NZ600121A
(en)
|
2001-11-13 |
2013-12-20 |
Univ Pennsylvania |
A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
|
US9217155B2
(en)
|
2008-05-28 |
2015-12-22 |
University Of Massachusetts |
Isolation of novel AAV'S and uses thereof
|
WO2010138263A2
(en)
|
2009-05-28 |
2010-12-02 |
University Of Massachusetts |
Novel aav 's and uses thereof
|
WO2011133874A1
(en)
|
2010-04-23 |
2011-10-27 |
University Of Massachusetts |
Multicistronic expression constructs
|
DK2561073T3
(en)
|
2010-04-23 |
2016-12-12 |
Univ Massachusetts |
Aav vectors targeted to central nervous system and methods of use thereof
|
KR20190123796A
(ko)
|
2011-04-29 |
2019-11-01 |
셀렉타 바이오사이언시즈, 인크. |
항체 반응을 감소시키기 위한 관용원성 합성 나노운반체
|
CA2856137A1
(en)
|
2011-11-22 |
2013-05-30 |
The Children's Hospital Of Philadelphia |
Virus vectors for highly efficient transgene delivery
|
US9255154B2
(en)
|
2012-05-08 |
2016-02-09 |
Alderbio Holdings, Llc |
Anti-PCSK9 antibodies and use thereof
|
JP2015529685A
(ja)
|
2012-09-17 |
2015-10-08 |
ザ・リサーチ・インスティテュート・アット・ネイションワイド・チルドレンズ・ホスピタルThe Research Institute Atnationwide Children’S Hospital |
筋萎縮性側索硬化症の処置のための組成物および方法
|
CN105612253A
(zh)
*
|
2013-03-15 |
2016-05-25 |
费城儿童医院 |
含有填充者/填充物多核苷酸序列的载体及其制备方法
|
EP2991685A4
(en)
|
2013-05-03 |
2016-11-16 |
Selecta Biosciences Inc |
LOCAL ADMINISTRATION, CONCOMITANT OF SYNTHETIC NANOSUPPORTS TOLEROGENIC TO REDUCE TYPE I AND TYPE IV HYPERSENSITIVITY
|
PE20160188A1
(es)
*
|
2013-07-22 |
2016-04-27 |
Philadelphia Children Hospital |
Variantes aav y composiciones, metodos y usos para transferencia de gen a celulas, organos y tejidos
|
CN105980401A
(zh)
|
2013-10-03 |
2016-09-28 |
现代治疗公司 |
编码低密度脂蛋白受体的多核苷酸
|
CA2929669A1
(en)
|
2013-11-05 |
2015-05-14 |
The Research Institute At Nationwide Children's Hospital |
Compositions and methods for inhibiting nf-.kappa.b and sod-1 to treat amyotrophic lateral sclerosis
|
US10072251B2
(en)
|
2014-02-19 |
2018-09-11 |
University Of Massachusetts |
Recombinant AAVS having useful transcytosis properties
|
DK3119797T3
(da)
|
2014-03-18 |
2021-03-15 |
Univ Massachusetts |
Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose
|
HUE046454T2
(hu)
*
|
2014-03-21 |
2020-03-30 |
Genzyme Corp |
Retinitis pigmentosa génterápia
|
US10975391B2
(en)
|
2014-04-25 |
2021-04-13 |
University Of Massachusetts |
Recombinant AAV vectors useful for reducing immunity against transgene products
|
BR112016024379A2
(pt)
|
2014-04-25 |
2017-10-10 |
Univ Pennsylvania |
variantes ldlr e seu uso em composições para reduzir os níveis de colesterol
|
WO2015187825A2
(en)
|
2014-06-03 |
2015-12-10 |
University Of Massachusetts |
Compositions and methods for modulating dysferlin expression
|
EP3151866B1
(en)
|
2014-06-09 |
2023-03-08 |
Voyager Therapeutics, Inc. |
Chimeric capsids
|
CN105396143B
(zh)
*
|
2014-09-01 |
2020-06-26 |
江苏命码生物科技有限公司 |
埃博拉病毒特异性的miRNA以及通过miRNA抑制埃博拉病毒的方法
|
MX2017002933A
(es)
*
|
2014-09-07 |
2017-05-30 |
Selecta Biosciences Inc |
Metodos y composiciones para atenuar las respuestas inmunes del vector de transferencia anti-viral de edicion del gen.
