A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects With Choroideremia Previously Treated With Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects With X-Linked Retinitis Pigmentosa Previously Treated With Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study

The objective of the study is to evaluate the long-term safety and efficacy of a sub-retinal injection of BIIB111 in participants with Choroideremia (CHM) who have been previously treated with BIIB111 and who have exited an antecedent study; these treated participants will be compared with untreated control participants who have exited the STAR (NCT03496012) study and BIIB112 in participants with X-linked retinitis pigmentosa (XLRP) who have been previously treated with BIIB112 and who have exited an antecedent study.

开始日期2018-06-04

申办/合作机构

NightstaRx Ltd.

NCT03496012 / Completed临床3期

A Randomised, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of BIIB111 in participants with choroideremia (CHM).

开始日期2017-12-11

申办/合作机构

Biogen, Inc.

NCT03507686 / Completed临床2期

An Open-Label Safety Study of Retinal Gene Therapy for Choroideremia With Bilateral, Sequential Administration of Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

The objective of the study is to evaluate the safety of bilateral, sequential sub-retinal administration of a single dose of BIIB111 in adult male participants with Choroideremia (CHM).

开始日期2017-11-29

申办/合作机构

Biogen, Inc.

100 项与 Timrepigene emparvovec 相关的临床结果

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100 项与 Timrepigene emparvovec 相关的转化医学

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100 项与 Timrepigene emparvovec 相关的专利（医药）

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项与 Timrepigene emparvovec 相关的文献（医药）

2023-10-01·Nature medicine1区 · 医学

Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial.

1区 · 医学

Article

作者: Gow, James A ; Fischer, M Dominik ; Panda, Sushil ; Lam, Byron L ; Sankila, Eeva-Marja K ; Pennesi, Mark E ; MacLaren, Robert E ; Girach, Aniz ; Yoon, Dan ; Zhao, Guolin

Choroideremia is a rare, X-linked retinal degeneration resulting in progressive vision loss. A randomized, masked, phase 3 clinical trial evaluated the safety and efficacy over 12 months of follow-up in adult males with choroideremia randomized to receive a high-dose (1.0 × 10¹¹ vector genomes (vg); n = 69) or low-dose (1.0 × 10¹⁰ vg; n = 34) subretinal injection of the AAV2-vector-based gene therapy timrepigene emparvovec versus non-treated control (n = 66). Most treatment-emergent adverse events were mild or moderate. The trial did not meet its primary endpoint of best-corrected visual acuity (BCVA) improvement. In the primary endpoint analysis, three of 65 participants (5%) in the high-dose group, one of 34 (3%) participants in the low-dose group and zero of 62 (0%) participants in the control group had ≥15-letter Early Treatment Diabetic Retinopathy Study (ETDRS) improvement from baseline BCVA at 12 months (high dose, P = 0.245 versus control; low dose, P = 0.354 versus control). As the primary endpoint was not met, key secondary endpoints were not tested for significance. In a key secondary endpoint, nine of 65 (14%), six of 35 (18%) and one of 62 (2%) participants in the high-dose, low-dose and control groups, respectively, experienced ≥10-letter ETDRS improvement from baseline BCVA at 12 months. Potential opportunities to enhance future gene therapy studies for choroideremia include optimization of entry criteria (more preserved retinal area), surgical techniques and clinical endpoints. EudraCT registration: 2015-003958-41 .

2023-04-01·American journal of ophthalmology

AAV2-Mediated Gene Therapy for Choroideremia: 5-Year Results and Alternate Anti-sense Oligonucleotide Therapy

Article

作者: MacLaren, Robert E ; Dimopoulos, Ioannis S ; Crichton, Paul ; Tennant, Matthew T ; Somani, Rizwan ; Xu, Manlong ; Zhai, Yi ; MacDonald, Ian M ; Radziwon, Alina ; Mah, Rachel

PURPOSE:

To assess the long-term safety and efficacy of AAV2-REP1 in choroideremia (CHM) patients, and to test a potential antisense oligonucleotide therapy for CHM.

DESIGN:

Extended, prospective phase 1/2 clinical trial and laboratory investigation.

