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2021-12-28

申办/合作机构

AbbVie, Inc.

[+1]

NCT05210803

/ Enrolling by invitationN/A

A Long-term Follow-Up Study to Evaluate the Safety and Efficacy of Suprachoroidal Administration of RGX-314 for Participants With Neovascular Age-Related Macular Degeneration

This is a prospective, observational study designed to evaluate the long-term safety and efficacy of RGX-314. Eligible participants are those who were previously enrolled in a clinical study of nAMD in which they received suprachoroidal space (SCS) administration of RGX-314. Enrollment of each participant in the current study should occur after the participant has completed either the end of study or early discontinuation visit in the previous (parent) clinical study. Participants will be followed for up to 5 years after RGX-314 administration (inclusive of the parent study). As such, the total study duration for each participant may vary depending on when they enroll in the current study following RGX-314 administration in the parent study.

开始日期2021-12-20

申办/合作机构

AbbVie, Inc.

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项与 Surabgene Lomparvovec 相关的文献（医药）

2025-01-01·Eye

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

Review

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

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

OBJECTIVEThis 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.DESIGNReview 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.

2024-06-28·Postgraduate Medical Journal

Advancements in the treatment of age-related macular degeneration: a comprehensive review

Review

作者: Papaioannou, Christos

AbstractAge-related macular degeneration (AMD) stands as a leading cause of irreversible blindness, particularly affecting central vision and impeding daily tasks. This paper provides a thorough exploration of AMD, distinguishing between its two main subtypes—Wet and Dry AMD—while shedding light on the prevalence and risk factors, including age, genetics, and smoking.The focus shifts to the current and future treatment landscape, examining both Dry and Wet AMD. Regarding Dry AMD, interventions such as antioxidant supplementation and ongoing clinical trials offer hope. Notable among these is Pegcetacoplan which is the only Food and Drug Administration (FDA)-approved medication, displaying promising results in reducing geographic atrophy lesions.For Wet AMD, anti-Vascular Endothelial Growth Factor therapies like Ranibizumab (Lucentis®) have been instrumental, and newer drugs like Faricimab and OPT-302 show comparable efficacy with extended dosing intervals. Additionally, gene therapies such as RGX-314 present a potential paradigm shift, reducing or eliminating the need for frequent injections. Biosimilars offer cost-effective alternatives.The paper also delves into the integration of technology and artificial intelligence in AMD management, highlighting the role of smartphone apps for patient monitoring and artificial intelligence algorithms for diagnosis and surveillance. Furthermore, patient perspectives on artificial intelligence demonstrate a positive correlation between understanding and trust.The narrative concludes with a glimpse into ground-breaking technologies, including retinal implants and bionic chips, offering hope for vision restoration. Overall, this paper underscores the multifaceted approach in addressing AMD, combining traditional and innovative strategies, paving the way for a more promising future in AMD treatment.

151

项与 Surabgene Lomparvovec 相关的新闻（医药）

2025-04-30

·PRNewswire

Roche's Susvimo FDA Approval Marks New Era in Diabetic Macular Edema Treatment Market | DelveInsight

