Ocugen Reports Positive 2-Year Results for OCU400 Gene Therapy in Retinitis Pigmentosa

January 16, 2025

2025年1月16日

Ocugen, Inc. has announced encouraging 2-year safety and efficacy results from its Phase 1/2 clinical trial of OCU400, a novel gene therapy for the treatment of retinitis pigmentosa (RP) across early to advanced stages in both pediatric and adult populations.

Ocugen，Inc.宣布，其OCU400的1/2期临床试验取得了令人鼓舞的2年安全性和有效性结果，OCU400是一种新型基因疗法，用于治疗儿童和成人早期至晚期视网膜色素变性（RP）。

Key Findings

主要调查结果

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Visual Acuity Improvement:

视力提高：

OCU400 demonstrated a 2-line gain (10 letters) on the ETDRS chart in low-luminance visual acuity (LLVA) in treated eyes compared to untreated fellow eyes.

OCU400在ETDRS图表上显示，与未治疗的对侧眼相比，治疗眼的低亮度视力（LLVA）有2行增益（10个字母）。

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This improvement was statistically significant (p=0.01) across all participants, regardless of genetic mutation.

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These findings validate OCU400’s gene-agnostic mechanism of action, making it a groundbreaking approach for RP treatment.

这些发现验证了OCU400的基因不可知作用机制，使其成为RP治疗的开创性方法。

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Durability of Results:

结果的持久性：

The 2-year findings align with results observed at the 1-year mark, confirming the long-term potential of OCU400 in preserving and improving visual function.

2年的研究结果与1年时观察到的结果一致，证实了OCU400在保持和改善视觉功能方面的长期潜力。

Expert Insights

专家见解

Dr. Syed M. Shah, Vice Chair for Research and Digital Medicine at Gundersen Health System, highlighted the significance of OCU400:

Gundersen Health System研究和数字医学副主席Syed M.Shah博士强调了OCU400的重要性：

“The broad spectrum of genes and mutations causing RP presents a unique challenge in developing treatments for this unmet need. This is where the promise of mutation-agnostic therapies becomes particularly compelling. OCU400’s demonstrated effectiveness across multiple mutations not only offers hope to RP patients but also opens new possibilities for treating other retinal diseases.'.

“导致RP的广泛基因和突变在为这种未满足的需求开发治疗方法方面提出了独特的挑战。这就是突变不可知疗法的前景变得特别引人注目的地方。OCU400在多种突变中表现出的有效性不仅为RP患者提供了希望，而且为治疗其他视网膜疾病开辟了新的可能性。”。

Dr. Huma Qamar, Chief Medical Officer at Ocugen, emphasized the therapy’s durability:

Ocugen首席医疗官Huma Qamar博士强调了这种疗法的持久性：

“Establishing the long-term safety and efficacy of OCU400 demonstrates the durability of this novel gene therapy. These 2-year low light visual acuity (LLVA) findings, which are the most sensitive measure of visual function, are consistent with the results observed at one year.”

“建立OCU400的长期安全性和有效性证明了这种新型基因疗法的持久性。这些2年低视力（LLVA）的发现是视觉功能最敏感的指标，与一年时观察到的结果一致。”

Next Steps

接下来的步骤

Ocugen is advancing the Phase 3 OCU400 liMeliGhT clinical trial, which remains on track for a Biologics License Application (BLA) submission in the first half of 2026.

Ocugen正在推进第三阶段OCU400引人注目的临床试验，该试验仍将在2026年上半年提交生物制剂许可证申请（BLA）。

Conclusion

结论

The 2-year results of OCU400 underscore its potential to address the challenges of RP with a mutation-agnostic approach, offering hope to patients across diverse genetic backgrounds. This innovative therapy is a promising step forward in treating one of the most challenging retinal diseases.

OCU400的2年结果强调了其通过突变不可知的方法解决RP挑战的潜力，为不同遗传背景的患者提供了希望。这种创新疗法是治疗最具挑战性的视网膜疾病之一的有希望的一步。