Avacincaptad pegol

In GATHER2 open-label extension study, IZERVAY reduced GA growth 37-40.5% vs. projected sham, with earlier intervention resulting in greater protection of retinal tissue area Study results found no new safety signals and no cases of retinal vasculitis or occlusive vasculitis after IZERVAY treatment of up to 3.5 years New U.S. real-world data from more than 10,000 GA patients support safety and tolerability of IZERVAY with low rates of discontinuation and adverse events TOKYO, Oct. 19, 2025 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") today announced the first results from the open-label extension trial of the Phase 3 GATHER2 study, which demonstrated monthly treatment with IZERVAY™ (avacincaptad pegol intravitreal solution) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) continued to reduce GA lesion growth for up to 3.5 years, with earlier intervention resulting in greater protection of retinal tissue area. IZERVAY was well tolerated, with no cases of retinal vasculitis or occlusive vasculitis. These findings, which indicate a cumulative treatment benefit with IZERVAY, were presented at the American Academy of Ophthalmology 2025 Annual Meeting (AAO 2025) during the Retina Subspeciality Day in Orlando, Fla. During the 18-month open-label extension (OLE) trial, GA patients who previously completed the GATHER2 study switched from IZERVAY monthly (EM) or every-other-month (EOM) treatment to IZERVAY EM, or from sham to IZERVAY EM. In the OLE study, disease progression, as measured by mean change in GA lesion growth area was reduced by 40.5% from month 24 (mm2/year) vs. projected sham in participants who switched from IZERVAY EM or EOM to IZERVAY EM at 3.5 years, and by 37% vs. projected sham in the IZERVAY EM group who previously received sham (p<0.001 for each). Earlier intervention with IZERVAY consistently resulted in greater protection of retinal tissue area: 2.92 mm2 in the group that switched from IZERVAY EM or EOM to IZERVAY EM at 3.5 years and 1.83 mm2 in participants treated with IZERVAY EM following sham. IZERVAY was well-tolerated with no new safety signals, no cases of retinal vasculitis or occlusive vasculitis, and no increased risk of intraocular inflammation. Arshad M. Khanani, MD, MA, FASRS, Director of Clinical Research at Sierra Eye Associates, Clinical Professor at University of Nevada, Reno, Nevada "The first reported findings from this open-label extension study corroborate the favorable efficacy and safety outcomes previously demonstrated in the 2-year GATHER2 trial. IZERVAY maintains a consistent safety profile and is associated with meaningful reduction in disease progression over time, with the most pronounced benefits observed in patients who initiated treatment at an earlier stage." A separate real-world study highlighted treatment patterns and safety of IZERVAY in patients with GA (n=10,181 patients, 13,391 eyes) treated in routine clinical practice. Based on data from the American Academy of Ophthalmology IRIS® Registry (Intelligent Research In Sight), this observational analysis included data from U.S. patients who received IZERVAY during its first year of approval by the U.S. Food and Drug Administration (FDA). This retrospective study found no new safety signals and low rates of discontinuation, supporting the safety and tolerability of IZERVAY. Key