Seven months after the FDA approved the reintroduction of Roche's eye implant Susvimo, the US agency has expanded its approved use to include treatment for diabetic macular edema, the primary cause of blindness related to diabetes. This marks the second approved indication for Susvimo, which was initially cleared in October 2021 as an alternative to frequent eye injections for wet age-related macular degeneration. LAS VEGAS, April 30, 2025 /PRNewswire/ -- Diabetic macular edema is a complication that develops from diabetic retinopathy, a widespread consequence of diabetes and a leading cause of irreversible vision loss in working-age adults globally. Diabetic retinopathy occurs due to long-term damage to the retina's small blood vessels, leading to fluid leakage and swelling—especially in the macula. As per DelveInsight's analysis, there were about 1.9 million prevalent cases of DME across the 7MM in 2023, with numbers expected to grow at a notable CAGR during the forecast period. The primary treatment for DME includes intravitreal injections, where the eye is numbed with drops before injecting medication directly into the vitreous humor. Common anti-VEGF drugs used include AVASTIN, EYLEA, and LUCENTIS. Additionally, corticosteroids are beneficial in cases of DME linked to inflammatory eye conditions and can be delivered via eye drops, oral tablets, or periocular injections to help reduce inflammation. The US FDA approved sustained-release corticosteroid implants for more serious or longer-lasting conditions, are OZURDEX, RETISERT, and ILUVIEN. Learn more about the diabetic macular edema treatment @ New Treatment for Diabetic Macular Edema EYLEA (Regeneron Pharmaceuticals) – Aflibercept is a recombinant fusion protein composed of the VEGF-binding regions derived from the extracellular domains of human VEGF receptors 1 and 2, linked to the Fc segment of human IgG1. It functions as a VEGF trap by binding to circulating vascular endothelial growth factors (VEGFs), thereby neutralizing VEGF-A, VEGF-B, and placental growth factor (PGF). This action inhibits the formation of new blood vessels in the tumor's choriocapillaris. In July 2014, the U.S. FDA approved EYLEA (aflibercept) Injection for treating diabetic macular edema (DME). Ranibizumab (Genentech) – Marketed as LUCENTIS, ranibizumab is a monoclonal antibody fragment (Fab) derived from the same original mouse antibody as bevacizumab. It is an anti-angiogenic therapy used to treat the "wet" form of age-related macular degeneration (AMD), a leading cause of vision loss in older adults. Ranibizumab has shown comparable effectiveness to bevacizumab. Developed by Genentech, it is marketed in the U.S. by Genentech and internationally by Novartis. The FDA approved ranibizumab for the treatment of DME in 2012. In February 2025, Roche announced that the FDA had approved Susvimo (ranibizumab injection) 100 mg/mL for treating diabetic macular edema (DME), a major cause of vision loss in adults with diabetes. This approval is based on positive one-year data from the phase III Pagoda trial, which demonstrated that Susvimo offered sustained improvements in vision for individuals with DME, with a safety profile consistent with what is already known for the drug. In the study, patients receiving Susvimo refills every six months experienced vision gains comparable to those receiving monthly intravitreal injections of 0.5 mg ranibizumab (9.6 letters versus 9.4 letters on an eye chart, equivalent to about two lines of vision improvement). Susvimo delivers a specially formulated version of ranibizumab continuously through the Port Delivery Platform, offering a less frequent dosing alternative compared to other treatments that may require monthly eye injections. The FDA initially approved Susvimo in 2021 for the treatment of neovascular age-related macular degeneration (nAMD). Regulatory reviews in other countries are currently underway. Learn more about the FDA-approved DME drugs @ Drugs for Diabetic Macular Edema Treatment Key players, such as Oculis, Rezolute, AsclepiX Therapeutics, and others, are evaluating their lead candidates in different stages of clinical development, respectively. They aim to investigate their products for the treatment of diabetic macular edema. The DME market has a promising outlook with the emerging therapies. Some of the drugs in the pipeline are OCS-01, RZ402, AXT107, and others. DME leads to significant vision loss in diabetics, affecting up to 7.9% of type 1 and 12.8% of type 2 diabetes patients. AbbVie, in collaboration with REGENEXBIO, is developing the gene therapy ABBV-RGX-314. This therapy targets wet age-related macular degeneration, diabetic retinopathy (DR), and other chronic retinal diseases, potentially offering a breakthrough in treating these conditions. Discover which therapies are expected to grab major DME market share @ Diabetic Macular Edema Market Report OCS-01 is a high-concentration dexamethasone eye drop developed for diabetic macular edema (DME), utilizing the proprietary Optireach solubilizing platform. This technology enhances drug solubility, allows for higher concentrations, prolongs ocular retention, and improves bioavailability in eye tissues, including the retina. Recently, in April 2025, Oculis