METHODS:

Five patients who received a single subfoveal injection of AAV2-REP1 were studied. The long-term safety was evaluated by ophthalmic examination, spectral domain optical coherence tomography, and fundus autofluorescence (FAF) for up to 5 years. Functional and structural changes were determined by different test modalities. Four antisense oligonucleotides (ASOs) were designed to treat the CHM c.1245-521A>G mutation, which was present in 2 patients within this trial.

RESULTS:

Subject P3 experienced a localized intraretinal immune response that resulted in a significant loss of preserved retinal pigment epithelium (RPE). P4 experienced an exacerbation of peripheral retinoschisis. P2 had a constant ≥15-letter best-corrected visual acuity (BCVA) gain in the treated eye, whereas P5 had ≥15-letter BCVA improvement once in the untreated eye. The preserved FAF areas declined more rapidly in the treated eyes compared to the untreated eyes (P = .043). A customized 25-mer ASO recovered 83.2% to 95.0% of the normal RNA and 57.5% of the normal protein in fibroblasts from 2 trial patients.

CONCLUSIONS:

Intraretinal inflammation triggered by AAV2-REP1 subretinal injection stabilized after 2 years but resulted in permanent damage to the retinal structure. Long-term progression of the disease was seen in both treated and untreated eyes, casting doubt as to the effectiveness of this approach in late-stage CHM. Alternative approaches such as ASO may have a therapeutic effect in a subgroup of CHM patients.

2020-01-01·Retina (Philadelphia, Pa.)2区 · 医学

CHANGES IN RETINAL SENSITIVITY AFTER GENE THERAPY IN CHOROIDEREMIA

2区 · 医学

Article

作者: Wilhelm, Barbara ; MacLaren, Robert E ; Ochakovski, G Alex ; Peters, Tobias ; Ueffing, Marius ; Seitz, Immanuel P ; Kortuem, Constanze ; Fischer, M Dominik ; Bartz-Schmidt, KarlUlrich ; Vaheb, Yousof ; Reichel, Felix F L ; Girach, Aniz ; Kuehlewein, Laura ; Zrenner, Eberhart ; Kahle, Nadine A ; Beier, Benjamin

Purpose::

Choroideremia (CHM) is a rare inherited retinal degeneration resulting from mutation of the CHM gene, which results in absence of functional Rab escort protein 1 (REP1). We evaluated retinal gene therapy with an adeno-associated virus vector that used to deliver a functional version of the CHM gene (AAV2-REP1).

Methods::

THOR (NCT02671539) is a Phase 2, open-label, single-center, randomized study. Six male patients (51–60 years) with CHM received AAV2-REP1, by a single 0.1-mL subretinal injection of 1011 genome particles during vitrectomy. Twelve-month data are reported.

Results::

In study eyes, 4 patients experienced minor changes in best-corrected visual acuity (−4 to +1 Early Treatment Diabetic Retinopathy Study [ETDRS] letters); one gained 17 letters and another lost 14 letters. Control eyes had changes of −2 to +4 letters. In 5/6 patients, improvements in mean (95% confidence intervals) retinal sensitivity (2.3 [4.0] dB), peak retinal sensitivity (2.8 [3.5] dB), and gaze fixation area (−36.1 [66.9] deg2) were recorded. Changes in anatomical endpoints were similar between study and control eyes. Adverse events were consistent with the surgical procedure.

Conclusion::

Gene therapy with AAV2-REP1 can maintain, and in some cases, improve, visual acuity in CHM. Longer term follow-up is required to establish whether these benefits are maintained.

