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2025-09-17

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

[+1]

NCT05407636

/ Recruiting临床3期

A Randomized, Partially Masked, Controlled, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD

ABBV-RGX-314 (also known as RGX-314) is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-vascular endothelial growth factor (VEGF) therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. ABBV-RGX-314 is being developed as a potential one-time treatment for wet AMD.

开始日期2022-01-13

申办/合作机构

AbbVie, Inc.

[+1]

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）

2025-05-24·EXPERT OPINION ON PHARMACOTHERAPY

Beyond the injection: delivery systems reshaping retinal disease management

Review

作者: Harris, Alon ; Ciulla, Thomas A. ; Akotoye, Christian ; Rowe, Lucas W.

INTRODUCTION:

Intravitreal injections remain the standard for treating common retinal diseases including age-related macular degeneration (AMD), diabetic macular edema (DME) and diabetic retinopathy. However, frequent administration creates significant treatment burden due to limited drug half-life and the chronic nature of these conditions.

AREAS COVERED:

This review summarizes emerging drug delivery techniques and therapies for retinal disease that have achieved FDA approval within the past five years or have advanced to Phase 3 development, including intravitreal sustained-release platforms and alternative delivery routes (suprachoroidal, subretinal, topical, and subcutaneous). Specific innovations discussed include the ranibizumab port delivery system, EYP-1901 (Duravyu, vorolanib implant), KSI-301 (tarcocimab tedromer), KSI-501, OTX-TKI (Axpaxli, axitinib implant), 4D-150, revakinagene taroretcel-lwey (Encelto, NT-501, encapsulated cell therapy), Xipere (triamcinolone acetonide injectable suspension), AU-011 (belzupacap sarotalocan targeted delivery), ABBV-RGX-314, elamipretide, and OCS-01 (high concentration dexamethasone).

EXPERT OPINION:

Promising innovations include sustained-release intravitreal implants, topical and subcutaneous delivery systems, and targeted methods like suprachoroidal and subretinal injections, each with unique advantages and limitations. Challenges include overcoming the blood-retinal barrier, surgical complications with implantable devices, and ensuring patient adherence. Advances in smart delivery systems, drug formulations, and predictive models, alongside interdisciplinary collaboration, will be crucial in achieving personalized, effective, and sustainable retinal therapies.

2025-01-01·EYE

Exploring new horizons in neovascular age-related macular degeneration: novel mechanisms of action and future therapeutic avenues

Review

作者: Shirian, Jonathan D ; Shirian, Jonathan D. ; Shukla, Priya ; Singh, Rishi P. ; Singh, Rishi P

Abstract:

Neovascular age-related macular degeneration (nAMD) can lead to significant vision impairment through the growth of abnormal neovascular membranes in the choroid. Despite advancements with current anti-vascular endothelial growth factor (VEGF) therapies, challenges such as frequent injections, inadequate response, and patient-related concerns persist. Emerging therapeutics aim to reduce vision-loss through a variety of mechanisms. Gene therapies, including RGX-314 and Ixo-vec, express an anti-VEGF protein, and 4D-150, expresses an anti-VEGF protein and a VEGF-C inhibitory miRNA. Anti-VEGF associated therapeutics include OPT-302, targeting VEGF-C and VEGF-D, BI 836880, which inhibits VEGF-A and Ang-2 activity, and Tarcocimab tedromer, inhibiting all VEGF-A isoforms. Agents with novel mechanisms of action include UBX1325, which inhibits an anti-apoptotic protein, Restoret (EYE103), a Wnt agonist, and the tyrosine kinase inhibitors, EYP-1901, OTX-TKI, CLS-AX, and KHK4951.

2024-12-01·AMERICAN JOURNAL OF OPHTHALMOLOGY

Subretinal Gene Therapy for Treatment of Retinal and Choroidal Vascular Diseases

作者: Gahn, Greggory M ; Khan, Hannah ; Aziz, Aamir A ; Khan, Huma ; Sulahria, Humza ; Mojumder, Ohidul ; Khanani, Ibrahim ; Khanani, Zoha A ; Khanani, Arshad M ; Mishra, Kapil

OBJECTIVE:

This review article discusses investigational subretinal gene therapies for retinal vascular diseases, including AVA-101, an adeno-associated viral (AAV) 2 vector expressing soluble vascular endothelial growth factor (VEGF) receptor 1, ABBV-RGX-314, an AAV8 vector expressing an anti-VEGF-A antibody fragment, and EXG102-031, an AAV8 vector expressing a recombinant protein that blocks VEGF family members and angiopoietin 2.