announced the completion of patient enrollment for its Phase 3 DIAMOND-1 and DIAMOND-2 clinical trials evaluating OCS-01 eye drops for diabetic macular edema (DME). These pivotal registration studies aim to support global regulatory filings, including a New Drug Application (NDA) submission and potential approval by the U.S. FDA. Over 800 patients were enrolled across 119 sites in the U.S. and other countries. The swift completion of enrollment in the DIAMOND program—comprising two double-masked, randomized, multi-center Phase 3 trials—is a significant milestone. These studies are assessing the efficacy and safety of OCS-01 over a 52-week treatment period in patients with DME. Topline results are anticipated in Q2 2026, followed by an NDA submission. If approved, OCS-01 would be the first topical eye drop treatment for DME, offering a novel therapeutic option for early intervention and for patients who do not adequately respond to anti-VEGF therapies. RZ402 is an oral small-molecule drug that selectively inhibits plasma kallikrein (PK) for the long-term treatment of DME. By blocking kallikrein activation, RZ402 aims to prevent bradykinin-driven vascular leakage and inflammation. Phase II results revealed a significant reduction in central subfield thickness (CST) across all dosing levels, with improvements of up to ~50 microns compared to placebo. The treatment was well tolerated and showed a favorable safety profile. AXT107 targets two validated mechanisms for treating retinal vascular diseases: it inhibits the pro-angiogenic receptor VEGFR2 and activates the vessel-stabilizing Tie2 receptor. These effects are mediated through its interaction with integrins αvβ3 and α5β1. AXT107 is formulated as a microparticulate suspension designed for intraocular injection and is currently undergoing Phase I/II clinical trials, according to the company's development pipeline. Discover more about drugs for DME in development @ Diabetic Macular Edema Clinical Trials The anticipated launch of these emerging therapies for DME are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the DME market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for DME is expected to grow from USD 3 billion in 2023, with a significant CAGR by 2034. Owing to the higher patient pool and higher diabetic macular edema treatment cost, according to the estimates, the United States had the highest market size in DME, i.e., ~60% of the total market size of DME in the 7MM, in 2023, followed by Germany and Japan. DelveInsight's analysis forecasts market growth due to the introduction of emerging therapies, expecting a rise in market size during the study period (2020–2034). The anticipated increase in market size is driven by advancements in treatment options, greater healthcare access, and a rising prevalence of the condition, which together foster higher demand for innovative and effective therapies. DelveInsight's latest published market report, titled as Diabetic Macular Edema Market Insight, Epidemiology, and Market Forecast – 2034 , will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the DME country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The DME market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of DME Total Diagnosed Prevalent Cases of DME Gender-specific Diagnosed Prevalent Cases of DME Age-Specific Diagnosed Prevalent Cases of DME Subgroup-specific Diagnosed Prevalent Cases of DME The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM DME market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this DME market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the DME market. Also, stay abreast of the mitigating factors to improve your market position in the DME therapeutic space. Related Reports Diabetic Macular Edema Epidemiology Forecast Diabetic Macular Edema Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted diabetic macular edema epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Diabetic Macular Edema Pipeline Diabetic Macular Edema Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key diabetic macular edema companies, including Mylan Pharmaceuticals, Kodiak Sciences, Celltrion, Exonate, Opthea, AsclepiX Therapeutics, Allgenesis Biotherapeutics, Ascentage Pharma, Rezolute, Ocuphire Pharma, Oxurion, MingSight Pharmaceuticals, Adverum Biotechnologies, among others. Diabetic Retinopathy Market Diabetic Retinopathy Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key diabetic retinopathy companies, including Genentech, Inc., Regeneron Pharmaceuticals, Roche, Opthea Limited, Regenxbio, Kodiak Sciences Inc, Ocuphire Pharma, Eisai Co Ltd, Apexian Pharmaceuticals, Oculis, among others. Diabetic Retinopathy Pipeline Diabetic Retinopathy Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key diabetic retinopathy companies, including Kodiak Sciences, Novartis, Regenxbio Inc., OcuTerra Therapeutics, Ocular Therapeutix, Bayer, RemeGen, Roche, Ocuphire Pharma, Adverum Biotechnologies, Boehringer Ingelheim, Palatin Technologies, Valo Health, EyePoint Pharmaceuticals, Kubota Vision, MingSight Pharmaceuticals, Oxurion, Aerie Pharmaceuticals, AsclepiX Therapeutics, Ocugen, Ashvattha Therapeutics, Stealth BioTherapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床结果临床3期上市批准临床2期