项与 Timrepigene emparvovec 相关的新闻（医药）

2023-06-16

·生物谷

多位全球眼科基因治疗专家领衔的初创携1.2亿美元亮相，Ⅱ期临床数据即将公布

对于 Beacon Therapeutics 在研的基因疗法，Dave Fellows 信心满满，“我们当然致力于向前发展，并希望在特定市场实现商业化，但我们不会‘关闭任何大门’。”他表示。英国当地时间 6 月 12 日，眼科基因治疗公司 Beacon Therapeutics 携 9600 万英镑（约 1.2 亿美元）首次亮相。 Beacon Therapeutics 专注于开发新一代基因疗法以恢复和改善视网膜疾病患者的视力，主要投资者 Syncona 和包括 Oxford Science Enterprises（OSE）在内的其他投资者向该公司提供资金以加速产品管线推进。 ▲图｜Beacon Therapeutics 首席执行官 Dave Fellows（来源：公司官网） “Beacon Therapeutics 结合了深厚的科学基础、强大的临床网络以及经验丰富的管理团队，以推动一个后期临床管线和两个临床前管线。凭借我们 AGTC-501 临床 Ⅱ 期试验的 12 个月数据，以及两个针对常见和罕见致盲性眼科疾病的高度创新和差异化的产品管线，希望我们能够引领并推动眼科基因治疗领域的快速发展。”Beacon Therapeutics 首席执行官 David Fellows 表示。该公司声称，“预计将在今年晚些时候公布关于AGTC-501 临床 Ⅱ 期试验的 12 个月数据。” 据悉，AGTC-501 是 Beacon Therapeutics 的核心管线，目前已经进入治疗 X 连锁视网膜色素变性（XLRP）的 Ⅱ 期临床试验阶段。值得注意的是，该管线是 Syncona 于 2022 年 11 月收购的 AGTC 的一部分。据官网资料显示，XLRP 主要由视网膜色素变性 GTP 酶调节因子（RPGR）基因突变引起，与该领域的其他方法不同，AGTC-501 能够正确表达全长 RPGR 蛋白，从而解决了由 XLRP 引起的整个光感受器损伤，包括视杆细胞和视锥细胞的损失等。 ▲图｜Beacon Therapeutics 在研产品管线（来源：公司官网）其他管线方面，该公司的第一个临床前管线是一种玻璃体内递送的基于 AAV 的新型干性年龄相关性黄斑变性（干性AMD）基因治疗药物；第二个临床前管线的适应症为视锥杆细胞营养不良（CRD），这是一种遗传性视网膜疾病，由钙粘蛋白相关家族成员 1（CDHR1）基因突变引起。这项技术已获得牛津大学眼科教授 Robert MacLaren 教授实验室的许可，而他本人也是 Beacon Therapeutics 的联合创始人，担任该公司的科学顾问，并将加入董事会。值得注意的是，不只是 Robert MacLaren，Beacon Therapeutics 的高管团队，包括 David Fellows、Nadia Waheed，以及 Abraham Scaria 等均在眼科基因治疗领域拥有数十年的从业经验。 Dave Fellows 曾是 Nightstar Therapeutics 的前首席执行官。Nightstar Therapeutics（纳斯达克股票代码 NITE）是一家总部位于英国伦敦的临床阶段基因治疗公司，成立于 2013 年，并于 2017 年在纳斯达克上市。该公司专注于使用腺相关病毒（AAV）基因疗法治疗遗传性视网膜疾病，其主要候选药物为 NSR-REP1，用于治疗无脉络膜症（一种罕见的退行性 X 连锁遗传性视网膜疾病，CHM）。2019 年，该公司被渤健以 8.77 亿美元收购。另一家视网膜疾病基因疗法开发公司 Gyroscope Therapeutics 成立于 2016 年，总部位于英国，其主要候选药物名为 GT005，是一种基于 AAV2 的基因疗法，通过视网膜下给药用于治疗继发于年龄相关性黄斑变性（AMD）的黄斑地图状萎缩（GA），并且该基因疗法已获得 FDA 授权治疗黄斑地图状萎缩患者的快速通道资格。2021 年，诺华宣布以 8 亿美元预付款和 7 亿美元的潜在额外里程碑付款收购 Gyroscope Therapeutics。 ▲图｜Beacon Therapeutics 首席医疗官 Nadia Waheed（来源：公司官网） Nadia Waheed 曾在 Gyroscope Therapeutics 参与研究开发 GT005 基因疗法，如今，她是 Beacon Therapeutics 公司的首席医疗官，“目前我们正在开发的这种基因疗法能够正确表达全长 RPGR 蛋白，该蛋白在罕见眼科疾病中发生突变，而这是解决疾病中视杆细胞和视锥细胞的关键，通过治疗，患者的夜视能力和阅读、写作等各种日常活动有望恢复，这看起来非常令人鼓舞。”Nadia Waheed 表示。 “目前，我们已收到欧洲监管机构的初步反馈，并正在与 FDA 就全球注册临床试验进行谈判。”她补充说。 ▲图｜Beacon Therapeutics 首席科学官 Abraham Scaria（来源：公司官网） Beacon Therapeutics 领导团队中的首席科学官 Abraham Scaria 曾是 AGTC 团队的成员。据悉，AGTC 于 1999 年在佛罗里达州注册成立，是一家临床阶段的眼科疾病基因治疗公司，其研发出一种名为 AGTC-401 的针对色盲患者的基因疗法，通过视网膜下注射采用 AAV 包载的功能性人类 CNGB3 基因，进而恢复视锥细胞的正常功能。值得一提的是，Abraham Scaria 此前还曾担任 Iveric Bio 首席战略官。就在不久前，安斯泰来宣布以 59 亿美元的价格收购 Iveric Bio。该公司其开发的 ACP 是一种研究性的新型补体 C5 蛋白抑制剂药物，用于治疗年龄相关性黄斑变性继发的黄斑地图状萎缩。今年 2 月，FDA 接受了其提交的 ACP 新药申请（ NDA ），目前 NDA 已获得优先审查，处方药用户费用法案（PDUFA）目标日期为 2023 年 8 月 19 日，意味着该公司今年有望成为第二家获得黄斑地图状萎缩药物批准的公司。（来源：公司官网） Beacon Therapeutics 是专注于眼科基因疗法的数家新兴生物技术公司之一，近年来加入该领域的公司还包括 Ray Therapeutics、 Complement Therapeutics、Avista Therapeutics，以及 Replay 的 Eudora。对于 Beacon Therapeutics 在研的基因疗法，Dave Fellows 信心满满，“我们当然致力于向前发展，并希望在特定市场实现商业化，但我们不会‘关闭任何大门’。”他表示。