DESIGN:

Review article CONCLUSION: Subretinal injection is a commonly used delivery route for investigational gene therapy agents which is theorized to provide relative immune privilege, thereby reducing the risk of inflammation, while providing high transgene expression in photoreceptors and retinal pigment epithelium. Subretinal injection of AVA-101 demonstrated safety and tolerability in Phase I and IIa trials, but failed to maintain visual acuity and control exudation. In contrast, subretinal injection of some doses of ABBV-RGX-314 have shown evidence of controlling exudation and the maintaining vision, as well as safety and tolerability, leading to two ongoing pivotal trials comparing subretinal delivery of two different doses of ABBV-RGX-314 versus intravitreal injections of 0.5mg ranibizumab or 2mg aflibercept. These preliminary results are an encouraging and welcome development in the search for efficacious, long-duration treatments for retinal vascular diseases.

162

项与 Surabgene Lomparvovec 相关的新闻（医药）

2025-06-19

·EINPresswire

Seeing Beyond Sight: New Frontiers in AMD Treatment-Gene Therapy and Biosimilars Take Center Stage | CI Intelligence

Pioneering AMD therapies-gene therapy, biosimilars, long‑acting biologics-promise durable vision gains and reduced injection burden for aging populations. AMD treatment is entering a transformative era, where precision gene therapy and extended-duration biologics are redefining care. The market is poised to benefit both patients and healthcare systems.” — DataM Intelligence AUSTIN, TX, UNITED STATES, June 19, 2025 / EINPresswire.com / -- Revolutionizing AMD Care: From Monthly Injections to One-Time Gene Therapies In a watershed moment for retinal health, the Age-Related Macular Degeneration (AMD) therapeutics market is poised for transformation. A new wave of innovative treatments—from gene therapy to long-acting anti-VEGF biologics and biosimilars—is reshaping care protocols and expanding access for millions worldwide. With the global burden of AMD projected to surpass 300 million by 2040, the urgency to reduce injection frequency, slow disease progression, and improve affordability has never been more critical. Download CI Sample Report: https://www.datamintelligence.com/strategic-insights/ci/age-related-macular-degeneration-amd 🌍 Market Snapshot: AMD Therapeutics Poised for Growth - The global AMD therapeutics market was valued at approximately $14.24 billion in 2024, with expectations to reach $24.22 billion by 2033, registering a 6–8% CAGR over the forecast period. - North America remains the leading market due to high disease prevalence and early access to biologics, while Asia-Pacific is emerging rapidly with aging populations and increasing healthcare access. - Biosimilar competition, reimbursement reforms, and gene therapy innovations are key drivers shaping this growth trajectory. 🧬 Disease Overview: AMD at a Glance Age-related macular degeneration affects the macula—the central portion of the retina responsible for sharp, straight-ahead vision. It is the leading cause of irreversible vision loss in people aged 50 and older. Two Forms of AMD: - Dry AMD (Atrophic): The most common form, accounting for about 80% of cases. Characterized by thinning of the macula and drusen deposits. - Wet AMD (Neovascular): Less common but more severe. Marked by abnormal blood vessel growth beneath the retina, leading to leakage, scarring, and rapid vision loss. 💊 Current Therapies: Anti-VEGF Dominates, But GA Drugs Rising Wet AMD: Approved Therapies - Eylea (Aflibercept): VEGF and PlGF inhibitor, long-standing market leader. - Lucentis (Ranibizumab): Anti-VEGF monoclonal antibody fragment, widely adopted globally. - Beovu (Brolucizumab): Longer dosing intervals, though safety remains under scrutiny. - Vabysmo (Faricimab): Dual inhibition of VEGF-A and Ang-2; allows for 16-week dosing intervals. - Off-label Avastin (Bevacizumab): Economical alternative, frequently used off-label. Recent Developments: Biosimilars: Byooviz (ranibizumab-nuna), Yesafili, and Opuviz (aflibercept biosimilars) are now FDA-approved, offering cost-effective alternatives with similar efficacy. Dry AMD / Geographic Atrophy (GA): - Syfovre (Pegcetacoplan): First FDA-approved therapy for GA, slows disease progression by up to 36% by inhibiting complement C3. - Izervay (Avacincaptad Pegol): Approved in 2023; targets complement C5 to reduce retinal degeneration in GA. - AREDS2 Supplements and Photobiomodulation Therapy offer supportive care for early or intermediate dry AMD. 