2025-04-27

·E药经理人

左手创新药，右手“老字号”，解码康弘药业业绩稳健的驱动力

在资本寒冬中，真正具有临床价值和技术壁垒的创新，永远不缺市场买单。中国药企通往FIC的道路或许荆棘密布，但创新无捷径，唯敢为人先。从一款改写国内眼底病治疗格局的康柏西普，到全球首个双载荷ADC药物，再到基因治疗管线的中美双线推进，康弘药业用30余年的坚守创新，实现着从传统药企到生物技术领跑者的蜕变。随着2024年年报的出炉，康弘药业的发展脉络愈发清晰。从数据来看，全年实现营业收入44.53亿元，同比增长12.51%；净利润为11.91亿元，同比增长14.02%。这样的稳健发展，在竞争激烈的医药市场中显得尤为难得。近年来，康弘药业的营收和利润保持着稳定的增长态势，2014-2023年营业收入年复合增长率为10.4%，净利润复合增长率为12.4%，而2024年的成绩更是在这一增长曲线上迈出了坚实的一步。从康柏西普到前沿技术领域的布局，康弘药业的突围之路，恰是中国创新药升级的缩影。01生物药拉动增长深入分析康弘药业的收入结构，生物药板块无疑是最大的亮点。2024年，康弘药业生物药业务营收23.43亿元，同比增长20.98%，占总营收的比重达52.61%，同比增加3.68个百分点。其中，作为上市11年的产品，康柏西普连续10年保持增长，已完成超过250万次注射，在眼底疾病用药市场龙头地位稳固。作为国内首个抗VEGF融合蛋白药物，康柏西普自2014年上市销售以来，就成为了康弘药业的业绩增长引擎。2015-2023年收入年复合增长率高达28%。数据的背后，是国内老龄化社会催生的刚性需求。目前，康柏西普已获批四大适应证，包括湿性年龄相关性黄斑变性（AMD）、糖尿病性黄斑水肿（DME）、脉络膜新生血管（CNV）及继发于视网膜静脉阻塞（RVO）的黄斑水肿。据统计，我国60岁以上人群湿性AMD患病率达2.79%，糖尿病患者中DME发病率超过6.8%，四大适应症对应患者超2000万人，潜在市场空间巨大。但是，传统抗VEGF药物需每月注射的痛点，让依从性成为行业最大瓶颈。康弘的破局之道颇具智慧——在保持疗效的同时，通过医保谈判将价格降至3452元/支（仅为雷珠单抗的87%），并创新推出“3+PRN”（初始3针负荷剂量后按需治疗）方案，将年均注射次数压缩至6-8次。这种“性价比+治疗便利性”的组合拳，使其在2022年新增RVO适应症后，市占率提升5%。从市场竞争格局来看，在国内眼科抗VEGF药物市场，主要参与者包括康柏西普、雷珠单抗、阿柏西普、法瑞西单抗等产品。根据PDB数据，2023年康柏西普样本医院销售额为5亿元，销售占比达36%，市占率较高，硬生生从诺华、拜耳等跨国巨头手中夺取国内市场份额。尽管市场中齐鲁制药的阿柏西普类似物、雷珠单抗类似物已经获批上市，还有多款生物类似物和抗 VEGF 创新药在研，但康柏西普凭借其多年积累的临床数据、医生信任度及患者依从性，依然保持着稳固的市场地位。随着老龄化趋势的加剧，眼底病用药市场规模持续扩大，康柏西普有望继续受益于市场扩大的趋势，实现业绩的持续增长。面对生物类似药的竞争，康弘的应对策略更显“老辣”。当齐鲁制药的阿柏西普类似物以2970元的低价杀入市场时，康弘同步推进高浓度剂型KH902-R10的临床试验。参考拜耳数据，高浓度阿柏西普可将96周注射次数从14次降至8次，若康弘新剂型落地，有望在“长效化”赛道上再筑护城河。02中药老字号寻新生中药业务在康弘药业的收入结构中也占据着重要地位，为公司提供了稳定的现金流。康弘药业旗下百年老字号济生堂拥有多款独家中药新药，如舒肝解郁胶囊、松龄血脉康胶囊和渴络欣胶囊等。其中，舒肝解郁胶囊是中国首个治疗轻中度抑郁症的中成药，与传统西药相比，药效相当且安全性更佳。据《2022年国民抑郁症蓝皮书》，目前，我国患抑郁症人数 9500万。独家品种舒肝解郁胶囊于2022年获得国家药监局批准成为中药二级保护品种。米内网数据显示，2021年舒肝解郁胶囊终端销售额5亿元，同比增长10.1%，2013-2021年复合增长率为12%。