基因疗法临床2期

2022-12-01

·药研网

Aduhelm头对头，礼来阿尔兹海默症新药3期达主要终点

2022年11月30日，礼来公布其药物donanemab达III期试验达6个月所有主要终点和次要终点，为早期症状性阿尔茨海默病患者的淀粉样蛋白斑块清除提供了首个Aduhelm头对头比较数据。该III期试验（TRAILBLAZER-ALZ 4）在阿尔茨海默病临床试验（CTAD）会议上公布。主要终点为大脑淀粉样斑块清除（大脑淀粉样斑块水平<24.1 Centiloids）。donanemab组6个月时这一比例为37.9%，Aduhelm组仅1.6%。在一个关键的次要终点中，donanemab组在6个月时脑部淀粉样蛋白水平与基线相比降低了65.2%，Aduhelm组为17.0%。安全性方面，ARIA是两组中最常见的治疗突发不良事件。Aduhelm组总的ARIA发生率为26.1%，其中4.3%为症状性的；在donanemab组，总的ARIA发生率为25.4%，其中2.8%有症状。布朗大学阿尔茨海默病研究中心副主任Stephen表示：令人鼓舞的是，donanemab在6个月后明显减少了大脑中淀粉样蛋白的积累和血液中的P-tau，值得注意的是，与Aduhelm相比，donanemab在6个月时更高的淀粉样蛋白清除率与较高的ARIA率无关。Donanemab是靶向N3pG（β淀粉样斑块的一种修饰形式）的研究性抗体药物，据悉，Donanemab加速批准的PDUFA日期为2023年2月，加速审批是基于早期的II期试验，将加速批准转变为完全批准的验证性试验也将在2023年中期读出数据。目前TRAILBLAZER-ALZ 4还将进行12个月和18个月的二级分析，该研究也是评估donanemab疗效和安全性临床项目的五项研究之一。值得一提的是，在过去的20年里，被称为“天坑”的AD领域有百余款新药均研发失败，临床失败率高达97.3%，辉瑞、礼来、罗氏等制药巨头也曾几度折戟。已上市的Aduhelm，由于其临床疗效数据问题而几经挫折，经历了上市申请撤回、限制使用、销售业绩惨淡、销售团队解散等一系列问题。Biogen 2022年一季度财报中，销售额280万美元，不及预期。同时，Biogen/卫材的另一款Aβ药物Lecanemab三期临床达主要终点，近期也因安全问题争议不断，lecanemab加速批准的PDUFA日期为2023年1月6日。扫码查询现货细胞系往期推荐泛癌种靶点Trop2，国内各家竞相角逐PROTAC全球布局一览国产PD-1销售盘点点击下方“药研网”，关注更多精彩内容