🔬 Emerging Therapies and Pipeline Highlights The AMD pipeline is robust, focusing on durable solutions and novel mechanisms of action. For Dry AMD (GA): - ALK-001 (Alkeus Pharmaceuticals): A modified vitamin A derivative in Phase II/III trials aimed at slowing photoreceptor degeneration. - Tinlarebant (Belite Bio): Oral therapy reducing retinal vitamin A buildup to slow GA progression (Phase III). For Wet AMD: - ABBV-RGX-314 (REGENXBIO/AbbVie): Gene therapy delivering anti-VEGF via subretinal or suprachoroidal injection. Phase III underway, with potential to eliminate frequent injections. - 4D-150 (4D Molecular Therapeutics): Intravitreal gene therapy targeting VEGF-A and VEGF-C for sustained delivery—Phase III trials are ongoing. Other Novel Agents: - UBX1325 (Unity Biotechnology): Senolytic targeting aging retinal cells to improve vision in AMD and DME. - Complement Inhibitors in Development: Aimed at upstream and downstream components (e.g., CFB, CFD, FD inhibitors). Ask For Customized CI Sample Report as per Your Business Requirement: https://www.datamintelligence.com/strategic-insights/ci/age-related-macular-degeneration-amd 📈 Market Opportunities & Forecasting - The wet AMD market continues to dominate revenue, but geographic atrophy (GA) is a growing segment due to newly approved treatments and expanding patient population. - Gene therapy and biosimilars are expected to drive a paradigm shift by improving durability and lowering long-term costs. - Market revenue is projected to grow from $14.24 billion (2024) to $24.22 billion by 2033, largely fueled by treatment innovation and the rising global prevalence of AMD. 🔍 Unmet Needs: The Next Frontier in AMD Care Despite progress, critical gaps remain: - Treatment Burden: Monthly injections are unsustainable for many elderly patients. Gene therapies and extended-interval dosing are key solutions. - Lack of Cure for Dry AMD: GA treatments slow degeneration but cannot reverse damage. - Global Access & Cost: High biologic prices remain a barrier in low- and middle-income countries. Biosimilars and oral agents offer scalable alternatives. - Delayed Detection: Early AMD often goes undiagnosed. Greater emphasis on screening and AI-enabled imaging is needed. - Personalized Medicine: Genotype-based AMD therapies remain underdeveloped, despite promising biomarker research. 🏆 Competitive Landscape and Strategic Positioning Key Players in Wet AMD: - Regeneron: Market leader with Eylea/Eylea HD. Facing competition from biosimilars and Roche’s Vabysmo. - Roche/Genentech: Innovator with Vabysmo and sustained delivery devices. - Novartis: With Beovu, pursuing longer dosing intervals. Leading in Dry AMD: - Apellis (Syfovre) and Astellas/Iveric Bio (Izervay) have pioneered complement-targeted approaches. Emerging Innovators: - REGENXBIO and Adverum Biotech: Leading the charge in gene therapy, aiming to provide one-time, durable treatment solutions. - Samsung Bioepis, Biocon, Polpharma: Disrupting the pricing landscape with Lucentis and Eylea biosimilars. Book Free CI Consultation Report: https://www.datamintelligence.com/strategic-insights/ci/age-related-macular-degeneration-amd 🎯 Target Opportunity Profile (TOP): Strategic Drug Benchmarking Ideal Drug Profile: - Wet AMD: ORR > 90%, injection frequency ≤ every 16 weeks, subcutaneous/gene therapy option. - GA: ≥30% reduction in lesion growth, good safety profile, ideally oral or less invasive. - Gene Therapy: One-time treatment, durable for 2–5+ years, minimal side effects. Benchmarking Snapshot: - Eylea HD: 12–16 week interval; market leader. - Vabysmo: Dual-target; fast-growing market share. - Syfovre & Izervay: First-movers in GA. - RGX-314 & 4D-150: Future disruptors with one-time treatment potential. - Yesafili, Opuviz: Cost-effective alternatives, expanding access. 💡 Conclusion: A Clearer Vision for Millions The Age-Related Macular Degeneration (AMD) landscape is evolving from high-frequency, high-cost injection therapies toward smarter, longer-lasting, and more accessible treatments. From breakthrough gene therapies to affordable biosimilars and the first-ever drugs for geographic atrophy, this is an era defined by innovation and opportunity. As the AMD patient population swells worldwide, driven by aging demographics, these advancements are not just medically vital—they’re a necessity for sustainable and equitable eye care. Read Related CI Report: 1. Atypical Hemolytic-Uremic Syndrome Therapeutic 2. Chronic Rhinosinusitis with Nasal Polyps Sai Kumar DataM Intelligence 4market Research LLP +1 877-441-4866 sai.k@datamintelligence.com Visit us on social media: LinkedIn X Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

