An Operationally Seamless Phase 2b/3, Multicenter, Randomized, Masked, Sham-controlled Study to Evaluate the Efficacy and Safety of Surabgene Lomparvovec (Sura-vec) Delivered Via Suprachoroidal Space (SCS) Injection Targeting Subjects With Diabetic Retinopathy Without Center Involved-Diabetic Macular Edema (CI-DME) (NAAVIGATE)

Diabetic Retinopathy (DR) is a common eye condition caused by diabetes, where high blood sugar levels damage the blood vessels in the back part of the eye (called the retina). Over time, this damage can lead to vision problems and even blindness if not treated. This study will assess surabgene lomparvovec (sura-vec) as a potential one-time gene therapy administered in the suprachoroidal space (SCS) for the treatment of diabetic retinopathy (DR) and prevention of vision-threatening events (VTEs) in participants with non-proliferative DR (NPDR) without center-involved diabetic macular edema (CI-DME).
This study will consist of 3 portions: a Phase 2b portion, a Phase 3 portion, and a bilateral treatment portion. Approximately 576 adult participants will be enrolled in the study across multiple sites in the United States and Puerto Rico.
In the Phase 2b and Phase 3 portions, participants will be randomized to different groups to receive sura-vec and prophylactic steroids or sham and artificial tears in their study eye. If assigned to sham, participants will be given an opportunity to cross over and receive treatment with sura-vec. In the bilateral treatment portion, participants will be enrolled to receive sura-vec and prophylactic steroids in both eyes. In all 3 portions, follow-up in the study will continue through 5 years following administration of sura-vec in each eye.
There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

开始日期2026-06-01

申办/合作机构

AbbVie, Inc.

NCT07007065

/ Recruiting临床3期

A Randomized, Controlled, Partially Masked, Phase 3b Study to Assess the Injection Burden, Efficacy, Safety, and Long-Term Preservation of Visual Acuity of Surabgene Lomparvovec (ABBV-RGX-314) in a Real-World Context in Subjects With Neovascular Age-Related Macular Degeneration (nAMD)

Neovascular age-related macular degeneration (nAMD), also known as "wet" AMD, is the abnormal growth of new blood vessels in the light-sensitive tissue at the back of the eye called the retina. The purpose of this study is to assess how safe and effective Surabgene Lomparvovec is in treating participants with Neovascular age-related macular degeneration (nAMD).
Surabgene Lomparvovec (ABBV-RGX-314) is an investigational gene therapy being developed for the treatment of neovascular age-related macular degeneration (nAMD). Participants will be placed into 1 of 3 groups, called treatment arms. Each group receives different treatment. Adult participants aged 50 and older years with a diagnosis of previously treated nAMD will be enrolled. Around 561 participants will be enrolled in the study at approximately 150 sites worldwide.
Participants in groups 1 and 2 will receive a single subretinal dose of ABBV-RGX-314. Participants in group 3 will receive Ranibizumab as needed throughout the study. Ranibizumab will be given as an intravitreal injection (injection into the jelly-like tissue that fills the eyeball injection), and ABBV-RGX-314 will be given as a subretinal (between the retina and the back of the eye) injection. The Assessment Period begins after randomization (1:1:1) to one of the ABBV-RGX-314 treatment groups or control at Week -2 and lasts up to 5 years.
There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular monthly visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

开始日期2025-11-05

申办/合作机构

AbbVie, Inc.

NCT06942520

/ Recruiting临床2期

A Randomized, Open Label, Controlled, Phase 2 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 AAV Gene Therapy Administered Via Subretinal Delivery in Participants With Center Involved Diabetic Macular Edema

Phase 2 open label, randomized, active controlled, dose-ranging trial in adults with Center Involved - Diabetic Macular Edema (CI - DME)

开始日期2025-03-18

申办/合作机构

Sierra Eye Associates

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100 项与 Surabgene Lomparvovec 相关的临床结果

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100 项与 Surabgene Lomparvovec 相关的转化医学

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100 项与 Surabgene Lomparvovec 相关的专利（医药）

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项与 Surabgene Lomparvovec 相关的文献（医药）

2026-07-01·GENE THERAPY

AAV-based gene therapies for neovascular AMD

Review

作者: Song, Jae Yoon ; Kwon, Chan You ; Yoo, Hee Chan ; Kim, Tae Hee

Abstract:

Neovascular age-related macular degeneration (nAMD) is a major cause of irreversible vision loss in the elderly, driven by choroidal neovascularization and dysregulated vascular endothelial growth factor (VEGF) signaling. While anti-VEGF injections have transformed management, their frequent administration imposes a substantial burden on patients and limits adherence. Adeno-associated virus (AAV)-based gene therapy offers sustained intraocular delivery of anti-angiogenic agents with a single treatment, potentially overcoming these limitations. This review summarizes the rationale for AAV use in ocular gene therapy, compares major delivery routes, and highlights leading clinical candidates, including RGX-314, ADVM-022, 4D-150, and NG101. Advances in vector engineering, promoter optimization, and immune modulation are discussed alongside key challenges such as preexisting immunity and inflammation. Future directions include next-generation capsids, combination regimens, and precision patient selection. Collectively, these developments position AAV-based gene therapy as a promising strategy to redefine the therapeutic landscape of nAMD.

2025-10-03·Translational Vision Science & Technology

Computational Fluid Dynamics Modeling of Intravitreal Ranibizumab Bolus Versus Subretinal ABBV-RGX-314 Transgene Product in Human Eyes

Article

作者: Jenny Park ; Mohammad Kazemi ; Jie Shen ; Mitalee Tamhane

Purpose:

ABBV-RGX-314 is being developed for neovascular age-related macular degeneration (nAMD). Computational fluid dynamics (CFDs) modeling in the eye enables simulation of drug distribution incorporating geometry and substructures of the eye across species. Given the similarity between ranibizumab and ABBV-RGX-314 transgene product (TP), ranibizumab intraocular pharmacokinetic (PK) data from literature were used to simulate intraocular drug distribution of ABBV-RGX-314 TP. This investigation aims to use CFD modeling to estimate retinal TP level based on aqueous humor (AH) TP level following subretinal (SR) injection of ABBV-RGX-314 in patients with nAMD.

Methods:

Ocular distribution of ranibizumab following a single intravitreal (IVT) injection was modeled in both monkey and human eyes independently. Following model validation, ABBV-RGX-314 TP distribution in human eyes was simulated following retinal transduction of ABBV-RGX-314.

Results:

Iterative simulations were performed to achieve similar AH ABBV-RGX-314 TP levels in patients with nAMD from phase I/IIa Study RGX-314-001. The CFD simulation estimated corresponding retinal TP concentrations of 1.86 to 5.50 µg/g at steady-state, which was assumed to be reached by 28 days and falls within the range of the estimated retinal ranibizumab trough retinal ranibizumab concentration (Ctrough; 0.718-5.37 µg/g) following monthly and every other month (EOM) dosing of 0.5 mg ranibizumab in patients with nAMD.

Conclusions:

The current study results predict that the 2 pivotal trial ABBV-RGX-314 doses (6.4E10 and 1.3E11 genome copies/eye) are expected to achieve and maintain sufficient retinal ABBV-RGX-314 TP levels for the treatment of nAMD.

