes pour traiter des hémoglobinopathies
|
WO2019150196A1
(en)
|
2018-02-05 |
2019-08-08 |
Crispr Therapeutics Ag |
Materials and methods for treatment of hemoglobinopathies
|
MA51869A
(fr)
|
2018-02-16 |
2020-12-23 |
Bayer Healthcare Llc |
Compositions et méthodes pour l'édition génique par ciblage du fibrinogène-alpha
|
WO2019183150A1
(en)
|
2018-03-19 |
2019-09-26 |
Casebia Therapeutics Limited Liability Partnership |
Novel rna-programmable endonuclease systems and uses thereof
|
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
|
WO2019236949A1
(en)
|
2018-06-08 |
2019-12-12 |
Avexis Inc. |
Cell-based assay for measuring drug product potency
|
GB201809588D0
(en)
|
2018-06-12 |
2018-07-25 |
Univ Bristol |
Materials and methods for modulating intraocular and intracranial pressure
|
CA3103485A1
(en)
|
2018-06-18 |
2019-12-26 |
Research Institute At Nationwide Children's Hospital |
Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
|
AU2019288291B2
(en)
|
2018-06-18 |
2024-09-26 |
Research Institute At Nationwide Children's Hospital |
Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
|
US20210277362A1
(en)
|
2018-06-29 |
2021-09-09 |
Research Institute At Nationwide Children's Hospital |
Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
|
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
|
JP7320601B2
(ja)
|
2018-09-11 |
2023-08-03 |
上▲海▼市公共▲衛▼生▲臨▼床中心 |
広域スペクトルな抗インフルエンザワクチン免疫原及びその使用
|
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
|
US20220042045A1
(en)
|
2018-12-21 |
2022-02-10 |
Genethon |
Expression cassettes for gene therapy vectors
|
EP3906308A1
(en)
|
2018-12-31 |
2021-11-10 |
Research Institute at Nationwide Children's Hospital |
Dux4 rna silencing using rna targeting crispr-cas13b
|
EP3921430A1
(en)
|
2019-02-04 |
2021-12-15 |
Research Institute at Nationwide Children's Hospital |
Adeno-associated virus delivery of cln3 polynucleotide
|
MX2021009404A
(es)
|
2019-02-04 |
2021-11-12 |
Res Inst Nationwide Childrens Hospital |
Administracion del virus adenoasociado de polinucleotido de cln6.
|
WO2020168362A1
(en)
|
2019-02-15 |
2020-08-20 |
Crispr Therapeutics Ag |
Gene editing for hemophilia a with improved factor viii expression
|
JP2022521025A
(ja)
|
2019-02-25 |
2022-04-04 |
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
|
CN113474461A
(zh)
|
2019-02-25 |
2021-10-01 |
诺华股份有限公司 |
治疗bietti晶体营养不良的组合物和方法
|
BR112021016570A2
(pt)
|
2019-02-26 |
2021-11-03 |
Res Inst Nationwide Childrens Hospital |
Entrega de vetor de vírus adenoassociado de beta-sarcoglicano e tratamento de distrofia muscular
|
SG11202109741VA
(en)
|
2019-03-12 |
2021-10-28 |
Crispr Therapeutics Ag |
Novel high fidelity rna-programmable endonuclease systems and uses thereof
|
AU2020253435A1
(en)
|
2019-04-01 |
2021-11-18 |
Tenaya Therapeutics, Inc. |
Adeno-associated virus with engineered capsid
|
SG11202111353QA
(en)
|
2019-04-15 |
2021-11-29 |
Univ Iowa Res Found |
Methods and compositions for transgene expression
|
SG11202111334SA
(en)
|
2019-04-15 |
2021-11-29 |
Univ Iowa Res Found |
Compositions and methods for treatment of cystic fibrosis
|
US20220193259A1
(en)
|
2019-04-15 |
2022-06-23 |
Sanford Research |
Gene therapy for treating or preventing visual effects in batten disease
|
WO2020225606A1
(en)
|
2019-05-08 |
2020-11-12 |
Crispr Therapeutics Ag |
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
|
SG11202113187WA
(en)
|
2019-05-30 |
2021-12-30 |
Gritstone Bio Inc |
Modified adenoviruses
|
WO2020264254A1
(en)
|
2019-06-28 |
2020-12-30 |
Crispr Therapeutics Ag |
Materials and methods for controlling gene editing
|
WO2021014428A1
(en)
|
2019-07-25 |
2021-01-28 |
Novartis Ag |
Regulatable expression systems
|
PL4017871T3
(pl)
|
2019-08-21 |
2024-07-08 |
Research Institute At Nationwide Children's Hospital |
Dostarczanie alfa-sarkoglikanu za pomocą wektora wirusa związanego z adenowirusami i leczenie dystrofii mięśniowej
|
US20230416776A1
(en)
|
2019-10-08 |
2023-12-28 |
Regents Of The University Of Minnesota |
Crispr-mediated human genome editing with vectors
|
WO2021077115A1
(en)
|
2019-10-18 |
2021-04-22 |
Research Institute At Nationwide Children's Hospital |
Gene therapy targeting cochlear cells
|
EP4045662A1
(en)
|
2019-10-18 |
2022-08-24 |
Research Institute at Nationwide Children's Hospital |
Materials and methods for the treatment of disorders associated mutations in the irf2bpl gene
|
EP4061831A1
(en)
|
2019-11-22 |
2022-09-28 |
Research Institute at Nationwide Children's Hospital |
Materials and methods for treatment of disorders associated with the ighmbp2 gene
|
EP4077687A1
(en)
|
2019-12-20 |
2022-10-26 |
Research Institute at Nationwide Children's Hospital |
Optimized gene therapy for targeting muscle in muscle diseases
|
KR20220155999A
(ko)
|
2020-02-18 |
2022-11-24 |
더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 |
X-연관 장애의 치료에서의 miRNA의 AAV-매개 표적화
|
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
|
BR112022025586A2
(pt)
|
2020-06-15 |
2023-03-07 |
Res Inst Nationwide Childrens Hospital |
Liberação de vetor de vírus adenoassociado para distrofias musculares
|
WO2022006253A2
(en)
|
2020-06-30 |
2022-01-06 |
University Of Iowa Research Foundation |
Methods and compositions for administering recombinant viral vectors
|
US20230374483A1
(en)
|
2020-07-08 |
2023-11-23 |
Regents Of The University Of Minnesota |