|
US10370432B2
(en)
|
2014-10-03 |
2019-08-06 |
University Of Massachusetts |
Heterologous targeting peptide grafted AAVS
|
WO2016054557A1
(en)
|
2014-10-03 |
2016-04-07 |
University Of Massachusetts |
Novel high efficiency library-identified aav vectors
|
RU2020140209A
(ru)
|
2014-10-21 |
2021-01-25 |
Юниверсити Оф Массачусетс |
Варианты рекомбинантных aav и их применения
|
RU2020109343A
(ru)
|
2014-11-05 |
2020-03-17 |
Вояджер Терапьютикс, Инк. |
Полинуклеотиды aadc для лечения болезни паркинсона
|
GB201420139D0
(en)
|
2014-11-12 |
2014-12-24 |
Ucl Business Plc |
Factor IX gene therapy
|
ES2878451T3
(es)
|
2014-11-14 |
2021-11-18 |
Voyager Therapeutics Inc |
Polinucleótidos moduladores
|
CN107109407A
(zh)
|
2014-11-14 |
2017-08-29 |
沃雅戈治疗公司 |
治疗肌萎缩性侧索硬化(als)的组合物和方法
|
EP3230441A4
(en)
|
2014-12-12 |
2018-10-03 |
Voyager Therapeutics, Inc. |
Compositions and methods for the production of scaav
|
WO2016131009A1
(en)
|
2015-02-13 |
2016-08-18 |
University Of Massachusetts |
Compositions and methods for transient delivery of nucleases
|
WO2016172008A1
(en)
|
2015-04-24 |
2016-10-27 |
University Of Massachusetts |
Modified aav constructions and uses thereof
|
WO2016190780A1
(en)
|
2015-05-22 |
2016-12-01 |
Dmitry Dmitrievich Genkin |
Extracellular dna as a therapeutic target in neurodegeneration
|
BR112017027956A2
(pt)
|
2015-06-23 |
2018-08-28 |
Childrens Hospital Philadelphia |
fator ix modificado e composições, métodos e usos para a transferência de gene para células, órgãos e tecidos
|
JP2018535929A
(ja)
|
2015-09-24 |
2018-12-06 |
バイオマリン ファーマシューティカル インコーポレイテッド |
アデノ随伴ウイルス第viii因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤
|
PT3365438T
(pt)
*
|
2015-10-21 |
2021-07-12 |
Us Health |
Adnc com codão otimizado para atp7a de tamanho reduzido e utilização para o tratamento de distúrbios de transporte de cobre
|
US11253576B2
(en)
|
2015-10-22 |
2022-02-22 |
University Of Massachusetts |
Methods and compositions for treating metabolic imbalance in neurodegenerative disease
|
CA3002980A1
(en)
|
2015-10-22 |
2017-04-27 |
University Of Massachusetts |
Prostate-targeting adeno-associated virus serotype vectors
|
IL281038B2
(en)
|
2015-11-13 |
2024-02-01 |
Baxalta Inc |
Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy in hemophilia A
|
EA036944B1
(ru)
|
2015-11-13 |
2021-01-19 |
Баксалта Инкорпорейтед |
Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
|
JP7444521B2
(ja)
*
|
2015-12-01 |
2024-03-06 |
スパーク セラピューティクス インコーポレイテッド |
臨床使用に適した無血清懸濁細胞培養システムにおいて組換えアデノ随伴ウイルス(aav)ベクターを産生するスケーラブルな方法
|
AU2016362477A1
(en)
|
2015-12-02 |
2018-06-14 |
Voyager Therapeutics, Inc. |
Assays for the detection of AAV neutralizing antibodies
|
WO2017100682A1
(en)
*
|
2015-12-11 |
2017-06-15 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for treating familial hypercholesterolemia
|
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
|
CA3012344A1
(en)
|
2016-02-12 |
2017-08-17 |
University Of Massachusetts |
Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
|
KR102479894B1
(ko)
*
|
2016-03-30 |
2022-12-21 |
스파크 테라퓨틱스, 인코포레이티드 |
재조합 단백질 및/또는 바이러스 벡터 생산용 세포주
|
WO2017176929A1
(en)
|
2016-04-05 |
2017-10-12 |
University Of Massachusetts |
Compositions and methods for selective inhibition of grainyhead-like protein expression
|
MX2018012605A
(es)
*
|
2016-04-15 |
2019-07-04 |
Res Inst Nationwide Childrens Hospital |
Administracion de vectores del virus adenoasociado de b-sarcoglicano y microarn-29 y el tratamiento de la distrofia muscular.