Translational Relevance:

CFD modeling effectively bridges limited human ocular PK data with rich preclinical data, supporting model-informed drug development (MIDD) for clinical dose selection.

2025-07-01·International ophthalmology clinics

Gene Therapy in Age-related Macular Degeneration

Review

作者: Zhou, Henry W ; Kim, Leo A

Recent advances in gene therapy and salient features of the current AMD therapeutic landscape have led to increased interest in applying gene therapy approaches to AMD. This review will discuss approaches to drug administration, viral and non-viral delivery vectors, and current trials in gene therapy for both wet and dry AMD. Drug administration routes include subretinal, intravitreal, and suprachoroidal approaches. Viral vectors include adenoviral, lentiviral, and adeno-associated viral (AAV) vectors. Non-viral vectors include lipid nanoparticle (LNP) and polymer-based vectors. Current trials in wet AMD include ADVM-022 and RGX-314. Current trials in dry AMD include GT-005 and JNJ-1887.

245

项与 Surabgene Lomparvovec 相关的新闻（医药）

2026-09-28

·靶点AI说

Kodiak两款ABC 平台长效眼科药Ⅲ期成功：抗VEGF之后，眼科真正的机会在哪里？

靶点AI说｜aiXcure 2026年9月28日，Kodiak Sciences公布DAYBREAK Ⅲ期研究结果。在治疗初治湿性年龄相关性黄斑变性（wAMD）患者中，Kodiak的两款候选药物——长效抗VEGF抗体-生物聚合物偶联物 Zenkuda（tarcocimab tedromer），以及同时抑制VEGF和IL-6的双功能分子 tabirafusp-ted（KSI-501）——均在一年最佳矫正视力改善方面达到相对于aflibercept的非劣效主要终点。(Kodiak Sciences Inc.) 这项结果值得关注的地方，不只是Kodiak终于获得了一组积极的Ⅲ期数据。它实际上展示了眼科生物药开发正在出现的两条不同路线：第一条路线，是把已经得到充分验证的VEGF机制做得更长效；第二条路线，则是在长效基础上进一步加入炎症通路，希望突破单纯VEGF抑制的疗效天花板。 ⸻ 一、Zenkuda并没有寻找新的靶点，而是在重新设计抗VEGF的药代动力学 Zenkuda原名tarcocimab tedromer/KSI-301，其核心仍然是抗VEGF，但Kodiak没有把竞争重点放在寻找新的血管生成靶点，而是利用自己的 ABC（Antibody Biopolymer Conjugate）平台，把抗体与高分子量生物聚合物连接起来。这个设计希望解决眼科抗VEGF治疗长期存在的核心矛盾：抗VEGF本身已经非常有效，但是药物在眼内维持有效浓度的时间仍然不够长。 Kodiak披露，Zenkuda在人体眼内的平均半衰期约为20天，大约是现有获批治疗的3倍。DAYBREAK使用的制剂同时强调早期快速起效和后期持续暴露，因此它追求的并不是简单提高峰值药物浓度，而是延长有效治疗窗口。(Kodiak Sciences Inc.) DAYBREAK采用了相当严格的“treat-to-dryness”策略，只要OCT仍检测到视网膜液体，就可以触发再次治疗。即使在这样的标准下，一年时仍有54%的Zenkuda患者能够达到24周，也就是约6个月的给药间隔。与此同时，Zenkuda达到一年BCVA非劣效主要终点，Kodiak报告的p值为0.0007；研究中Zenkuda组眼内炎症发生率为0%。(Kodiak Sciences Inc.) 因此，Zenkuda真正试图建立的产品属性可以概括为：抗VEGF疗效不打折，同时把部分患者从一年多次玻璃体注射逐渐推向一年约两次治疗。 ⸻ 二、Kodiak为什么还要做VEGF×IL-6？如果Zenkuda已经可以把VEGF抑制做到半年一次，那么为什么还需要第二个分子？因为另一个问题仍然没有解决：并不是所有视网膜血管疾病都完全由VEGF驱动。部分wAMD和DME患者即使接受充分抗VEGF治疗，仍可能存在视网膜液体、血视网膜屏障异常以及慢性炎症状态。 Kodiak因此设计了tabirafusp-ted。这个分子同时包含两种功能： VEGF trap负责抑制VEGF介导的血管生成和血管通透性，而抗IL-6部分负责抑制炎症及相关血视网膜屏障异常。它同样使用Kodiak的ABC长效技术。(Kodiak Sciences) DAYBREAK中，tabirafusp-ted同样达到一年视力非劣效主要终点，p值为0.0036，同时达到预设的解剖学关键次要终点，p值低于0.0001。眼内炎症发生率为0.4%。(Kodiak Sciences Inc.) 但是这里有一个非常重要的研发信号。 Kodiak并没有宣布VEGF×IL-6在整个wAMD人群中已经证明优于单纯抗VEGF。相反，公司明确表示，下一步将进行post-hoc分析，寻找哪些治疗初治wAMD患者亚群真正能够从IL-6抑制中获益。(Kodiak Sciences Inc.) 这可能比“非劣效”本身更加重要。 ⸻ 三、AI视角：未来眼科双抗不应该先问“还能加什么靶点”，而应该先问“哪些患者需要第二个靶点” 过去抗体工程很容易形成一种研发思维： VEGF有效，那么VEGF×Ang2是否更好？ VEGF仍然有残余液体，那么VEGF×IL-6是否更好？如果还有炎症和纤维化，再加入第三条通路是否更好？但眼科尤其不适合无限增加靶点。因为玻璃体腔是一个体积有限、免疫环境特殊，而且对炎症极其敏感的给药空间。因此，下一代眼科多特异性药物更合理的研发模式可能是：先利用OCT影像、液体分布、病灶形态、既往抗VEGF反应、aqueous humor蛋白组以及炎症因子建立患者亚型，再决定应该增加哪一个第二机制。