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Telomir报告了口服AMD疗法Telomir-1的有希望的临床前结果

Telomir Reports Promising Preclinical Results for Oral AMD Therapy Telomir-1

OBN 等信源发布 2025-06-04 18:25

可切换为仅中文


Telomir Reports Promising Preclinical Results for Oral AMD Therapy Telomir-1

Telomir报告了口服AMD疗法Telomir-1的有希望的临床前结果

June 04, 2025

2025年6月4日

Telomir Pharmaceuticals has released encouraging preclinical data demonstrating the potential of its investigational oral therapy, Telomir-1, for age-related macular degeneration (AMD).

Telomir制药公司发布了鼓舞人心的临床前数据,展示了其研究中的口服疗法Telomir-1在治疗年龄相关性黄斑变性(AMD)方面的潜力。

Retinal Restoration and Functional Recovery in AMD Model

年龄相关性黄斑变性(AMD)模型中的视网膜修复与功能恢复

Telomir-1 showed notable therapeutic effects in a preclinical model, reversing central vision impairment, restoring retinal structure, and significantly reducing oxidative stress—achieving improvements across several FDA-recognized surrogate endpoints relevant to AMD.

Telomir-1在临床前模型中显示出显著的治疗效果,逆转了中心视力损伤,恢复了视网膜结构,并显著降低了氧化应激——在与AMD相关的一些FDA认可的替代终点上取得了改善。

“The preclinical success achieved in this AMD model is truly remarkable,” said Itzchak Angel, PhD, Chief Scientific Advisor for Telomir Pharmaceuticals. “Telomir-1, when studied orally, restored both structure and function in the retina, demonstrating not just neuroprotection but true regenerative capacity––a property rarely seen in ophthalmic drug development.”.

“在这一AMD模型中取得的临床前成功确实令人瞩目,”Telomir Pharmaceuticals的首席科学顾问Itzchak Angel博士说道。“Telomir-1在口服研究中恢复了视网膜的结构和功能,不仅展示了神经保护作用,还展现了真正的再生能力——这是眼科药物开发中很少见到的特性。”

Genetically Modified AMD Model

转基因AMD模型

The data were obtained from a genetically engineered zebrafish model (Sen57wrn-/-ND6-/+), designed to mimic dry AMD and geographic atrophy (GA). This model integrates genetic markers associated with premature aging (WRN), mitochondrial dysfunction (ND6), and chronic senescence (Sen57). Prior to treatment, 18-month-old zebrafish displayed:.

数据来源于一种基因工程斑马鱼模型(Sen57wrn-/-ND6-/+),该模型旨在模拟干性年龄相关性黄斑变性(AMD)和地理性萎缩(GA)。此模型整合了与早衰(WRN)、线粒体功能障碍(ND6)以及慢性衰老(Sen57)相关的遗传标记。在治疗之前,18个月大的斑马鱼表现出:

• Visual impairment

• 视力障碍

• Sluggish and uncoordinated swimming

• 缓慢且不协调的游泳

• Elevated oxidative stress (ROS levels 4x higher than controls)

• 氧化应激升高(ROS 水平比对照组高 4 倍)

• 15% retinal degeneration and 15% mortality rate

• 15%的视网膜退化和15%的死亡率

Structural Degeneration Observed

观察到结构退化

Key findings from retinal histology included:

视网膜组织学的关键发现包括:

• Thinning of the Inner Nuclear Layer (INL) – critical for signal processing

• 内核层(INL)变薄——对信号处理至关重要

• Degradation of the Ganglion Cell Layer (GLC) – responsible for transmitting visual signals

• 视网膜神经节细胞层(GLC)退化 - 负责传递视觉信号

• Reduced Outer Plexiform Layer (OPL) volume – crucial for detecting light and contrast

• 外丛状层(OPL)体积减少——对检测光线和对比度至关重要

Oral Telomir-1 Treatment Reversed Damage

口服Telomir-1治疗逆转了损伤

Following a 14-day oral dosing regimen, treated zebrafish exhibited significant recovery:

在为期14天的口服给药方案后,经治疗的斑马鱼表现出显著的恢复:

• Restored INL thickness – reinstating signal processing

• 恢复INL厚度 – 重新建立信号处理

• Improved GLC structure – reactivating visual signal transmission

• 改进的GLC结构——重新激活视觉信号传输

• Augmented Inner Plexiform Layer (IPL) – enhancing visual processing

• 增强的内丛状层 (IPL) – 提升视觉处理能力

• Recovery of OPL structure – maintaining photoreceptor input

• 恢复OPL结构——保持感光细胞输入

Behaviorally, the zebrafish returned to active, coordinated swimming with appropriate visual responses. ROS levels dropped by up to 50%, and no mortality occurred in the treated group.

行为上,斑马鱼恢复了活跃、协调的游泳,并表现出适当的视觉反应。活性氧水平下降了多达50%,并且治疗组没有出现死亡。

“Collectively, these results demonstrate Telomir-1’s ability to restore visual function, reverse retinal degeneration, reduce oxidative stress, and improve survival—all from a short oral treatment regimen,” the company stated.

“总体而言,这些结果证明了Telomir-1能够恢复视觉功能、逆转视网膜退化、减少氧化应激并提高存活率——且仅需短暂的口服治疗疗程,”该公司表示。

Advancing Toward a New Paradigm in AMD Treatment

迈向AMD治疗的新范式

Erez Aminov, Chairman and CEO of Telomir Pharmaceuticals, emphasized the broader implications of the findings:

特拉米尔制药公司董事长兼首席执行官埃雷兹·阿米诺夫强调了这些研究结果的更广泛影响:

“This breakthrough reinforces our vision at Telomir: to redefine how we treat age-related diseases by going beyond symptom management and targeting the root mechanisms of degeneration. To our knowledge, no oral drug has ever demonstrated this level of retinal restoration and vision recovery in any AMD model––this is a meaningful leap forward for patients and the field.”.

“这一突破进一步坚定了我们在Telomir的愿景:通过超越症状管理,针对退化的根本机制,重新定义我们治疗与年龄相关疾病的方式。据我们所知,没有任何口服药物在任何AMD模型中展现出如此程度的视网膜修复和视力恢复——这对患者和整个领域来说都是一个有意义的巨大进步。”