Sepul Bio Doses First Patient in Phase 2b Trial of Ultevursen for USH2A-Associated Retinitis Pigmentosa

在Ultevursen治疗USH2A相关性视网膜色素变性的2b期临床试验中，Sepul Bio Doges是第一位患者

December 20, 2024

Sepul Bio has announced the dosing of the first patient in the LUNA clinical study, a Phase 2b trial evaluating ultevursen for the treatment of USH2A-associated retinitis pigmentosa (RP). This milestone represents renewed hope for individuals affected by this debilitating genetic condition.

Sepul Bio宣布了LUNA临床研究中第一名患者的剂量，这是一项评估ultevursen治疗USH2A相关性视网膜色素变性（RP）的2b期临床试验。这一里程碑代表了受这种令人衰弱的遗传病影响的个体的新希望。

Understanding USH2A-Associated Retinitis Pigmentosa

了解USH2A相关性视网膜色素变性

USH2A (Usher Syndrome Type 2a) is the most common cause of autosomal recessive retinitis pigmentosa, a condition affecting the retina and inner ear.

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Key Facts:

关键事实：

• Mutations in exon 13 of the USH2A gene result in syndromic and non-syndromic forms of RP.

•USH2A基因第13外显子突变导致RP的综合征和非综合征形式。

• Symptoms include progressive vision loss, often accompanied by hearing impairment.

•症状包括进行性视力丧失，常伴有听力障碍。

• Currently, there are no approved treatments for these patients.

•目前，这些患者尚无批准的治疗方法。

About Ultevursen

关于Ultevursen

Ultevursen, developed by Sepul Bio, is an RNA therapy designed to specifically target mutations in exon 13 of the USH2A gene. By addressing the underlying genetic cause, ultevursen aims to preserve vision in patients with RP.

由Sepul Bio开发的Ultevursen是一种RNA疗法，旨在专门针对USH2A基因第13外显子的突变。通过解决潜在的遗传原因，ultevursen旨在保护RP患者的视力。

LUNA Clinical Trial Overview

LUNA临床试验概述

The LUNA study is a two-year, double-masked, randomized, sham-controlled Phase 2b trial evaluating the safety and efficacy of ultevursen.

LUNA研究是一项为期两年，双盲，随机，假对照的2b期试验，评估ultevursen的安全性和有效性。

Key Details:

关键细节：

• Participants: 81 adults and children aged 8 years and older.

•参与者：81名8岁及以上的成年人和儿童。

• Objective: Assess the potential of ultevursen to slow or halt the progression of vision loss in patients with USH2A-associated RP.

•目标：评估ultevursen减缓或阻止USH2A相关RP患者视力丧失进展的潜力。

Community Support

社区支持

Krista Vasi, MPA, Executive Director of the Usher Syndrome Coalition, highlighted the significance of the trial:

Usher综合征联盟执行董事、MPA克里斯塔·瓦西（KristaVasi）强调了该试验的重要性：

“The initiation of enrollment in the LUNA study is an exciting milestone for the Usher syndrome community. We have been eagerly waiting for the re-initiation of the ultevursen program, and today’s announcement reflects renewed hope for the community.”

“LUNA研究的开始对Usher综合征社区来说是一个激动人心的里程碑。我们一直在热切等待ultevursen项目的重新启动，今天的宣布反映了社区的新希望。”

The Usher Syndrome Coalition, a global organization supporting individuals with Usher syndrome, maintains the largest international contact database of affected individuals from 76 countries.

Usher综合征联盟是一个支持Usher综合征患者的全球组织，它维护着来自76个国家的受影响个体的最大国际联系数据库。

Sepul Bio’s Commitment

Sepul Bio的承诺

As a business unit of Laboratoires Théa, France-based Sepul Bio is dedicated to advancing RNA therapies for inherited retinal diseases. The launch of the LUNA study aligns with the company’s mission to address significant unmet medical needs in retinal health.

作为Laboratoires Théa的一个业务部门，总部位于法国的Sepul Bio致力于推进遗传性视网膜疾病的RNA疗法。LUNA研究的启动符合该公司的使命，即解决视网膜健康方面尚未满足的重大医疗需求。

Conclusion

结论

The dosing of the first patient in the LUNA study marks a significant step forward in the development of ultevursen as a potential treatment for USH2A-associated retinitis pigmentosa. With its focus on exon 13 mutations, Sepul Bio is bringing hope to patients and families affected by this challenging condition..

LUNA研究中第一位患者的剂量标志着ultevursen作为USH2A相关性视网膜色素变性的潜在治疗方法的发展迈出了重要的一步。Sepul Bio专注于第13外显子突变，为受这种挑战性疾病影响的患者和家庭带来了希望。。