临床3期财报临床2期加速审批临床结果

2022-11-22

·Business Wire

AAV Vectors in Gene Therapy - Pipeline Insight, 2022 - ResearchAndMarkets.com

DUBLIN--( BUSINESS WIRE )--The "AAV vectors in gene therapy - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering. AAV vectors in gene therapy: Overview Out of the several viral vectors that have been used to date for delivering the genes of interest, the Adeno-associated viral (AAV) vector appears to be the safest and effective vehicle and can maintain long-term gene and protein expression following a single injection of the vector. AAV vectors are the leading viral vectors for gene delivery to treat a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs, and harnessing revolutionary biotechnologies have contributed extensively to the growth of the gene therapy field. AAV Vectors in Gene Therapy Emerging Drugs GS010: Gensight Biologics LUMEVOQ (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. "LUMEVOQ" was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018. Valoctocogene roxaparvovec: BioMarin Pharmaceutical Valoctocogene roxaparvovec is an investigational AAV5 gene therapy under regulatory review for the treatment of severe hemophilia A. It is currently in preregistration stage of development. In June 2021, BioMarin resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA). In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase III GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA. DTX401: Ultragenyx Pharmaceutical DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase-? under control of the native promoter. DTX401 is administered as a single intravenous infusion and has been shown in preclinical studies to improve G6Pase-? activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression. In a Phase I/II clinical study, all nine patients showed a clinical response, with significant reductions in the need for cornstarch and improvements in glucose control and other metabolic parameters compared to baseline. The drug is currently being evaluated in Phase III clinical trial to treat patients with Glycogen storage disease type I. AAV5-RPGR: MeiraGTx AAV-RPGR is an investigational gene therapy for the treatment of patients with X-linked retinitis pigmentosa (XLRP) caused by disease-causing variants in the eye specific form of the RPGR gene (RPGR ORF15). AAV-RPGR is designed to deliver functional copies of the RPGR gene to the subretinal space in order to improve and preserve visual function. MeiraGTx and development partner Janssen are currently conducting a Phase III clinical trial of AAV-RPGR in patients with XLRP with disease-causing variants in RPGR ORF15. Timrepigene emparvovec: Biogen Timrepigene emparvovec is an AAV2 vector administered by subretinal injection, which aims to provide a functioning CHM gene and expression of the REP-1 protein to restore membrane trafficking and thereby slow, stop or potentially reverse decline in vision. Data from the Phase 1/2 studies demonstrated a slower rate of decline in visual acuity in patients treated with timrepigene emparvovec compared to untreated patients in the natural history study. In addition, some patients treated with timrepigene emparvovec showed improvements in visual acuity. The studies also demonstrated that timrepigene emparvovec was generally well tolerated with an acceptable safety profile. The safety and efficacy of a single subretinal injection of timrepigene emparvovec is currently being evaluated in the ongoing Phase III STAR study. AMT 061: UniQure AMT-061 (etranacogene dezaparvovec) is an experimental gene therapy that uniQure is developing to treat hemophilia B. AMT-061 uses a modified and harmless adeno-associated virus 5 (AAV5) to deliver a highly functional copy of the F9 gene, called FIX-Padua, to patients' cells. The FIX-Padua gene version was shown to result in FIX clotting activity eight times greater than that associated with the standard F9 gene. As such, the one-time therapy - administrated directly into the bloodstream - is expected to increase FIX levels, helping to prevent and control bleeds. If approved, the therapy will be marketed globally by CSL Behring, which closed a commercialization and licensing agreement with uniQure in May 2021. UniQure and CSL Behring expect to file a regulatory application to the U.S. Food and Drug Administration (FDA) in early 2022 seeking AMT-061's approval for hemophilia B. Research and Development. RGX-314: REGENXBIO RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina. REGENXBIO is advancing research in