Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

计划入组 81 人 · 首次发布于 2024 年 10 月 4 日

Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene

计划入组 20 人 · 首次发布于 2018 年 12 月 19 日

Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

计划入组 81 人 · 首次发布于 2026 年 9 月 1 日

USH2A gene-targeting RCas13d

USH2A gene therapy (Usher syndrome)

USH2A gene therapy (adeno-associated virus based vector, retinitis pigmentosa)

JIUYUAN GENE

Triple-Gene LLC

United States

MOMOTARO-GENE INC.

Japan

Lyora Therapeutics Launches With $2.5M Seed Funding to Advance Genetic Medicines for Inherited Retinal Diseases

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