例如，对于一个标准VEGF高度依赖型患者，最好的药物可能不是复杂双抗，而只是一个安全、强效并且能够维持6个月的抗VEGF。但是对于存在持续性液体、炎症特征或者血视网膜屏障异常的患者，VEGF×IL-6、VEGF×Ang2或者其他组合才可能真正体现第二靶点价值。这也是为什么Kodiak后续针对IL-6获益人群的分层分析值得重点关注。 ⸻ 四、眼科下一阶段还有哪些值得布局的策略？ 1. 第一类机会：把“半年一次”进一步推向“一次治疗、长期表达” Zenkuda代表的是蛋白药长效化。但另外一条路线正在快速推进：让眼睛自己持续产生抗VEGF蛋白。 4D Molecular Therapeutics的4D-150采用单次玻璃体内AAV给药，同时表达aflibercept并利用RNA干扰抑制VEGF-C。2026年公布的PRISM Ⅱb两年数据显示，在Ⅲ期剂量组中仍观察到持续的治疗负担下降，目前项目已经进入Ⅲ期开发。(4D Molecular Therapeutics) AbbVie/REGENXBIO开发的sura-vec（ABBV-RGX-314）则通过AAV表达抗VEGF抗体片段，其湿性AMD关键性研究数据预计于2026年第四季度公布。(Regenxbio) 因此眼科长效治疗实际上已经形成三级竞争：长效蛋白 → 植入式持续释放 → 一次性基因治疗。真正需要比较的不只是BCVA，而是多年累计注射次数、安全性、可逆性、炎症风险以及总治疗成本。 ⸻ 2. 第二类机会：可补充、可逆的眼内“药物仓库” 基因治疗最大的优势是长期表达，但最大的挑战同样来自长期表达。一旦表达水平过高、过低或者出现安全性问题，很难像普通药物一样简单停药。因此，可补充的眼内储库可能形成另一个重要方向。 Roche的Susvimo采用可重复补充的Port Delivery System持续释放ranibizumab。2026年9月欧盟批准其用于nAMD，采用每24周补充一次的方案；Roche披露长期研究中约95%的患者在计划补充前不需要额外抗VEGF治疗。(罗氏) 这说明未来眼科药物的竞争单位可能不再只是“一个抗体”，而是：抗体 + formulation + implant + refill system。 ⸻ 3. 第三类机会：从VEGF×Ang2继续向“VEGF×疾病第二驱动因素”扩展 Faricimab已经验证了VEGF×Ang2双通路模式具有临床可行性。 Kodiak现在进一步测试VEGF×IL-6。下一步值得研究的并不是机械地寻找更多双抗组合，而是建立疾病机制矩阵。例如： VEGF + inflammation 可以研究IL-6、IL-1家族以及其他与血视网膜屏障炎症相关的机制。 VEGF + vascular stability 可以继续围绕Ang/Tie等血管稳定机制展开。 VEGF + fibrosis 对于长期nAMD治疗后形成纤维化和瘢痕、最终导致不可逆视力损失的患者，抗VEGF并不能解决所有问题，因此抗纤维化可能成为非常重要的第二机制。 VEGF + complement / innate immunity AMD本身与补体及先天免疫异常高度相关，但这种组合需要特别谨慎地区分湿性AMD的渗漏控制与AMD基础疾病进展。真正有价值的双抗应该解决一个明确的抗VEGF残余疾病表型，而不是单纯增加一个“理论上相关”的靶点。 ⸻ 4. 第四类机会：眼科抗体真正值得做的是“小型化 + 高浓度 + 长效化” 眼科与系统给药抗体存在一个非常大的差异：给药体积非常有限。因此，同样一个双抗分子，系统给药时可以接受的黏度、聚集倾向和表达量，在玻璃体注射制剂中可能完全不可接受。未来眼科抗体工程的重要指标应该同时包括：高效价、低黏度、高溶解度、高浓度制剂稳定性、低聚集、低免疫原性以及良好的玻璃体扩散。因此Fab、VHH、scFv、VEGF trap以及不同的小型双功能蛋白都值得重新评估。眼科双抗不一定需要传统150 kDa IgG结构。谁能在50–100 mg/mL甚至更高浓度下仍保持良好的稳定性和可制造性，谁可能拥有非常实际的产品优势。 ⸻ 五、AI在眼科药物开发中最值得切入的地方，可能不是“设计一个新抗体” DAYBREAK本身已经透露了一个很有意思的方向。 Kodiak在研究中采用了基于疾病活动度进行个体化再治疗的策略，并提到AI-guided retreatment algorithm。这意味着眼科可能是AI真正容易形成闭环的治疗领域之一。(Kodiak Sciences Inc.) 原因很简单：眼科拥有大量高度结构化、可以连续量化的影像数据。 OCT可以告诉我们视网膜液体是否存在、位于哪里、体积多少以及治疗后如何变化。因此未来可以形成： OCT影像 + 临床表型 + 分子标志物 + 药代动力学 + 给药历史 → AI患者分型 → 药物选择 → 给药间隔预测。 AI甚至可以进一步回答：这个患者到底只需要VEGF？还是需要VEGF×Ang2？还是属于IL-6高炎症型患者，更适合VEGF×IL-6？以及这个患者究竟应该Q8W、Q16W还是Q24W？如果能够做到这一点，眼科药物研发就会从今天的“所有患者接受同一种分子”逐渐走向：不同疾病生物学对应不同分子，不同药物清除速度对应不同给药间隔。 ⸻ 六、从药物研发角度，我更关注五个机会 Kodiak这次Ⅲ期结果之后，眼科值得关注的机会已经不只是再做一个抗VEGF。第一，是半年级长效蛋白。通过聚合物偶联、Fc/蛋白工程、高浓度制剂或者其他眼内滞留技术，在不改变成熟靶点的情况下显著降低注射频率。第二，是机制驱动的双功能分子。围绕VEGF之外真正导致抗VEGF残余疾病的炎症、血管稳定和纤维化机制设计第二靶点，而不是简单堆叠热门靶点。第三，是一次性或超长效基因治疗。 AAV持续表达抗VEGF甚至双通路分子，有机会把治疗频率从“每半年一次”进一步变成“多年一次”。第四，是可补充、可调整的持续递送系统。它可能在普通蛋白药和不可逆长期表达的基因治疗之间找到一个非常重要的产品位置。第五，也是AI最值得进入的方向，是建立“患者亚型—分子机制—给药间隔”的闭环。未来真正有竞争力的眼科平台，可能不是拥有一个最强的抗VEGF抗体，而是能够回答三个问题：这个患者还需要抑制什么？这个药物怎样才能在眼内维持更久？这个患者什么时候才真正需要下一针？ ⸻ 靶点AI说 Kodiak DAYBREAK真正值得关注的，不是“又有两款药做到了对aflibercept非劣效”。更重要的是，它把下一代视网膜药物的两种产品逻辑放在了同一个Ⅲ期研究中： Zenkuda代表“成熟靶点 × 极致长效化”，tabirafusp-ted代表“长效化 × 多机制干预”。而从整个行业来看，另一边还有Susvimo代表的持续递送，以及4D-150、sura-vec代表的一次性基因治疗。所以，下一轮眼科竞争可能已经不再是：谁拥有更强的抗VEGF。而是：谁能够在保持视力和解剖学控制的同时，把治疗从每月一次推到半年一次、几年一次；并进一步通过患者分层，找到真正需要第二条疾病通路的人群。这可能才是Kodiak这组DAYBREAK数据对眼科药物研发最大的启示。