two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye. According to the company's pipeline, the drug is currently in the Phase III stage of development. SPK-8011: Spark Therapeutics Investigational SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the AAV-LK03 capsid, also referred to as Spark200, contains a codon-optimized human factor VIII gene under the control of a liver-specific promoter. The Food and Drug Administration (FDA) granted orphan-disease designation and breakthrough therapy designation in the US, while the European Commission has granted orphan designation to SPK-8011. NFS-01: Neurophth Investigational NR082, a novel recombinant adeno-associated viral serotype 2 vector (rAAV2) containing a codon-optimized of NADH-dehydrogenase subunit 4 (ND4) gene under the control of the cytomegalovirus promoter and enhancer, is a novel ophthalmic injection that is being developed for the treatment of Leber hereditary optic neuropathy (LHON) associated with ND4 mutations. It is currently being evaluated in Phase II/III clinical trial. GT 005: Gyroscope Therapeutics GT005 is designed as an AAV2-based one-time investigational gene therapy for GA secondary to AMD that is delivered under the retina. GT005 aims to restore balance to an overactive complement system, a part of the immune system, by increasing production of the CFI protein. Complement overactivation has been strongly correlated with the development and progression of AMD. The CFI protein regulates the activity of the complement system. It is believed that increasing CFI production could dampen the system's overactivity and reduce inflammation, with the goal of preserving a person's eyesight. Gyroscope is also evaluating GT005 in two Phase II clinical trials. Further product details are provided in the report AAV vectors in gene therapy: Therapeutic Assessment This segment of the report provides insights about the different AAV vectors in gene therapy drugs segregated based on following parameters that define the scope of the report, such as: Major Players in AAV vectors in gene therapy There are approx. 70+ key companies which are developing the therapies for AAV vectors in gene therapy. The companies which have their AAV vectors in gene therapy drug candidates in the most advanced stage, i.e. Preregistration include, BioMarin Pharmaceutical. Phases The publisher's report covers around 235+ products under different phases of clinical development like Late stage products (Phase III) Mid-stage products (Phase II) Early-stage product (Phase I) along with the details of Pre-clinical and Discovery stage candidates Discontinued & Inactive candidates Route of Administration Key Players BioMarin Pharmaceutical Gensight Biologics PTC therapeutics Ultragenyx Pharmaceutical MeiraGTx Pfizer Biogen uniQure Pfizer Ultragenyx Pharmaceutical REGENXBIO Biogen Spark therapeuics (Roche) Sarepta therapeutics Neurophth Therapeutics LYSOGENE Gyroscope Therapeutics Nanoscope Therapeutics Homology medicines Ultragenyx Pharmaceutical Passage Bio Freeline therapeutics Astellas Pharma Aspa therapeutics Adrenas Therapeutics ESTEVE Sio Gene Therapies Amicus therapeutics 4D Molecular therapeutics Taysha Gene Therapies Atsena Therapeutics BioMarin Pharmaceutical Abeona Therapeutics REGENXBIO uniQure Taysha Gene Therapies Asklepios BioPharmaceutical Sarepta Therapeutics Abeona Therapeutics Forge Biologics Sangamo therapeuics Bayer LogicBio therapeutics Solid Biosciences Applied Genetic Technologies Spark therapeuics (Roche) Eli lilly and company Lexeo Therapeutics Spark therapeutics Gensight Biologics Alcyone Lifesciences MeiraGTx Audentes therapeutics Vivet Therapeutics Takeda Astellas Gene Therapies Homology Medicines Adverum Biotechnologies Libella Gene Therapeutics Rocket Pharmaceuticals Brain Neurotherapy Bio, Inc. Ocugen Asklepios BioPharmaceutical Neurogene Jaguar Gene Therapy, LLC Axovia Therapeutics Coave therapeutics Novartis/ Arctos medical Novartis/Vedere Bio REGENXBIO Inc. StrideBio Abeona therapeutics Sanofi/ Sirion therapeutics Capsida Biotherapeutics Kriya therapeutics Neurophth Poseida Therapeutics, BridgeBio Pharma DiNAQOR/ Biomarin ViGeneron AskBio/Selecta Biosciences Prevail therapeutics Intas Pharmaceuticals Tenaya Therapeutics Scout Bio therapeutics Ocugen Oyster Point pharmaceuticals Cyprium Therapeutics Solid Biosciences Amicus Therapeutics Coave therapeutics AvantiBio Voyager Therapeutics/ Pfizer Aruvant Sciences Alcyone Lifesciences Gene Therapy Research Institution Co., Ltd. For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/ylu69s .