|
WO2017181105A1
(en)
|
2016-04-15 |
2017-10-19 |
University Of Massachusetts |
Methods and compositions for treating metabolic imbalance
|
WO2017189964A2
(en)
|
2016-04-29 |
2017-11-02 |
Voyager Therapeutics, Inc. |
Compositions for the treatment of disease
|
EP3448874A4
(en)
|
2016-04-29 |
2020-04-22 |
Voyager Therapeutics, Inc. |
COMPOSITIONS FOR TREATING A DISEASE
|
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
|
KR20220108216A
(ko)
|
2016-05-18 |
2022-08-02 |
보이저 테라퓨틱스, 인크. |
헌팅톤 질환을 치료하기 위한 조성물 및 방법
|
US11882815B2
(en)
|
2016-06-15 |
2024-01-30 |
University Of Massachusetts |
Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
|
WO2018022511A1
(en)
|
2016-07-25 |
2018-02-01 |
The Trustees Of The University Of Pennsylvania |
Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
|
BR112019001532A2
(pt)
|
2016-07-26 |
2019-09-10 |
Biomarin Pharm Inc |
proteínas de capsídeo de vírus adenoassociado inovadoras
|
EP4219724A3
(en)
|
2016-08-23 |
2023-09-27 |
Akouos, Inc. |
Compositions and methods for treating non-age-associated hearing impairment in a human subject
|
CN106344932B
(zh)
*
|
2016-08-26 |
2019-12-27 |
首都医科大学附属北京同仁医院 |
一种用于提高视网膜退行性疾病基因治疗效果的组合物及其应用
|
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
|
US10457940B2
(en)
|
2016-09-22 |
2019-10-29 |
University Of Massachusetts |
AAV treatment of Huntington's disease
|
CN110506119A
(zh)
|
2016-10-13 |
2019-11-26 |
马萨诸塞大学 |
Aav衣壳设计
|
WO2018082093A1
(en)
*
|
2016-11-07 |
2018-05-11 |
Wuhan Institute Of Virology, Chinese Academy Of Sciences |
Anterograde monosynaptic transneuronal tracer system
|
WO2018131551A1
(ja)
|
2017-01-13 |
2018-07-19 |
学校法人自治医科大学 |
肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのaavベクター
|
WO2018152485A1
(en)
|
2017-02-20 |
2018-08-23 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for treating familial hypercholesterolemia
|
AU2018236123B2
(en)
|
2017-03-11 |
2024-04-18 |
Selecta Biosciences, Inc. |
Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
|
MA52112B1
(fr)
*
|
2017-03-17 |
2023-08-31 |
Res Inst Nationwide Childrens Hospital |
Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique du muscle pour traiter la dystrophie musculaire
|
JP7246359B2
(ja)
|
2017-04-03 |
2023-03-27 |
エンコーデッド セラピューティクス, インコーポレイテッド |
組織選択的導入遺伝子発現
|
EP3618839A4
(en)
|
2017-05-05 |
2021-06-09 |
Voyager Therapeutics, Inc. |
COMPOSITIONS AND TREATMENT METHODS FOR AMYOTROPHIC LATERAL SCLEROSIS (ALS)
|
JP2020518259A
(ja)
|
2017-05-05 |
2020-06-25 |
ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. |
ハンチントン病治療組成物および方法
|
AU2018264996A1
(en)
|
2017-05-09 |
2019-12-05 |
University Of Massachusetts |
Methods of treating Amyotrophic Lateral Sclerosis (ALS)
|
EP3624858A4
(en)
*
|
2017-05-19 |
2021-06-23 |
Encoded Therapeutics, Inc. |
HIGH ACTIVITY REGULATORY ELEMENTS
|
CN110945127B
(zh)
|
2017-05-22 |