real-world study results include: In patients who received [IZERVAY/ACP] at least twice, the mean interval between injections per patient eyes (n=11,799) was 7.0 weeks While nearly 9 in 10 patients (87.1%) had GA in both eyes, 68.5% received IZERVAY in one eye 22.7% of patient eyes received anti-VEGF therapy in addition to IZERVAY, of which nearly 90% had previously been on anti-VEGF treatment Overall, 3.6% of patients (2.9% of eyes) experienced any adverse event (AE) during the 25-week study follow-up period, for an event rate of 103 per 10,000 IZERVAY injections Marci English, Senior Vice President, Biopharma and Ophthalmology Development, Astellas Pharma "IZERVAY continues to be a trusted treatment option for countless patients living with GA and the fear of vision loss. At Astellas, we are committed to deepening our understanding of this condition, and our latest findings shed light on both disease progression and treatment strategies. The ongoing research and data continue to suggest the crucial need for earlier detection and intervention to support patients in their journey to preserve their vision for as long as possible." Another Astellas study at AAO 2025 examined the relationship between structure and function in GA by analyzing ellipsoid zone (EZ) integrity and low luminance deficit (LLD) in eyes with GA treated with IZERVAY. This post hoc analysis of pooled 18-month data from the GATHER1 and GATHER2 studies demonstrated a structure-function link in GA between photoreceptor health, measured by EZ integrity, and visual function, captured by LLD. Treatment with IZERVAY slowed progressive loss of photoreceptor signal intensity in the EZ compared to sham over 18 months, particularly in eyes with greater baseline LLD, i.e., more severe disease. The findings support the structure-function link between EZ integrity and LLD as biomarkers to help measure GA progression and the impact of treatment. The results also suggest LLD may be a better indicator of visual function in GA than best-corrected visual acuity (BVCA). IZERVAY is approved for the treatment of GA in the United States, Australia and conditionally in Japan. Astellas continues to engage with regulatory authorities around the world to bring ACP to GA patients globally. About IZERVAY ™ (avacincaptad pegol intravitreal solution) INDICATION AND IMPORTANT SAFETY INFORMATION What is IZERVAY™? IZERVAY (avacincaptad pegol intravitreal solution) is a prescription eye injection, used to treat geographic atrophy (GA), the advanced form of dry age-related macular degeneration (AMD). What is the most important information I should know about IZERVAY? Do NOT receive IZERVAY if you: Have an infection in or around your eye Have active swelling in or around your eye that may include pain and redness IZERVAY can cause serious side effects: Eye injections like the one for IZERVAY can cause an eye infection (endophthalmitis) or separation of layers of the retina (retinal detachment). Call your healthcare provider right away if you have redness of the eye, eye pain, increased discomfort, worsening eye redness, blurred or decreased vision, an increased number of small specks floating in your vision, flashes of light, or increased sensitivity to light. There is a risk of developing wet AMD with IZERVAY. You should report any symptoms (visual distortions such as straight