临床3期孤儿药基因疗法引进/卖出临床结果

100 项与 Timrepigene emparvovec 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB16018

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
X连锁视网膜色素变性	临床3期	美国	NightstaRx Ltd.	2018-06-04
X连锁视网膜色素变性	临床3期	巴西	NightstaRx Ltd.	2018-06-04
X连锁视网膜色素变性	临床3期	加拿大	NightstaRx Ltd.	2018-06-04
X连锁视网膜色素变性	临床3期	丹麦	NightstaRx Ltd.	2018-06-04
X连锁视网膜色素变性	临床3期	芬兰	NightstaRx Ltd.	2018-06-04
X连锁视网膜色素变性	临床3期	法国	NightstaRx Ltd.	2018-06-04
X连锁视网膜色素变性	临床3期	德国	NightstaRx Ltd.	2018-06-04
X连锁视网膜色素变性	临床3期	荷兰	NightstaRx Ltd.	2018-06-04
X连锁视网膜色素变性	临床3期	英国	NightstaRx Ltd.	2018-06-04
无脉络膜症	临床3期	美国	-	2017-09-21

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03507686 (CTgov)	临床2期	无脉络膜症	66	BIIB111 (Treatment Period 1: BIIB111)	鹹鏇窪壓鹹憲襯蓋襯淵(壓築膚構簾網膚鏇襯觸) = 願襯襯顧獵齋築願顧壓廠鬱觸窪構窪醖鹹夢憲 (網鹽蓋壓淵廠鬱窪廠繭, 鹹選製積鹽窪網網築窪 ~ 繭選壓構齋鬱選淵醖鹹) 更多	-	2023-09-18
NCT03507686 (CTgov)	临床2期	无脉络膜症	66	BIIB111 (Treatment Period 2: BIIB111)	鹹鏇窪壓鹹憲襯蓋襯淵(壓築膚構簾網膚鏇襯觸) = 願鬱壓醖鏇積齋網製憲廠鬱觸窪構窪醖鹹夢憲 (網鹽蓋壓淵廠鬱窪廠繭, 獵構願簾餘積簾壓範餘 ~ 夢鑰遞鹹蓋襯鹹繭願夢) 更多	-	2023-09-18
NCT02553135 (CTgov)	临床2期	无脉络膜症	6	Injection of AAV2-REP1 (10e11 vg)	繭艱鹽餘鹹鹹觸齋繭鬱(糧願衊觸壓憲範壓夢衊) = 淵遞鏇構顧構壓鹽築構鏇構遞製鬱鏇繭憲顧廠 (衊鹹獵觸淵顧構淵襯淵, 鹹築鹹鹹鹹餘蓋積網齋 ~ 鹹選選顧獵顧築蓋鬱選) 更多	-	2019-07-29
NSR-REP1 Phase 1/2I/II (AAO2018)	临床1/2期	无脉络膜症 AAV2 vector encoding REP1	32	NSR-REP1	膚襯鏇醖鑰膚願糧簾淵(網構襯廠鹽繭願蓋遞廠) = 廠網夢廠鏇醖構壓襯鏇顧窪糧醖積網網鹹憲襯 (憲觸積膚窪艱醖選鹹衊, -0.1 ~ 5.7)	积极	2018-10-29
PO221 (AAO2018)	临床1/2期	无脉络膜症 AAV2-NABs	42	NSR-REP1 gene therapy	構鏇繭廠齋窪鏇膚願繭(顧顧廠齋願夢觸壓構糧) = 壓壓繭繭艱選襯鹹觸廠醖鹽積鏇蓋鏇築膚餘築 (艱餘構顧蓋襯膚繭獵壓 )	积极	2018-10-28
NCT01461213 (Pubmed) 人工标引	临床1/2期	无脉络膜症	6	AAV.REP1	壓獵窪衊繭齋鏇窪鏇餘(簾鑰衊齋選簾糧齋顧夢) = 齋廠製齋壓願選構遞廠糧繭餘簾蓋壓獵遞淵窪 (觸獵鹹衊醖窪鏇蓋窪鹹 ) 更多	积极	2014-03-29
NCT01461213 (Pubmed) 人工标引	临床1/2期	无脉络膜症	6	Placebo	簾醖襯醖積醖膚夢衊鹽(獵餘壓願夢夢繭積鑰簾) = 鹹繭齋壓鏇鹽膚窪壓淵廠壓網鹽製鹹鹽蓋積製 (艱淵壓蓋膚襯鬱觸廠襯, 0.9)	积极	2014-03-29