2024-07-12 |
武田药品工业株式会社 |
用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体
|
JOP20190269A1
(ar)
|
2017-06-15 |
2019-11-20 |
Voyager Therapeutics Inc |
بولي نوكليوتيدات aadc لعلاج مرض باركنسون
|
AU2018302016A1
(en)
|
2017-07-17 |
2020-02-06 |
The Regents Of The University Of California |
Trajectory array guide system
|
JP2020530834A
(ja)
*
|
2017-07-17 |
2020-10-29 |
スパーク セラピューティクス インコーポレイテッドSpark Therapeutics, Inc. |
アフェレーシスの方法及び使用
|
CA3071978A1
(en)
|
2017-08-03 |
2019-02-07 |
Voyager Therapeutics, Inc. |
Compositions and methods for delivery of aav
|
US20210130845A1
(en)
*
|
2017-09-08 |
2021-05-06 |
Poseida Therapeutics, Inc. |
Compositions and methods for chimeric ligand receptor (clr)-mediated conditional gene expression
|
US11118192B2
(en)
|
2017-09-20 |
2021-09-14 |
4D Molecular Therapeutics Inc. |
Adeno-associated virus variant capsids and methods of use thereof
|
AU2018338188A1
(en)
|
2017-09-22 |
2020-04-02 |
University Of Massachusetts |
SOD1 dual expression vectors and uses thereof
|
JP7502991B2
(ja)
|
2017-10-16 |
2024-06-19 |
ボイジャー セラピューティクス インコーポレイテッド |
筋萎縮性側索硬化症(als)の治療
|
US20200237799A1
(en)
|
2017-10-16 |
2020-07-30 |
Voyager Therapeutics, Inc. |
Treatment of amyotrophic lateral sclerosis (als)
|
CN107760719B
(zh)
*
|
2017-10-24 |
2020-09-22 |
北京领柯生物科技有限公司 |
柯萨奇病毒在过继免疫基因递送系统中的应用
|
CA3083472A1
(en)
|
2017-11-27 |
2019-05-31 |
4D Molecular Therapeutics Inc. |
Adeno-associated virus variant capsids and use for inhibiting angiogenesis
|
WO2019109051A1
(en)
|
2017-12-01 |
2019-06-06 |
Encoded Therapeutics, Inc. |
Engineered dna binding proteins
|
CN111727243A
(zh)
*
|
2017-12-19 |
2020-09-29 |
北卡罗来纳-查佩尔山大学 |
用于跨越血-脑屏障递送病毒载体的方法和组合物
|
US11634728B2
(en)
|
2017-12-29 |
2023-04-25 |
Helixmith Co., Ltd |
Adeno-associated virus (AAV) vector having hybrid HGF gene introduced thereto
|
EP3740581A4
(en)
*
|
2018-01-16 |
2021-10-27 |
CLS Therapeutics Limited |
TREATMENT OF DISEASES BY LIVER EXPRESSION OF AN ENZYME WITH DESOXYRIBONUCLEASE (DNASE) ACTIVITY
|
US10610606B2
(en)
|
2018-02-01 |
2020-04-07 |
Homology Medicines, Inc. |
Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
|
EP3755795A4
(en)
|
2018-02-19 |
2022-07-20 |
Homology Medicines, Inc. |
ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RECOVERING F8 GENE FUNCTION AND METHODS OF USE THEREOF
|
TN2020000187A1
(en)
|
2018-04-05 |
2022-04-04 |
Univ Devry Val Dessonne |
Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism
|
SG11202010830WA
(en)
|
2018-05-09 |
2020-11-27 |
Biomarin Pharm Inc |
Methods of treating phenylketonuria
|
TW202005978A
(zh)
|
2018-05-14 |
2020-02-01 |
美商拜奧馬林製藥公司 |
新穎肝靶向腺相關病毒載體
|
MX2021000582A
(es)
|
2018-07-16 |
2021-04-12 |
Baxalta Inc |
Terapia genica de hemofilia a mediante el uso de vectores virales que codifican variantes del factor viii (fviii) recombinantes con mayor expresion.