临床3期临床结果临床终止

2026-09-26

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基因测序与药物研发每日资讯 · 2026-09-26

基因测序与药物研发每日资讯 · 2026 年 9 月 26 日当日资讯摘要基因测序：英国 NHS 启动 210 万英镑试点，用纳米孔测序把脑瘤分子分型从平均 26 天压缩到最短两小时。[1] 药物研发 · 基因与细胞治疗：全人源 BCMA/GPRC5D 双靶 CAR-T 首个人体数据读出，9 例高危患者客观缓解率 88.9%，细胞制备仅需 3 至 4 天。[7] 监管获批 · 投资并购：FDA 批准口服 ALK2 抑制剂用于进行性骨化性纤维发育不良，第 24 周新异位骨化体积平均减少 3.2 cm³，安慰剂组增加 24.6 cm³。[15]一、🧬 基因测序（4 条）1. 纳米孔把脑瘤分型压到两小时 2026-09-25 · 诺丁汉大学 / Oxford Nanopore · NHS England 英国 NHS 基因组学部门投入 210 万英镑，启动为期两年的脑瘤快速基因组诊断试点，把诺丁汉大学团队开发的纳米孔分型方案推向英格兰六家研究中心。英国每年新发脑瘤逾 1.2 万例，常规检测需外送专科实验室，患者往往要等上数周才能拿到分型结果。[1] 该方案用纳米孔测序靶向读取关键 DNA 区段，配合 ROBIN 软件完成肿瘤分类，把分型时间从平均 26 天压缩至最短两小时；部分病例可在手术仍在进行时拿到分子诊断，帮助外科医生实时判断切除范围、保护健康脑组织。项目由中部与南部、东部两大基因组医学服务牵头，长期目标是推广至英格兰所有神经病理中心。[2]2. 空间转录组降噪有了新算法 2026-09-24 · 《自然》系列期刊 · 加州大学伯克利分校 / 魏茨曼科学研究所研究团队 9 月 24 日发布 resolVI，一种面向测序型空间转录组的概率模型，可接在任何细胞分割算法之后运行，用于校正分子错配、批次效应与扩散带来的假信号。源码已并入开源框架 scvi-tools，兼容 Stereo-seq 与 Visium HD 等平台数据。[3] 模型把观测到的基因计数拆分为真实表达、邻位扩散与组织背景三部分，在小鼠脑与人类肝癌数据中显著压低了不同谱系标记基因的错误共表达。约 140 万个细胞、52 张切片的分析可在单张消费级显卡上六小时内完成；与 Baysor、ProSeg 等分割工具的对比显示其均能稳定减少错误信号，且不依赖预设的标记基因约束。[4]3. 转座子测序绘出噬菌体基因图谱 2026-09-26 · Nature Microbiology · 国际研究团队研究团队提出 HIDEN-SEQ，一种适用于噬菌体的转座子插入测序方法，可把病毒基因与可筛选表型系统性关联起来。方法以模式噬菌体 T4 完成验证，轻松重现了数十年研究所积累的基因必需性图谱，为后续向非模式噬菌体推广奠定基础。[5] 该方法易移植到多种非模式噬菌体，能在不同细菌宿主与培养条件下识别条件必需基因，并定位到此前未知、可匹配至特定抗病毒防御系统的抗防御因子。噬菌体基因组中大量功能未知的「暗物质」基因，一直是噬菌体疗法理性设计的最大障碍。[5]4. 人类基因组里发现活跃转座子 2026-09-24 · Science · 首都师范大学 / 康奈尔大学首都师范大学孙成课题组与康奈尔大学团队合作，9 月 24 日在《科学》发文确认 BC200 是一个仍具转座活性的人类基因。团队先在侵染人类的软疣病毒基因组中发现源自 BC200 的插入序列，两次病毒插入事件均发生在距今 15 万年内，恰对应现代人类的演化阶段。[6] 对 908 份不同人群测序数据的分析进一步鉴定出 8 个参考基因组中不存在的新插入位点，证明其在人群中具备活跃转座能力。传统观点认为转座子一旦被「征用」为功能基因便彻底丧失移动能力，这项研究提示二者之间存在长期进化权衡。[6]二、💊 药物研发 · 基因与细胞治疗（5 条）5. 双靶点 CAR-T 三天完成制备 2026-09-25 · 2026 IMS 年会 · 驯鹿生物 / 北京大学人民医院 2026 年国际骨髓瘤学会年会上，驯鹿生物报告了全人源 BCMA/GPRC5D 双靶 CAR-T 产品 RD140 的首个人体 I 期数据。9 例经多线治疗的高危复发难治骨髓瘤或浆细胞白血病患者中，客观缓解率 88.9%，高剂量组达到 100%，微小残留病阴性率同为 88.9%。[7] 安全性上未观察到包括 ICANS 在内的神经毒性事件，细胞因子释放综合征以低级别为主。RD140 基于 FasT 快速制备平台，细胞生产仅需 3 至 4 天，对疾病进展迅速、等待窗口极窄的患者意义突出；该研究由北京大学人民医院路瑾教授团队以壁报形式报告。[7]6. 单次 CAR-T 输注五年随访结果 2026-09-25 · 2026 IMS 年会 · 强生 / 传奇生物强生在国际骨髓瘤学会年会公布 CARTITUDE-2 研究队列 A 的五年随访结果：20 例接受过一至三线治疗、且来那度胺难治的复发难治骨髓瘤患者中，10 例在单次输注五年后仍存活且未进展，全部未接受任何维持治疗。[8] 该亚组中位随访 60.7 个月，中位无进展生存 60.5 个月，五年总生存率 69.2%；3 例完成五年骨髓检测的患者在 10⁻⁶ 灵敏度下均为 MRD 阴性，其中 5 例携带至少一项高危细胞遗传学特征。公司同时提示样本量仅 20 例，尚不足以支撑监管结论。[8]7. 口服 CELMoD 三期拿下 MRD 终点 2026-09-25 · 企业通稿 · 百时美施贵宝百时美施贵宝公布 III 期 EXCALIBER-RRMM 的预设分析结果：iberdomide 联合达雷妥尤单抗与地塞米松，对比达雷妥尤单抗联合硼替佐米与地塞米松，MRD 阴性完全缓解率 41.1% 对 20.7%，比值比 2.75，P 值小于 0.0001。[9] 该研究纳入 420 例可评估的复发难治骨髓瘤患者，中位随访 15.7 个月，MRD 阴性 CR 率与无进展生存为共同主要终点。该药已于今年 8 月获 FDA 加速批准上市，公司表示本次完整数据将在随后召开的学术会议上正式公布。[9]8. 石药免化疗双靶向方案三期成功 2026-09-24 · 港交所公告 · 石药集团石药集团公告，贝柯妥塔单抗联合奥希替尼一线治疗携带 EGFR 20 号外显子插入突变的局部晚期或转移性非小细胞肺癌，在预设期中分析达到主要终点，对照组为铂类联合培美曲塞化疗。该突变异质性强、脑转移比例高，一线治疗仍存在明显未被满足的需求。[10] 经独立审查委员会依据 RECIST 1.1 评估的无进展生存期达到期中分析预设的统计学优效界值，并显示总生存获益趋势，联合方案安全性良好。该组合从胞外与胞内同时作用于 EGFR 信号通路，属不含化疗的双靶向方案；公司在经典突变非鳞 NSCLC 与晚期结直肠癌的关键 III 期研究也在推进中。[11]9. 眼底基因治疗三年数据出炉 2026-09-24 · 视网膜学会年会 · REGENXBIO / 艾伯维 REGENXBIO 在视网膜学会第 59 届年会公布 II 期 ALTITUDE 三年随访：非增殖期糖尿病视网膜病变患者接受单次脉络膜上腔注射 sura-vec 后，10 例完成三年访视的剂量组受试者中 6 例改善超过 2 级糖尿病视网膜病变严重度量表，且均未接受额外治疗。[12] 这些受试者在此期间未发生任何威胁视力事件；17 例三年安全性随访未见新的药物相关信号，短程预防性局部激素方案下也未观察到眼内炎症。该疗法以 AAV8 载体递送抗 VEGF 抗体片段，美国糖尿病视网膜病变患者近 1000 万，相关剂量正在 IIb/III 期 NAAVIGATE 研究中验证。[13]三、📋 监管获批 · 投资并购（5 条）10. 肾癌二线迎来全口服组合 2026-09-25 · 企业通稿 · 默沙东 / 卫材 FDA 批准 belzutifan 联合仑伐替尼，用于 PD-1 或 PD-L1 抑制剂治疗后的晚期透明细胞肾细胞癌，成为该人群首个 HIF-2α 抑制剂联合多靶点酪氨酸激酶抑制剂的全口服方案，两药均为口服用药。[14] 批准基于 III 期 LITESPARK-011 的预设期中分析：联合组中位无进展生存 14.6 个月，对照卡博替尼组 10.6 个月，疾病进展或死亡风险降低 26%，客观缓解率 53% 对 40%。最终分析中总生存这一共同主要终点未达统计学显著，完整数据将在 ESMO 2026 公布。[14]11. 进行性骨化症有了口服药 2026-09-25 · 媒体报道 · Mirum Pharmaceuticals / Incyte FDA 批准口服 ALK2 抑制剂 zilurgisertib 片剂，用于减少 12 岁及以上进行性骨化性纤维发育不良（FOP）患者的全新生异位骨化体积，成为该病第三个获批疗法，也是首个覆盖低至 12 岁人群的口服药物。推荐起始剂量为每日一次 100 mg。[15] 依据来自随机双盲安慰剂对照的 II 期 PROGRESS 研究：第 24 周时治疗组全新生异位骨化体积平均减少 3.2 cm³，安慰剂组增加 24.6 cm³；新发骨化病灶人数相对减少 81%，但预设主要终点未达统计学显著。标签含胚胎胎儿毒性警告。[15]12. 