临床3期上市批准基因疗法

2025-06-19

·动脉网

眼科药物进入长效疗法竞争，中国企业上牌桌

眼底治疗市场正孕育着一场大的变革。5月底，拜耳和诺华新的抗VEGF药物相继在国内获批，在传统抗VEGF药物主导眼底疾病治疗市场十年后，市场迎来了一丝新意。近年来，随着双靶点药物（法瑞西单抗）、长效制剂（布西珠单抗）以及基因疗法（如AAV载体药物）的接连登场，眼底治疗周期从“月注射”延至“季度注射”，疗效与依从性革命也在重塑临床标准。再加上医保的助推，使得市场增量得以持续释放。这场由技术、价格和渠道三维驱动的眼底药物竞争，正从红海厮杀走向价值重构，唯有突破“跟随式创新”的企业，方能占据市场的制高点。01被搅浑的眼底治疗市场上市10多年的雷珠单抗开始显露疲态。仰仗着原研以及先发的优势，诺华的雷珠单抗本来牢牢把持着国内眼底治疗市场的头把交椅，可随着竞争者的不断涌现，市场起了变化。从康弘（康柏西普）和拜耳（阿柏西普），到2024年获批的罗氏（法瑞西单抗）和齐鲁制药（阿柏西普、雷珠单抗仿制药）的加入，雷珠单抗已经疲于应对。雷珠单抗近10年销售情况及增长率，图源摩熵医药据摩熵医药数据显示，雷珠单抗在经历10年的高速增长后，销售开始放缓。尽管2024年仍然取得了同比1.37%的增长，但相较于之前动辄20%、30%的增长速度已然降低。除了外部对手的竞争，雷珠单抗还面临着仿制药的冲击，目前齐鲁制药的雷珠单抗注射液已经获批，华东医药的正在审批中，他们的出现还将进一步分食市场份额。诺华雷珠单抗的市场统治力已经开始松动。另一方面，竞争者们也交出了不错的成绩单。康弘药业推出的康柏西普继续保持着较高的增长率，2024年同比增速达17.53%。与雷珠单抗相比，康柏西普的定价较低，从上市时约6800元/支，此后一路降价，在纳入医保后更是将价格降至初上市的50%不到。再加上相较雷珠单抗每年注射次数更少，使得康柏西普在国内市场份额迅速提升，并且反超了雷珠单抗。三款药物的份额占比变化，数据源自摩熵医药此后，拜耳阿柏西普的加入则让竞争进入白热化。自2018年国内获批上市，2019年纳入医保目录后，阿柏西普的销售额开始一路狂飙，2024年销售超9亿元。更值得关注的是，由于阿柏西普的专利到期，国内有不少企业布局了仿制药，其中齐鲁制药进度最快，于2023年底获批。2024年，齐鲁制药的阿柏西普正式上市，尽管总体销售额并不算高，但分季度来看，其增长速度还是相当惊人。并且不仅仅是齐鲁制药，像欧康维视与博安生物合作开发的阿柏西普眼内注射溶液也已进入审批阶段，后续迈威生物以及景泽生物的相关仿制药也处于临床3期阶段，此外国内还有多款仿制药在研。仿制药的上市通常也意味着更低价格的出现，这势必进一步加剧竞争的烈度。除了国内药企，MNC也在加入这个战局。罗氏的法瑞西单抗是全球首个眼底双抗药物，继2022年在美国获批后于2023年底国内获批。上市后，法瑞西单抗迅速放量，2024年全球销售额超40亿美元，尽管国内市场是上市首年占比较低，但从季度增长来看，其增速相当惊人，这还是在单支售价9000元的基础上。值得注意的是，从2025年开始，法瑞西单抗已被纳入医保目录，同时价格降至3608元，也就是说，法瑞西单抗今年迅速放量已是必然，现有市场格局肯定会受到巨大冲击。法瑞西单抗上市首年销售情况，图源摩熵医药总的来看，雷珠单抗在经历多年辉煌后，后劲已然不足，对市场的掌控大不如前。以康柏西普和阿柏西普为代表的药物对其发起了冲击，特别是随着更多阿柏西普仿制药的上市以及法瑞西单抗纳入医保，市场将陷入进一步的洗牌局面。在这个巨大变革的当下，各家都开始出牌以应对更白热化的竞争。02眼科进入长效VEGF竞争时代与现有的药物做出差异化优势成药企的选择。