至2023年，舒肝解郁胶囊的终端销售额达6.8亿元，在化药主导的抗抑郁市场中，凭借“现代循证医学+传统组方智慧”融合的方式，撕开了一道口子。目前，舒肝解郁胶囊新增焦虑症适应症的Ⅲ期临床试验正在稳步推进，随着精神健康问题日益受到关注，有望覆盖更多用药群体，进一步提升业绩。在产业升级方面，全面提升中药生产线的自动化、智能化水平，推进节能降耗、降本增效。为了保障产品质量和稳定供给，康弘药业构筑了中药材全产业链质量管理体系，将中药的质量管理延伸至中药材种植及加工环节，并建立药材信息追溯系统和可视化系统，同时积极探索中药材种植技术，并在全国建立了多个中药材种植基地，保障了舒肝解郁胶囊、松龄血脉康胶囊、渴络欣胶囊等多个临床所需创新中药的稳定供给。03All in 创新康弘始终坚持创新战略，以眼科、精神神经、肿瘤三大核心治疗领域为主线，持续加大研发投入。随着去年4月FDA的一纸批文，全球眼科创新的目光聚焦至康弘药业，其自主研发的KH658基因治疗药物获准开展用于治疗新生血管性（湿性）年龄相关性黄斑变性（nAMD）的临床试验，这是康弘药业继KH631之后，第二款进入中美双报阶段的AAV基因疗法。目前，KH631已进入Ⅱ期临床试验阶段，KH658Ⅰ期临床试验正稳步推进。这些阶段性成果的背后，是一场关乎技术路线的生死竞速。与传统抗VEGF药物“治标不治本”不同，基因治疗通过AAV病毒载体将治疗基因导入视网膜细胞，使患者自体持续表达抗VEGF蛋白，理论上可实现“一次治疗终生有效”。其中，在解决载体递送效率、免疫原性及长期安全性等问题，康弘采用AAV载体优化设计，降低免疫反应风险，同时采用差异化给药途径（如脉络膜上腔注射），具有给药方式简便、安全、转导细胞效率高的特点。放眼全球，REGENXBIO与艾伯维合作的ABBV-RGX-314已进入美国临床Ⅲ期阶段，华福证券研报指出，若2025年关键临床数据积极，康弘有望成为全球第三家获批眼底病基因治疗的企业，占得千亿级市场先机。如果说基因治疗是康弘的未来引擎，那么KH815的横空出世，则让全球看到了中国药企在ADC领域的颠覆创新。2025年4月16日，CDE官网最新公示，康弘药业全资子公司康弘生物申报的I类新药注射用KH815获批临床，拟开发治疗晚期实体瘤。这是康弘生物研发的一种具有抗耐药潜力的靶向滋养层细胞表面抗原2（TROP2）的新型双载荷（dual-payload）抗体偶联药物。该产品于今年3月在澳大利亚获批I期临床研究。TROP2是一种广泛表达的糖蛋白抗原，可促进肿瘤细胞增殖、侵袭、转移扩散等过程，其高表达与肿瘤患者生存期缩短及不良预后密切相关。公开资料显示，KH815由人源化的IgG1抗体（hRS7）组成，该抗体直接针对TROP2，结合拓扑异构酶I抑制剂（TOP1i）和RNA聚合酶II抑制剂（RNA POL IIi）。KH815的双载荷使其能够同时抑制RNA合成和诱导DNA双链断裂，具有双效协同机制。体内药效研究显示，KH815在多个瘤种的CDX模型和PDX型中均表现出剂量依赖的抑制肿瘤生长作用，并且在多个喜树碱类ADC耐药的CDX和PDX模型中也显示出抗肿瘤作用。曾有人将双载荷ADC比作是一款未来的“核武器”：双载荷不是简单的1+1，而是要像交响乐指挥家，让两种毒素在时空上精准协同。而这背后，是康弘对ADC技术路线的深刻洞察。华福证券测算，若KH815后续顺利上市，首年销售额有望突破15亿元，带动公司估值重塑。整体而言，康弘药业在 2024 年交上了一份令人满意的答卷。其营收业绩和利润的稳健增长，得益于生物药、中药等各业务板块的协同发展。而在创新战略的驱动下，丰富的在研管线为公司的未来发展奠定了坚实基础。站在2025年的十字路口，康弘的航道上并非全是风平浪静。基因治疗与双载荷的前景尚未可知，目前全球尚无眼底病基因治疗产品获批上市。在创新药研发“高风险高回报”的丛林法则下，在未被满足的临床需求领域，敢为人先者方有定价权。“丛林竞争”固然激烈，不过凭借着强大的创新实力、优质的产品管线以及稳健的经营策略，康弘药业有望在未来的市场竞争中继续保持领先地位，为患者带来更多新的治疗选择。一审| 黄佳二审| 李芳晨三审| 李静芝