欧盟批准首个脑型肾上腺脑白质营养不良药 2026-09-25 · 企业通稿 · Neuraxpharm / Minoryx 欧盟委员会 9 月 21 日以「特殊情形」批准口服 PPARγ 激动剂 leriglitazone，用于 2 至 12 岁、脑部 MRI 无钆强化病灶且神经功能评分为 0 或 1 的男性脑型肾上腺脑白质营养不良患者，为该病首个获批的药物疗法。[16] 关键 II/III 期 NEXUS 研究中，20 例可评估患儿经 96 周治疗后 7 例病情停止进展，而 EMA 设定的自然病程对照预期仅约 10%。此前唯一获批的基因疗法 Skysona 已于 2024 年退市，该药预计年底前在德国率先上市。[17]13. 基因泰克十五亿美元押注 AI 双抗 2026-09-24 · 企业通稿 · 罗氏基因泰克 / Earendil Labs 基因泰克与 AI 驱动的生物药公司 Earendil Labs 达成肿瘤双特异性抗体研发合作，首付 5500 万美元，含开发、注册与销售里程碑在内总额超过 15 亿美元，Earendil 另有资格获得净销售额的分级分成。靶点组合未披露。[18] Earendil 负责抗体发现直至早期临床开发，基因泰克接手后续全球开发与商业化，意味着首次人体研究成本留在技术方。这是该公司 17 个月内第三笔大型药企合作，赛诺菲此前两次引进其自免双抗；其关联方华深智药今年 6 月完成 7.87 亿美元融资。[19]14. RNAi 企业 ADARx 登陆纳斯达克 2026-09-25 · 企业通稿 / 媒体报道 · ADARx Pharmaceuticals / 艾伯维 siRNA 药企 ADARx 把扩大后的首次公开募股定价在每股 17 美元，发行 2625 万股，预计募资约 4.463 亿美元，9 月 25 日以代码 ADRX 在纳斯达克全球精选市场开始交易，发行规模高于原计划的约 2190 万股。[20] 艾伯维同步以同价参与私募配售，出资上限 1 亿美元，两笔交易合计约 5.352 亿美元，预计 9 月 28 日交割。核心产品 onvuzosiran 用于遗传性血管性水肿，早期研究显示血浆激肽释放酶最多下降 93%，III 期初步数据预计 2027 年底读出。[21]趋势点评纳米孔正在从「能做」走向「临床常规可用」。英国这次试点最值得注意的不是读长或准确率，而是把流程标准固化进医院网络——26 天到两小时的压缩，改变的是手术决策窗口。算法侧同样如此，resolVI 这类工具在补的空间组学短板，恰恰是技术进入临床前必须补的误差账。细胞治疗的主线已从「能不能缓解」转向「缓解能撑多久」。Carvykti 五年随访与 RD140 的 MRD 阴性率，分别回应了持久性与深度两个维度。与此同时，制备周期被压到三到四天，意味着竞争正从疗效扩展到可及性与成本。本期三项获批全部落在罕见病与难治肿瘤，监管机构对替代终点和小样本证据的容忍度仍在提高。资本端回到平台型交易与 IPO 两条线：AI 双抗授权总额高、首付低，风险更多留在技术方，与去年以来的交易结构一脉相承。关于 NextSeq 亦镁诺 NextSeq 亦镁诺（上海亦镁诺生物科技有限公司）专注组学数据分析与AI建模，携手美国合作伙伴 AmpSeq（实验室位于 Maryland、Boston 及 Connecticut），为科研机构与药企提供从快速测序到生信分析的端到端服务。在分析端，我们支持定制化生信Pipeline开发，并将AI建模能力拓展至肿瘤新抗原预测、mRNA/siRNA设计等药物开发场景；在CGT领域，具备面向长期安全性评估的NGS检测与解读能力。AmpSeq美国实验室同步提供Nanopore及NGS快速测序和交付服务，以灵活方案覆盖从深度分析到快速交付的多样化需求。如需了解，欢迎在公众号留言，或访问 nextseq.cn / ampseq.com。资料来源 📎 本文全部参考文献已汇总，点击底部「阅读原文」一键查看。 [1] 诺丁汉大学 — University of Nottingham innovation to drive NHS rollout of ultra-rapid brain tumour genetic testing（2026-09-25）｜机构通稿：试点金额 210 万英镑、为期两年、覆盖六家 NHS 中心、26 天压缩至两小时、英国年新发脑瘤逾 1.2 万例、ROBIN 软件。https://www.nottingham.ac.uk/news/nhs-roll-out-brain-tumour-genetic-test [2] Oxford Nanopore Technologies — Oxford Nanopore technology supports NHS pilot of rapid genomic testing for brain tumours（2026-09-25）｜企业通稿：纳米孔测序平台支持 NHS 试点、术中早期肿瘤分型、公司首席执行官表态。https://nanoporetech.com/zh/news/oxford-nanopore-technology-supports-nhs-pilot-of-rapid-genomic-testing-for-brain-tumours [3] Brief — ResolVI: addressing noise and bias in spatial transcriptomics（Nature, 2026-09-24）｜论文报道：resolVI 模型原理、扩散与分割误差校正、小鼠脑与人类肝癌 CosMx 数据验证、140 万细胞 52 切片六小时、scvi-tools 开源。https://www.brief.news/life-sciences/2026/09/24/resolvi-elevates-spatial-transcriptomics [4] bioRxiv — ResolVI - addressing noise and bias in spatial transcriptomics（预印本）｜预印本：模型架构、分子错配与批次效应校正、与 Baysor/ProSeg 等分割工具的对比实验设计。https://www.biorxiv.org/content/10.1101/2025.01.20.634005v1 [5] 生物通 — 系统绘制噬菌体基因必需性图谱并揭示新型抗防御因子（Nature Microbiology, 2026）｜论文报道：HIDEN-SEQ 方法原理、T4 噬菌体基因必需性图谱重现、非模式噬菌体可移植性、抗防御因子发现。https://www.ebiotrade.com/newsf/2026-9/20260926000806957.htm [6] 腾讯新闻 — 首都师大团队在《科学》刊发 BC200 基因转座研究成果（2026-09-25）｜媒体报道：BC200 转座子发现、软疣病毒插入事件与 15 万年时间窗、908 份人群测序数据与 8 个新插入位点、与康奈尔大学合作。https://news.qq.com/rain/a/20260925A06YGX00 [7] PR Newswire — IASO Bio Presents First-in-Human Phase 1 Data of RD140, a Fully Human BCMA/GPRC5D Dual-Targeted FasT CAR-T（2026-09-25）｜企业通稿：9 例患者 ORR 88.9%、高剂量组 100%、MRD 阴性率 88.9%、无 ICANS、3 至 4 天制备、IMS 2026 壁报 PA-200。https://www.prnewswire.com/news-releases/2026-ims--iaso-bio-presents-first-in-human-phase-1-data-of-rd140-a-fully-human-bcmagprc5d-dual-targeted-fast-car-t-302890377.html [8] Europe Pharma Today — Carvykti five-year data in 20 patients（2026-09-25）｜行业快讯：CARTITUDE-2 队列 A 五年随访、10/20 无进展生存、中位随访 60.7 个月、中位 PFS 60.5 个月、五年 OS 69.2%、10⁻⁶ MRD 阴性。https://europepharmatoday.com/news/carvykti-cartitude-2-five-year-ims-september-2026 [9] Business Wire — Bristol Myers Squibb Announces First Presentation of Results for ZENBEXUS (iberdomide) in Combination with Daratumumab from Phase 3 EXCALIBER-RRMM Trial（2026-09-25）｜企业通稿：420 例可评估患者、中位随访 15.7 个月、MRD 阴性 CR 41.1% 对 20.7%、比值比 2.75、P<0.0001、双主要终点设计。