lines seeming bent, deterioration in vision, dark spots, loss of central vision) to your healthcare provider to monitor. IZERVAY may cause a temporary increase in eye pressure after the injection. Your healthcare provider will monitor this after each injection. Before receiving IZERVAY tell your healthcare provider about all of your medical conditions including if you: Have a history of seeing flashes of light or small specks floating in your vision and if you have a sudden increase of size and number of these specks. Have high pressure in the eye or if you have glaucoma. Are pregnant or breastfeeding, think you may be pregnant, or are planning to have a baby, ask your doctor for advice before taking this medicine. Are taking any medications, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Tell your healthcare provider about any medicine you take. What should I avoid while receiving IZERVAY? Your vision may be impaired after receiving an eye injection or after an eye exam. Do not drive or use machinery until your vision has recovered sufficiently. What are the most common side effects of IZERVAY? Blood in the white of the eye Increase in eye pressure Blurred vision Wet age-related macular degeneration These are not all the possible side effects of IZERVAY. Tell your healthcare provider about any side effect that bothers you or that does not go away. Call your healthcare provider for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-FDA-1088. Please see full Prescribing Information for more information. About Geographic Atrophy  Age-related macular degeneration (AMD) is the major cause of moderate and severe loss of central vision in aging adults, affecting both eyes in the majority of patients. The macula is a small area in the central portion of the retina responsible for central vision. As AMD progresses, the loss of retinal cells and the underlying blood vessels in the macula results in marked thinning and/or atrophy of retinal tissue. Geographic atrophy, associated with AMD, leads to further irreversible loss of vision in these patients.  About the GATHER Clinical Trials IZERVAY met its primary endpoint in the GATHER1 (NCT02686658) clinical trial and the GATHER2 (NCT04435366) clinical trial, both of which were randomized, double-masked, sham-controlled, multicenter Phase 3 clinical trials. These trials evaluated the safety and efficacy of monthly 2 mg intravitreal administration of IZERVAY in patients with GA secondary to AMD. For the first 12 months in both trials, patients were randomized to receive either IZERVAY 2 mg or sham monthly. There were 286 participants enrolled in GATHER1 and 448 participants enrolled in GATHER2. The primary efficacy endpoints in both pivotal studies were based on GA area measured by fundus autofluorescence at three time points: baseline, month 6, and month 12. Safety was evaluated in over 700 patients with GA across the two trials. In year 2 of the GATHER2 study, patients treated with IZERVAY in year 1 were re-randomized to receive either IZERVAY dosed monthly (EM, n=96) or every other month (EOM, n=93); patients who received sham in year 1 continued to receive sham in year 2 (n=203). IZERVAY is continuing to be evaluated in an open-label extension study.  