刚过去的5月对于眼底治疗市场特别精彩，先是5月22日国家药监局批准拜耳的阿柏西普（8mg）用于治疗新生血管（湿性）年龄相关性黄斑变性（nAMD），相较于之前2mg版本，高剂量版被批准在初始3个月，连续每月注射一次，然后根据医生的判断，治疗间隔可延长至每4个月一次。5月23日，罗氏的Susvimo（ranibizumab）100mg/mL也被FDA批准上市，它的特点在于每9个月仅需一次补充给药的用于治疗糖尿病视网膜病变的方案。面对雷珠单抗市场竞争力的逐渐减弱，诺华拿出了布西珠单抗进行应对，并于5月底在国内获批。诺华希望以其独特的小分子结构特点带来的诸多特性和超长用药间隔的优势，能重塑DME治疗格局，开创一个强效新时代。药品用药间隔对比，据公开资料收集整理目前，无论是基于VEGF受体胞外域与人IgG1 Fc段融合蛋白结构的阿柏西普还是基于双特异性抗体技术平台开发的法瑞西单抗，其分子大小分别为115kDa和150kDa。研究表明，能够在人类视网膜上自由扩散的分子大小约为76.5kDa。也就是说，这类大分子药物会存在一些难以解决的问题，如视网膜组织屏障限制了大分子药物向深层组织的扩散，导致靶部位有效浓度不足；Fc片段与眼内免疫细胞表面受体结合进入全身循环后增加心血管风险；双通路抑制策略中Ang-2通路作用机制尚未完全探明等。对手的短板自然是差异化的绝佳切入点。诺华布西珠单抗仅由抗体重链可变区（VH）和轻链可变区（VL）通过柔性多肽连接（Linker）连接而成，是一个仅有26kDa大小的超紧凑型蛋白结构。这种设计不同于传统抗体可能引发免疫副作用的Fc片段，只选择保留抗原识别所需的最小功能单元，同时又有对靶标的完全结合能力，其超小分子量会带来快速渗透的特性，能高效跨越视网膜色素上皮屏障和内界膜屏障，为眼科治疗带来新的路径。简单来说，布西珠单抗通过特殊的结构设计，从穿透能力、浓度控制和高亲和力三个维度入手，相比竞争对手，既做到了药物起效更快，同时又延长了药物持续时间。为今后的市场竞争做好了充分的准备。围绕长效药物，不仅是MNC，本土药企也在持续发力加速研发进程。03中国创新药的眼科解法中国药企开始走上牌桌。一直以来，国产抗VEGF药物主要集中在仿制药开发层面上，特别是随着阿柏西普关键专利陆续到期，更是有众多药企参与其中。然而，并非所有企业都愿意只扮演跟随者的角色，已经有越来越多的企业意识到要想从眼底治疗市场中分得一杯羹，需要具备突破性价值的、拥有真正创新机制的产品。在ARVO 2025（视觉与眼科学研究学会）年会上，信达生物和荣昌生物分别口头报告了旗下相关药物的重大进展，也标志着国产抗VEGF眼科药物从仿制跟随到创新竞争的新阶段。年会上，信达公布其全球首创VEGFR-CR1双靶药依莫芙普（IBI302）2期临床数据（NCT05403749）。该研究持续持续52周，主要终点为：第40周时研究眼最佳矫正视力评分较基线的改变。IBI302在传统VEGF结合域之外增加C端补体结合域轻补体活化介导的炎症反应，达到同时抑制VEGF介导的新生血管生成和补体活化通路的治疗作用。整体而言，研究达到了主要目标，从间隔用药时间来看，超过80%的IBI302组受试者能够以Q12W给药间隔（阿柏西普为Q8W）维持视力获益；视力改善程度与阿柏西普相当；解剖学疗效指标改善；更重要的是在抗黄斑萎缩获益趋势方面，IBI302和阿柏西普组1年新发黄斑萎缩的比例分别为4.9%和8.3%，IBI302治疗后新发比例相较阿柏西普组下降40%，显示其具有抑制黄斑萎缩的潜力。就在亮相ARVO大会后，信达宣布IBI302治疗糖尿病黄斑水肿2期临床研究完成首例受试者给药，并表示将挑战本领域全球当前最高的治疗标准，即同另一个双靶点创新药物罗氏法瑞西单抗进行头对头临床。