https://www.joplinglobe.com/region/national_business/bristol-myers-squibb-announces-first-presentation-of-results-for-zenbexus-iberdomide-in-combination-with-daratumumab/article_c8afbdbf-d622-556f-8b42-afd4081fbf25.html [10] 香港交易所 — 石药集团自愿公告：贝柯妥塔单抗（JMT101）联合奥希替尼一线治疗 EGFR ex20ins NSCLC 的 III 期临床试验取得阳性顶线结果（2026-09-24）｜监管披露文件：随机开放平行对照设计、对照为铂类联合培美曲塞、IRC 依 RECIST 1.1 评估的 PFS 主要终点、IDMC 决议、OS 获益趋势。https://www1.hkexnews.hk/listedco/listconews/sehk/2026/0924/2026092401983_c.pdf [11] FirstWord PHARMA — Phase III Clinical Trial of Becotatug (JMT101) in Combination With Osimertinib Achieves Positive Topline Results（2026-09-24）｜行业快讯：公告英文全文、免化疗双靶向机制、在研的经典突变非鳞 NSCLC 与晚期结直肠癌 III 期、EGFR ex20ins 疾病负担。https://firstwordpharma.com/story/8095930 [12] REGENXBIO — Presents Positive Three-Year Durability Data for Surabgene Lomparvovec in Diabetic Retinopathy at Retina Society Annual Scientific Meeting（2026-09-24）｜企业通稿：6/10 受试者 DRSS 改善超 2 级、无额外治疗、17 例三年安全性、剂量 1.0×10¹² GC/眼、NAAVIGATE IIb/III 期。https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-presents-positive-three-year-durability-data-surabgene [13] Pharmacally — Sura-vec Shows Three-Year DRSS Improvement in Diabetic Retinopathy（2026-09-24）｜论文报道：ALTITUDE 试验编号 NCT04567550、数据截止 2026-08-17、AAV8 载体抗 VEGF 抗体片段机制、美国患者近 1000 万。https://pharmacally.com/sura-vec-three-year-altitude-diabetic-retinopathy-data [14] Eisai — U.S. FDA Approves WELIREG (belzutifan) Plus LENVIMA (lenvatinib) for Certain Previously Treated Adult Patients With Advanced ccRCC（2026-09-25）｜企业通稿：LITESPARK-011 数据、中位 PFS 14.6 对 10.6 个月、HR 0.74、ORR 53% 对 40%、OS 未达显著、ESMO 2026 公布。https://us.eisai.com/press-releases/us-fda-approves-welireg-belzutifan-plus-lenvima-lenvatinib-for-certain-previously-treated-adult-patients-with-advanced-renal-cell-carcinoma-with-a-clear-cell-component-ccrcc [15] Consultant Live — Zilurgisertib Receives FDA Approval for FOP in Patients 12 and Older（2026-09-25）｜媒体报道：PROGRESS II 期设计与 63 例入组、第 24 周新 HO 体积 −3.2 cm³ 对 +24.6 cm³、新病灶相对减少 81%（P=0.0986）、推荐剂量与标签警告。https://www.consultantlive.com/view/zilurgisertib-receives-fda-approval-for-fop-in-patients-12-and-older [16] Neuraxpharm — European Commission grants marketing authorisation for NEZGLYAL (leriglitazone)（2026-09-25）｜企业通稿：9 月 21 日欧盟委员会批准、7 月 23 日 CHMP 积极意见、适应症人群界定、NEXUS 与 ADVANCE 研究、PPARγ 激动剂机制。https://www.neuraxpharm.com/news/european-commission-grants-marketing-authorisation-for-nezglyal-leriglitazone-the-first-pharmacological-treatment-approved-for-cerebral-adrenoleukodystrophy-cald-a-rare-neurodegenerative-d/ [17] Europe Pharma Today — EC authorises Nezglyal for childhood cALD（2026-09-21，企业 2026-09-25 确认）｜政策转述：EMA EPAR 依据、23 例主研究中 7/20 病情停止进展、自然病程对照 10%、德国年底前首发。https://europepharmatoday.com/news/ec-nezglyal-leriglitazone-cald-september-2026 [18] PR Newswire — Earendil Labs Announces Research Collaboration with Genentech to Discover and Develop Therapeutic Bispecific Antibodies in Oncology（2026-09-24）｜企业通稿：5500 万美元首付、总额逾 15 亿美元、分级销售分成、分工安排（Earendil 至早期临床、基因泰克后续全球开发）。https://www.prnewswire.com/news-releases/earendil-labs-announces-research-collaboration-with-genentech-to-discover-and-develop-therapeutic-bispecific-antibodies-in-oncology-302888622.html [19] BioSpace — Genentech makes $1.5B+ deal with AI heavyweight Earendil for cancer bispecifics（2026-09-25）｜行业快讯：赛诺菲 2025 年 1.845 亿美元授权与 2026 年第二次合作、7.87 亿美元融资、罗氏同日与 Atavistik 的 20 亿美元交易。https://www.biospace.com/deals/genentech-makes-1-5b-deal-with-ai-heavyweight-earendil-for-cancer-bispecifics [20] BioSpace — ADARx Pharmaceuticals Announces Pricing of Upsized $446.3 Million Initial Public Offering（2026-09-24）｜企业通稿：每股 17 美元、2625 万股、募资约 4.463 亿美元、9 月 25 日以 ADRX 挂牌、艾伯维同步私募上限 1 亿美元、合计 5.352 亿美元。https://www.biospace.com/press-releases/adarx-pharmaceuticals-announces-pricing-of-upsized-446-3-million-initial-public-offering [21] Yahoo Finance / BioPharma Dive — RNAi drug specialist ADARx raises $446M IPO（2026-09-25）｜媒体报道：IPO 定价位于 15 至 17 美元区间上限、发行规模高于原计划、onvuzosiran 血浆激肽释放酶最多下降 93%、III 期初步数据 2027 年底。https://finance.yahoo.com/healthcare/articles/rnai-drug-specialist-adarx-raises-003600444.html 关注 NextSeq亦镁诺，每日获取基因测序与药物研发最新资讯。