财报抗体药物偶联物申请上市临床研究

2025-03-20

·PipelineReview

REGENXBIO reports positive biomarker data from AFFINITY DUCHENNE trial of RGX-202 gene therapy

ROCKVILLE, MD, USA I March 19, 2025 I REGENXBIO Inc. (Nasdaq: RGNX ) today reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE ® trial of RGX-202, a differentiated investigational gene therapy for Duchenne muscular dystrophy (Duchenne). Results were presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. “RGX-202 is the only next generation gene therapy for Duchenne in a pivotal phase trial. The new data from the age 1-3 cohort builds on the favorable safety and efficacy profile seen in ages 4 and older and reinforces the potential of RGX-202 to serve a wide age range of patients,” said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. “The consistent, robust microdystrophin levels seen across the age range as well as the functional improvements previously reported support RGX-202’s potential to alter the course of this devastating disease. We look forward to sharing additional Phase I/II functional data in the first half of 2025. We also continue to rapidly advance the pivotal trial towards completing enrollment this year and BLA submission mid-2026.” “Patients with Duchenne continue to be in need of treatment options that could meaningfully impact the course of disease,” said Carolina Tesi-Rocha, M.D., Stanford Children’s Hospital. “The microdystrophin expression and biomarker data presented represent key indicators of potential therapeutic effect. Combined with the safety and functional data to date, I am highly encouraged by the profile of RGX-202.” AFFINITY DUCHENNE Phase I/II Interim Data Updates Biomarker Data New biomarker data from two patients who received the pivotal dose of RGX-202 were presented at MDA and continue to support consistent, robust expression and transduction of RGX-202 microdystrophin across all ages. In a patient aged 3 at dosing, microdystrophin expression was measured to be 122.3% compared to control. Patients under 4 years old have no access to gene therapy, and REGENXBIO is the only gene therapy sponsor recruiting patients in this age group in the U.S. In a patient aged 7 years old, RGX-202 microdystrophin expression was measured to be 31.5% compared to control. In all patients, RGX-202 was appropriately localized to the sarcolemma, demonstrating the differentiated construct with the CT-Domain is appropriately targeting the muscle. RGX-202 microdystrophin expression results in ambulatory patients aged 8+ are the highest reported microdystrophin levels across approved or investigational gene therapies. To support a Biologics License Application (BLA) using the accelerated approval pathway, the primary endpoint in the pivotal phase of AFFINITY DUCHENNE is the proportion of participants whose RGX-202 microdystrophin expression is ≥10% at Week 12. RGX-202 also continues to demonstrate the highest reported vector genome copies (4.9-55.4) measured by qPCR across approved or investigational gene therapies. Safety and Tolerability Data As of February 21, 2025, RGX-202 was well tolerated with no serious