这一行为凸显了信达对于IBI302的自信。在信达之后，荣昌生物也在年会上将自家自主研发的全球首创VEGF/FGF双靶点融合蛋白药物RC28亮相，它通过同时抑制血管内皮生长因子（VEGF）和成纤维细胞生长因子-2（FGF-2）通路，展现出优异的临床疗效。同时，RC28的人源化设计，可以有效延长其半衰期、减少给药频率、减轻患者不适。目前，RC28正在进行头对头阿柏西普的DME适应症3期研究，此外，和阿柏西普头对头AMD的临床3期也在进行中。据公开信息，RC28分别将于2025年下半年和2026上半年分别提交两项适应症的上市申请。荣昌生物曾表示将会把RC28权益BD出去，照当前进度来看，交易或将在NDA之前完成。此外，恒瑞医药的HR19034也值得关注，这是一款已经进入临床3期的抗VEGF双抗药物，通过同时靶向VEGF两个不同表位来高效阻断VEGF信号通路，从而抑制新生血管生成达到治疗湿性AMD的目的。总的来看，国内企业在设计双靶点药物时目的很明确，一是显著降低治疗频率提高患者依从性；二是提供额外辅助功效如RC28的抗纤维化和IBI302的抗血管渗出，都有效提升了整体疗效。04创新还在继续基因疗法虽在其他领域有所退潮，但在眼底治疗中却异常火热。据摩熵医药数据，目前全球范围内有300多款以AAV作为病毒载体的基因治疗药物处于临床研发阶段，而眼科领域就有80多款。之所以如此，在于眼科的基因治疗相对安全。一方面相对封闭的眼部环境使其具有免疫豁免特性，同时眼睛体积又小，病毒用量低，对于身体其他部位基本没有影响。另一方面，眼底细胞不会再生和分裂，而许多眼科疾病由单基因突变引起，更使其成为基因治疗的理想对象。在年龄相关性黄斑变性（AMD）治疗过程中，抗VEGF治疗能减缓部分患者的疾病进展和改善视力，但需要频繁的眼部注射，患者依从性较差且对部分患者效果有限。基因治疗可以将表达抗VEGF的病毒载体转染到眼内组织，能够实现治疗性蛋白的长期内源性表达，解决患者频繁注射、长期治疗依从性不佳的难题。以当前全球进度较快的眼科基因疗法ABBV-RGX-314（临床3期）为例，此前2期临床数据显示，在9个月的观察期内，抗VEGF注射需求暴降97%。此外，所有患者仅需要0次或1次抗VEGF疗法补充注射，其中78%的患者可以完全脱离补充治疗。也就是说，大部分患者在接受一次ABBV-RGX-314治疗后，很长一段时间内都无需再进行频繁的抗VEGF注射，大幅减少了治疗频次。值得注意的是，该管线是艾伯维在2021年9月以最高17.52亿美元的交易金额从ReGenXBio那里获得。这显示出艾伯维对于基因疗法打破传统治疗局限，获取可观市场份额的商业化自信。国内AMD适应症进入临床阶段的基因治疗管线，数据源自摩熵医药目前，国内眼科基因治疗领域的研发也相当活跃，虽然湿性老年性黄斑变性和糖尿病视网膜病变这两大眼底疾病的基因治疗管线都没有达到临床3期，但国内目前已经进入临床阶段的基因治疗管线已近10条。未来3年，或将成为基因疗法在眼科领域发展的关键窗口期，若艾伯维的RGX-314能顺利上市，则能成功刺激全球市场，而中国管线落后2年左右的进度则完全能赶上这波浪潮，眼底治疗市场的剧烈变革，中国药企必将成为牌桌上的重要参与者。*封面图片来源：神笔PRO如果您认同文章中的观点、信息，或想进一步讨论，请与我们联系；也可加入动脉网行业社群，结交更多志同道合的好友。近期推荐声明：动脉网所刊载内容之知识产权为动脉网及相关权利人专属所有或持有。未经许可，禁止进行转载、摘编、复制及建立镜像等任何使用。文中如果涉及企业信息和数据，均由受访者向分析师提供并确认。动脉网，未来医疗服务平台