2026-09-24

·PRNewswire

REGENXBIO Presents Positive Three-Year Durability Data for Surabgene Lomparvovec in Diabetic Retinopathy at Retina Society Annual Scientific Meeting

Majority of Dose Level 3 participants with NPDR demonstrated a > 2-step DRSS improvement at 3 years post-one-time, in-office sura-vec gene therapy treatment, with no vision-threatening events and no additional treatment for diabetic retinopathy No IOI observed with short-course prophylactic topical steroids ROCKVILLE, Md., Sept. 24, 2026 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced positive long-term follow-up data from the Phase II ALTITUDE® study of investigational surabgene lomparvovec (sura-vec, ABBV-RGX-314) in diabetic retinopathy (DR) using suprachoroidal delivery. These new data were presented at the Retina Society 59th Annual Scientific Meeting in Los Angeles. "These latest long-term results are highly encouraging and reinforce the potential of sura-vec to change the treatment landscape for the millions of patients with non-proliferative diabetic retinopathy," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. "Building on the positive data seen at two and two and a half years after a single treatment, the three-year data continues to show meaningful improvements in DRSS alongside a reduction in disease progression. The durability of these results strengthens our conviction in sura-vec as a potentially transformative, one-time treatment." "For patients with non-proliferative diabetic retinopathy, the goal is to intervene before the disease progresses to vision-threatening complications and irreversible vision loss. However, current preventive treatment requires repeat injections, which is a significant burden for a population that is largely working-age adults," said Nikolas London, M.D., FACS, FASRS, Retina Consultants San Diego, ALTITUDE study investigator. "These three-year ALTITUDE results are encouraging and support continued study of sura-vec as a one-time, in-office treatment with the potential to change the course of the disease. A single treatment that reduces or eliminates the need for ongoing intraocular injections, and reduces the risk of vision-threatening complications, would be a meaningful change for our diabetic patients." Phase II ALTITUDE Long-term follow up data (as of August 17, 2026) In long-term follow-up from the Phase II ALTITUDE® trial, a single in-office administration of sura-vec using suprachoroidal delivery in participants with non-proliferative DR (NPDR) at Dose Level 3 (1.0×10¹² GC/eye) with short-course prophylactic topical steroids demonstrated a durable efficacy profile at three years with no new sura-vec-related safety signals observed during long-term follow up. 60% of all Dose Level 3 participants with three-year visits (n=6/10) achieved >2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) without additional treatment for diabetic retinopathy.1 These participants did not experience any vision-threatening events. Additionally, the majority of participants (n=3/4) who achieved a 1-step DRSS improvement at one year without supplemental anti-VEGF injections went on to achieve a >2-step DRSS improvement by three years without additional treatment for diabetic retinopathy. There were no new sura-vec-related safety signals and no intraocular inflammation observed through three years (n=17) with short-course prophylactic topical steroids. These data are consistent with previously presented two and a half-year Dose Level 3 NPDR data from the ALTITUDE trial and support the potential of one-time in-office sura-vec to modify the underlying disease and decrease risk of vision-threatening events. Dose Level 3 is being evaluated in the Phase IIb/III NAAVIGATE trial of sura-vec in NPDR. About Surabgene Lomparvovec (sura-vec, ABBV-RGX-314) Sura-vec is a one-time investigational gene therapy designed to deliver sustained treatment effect in wet AMD, diabetic retinopathy and other chronic retinal conditions. Sura-vec uses the NAV® AAV8 vector to encode an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). The sura-vec transgene protein inhibits the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.2 About Diabetic Retinopathy Diabetic retinopathy (DR) is the leading cause of vision loss in adults between 24 and 75 years of age worldwide.3 DR affects nearly 10 million people in the United States alone.4 The spectrum of DR severity ranges from non-proliferative diabetic retinopathy (NPDR) to proliferative diabetic retinopathy (PDR).4 As DR progresses, a large proportion of patients develop vision-threatening events, including diabetic macular edema (DME) and neovascularization that can lead to blindness.5 Current treatment options for patients with NPDR typically include "watchful waiting" or anti-VEGF treatment. For patients with PDR, current treatment options include anti-VEGF treatment or retinal laser; surgical treatment may be required for advanced PDR.2 ABOUT REGENXBIO Inc. REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA® and ITVISMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit . FORWARD-LOOKING STATEMENTS This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials, the timing, availability and interpretation of clinical data. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including risks related to the FDA's review process, the timing of enrollment, commencement, completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of any milestone payments, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2025, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at . All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. Zolgensma® and ITVISMA® are registered trademarks of Novartis AG. All other trademarks referenced herein are registered trademarks of REGENXBIO. Contacts: Dana Cormack Corporate Communications dcormack@regenxbio.com Investors: George E. MacDougall Investor Relations IR@regenxbio.com 1 One participant who achieved a ≥2-step DRSS improvement at Year 2 received two supplemental anti-VEGF injections after Year 2 despite no evidence of disease progression or center-involved DME in the study eye; investigator elected to treat the study eye concurrently with treatment of the fellow eye for center-involved DME. Participant was lost to follow-up and had no Year 3 data. *These are interim results from analyses performed by REGENXBIO for an ongoing trial. 2 Penn JS, Madan A, Caldwell RB, et al. Vascular endothelial growth factor in eye disease. Prog Retin Eye Res. 2008;27(4):331-71. 3 Cheung N, Mitchell P, Wong TY. Diabetic retinopathy. Lancet. 2010;376(9735):124–36. 4 Lundeen EA, Burke-Conte Z, Rein DB, Wittenborn JS, Saaddine J, Lee AY, Flaxman AD. Prevalence of Diabetic Retinopathy in the US in 2021. JAMA Ophthalmology. 2023;141(8):747-754. 5 Berrocal MD, Alexandra Acabá. Current Management of Diabetic Retinopathy, 2018 SOURCE REGENXBIO Inc. 21% more press release views with Request a Demo