|
CN113614104A
(zh)
|
2019-01-16 |
2021-11-05 |
百深公司 |
用于a型血友病的基因疗法的编码表达增加的重组fviii变体的病毒载体
|
US20220143215A1
(en)
*
|
2019-02-12 |
2022-05-12 |
Spacecraft Seven, Llc |
Gene therapy vectors for treatment of danon disease
|
BR112021016501A2
(pt)
|
2019-02-25 |
2021-10-26 |
Novartis Ag |
Composições e métodos para tratar distrofia cristalina de bietti
|
US20220154211A1
(en)
|
2019-02-25 |
2022-05-19 |
Novartis Ag |
Compositions and methods to treat bietti crystalline dystrophy
|
CN109999182B
(zh)
*
|
2019-03-29 |
2023-03-07 |
四川大学华西医院 |
凝血因子在制备抗肿瘤药物中的应用
|
EP4022057A1
(en)
|
2019-08-27 |
2022-07-06 |
Vertex Pharmaceuticals Incorporated |
Compositions and methods for treatment of disorders associated with repetitive dna
|
AU2020384294A1
(en)
|
2019-11-14 |
2022-06-02 |
Biomarin Pharmaceutical Inc. |
Treatment of hereditary angioedema with liver-specific gene therapy vectors
|
CN113025618B
(zh)
*
|
2019-12-24 |
2024-02-06 |
朗信启昇(苏州)生物制药有限公司 |
一种x连锁遗传性视网膜劈裂症的基因治疗方案与应用
|
TW202140791A
(zh)
|
2020-01-13 |
2021-11-01 |
美商霍蒙拉奇醫藥公司 |
治療苯酮尿症之方法
|
AU2021225035A1
(en)
|
2020-02-21 |
2022-10-13 |
Akouos, Inc. |
Compositions and methods for treating non-age-associated hearing impairment in a human subject
|
AR122409A1
(es)
|
2020-04-03 |
2022-09-07 |
Biomarin Pharm Inc |
Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas
|
TW202208632A
(zh)
|
2020-05-27 |
2022-03-01 |
美商同源醫藥公司 |
用於恢復pah基因功能的腺相關病毒組成物及其使用方法
|
WO2021257497A1
(en)
*
|
2020-06-15 |
2021-12-23 |
Sarepta Therapeutis, Inc. |
Adeno-associated virus antibodies and fragments thereof
|
CN111500635B
(zh)
*
|
2020-07-02 |
2020-10-09 |
北京大学第三医院(北京大学第三临床医学院) |
一种包含携带核酸分子的载体的试剂盒
|
CN115927398A
(zh)
*
|
2020-07-29 |
2023-04-07 |
舒泰神(北京)生物制药股份有限公司 |
一组肝靶向新型腺相关病毒的获得及其应用
|
JP2023538519A
(ja)
|
2020-08-07 |
2023-09-08 |
スペースクラフト セブン リミテッド ライアビリティ カンパニー |
Aavベクターを使用したプラコフィリン-2(pkp2)遺伝子治療
|
US20220096606A1
(en)
|
2020-09-09 |
2022-03-31 |
Vertex Pharmaceuticals Incorporated |
Compositions and Methods for Treatment of Duchenne Muscular Dystrophy
|
US12071633B2
(en)
|
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(en)
|
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|
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(pt)
|
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|
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(en)
*
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|
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(en)
|
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(ko)
|
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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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|
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(zh)
*
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