About Astellas Astellas is a global life sciences company committed to turning innovative science into VALUE for patients. We provide transformative therapies in disease areas that include oncology, ophthalmology, urology, immunology and women's health. Through our research and development programs, we are pioneering new healthcare solutions for diseases with high unmet medical need. Learn more at . Cautionary Notes In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management's current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas' intellectual property rights by third parties. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice. SOURCE Astellas Pharma Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

今天，小编带大家认识一位四院院士——谭蔚泓，一位以化学“解码”生命的科学家，其科研生涯与核酸适体、癌症靶向治疗及生物纳米技术等领域紧密相连。1960年出生于湖南益阳农家，他在改革开放的浪潮中迎来人生转折——1978年考入湖南师范大学化学系，成为恢复高考后的首批大学生。本科毕业后，他并未止步，于1985年在中国科学院山西煤炭化学研究所获物理化学硕士学位，由此踏上科研之路。随后，他于1987年赴美国密歇根大学深造，1992年获物理化学博士学位。在密歇根求学期间，他不仅拓展了学术视野，更形成了贯穿一生的科研哲学：“智商、情商与乐商的平衡是成功的关键”。从材料与催化科学起步，到以核酸适体为“分子钥匙”开创癌症靶向诊疗新范式，谭蔚泓逐步成长为四院院士（中国科学院、发展中国家科学院、欧洲科学院、美国科学促进会会士），并领衔国家级医学研究所，实现了从农家少年到国际知名科学家的跨越。 近日，中国科学院杭州医学研究所谭蔚泓院士、方晓红教授、渠凤丽研究员团队综述了介绍了FNA在推动分子生物学和精准医疗发展方面的变革性影响，强调了其结构的多功能性和功能潜力。他们探讨了关键FNA（例如适体和脱氧核糖核酸酶）的生产方法、结构原理和生物学作用，以及旨在优化其稳定性、亲和力和催化效率的工程策略。此外，本综述还讨论了FNA的临床试验、挑战和前景，将其定位为生物医学应用的强大分子工具，可以应对精准医疗、靶向治疗和诊断领域的复杂挑战。相关研究成果以题为“Molecular bioengineering of functional nucleic acids”发表在最新一期《Nature Reviews Bioengineering》上。 【适配体：高特异性识别与多功能应用】 FNAs通过碱基互补配对折叠形成特定的二级与三级结构，具备识别、催化、调控与治疗等多种生物学功能，如图1所示。适配体是一类由短链单链DNA或RNA组成的FNAs，能折叠成特定三维结构，以高亲和力和特异性识别并结合各类靶标，包括蛋白质、小分子、离子乃至整个细胞（图2b）。其筛选依赖于系统性配体进化指数富集技术（SELEX），通过多轮负选与正选，从随机核酸库中筛选出高亲和力序列（图2a）。适配体凭借其柔性磷酸骨架和丰富的可旋转键，可形成茎环、凸起、假结、G-四链体等复杂结构，进一步组装成结合口袋，通过氢键、范德华力、静电与疏水作用实现精准识别，甚至呈现“诱导契合”式结合（图2c-e）。例如，RNA适配体IIF7t与凝血因子Xa通过15个氢键形成高度互补界面，展现出强结合力与特异性。在应用中，适配体可作为拮抗剂（如已获批的Macugen与Izervay）、激动剂（如激活MET受体的二价DNA适配体）或靶向递送载体（如AS1411-阿霉素偶联物），在治疗、诊断与靶向递送中发挥重要作用。 图 1. 功能核酸的结构和类型 图 2. 适配体的选择、结合和应用 【DNA酶：具有催化活性的DNA分子】 DNA酶是一类具有内在酶活性的单链DNA，能催化RNA/DNA切割、连接、磷酸化等反应（图3a）。其结构包括催化核心与底物结合域，前者形成发夹、G-四链体等二级结构以稳定活性中心，后者通过互补配对识别底物（图3a）。金属离子作为辅因子在催化中起关键作用：Mg²⁺可中和磷酸骨架负电荷，Pb²⁺可降低水分子pKₐ以促进RNA切割，而Ce³⁺与Na⁺的协同作用则可实现高效磷酸二酯键水解（图3b）。DNA酶在基因调控、金属离子检测、疾病诊断与治疗中广泛应用。例如，10–23 DNA酶可切割几乎任何目标RNA序列，用于沉默癌症相关基因；自锁变构DNA酶生物传感器可实现SARS-CoV-2病毒RNA的无扩增检测；而RNA连接DNA酶（如9DB1与7DES）则可用于RNA标记与修复（图3c-e）。 图 3. DNA 酶的酶学原理和生物学功能 【设计与工程策略：优化FNAs性能】 为提升FNAs在复杂生物环境中的稳定性与功能，研究人员发展了多种工程策略（图4a-e）。SELEX技术通过自动化、微流体与活体筛选等手段提升筛选效率与靶标适应性。结构工程包括截短非必需区、构建多价结构、环化与镜像核酸（Spiegelmer）等，以增强稳定性与亲和力（表1）。化学修饰涵盖碱基、糖环、磷酸与末端的改造，如2′-氟/氧甲基修饰、锁核酸（LNA）、磷硫酰化与3′-倒置dT封端等，显著提升核酸酶抗性与药代动力学性能（图4c）。人工碱基（如Z–P、d5SICS–dNaM、Ds–Px等）通过非标准氢键或疏水作用形成新碱基对，极大扩展了核酸库的多样性与功能潜力（图4d）。此外，FNAs还可与小分子、蛋白质、纳米颗粒等共轭，构建靶向递送系统（如ApDC、DNAzyme-纳米框架），实现精准药物递送与基因调控（图4e）。 图 4. FNA 的设计和工程策略 【临床转化：从实验室到病床的挑战与进展】 目前已有两款适配体药物获批：Macugen（靶向VEGF）用于湿性年龄相关性黄斑变性，Izervay（靶向C5）用于地理萎缩。此外，多项适配体与DNA酶候选药物处于临床试验阶段，如靶向vWF的ARC1779、靶向GATA3的SB010 DNA酶等。然而，FNAs的临床转化仍面临核酸酶降解、组织穿透性差、免疫原性、金属离子依赖性（DNA酶）及递送效率低等挑战。化学修饰（如PEGylation、2′-O-甲基、LNA）与靶向共轭策略被广泛用于改善其药代动力学与治疗效果。监管审批方面，由于FNAs相较于小分子与单抗经验较少，审批周期可能延长2–5年。未来，需进一步优化筛选平台、开发智能递送系统，并借助人工智能与多组学技术推动FNAs向临床应用的加速迈进。 【总结与展望】 FNAs作为一类可编程、结构多样、功能可定制的人工合成分子，在精准医学、基因调控与分子诊断中展现出巨大潜力。尽管目前仅有少数适配体药物获批，且DNA酶尚未进入市场，但通过持续的工程优化——包括SELEX平台升级、结构设计、化学修饰、人工碱基引入以及智能递送系统的构建——FNAs有望在未来突破现有瓶颈，成为解决复杂生物医学问题的关键工具。跨学科技术的融合，如人工智能辅助设计、高通量筛选与单细胞测序，将进一步提升FNAs的功能性与临床应用范围，为精准医疗提供更高效、安全的诊断与治疗策略。 「BioMed科技」关注生物医药×化学材料交叉前沿研究进展！交流、合作，请添加杨主编微信！ 原文链接： https://www.nature.com/articles/s44222-025-00361-y 来源：BioMed科技声明：仅代表作者个人观点，作者水平有限，如有不科学之处，请在下方留言指正！