临床结果基因疗法临床2期

100 项与 Surabgene Lomparvovec 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
糖尿病性黄斑水肿	临床3期	美国	AbbVie, Inc.	2026-06-01
脉络膜新生血管	临床3期	美国	AbbVie, Inc.	2022-01-13
脉络膜新生血管	临床3期	日本	AbbVie, Inc.	2022-01-13
脉络膜新生血管	临床3期	加拿大	AbbVie, Inc.	2022-01-13
脉络膜新生血管	临床3期	法国	AbbVie, Inc.	2022-01-13
脉络膜新生血管	临床3期	德国	AbbVie, Inc.	2022-01-13
脉络膜新生血管	临床3期	匈牙利	AbbVie, Inc.	2022-01-13
脉络膜新生血管	临床3期	意大利	AbbVie, Inc.	2022-01-13
脉络膜新生血管	临床3期	西班牙	AbbVie, Inc.	2022-01-13
脉络膜新生血管	临床3期	英国	AbbVie, Inc.	2022-01-13

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适应症

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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


NCT04832724 (ARVO2026)	临床2期	年龄相关性黄斑变性	60	sura-vec	壓壓襯願觸艱獵憲積選(窪醖顧選鏇鹹願鹹廠願) = 醖蓋窪壓襯衊蓋遞積憲構構繭觸積鏇艱簾餘築 (憲齋鏇夢繭積窪淵願網, 0.872 ~ 0.942) 更多	积极	2026-05-03
ARVO2026	临床1/2期	湿性年龄相关性黄斑变性	11	ABBV-RGX-314 (bilateral nAMD)	膚醖齋齋築繭顧鑰膚鹽(淵膚繭選餘艱壓齋壓選) = 憲鹹願醖齋艱蓋築艱淵壓積範繭範顧鏇淵窪廠 (鹽壓襯糧膚淵願築簾齋 ) 更多	积极	2026-05-03
EURETINA2025	临床1/2期	湿性年龄相关性黄斑变性	10	ABBV-RGX-314 (bilateral choroidal neovascularization secondary to age-related macular degeneration)	積憲衊鏇築餘鑰艱憲鬱(觸構構廠憲膚鹽夢淵蓋) = 觸鏇衊窪膚糧齋衊壓蓋夢鹽襯衊構膚構窪壓艱 (衊糧夢願製觸窪廠膚餘 ) 更多	积极	2025-09-04
NCT04514653 (AAVIATE, Corporate Publications) 人工标引	临床2期	湿性黄斑变性	106	ABBV-RGX-314	網顧衊餘鹽獵艱鹽壓繭(憲製鹹繭鏇糧願製餘簾) = in the study eye were mild or moderate and included conjunctival hemorrhage, increased intraocular pressure, episcleritis, and conjunctival hyperemia. 齋餘遞選觸顧繭範淵窪 (製醖繭簾製窪簾鹽遞壓 ) 更多	积极	2024-01-16
EURETINA2023	N/A	糖尿病性黄斑水肿	-	RGX-314 at a dose level of 2.5 x E11 GC/eye	窪積願簾願糧廠願觸夢(積餘觸壓鏇選顧鏇獵廠) = three cases of mild intraocular inflammation through 6 months which resolved with topical corticosteroids 鏇壓衊觸淵糧獵廠蓋窪 (簾顧鏇鑰觸醖鹹憲築觸 ) 更多	-	2023-10-05
EURETINA2023	N/A	糖尿病性黄斑水肿	-	RGX-314 at an increased dose level of 5 x E11 GC/eye		-	2023-10-05
EURETINA2023	N/A	年龄相关性黄斑变性	-	RGX-314	顧淵範糧餘糧糧襯鑰獵(築鹹製範簾願鹹廠衊觸) = No new drug-related ocular adverse events (AEs) have been reported in Cohorts 1-4 獵選餘餘鏇網顧構顧顧 (繭繭鹹糧鬱網夢廠繭鹹 ) 更多	-	2023-10-05
Phase I/IIa (ARVO2023)	临床1/2期	湿性年龄相关性黄斑变性	42	RGX-314	築積膚範網築鹽觸觸餘(鹽鏇繭艱艱蓋憲繭積範) = no new drug-related ocular adverse events reported in Cohorts 1-4 and one drug-related adverse event of significantly decreased BCVA in Cohort 5 鹹膚鑰襯網窪選壓憲鹹 (願願獵壓簾醖憲窪遞獵 )	积极	2023-04-23
NCT04567550 (ALTITUDE, Corporate Publications) 人工标引	临床2期	糖尿病性视网膜病变	60	RGX-314	鹽憲糧衊鏇糧獵鏇蓋憲(構齋鹹淵壓憲壓鏇衊齋) = 鏇壓鑰鑰糧積網築膚鑰選夢繭糧醖鑰築艱鑰艱 (鬱齋廠憲鹽夢觸衊糧觸 ) 更多	积极	2022-11-02
NCT04567550 (ALTITUDE, Corporate Publications) 人工标引	临床2期	糖尿病性视网膜病变	60	control	鹽憲糧衊鏇糧獵鏇蓋憲(構齋鹹淵壓憲壓鏇衊齋) = 艱築築蓋糧夢廠觸鹹願選夢繭糧醖鑰築艱鑰艱 (鬱齋廠憲鹽夢觸衊糧觸 ) 更多	积极	2022-11-02
NCT04567550 (ALTITUDE, Corporate Publications) 人工标引	临床2期	糖尿病性视网膜病变	20	RGX-314	蓋簾選顧壓膚遞範製鹽(繭鏇築淵鬱觸鹹網窪積) = 獵鏇蓋壓艱願獵餘齋齋鹽獵繭膚醖憲廠簾築糧 (糧淵廠襯築膚鏇鹹鹽顧 ) 更多	积极	2022-09-13
NCT04567550 (ALTITUDE, Corporate Publications) 人工标引	临床2期	糖尿病性视网膜病变	20	Observational	蓋簾選顧壓膚遞範製鹽(繭鏇築淵鬱觸鹹網窪積) = 廠遞顧窪膚窪膚簾鏇鹽鹽獵繭膚醖憲廠簾築糧 (糧淵廠襯築膚鏇鹹鹽顧 ) 更多	积极	2022-09-13
NCT04514653 (AAVIATE, PRNewswire) 人工标引	临床2期	湿性年龄相关性黄斑变性	19	RGX-314	積鑰襯憲獵繭膚顧繭鹽(鏇廠獵繭獵蓋憲蓋觸廠) = 鏇積遞願糧獵膚顧簾襯艱製淵遞憲廠製膚構餘 (壓積糧壓鹹糧鬱艱糧鹽 ) 更多	积极	2021-10-01
NCT04514653 (AAVIATE, PRNewswire) 人工标引	临床2期	湿性年龄相关性黄斑变性	19	Ranibizumab	積鑰襯憲獵繭膚顧繭鹽(鏇廠獵繭獵蓋憲蓋觸廠) = 繭構鑰構衊鬱鑰醖鏇願艱製淵遞憲廠製膚構餘 (壓積糧壓鹹糧鬱艱糧鹽 ) 更多	积极	2021-10-01