专利到期基因疗法

2025-06-17

·ir.clearsidebio.com

Clearside Biomedical Announces Multiple Presentations to be Featured at the Clinical Trials at the Summit Meeting

- Broad Use of Suprachoroidal Injection Platform Highlighted Across Retinal Indications - - CLS-AX Combines the Flexibility of Anti-VEGF Therapies with the Long-Lasting Benefits of a Tyrosine Kinase Inhibitor - ALPHARETTA, Ga., June 17, 2025 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD) (“Clearside” or the “Company”), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), announced today that the use of its SCS delivery platform will be featured in multiple presentations at the Clinical Trials at the Summit (CTS) Meeting on June 21, 2025 in Las Vegas, Nevada. Victor Chong, MD, MBA, Chief Medical Officer and EVP, Head of Research and Development, commented, “Our proprietary SCS Microinjector® platform is an established, in-office solution for delivering vision-preserving therapies to the back of the eye. The pipeline of promising clinical programs using our platform underscores its versatility across multiple treatment modalities. We remain committed to redefining care with therapies that offer both extended durability and flexible dosing to address serious retinal diseases.” Presentations Related to Clearside and its Development Partners Title: Suprachoroidal CLS-AX for nAMD: Phase 3 Program Update Presented by: Sobha Sivaprasad, MD, Professor of Retinal Clinical Research, Consultant Ophthalmologist, Moorfields Eye Hospital, London, UK Partner: REGENXBIO Title: Update on One-Time Suprachoroidal ABBV-RGX-314 for the Treatment of DR Presented by: Anna Abolian, OD, Senior Medical Director, Clinical Development Lead, REGENXBIO Inc. Partner: Aura Biosciences Title: Bel-sar - a New Treatment Paradigm in Choroidal Melanoma: Update on the Global Phase 3 CoMpass Trial Presented by: Jennifer Lim, MD, Professor of Ophthalmology, Director, Retina Service, Department of Ophthalmology and Visual Sciences, University of Illinois School of Medicine About CLS-AX (axitinib injectable suspension) Clearside is developing CLS-AX as a longer-acting therapy for the treatment of retinal diseases. CLS-AX (axitinib injectable suspension) is a proprietary suspension of axitinib for suprachoroidal injection. Axitinib is a tyrosine kinase inhibitor (TKI), currently approved as an oral tablet formulation to treat advanced renal cell carcinoma, that achieves pan-VEGF blockade, directly inhibiting VEGF receptors-1, -2, and -3 with high potency and specificity. Clearside believes this broad VEGF blockade may have efficacy advantages over existing retinal therapies by acting at a different level of the angiogenesis cascade and may benefit patients who sub-optimally respond to current, more narrowly focused anti-VEGF therapies. Suprachoroidal injection of this proprietary suspension of axitinib has demonstrated meaningful potential in Phase 1/2a and Phase 2b wet AMD clinical trials in which CLS-AX was well tolerated and demonstrated a positive safety profile. With suprachoroidal administration of axitinib, there is the potential to achieve prolonged duration and targeted delivery to affected tissue layers by compartmentalizing axitinib behind the retina, thereby limiting drug exposure to the front of the eye. About Clearside’s Suprachoroidal Space (SCS®) Injection Platform and SCS Microinjector® Clearside’s patent protected, proprietary suprachoroidal space (SCS®) injection treatment approach offers unprecedented access to the back of the eye, where sight-threatening disease often occurs. The Company’s unique platform is inherently flexible and intended to work with established and new formulations of medications. Clearside’s patented SCS Microinjector® can deliver a wide variety of drug candidates into the suprachoroidal space, providing targeted delivery to potentially improve efficacy and compartmentalization of medication to reduce or eliminate toxic effects on non-diseased cells. The SCS Microinjector is comprised of a syringe with a custom-designed hub and two 30-gauge hollow microneedles of varying lengths, each approximately one millimeter, optimizing insertion and suprachoroidal administration of drugs. About Clearside Biomedical, Inc. Clearside Biomedical, Inc. is a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®) to improve patient outcomes. Clearside’s SCS injection platform, utilizing the Company’s patented SCS Microinjector®, enables an in-office, repeatable, non-surgical procedure for the targeted and compartmentalized delivery of a wide variety of therapies to the macula, retina, or choroid to potentially preserve and improve vision in patients with sight-threatening eye diseases. Clearside is developing its own pipeline of small molecule product candidates for administration via its SCS Microinjector. The Company’s lead program, CLS-AX (axitinib injectable suspension), is in development for the treatment of neovascular age-related macular degeneration (wet AMD). Planning for a Phase 3 program is underway. In addition, Clearside is evaluating various small molecules for the potential long-acting treatment of geographic atrophy (GA). Clearside developed and gained approval for its first product, XIPERE® (triamcinolone acetonide injectable suspension) for suprachoroidal use, which is available in the U.S. through a commercial partner. Clearside also strategically partners its SCS injection platform with companies utilizing other ophthalmic therapeutic innovations. For more information, please visit clearsidebio.com or follow us on LinkedIn and X. Cautionary Note Regarding Forward-Looking Statements Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as “believe”, “expect”, “may”, “plan”, “potential”, “will”, and similar expressions, and are based on Clearside’s current beliefs and expectations. These forward-looking statements include statements regarding the clinical development of Clearside’s product candidates, and the potential benefits of CLS-AX and XIPERE®, Clearside’s suprachoroidal delivery technology and Clearside’s SCS Microinjector®. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include uncertainties inherent in the conduct of clinical trials, Clearside’s reliance on third parties over which it may not always have full control, Clearside's ability to raise additional capital, and other risks and uncertainties that are described in Clearside’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the U.S. Securities and Exchange Commission (SEC) on March 27, 2025, and Clearside’s other periodic reports filed with the SEC. Any forward-looking statements speak only as of the date of this press release and are based on information available to Clearside as of the date of this release, and Clearside assumes no obligation to, and does not intend to, update any forward-looking statements, whether as a result of new information, future events or otherwise. Investor and Media Contacts: Jenny Kobin Remy Bernarda ir@clearsidebio.com Source: Clearside Biomedical, Inc.