adverse events (SAEs) and no AEs of special interest (AESIs). Common drug-related AEs included nausea, vomiting and fatigue. All resolved and are typically anticipated with gene therapy administration. A thorough, proactive, short-course immune modulation regimen in combination with industry-leading product purity levels of more than 80% full capsids may contribute to the favorable safety profile seen in patients receiving RGX-202 to date. As reported in November 2024, the first five participants in the Phase I/II portion of the AFFINITY DUCHENNE trial all showed functional improvements that exceeded external natural history controls, demonstrating evidence of RGX-202 positively impacting disease trajectory. ( press release ). Patients demonstrated stable or improved function on the North Star Ambulatory Assessment (NSAA) and timed function tests. REGENXBIO plans to share additional interim functional data in the first half of 2025. About RGX-202 RGX-202 is a potential best-in-class investigational gene therapy designed for improved function and outcomes in Duchenne. RGX-202 is the only gene therapy approved or in late-stage development for Duchenne with a differentiated microdystrophin construct that encodes key regions of naturally occurring dystrophin, including the C-Terminal (CT) domain. In preclinical studies, the CT domain has been shown to protect the muscle from contraction-induced stress and improve its ability to repair itself. Additional design features may potentially improve gene expression, increase protein translation efficiency and reduce immunogenicity. RGX-202 is designed to support the delivery and targeted expression of microdystrophin throughout skeletal and heart muscle using the NAV ® AAV8 vector and a well-characterized muscle-specific promoter (Spc5-12). RGX-202 is manufactured using REGENXBIO’s proprietary, high-yielding NAVXpress™ suspension-based platform process. About Duchenne Muscular Dystrophy Duchenne is a severe, progressive, degenerative muscle disease, affecting 1 in 3,500 to 5,000 boys born each year worldwide. Duchenne is caused by mutations in the Duchenne gene which encodes for dystrophin, a protein involved in muscle cell structure and signaling pathways. Without dystrophin, muscles throughout the body degenerate and become weak, eventually leading to loss of movement and independence, required support for breathing, cardiomyopathy and premature death. 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 ® . REGENXBIO’s investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.REGENXBIO.com . SOURCE: REGENXBIO

基因疗法临床结果

100 项与 Surabgene Lomparvovec 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
湿性年龄相关性黄斑变性	临床3期	德国	AbbVie, Inc.	2021-12-28
湿性年龄相关性黄斑变性	临床3期	匈牙利	AbbVie, Inc.	2021-12-28
湿性年龄相关性黄斑变性	临床3期	法国	AbbVie, Inc.	2021-12-28
湿性年龄相关性黄斑变性	临床3期	英国	AbbVie, Inc.	2021-12-28
湿性年龄相关性黄斑变性	临床3期	日本	AbbVie, Inc.	2021-12-28
湿性年龄相关性黄斑变性	临床3期	美国	AbbVie, Inc.	2021-12-28
湿性年龄相关性黄斑变性	临床3期	波多黎各	AbbVie, Inc.	2021-12-28
湿性年龄相关性黄斑变性	临床3期	意大利	AbbVie, Inc.	2021-12-28
湿性年龄相关性黄斑变性	临床3期	西班牙	AbbVie, Inc.	2021-12-28
湿性黄斑变性	临床3期	美国	AbbVie, Inc.	2020-12-29

