4D Molecular Therapeutics (4DMT) has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to 4D-150 for the treatment of diabetic macular edema (DME). This follows a previous RMAT designation for the same investigational therapy in wet age-related macular degeneration (wet AMD)..

4D Molecular Therapeutics（4DMT）宣布，美国食品药品监督管理局（FDA）已授予4D-150用于治疗糖尿病性黄斑水肿（DME）的再生医学先进疗法（RMAT）资格。此前，该在研疗法还获得了用于湿性年龄相关性黄斑变性（湿性AMD）的RMAT资格。

In a company press release, David Kirn, MD, Co-Founder and CEO of 4DMT, commented on the regulatory milestone:

在公司新闻稿中，4DMT联合创始人兼首席执行官David Kirn博士就这一监管里程碑发表了评论：

“This milestone validates the potential of 4D-150 to address the significant unmet needs of patients with DME, a second large market retinal vascular disease indication after wet AMD. The RMAT designation is based on the review of our results to date from our ongoing 4D-150 SPECTRA DME study, underscoring the potential of 4D-150 to sustain visual acuity improvements while dramatically reducing treatment burden for patients.

“这一里程碑验证了4D-150在满足糖尿病性黄斑水肿（DME）患者显著未满足需求方面的潜力，这是继湿性年龄相关性黄斑变性（AMD）之后的第二大视网膜血管疾病市场。RMAT指定是基于我们正在进行的4D-150 SPECTRA DME研究截至目前的结果审查，突显了4D-150在维持视力改善的同时大幅减少患者治疗负担的潜力。”

This designation in DME follows the RMAT designation granted for 4D-150 in wet AMD, and to our knowledge, 4D-150 is the first investigational medicine to be granted the designation in both indications.”.

DME中的这一指定是继4D-150在湿性AMD中获得RMAT指定之后的又一成果，据我们所知，4D-150是首个在这两个适应症中均获得该指定的在研药物。

RMAT Designation: Accelerating the Development of Regenerative Therapies

RMAT指定：加速再生疗法的开发

The RMAT designation program was created by the FDA to expedite development and review of regenerative medicine therapies intended to treat, modify, reverse, or cure serious conditions. This designation offers sponsor companies the benefits of fast track and breakthrough therapy programs, including:.

RMAT指定计划由FDA创建，旨在加速开发和审查用于治疗、改善、逆转或治愈严重疾病的再生医学疗法。该指定为赞助公司提供了快速通道和突破性疗法计划的好处，包括：。

• Frequent and early FDA interaction

• 频繁且早期的FDA互动

• Rolling review opportunities

• 滚动审查机会

• Streamlined clinical development paths

• 简化的临床开发路径

4D-150: A Long-Term Anti-VEGF Gene Therapy for Retinal Diseases

4D-150：一种用于视网膜疾病的长效抗VEGF基因疗法

4D-150 is a potential backbone gene therapy developed to deliver long-term expression of anti-VEGF agents—specifically aflibercept and anti–VEGF-C—to the retina via a single intravitreal injection. Designed for both DME and wet AMD, 4D-150 aims to sustain improvements in visual acuity while reducing treatment burden and preserving safety with good tolerability..

4D-150 是一种潜在的骨干基因疗法，通过单次玻璃体内注射将抗VEGF药物（特别是阿柏西普和抗VEGF-C）长期表达递送至视网膜。该疗法针对糖尿病性黄斑水肿（DME）和湿性年龄相关性黄斑变性（wet AMD），旨在持续改善视力，同时减轻治疗负担，并在安全性与良好耐受性方面保持优势。

Interim Data Supports Clinical Advancement and Regulatory Readiness

中期数据支持临床进展和监管准备就绪

In January 2025, 4DMT announced positive 32-week interim results from Part 1 of the SPECTRA trial in DME. Key findings include:

2025年1月，4DMT宣布了SPECTRA试验DME部分第1阶段的积极32周中期结果。关键发现包括：

• Well-tolerated safety profile with no intraocular inflammation observed at any dose

• 良好的安全耐受性，在任何剂量下均未观察到眼内炎症

• At the 3E10-vg/eye dose, patients achieved +8.4 letters gain in best corrected visual acuity (BCVA) and –194 µm reduction in central subfield thickness (CST) through week 32

• 在3E10-vg/眼剂量下，患者在第32周时最佳矫正视力（BCVA）提高了+8.4个字母，中心子野厚度（CST）减少了194微米。

These findings, alongside data from the PRISM trial, supported the FDA’s acceptance of a single Phase 3 clinical trial as the basis for a biologics license application (BLA) submission for 4D-150 in DME.

这些发现以及来自 PRISM 试验的数据，支持了 FDA 接受将单个 III 期临床试验作为 4D-150 在 DME 中生物制品许可申请 (BLA) 的提交依据。

Focused Pipeline Strategy and Updated Development Plans

专注的管道战略和更新的发展计划

Earlier in 2025, 4DMT also announced a strategically focused pipeline, with updates to its Phase 3 4FRONT program, new guidance for trial design, and revised cash runway projections. These updates reflect the company’s commitment to advancing 4D-150 and its broader ophthalmology portfolio efficiently..

2025年早些时候，4DMT还宣布了一条战略重点明确的研发管线，包括对其三期4FRONT项目的更新、新的试验设计指导以及修订后的现金跑道预测。这些更新反映了公司致力于高效推进4D-150及其更广泛的眼科产品组合。