临床3期临床结果上市批准临床1期

100 项与 Surabgene Lomparvovec 相关的药物交易

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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
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	波多黎各	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


NCT04514653 (AAVIATE, Corporate Publications) 人工标引	临床2期	湿性黄斑变性	106	ABBV-RGX-314	鬱遞範糧鑰壓鑰衊餘鑰(醖鏇鹽淵糧鑰衊窪窪積) = in the study eye were mild or moderate and included conjunctival hemorrhage, increased intraocular pressure, episcleritis, and conjunctival hyperemia. 膚範簾淵簾積鑰構廠淵 (醖簾鹹觸襯觸製廠積願 ) 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	顧鑰鬱鏇鏇築壓蓋襯淵(網鑰鏇夢膚淵壓鏇餘淵) = three cases of mild intraocular inflammation through 6 months which resolved with topical corticosteroids 窪淵鏇製鹹顧願壓艱鬱 (壓膚夢獵築夢簾鏇齋遞 ) 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	鏇鑰鏇範願夢範壓觸齋(製網簾醖繭鑰鏇築窪構) = No new drug-related ocular adverse events (AEs) have been reported in Cohorts 1-4 淵網餘淵構觸壓糧觸遞 (網衊艱糧蓋襯蓋願鹹餘 ) 更多	-	2023-10-05
ARVO2023	临床1/2期	湿性年龄相关性黄斑变性	42	RGX-314	鬱製構鏇獵範膚積齋鹽(淵憲鬱襯積襯鏇遞獵獵) = 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 夢齋蓋遞艱齋憲夢襯觸 (壓觸窪窪選範簾鹽願顧 )	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	願積餘鏇積獵獵糧簾願(糧糧顧願糧繭淵簾糧築) = 觸製積構夢膚壓願膚願選獵積簾網願糧鑰觸壓 (壓築網範膚廠齋艱餘壓 ) 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	願積餘鏇積獵獵糧簾願(糧糧顧願糧繭淵簾糧築) = 繭憲鑰鹹餘齋鹹衊餘積選獵積簾網願糧鑰觸壓 (壓築網範膚廠齋艱餘壓 ) 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	餘顧膚構網醖鹹築鹹膚(窪壓願夢蓋窪窪糧網窪) = 憲製窪遞淵選窪蓋築衊餘膚簾顧廠築衊繭淵觸 (簾網鏇夢蓋遞窪衊網鑰 ) 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	餘顧膚構網醖鹹築鹹膚(窪壓願夢蓋窪窪糧網窪) = 衊選齋獵網壓膚構憲獵餘膚簾顧廠築衊繭淵觸 (簾網鏇夢蓋遞窪衊網鑰 ) 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	淵糧獵願憲簾鏇衊夢齋(製簾夢積餘窪壓衊鬱鬱) = 網網衊廠遞艱鑰衊築淵窪範膚顧鏇積餘窪遞願 (鹽廠淵窪簾壓壓積願簾 ) 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	淵糧獵願憲簾鏇衊夢齋(製簾夢積餘窪壓衊鬱鬱) = 構願鏇壓鹽簾淵遞繭鏇窪範膚顧鏇積餘窪遞願 (鹽廠淵窪簾壓壓積願簾 ) 更多	积极	2021-10-01
EURETINA2021	N/A	湿性年龄相关性黄斑变性	-	RGX-314	蓋築積糧網範鑰繭醖廠(繭顧鹹鹽觸糧窪獵鑰衊) = 20 serious adverse events (SAEs) reported in 13 patients, including one possibly drug-related SAE of significant decrease in vision in Cohort 5 鹽衊夢遞鏇網願齋鑰獵 (觸選壓製繭獵夢鬱鬱築 ) 更多	-	2021-09-01
RGX-314 (ASGCT2020)	临床1/2期	湿性年龄相关性黄斑变性	42	RGX-314 gene therapy	壓窪夢鏇選夢願遞窪壓(簾簾簾蓋衊製鹽襯餘窪) = improved central retinal thickness (-83 µm) 襯壓餘廠夢積簾積鹹繭 (觸鬱廠簾觸範餘蓋壓蓋 ) 更多	积极	2020-04-28