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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	(窪蓋選鹹膚膚構鬱壓淵) = in the study eye were mild or moderate and included conjunctival hemorrhage, increased intraocular pressure, episcleritis, and conjunctival hyperemia. 鹹範鬱鹽夢艱獵膚窪鹽 (窪鏇遞鹹鏇衊艱齋選鑰 ) 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	網蓋簾艱選網築糧衊選(鑰獵夢選簾鏇網選願繭) = three cases of mild intraocular inflammation through 6 months which resolved with topical corticosteroids 願鏇廠蓋構範構製膚製 (鬱齋壓觸醖繭蓋淵遞繭 ) 更多	-	2023-10-05
				RGX-314 at an increased dose level of 5 x E11 GC/eye
EURETINA2023	N/A	年龄相关性黄斑变性	-	RGX-314	製糧鬱鹽憲製醖積膚範(餘遞鑰繭簾製衊夢鏇觸) = No new drug-related ocular adverse events (AEs) have been reported in Cohorts 1-4 蓋獵夢觸夢鬱襯艱夢窪 (艱積襯鑰鹽廠艱憲範網 ) 更多	-	2023-10-05
NCT04567550 (ALTITUDE, Corporate Publications) 人工标引	临床2期	糖尿病性视网膜病变	60	RGX-314	(憲醖顧艱鬱衊衊鏇築築) = 鬱顧膚夢鬱鏇膚鹽襯蓋鏇顧夢鹽遞艱壓簾壓鑰 (繭淵糧餘蓋齋構淵積積 ) 更多	积极	2022-11-02
				control	(憲醖顧艱鬱衊衊鏇築築) = 淵衊夢壓膚繭範構鬱製鏇顧夢鹽遞艱壓簾壓鑰 (繭淵糧餘蓋齋構淵積積 ) 更多
NCT04567550 (ALTITUDE, Corporate Publications) 人工标引	临床2期	糖尿病性视网膜病变	20	RGX-314	(選鹽鹽顧繭鑰艱夢壓蓋) = 膚製鑰糧膚網艱顧艱廠選窪淵願鏇壓鹽衊遞膚 (構製餘衊獵簾醖淵齋憲 ) 更多	积极	2022-09-13
				Observational	(選鹽鹽顧繭鑰艱夢壓蓋) = 鬱獵範獵構蓋夢鬱襯糧選窪淵願鏇壓鹽衊遞膚 (構製餘衊獵簾醖淵齋憲 ) 更多
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	(衊襯鑰選願鑰鹹鏇膚願) = 選遞願選網蓋選製餘膚築遞鑰窪糧鹽觸鑰願網 (襯獵憲網鑰築鏇願觸鏇 ) 更多	积极	2021-10-01
				Ranibizumab	(衊襯鑰選願鑰鹹鏇膚願) = 鬱簾遞蓋壓構顧顧簾糧築遞鑰窪糧鹽觸鑰願網 (襯獵憲網鑰築鏇願觸鏇 ) 更多
EURETINA2021	N/A	湿性年龄相关性黄斑变性	-	RGX-314	(遞憲憲夢夢糧製醖鬱艱) = 20 serious adverse events (SAEs) reported in 13 patients, including one possibly drug-related SAE of significant decrease in vision in Cohort 5 衊範淵鬱鹽鬱製襯糧窪 (願構獵窪糧鑰鏇艱齋衊 ) 更多	-	2021-09-01