Modified hexosaminidase and uses thereof
|
WO2022018638A1
(en)
|
2020-07-21 |
2022-01-27 |
Crispr Therapeutics Ag |
Genome-editing compositions and methods to modulate faah for treatment of neurological disorders
|
WO2022026927A1
(en)
|
2020-07-30 |
2022-02-03 |
Shape Therapeutics Inc. |
Stable cell lines for inducible production of raav virions
|
JP2023541108A
(ja)
|
2020-08-06 |
2023-09-28 |
グリットストーン バイオ インコーポレイテッド |
マルチエピトープワクチンカセット
|
US20230414787A1
(en)
|
2020-08-27 |
2023-12-28 |
University Of Iowa Research Foundation |
Gene knock-out for treatment of glaucoma
|
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
|
JP2023541444A
(ja)
|
2020-09-15 |
2023-10-02 |
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 |
筋ジストロフィー患者における多様なdmd変異の補正のためのaav媒介性の相同性非依存的標的化組み込み遺伝子編集
|
EP4217375A1
(en)
|
2020-09-28 |
2023-08-02 |
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
|
AU2021385595A1
(en)
|
2020-11-30 |
2023-06-29 |
Research Institute At Nationwide Children's Hospital |
Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)
|
WO2022133246A1
(en)
|
2020-12-17 |
2022-06-23 |
Vertex Pharmaceuticals Incorporated |
Compositions and methods for editing beta-globin for treatment of hemaglobinopathies
|
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)
|
US20240093191A1
(en)
|
2021-02-03 |
2024-03-21 |
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
|
EP4288556A1
(en)
|
2021-02-05 |
2023-12-13 |
Regents of the University of Minnesota |
Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses
|
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
|
EP4305157A1
(en)
|
2021-03-09 |
2024-01-17 |
Huidagene Therapeutics (Singapore) Pte. Ltd. |
Engineered crispr/cas13 system and uses thereof
|
US20240189452A1
(en)
|
2021-04-13 |
2024-06-13 |
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
|
EP4323011A1
(en)
|
2021-04-15 |
2024-02-21 |
Spirovant Sciences, Inc. |
Methods and compositions for treatment of cystic fibrosis
|
AU2022262420A1
(en)
|
2021-04-23 |
2023-11-16 |
Research Institute At Nationwide Children's Hospital |
Products and methods for treating muscular dystrophy
|
EP4334447A1
(en)
|
2021-05-07 |
2024-03-13 |
UCL Business Ltd |
Abca4 genome editing
|
WO2022245675A1
(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
|
EP4352214A1
(en)
|
2021-06-11 |
2024-04-17 |
Bayer AG |
Type v rna programmable endonuclease systems
|
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)
|
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|
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(ja)
|
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|
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|
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|
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|
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|
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|
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|
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|
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|
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|
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|
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|
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|
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(en)
|
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|
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(en)
|
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|
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(zh)
|
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|
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|
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|
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|
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|
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|
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|
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|
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|
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(en)
|
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|
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(en)
|
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|
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|
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|
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(en)
|
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|
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(en)
|
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|
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(en)
|
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|
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|
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|
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(en)
|
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|
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(en)
|
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|
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(en)
|
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|
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(en)
|
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|