价格

EYEDNA THERAPEUTICS

63 B AV LEDRU ROLLIN, Paris 12, Île-de-France, 法国

别名:

eyedna tx

概览

试验阶段分布

阶段

试验数

占比

1 期

66.7%

2 期

33.3%

试验状态分布

招募中

66.7%

终止

33.3%

药物批准

暂无批准数据

资讯

EyeDNA's HORA-PDE6b Receives FDA Rare Pediatric Disease Designation for PDE6b Retinitis Pigmentosa

- EyeDNA Therapeutics' HORA-PDE6b, a novel gene therapy, has received Rare Pediatric Disease Designation from the FDA for PDE6b-related retinitis pigmentosa. - HORA-PDE6b delivers a functional PDE6B gene, potentially halting retinal degeneration in patients with PDE6b deficiency, addressing a critical unmet need. - Positive 24-month follow-up data from a Phase I/II study showcased the safety and efficacy of HORA-PDE6b, supporting its accelerated development. - The designation grants EyeDNA a Priority Review Voucher, potentially expediting the approval process for other products and enhancing market entry.

Dec 18, 2024

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EYEDNA THERAPEUTICS

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该机构主办的试验（3）

Safety and Efficacy of a Unilateral Subretinal Administration of HORA PDE6B in Patients with Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expressio

Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene

Safety and Efficacy of a Unilateral Subretinal Administration of HORA PDE6B in Patients with Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression

药物批准

资讯

EyeDNA's HORA-PDE6b Receives FDA Rare Pediatric Disease Designation for PDE6b Retinitis Pigmentosa