Gene Therapy in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy

计划入组 9 人 · 首次发布于 2023 年 5 月 15 日

Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy

计划入组 4 人 · 首次发布于 2020 年 8 月 18 日

Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations

计划入组 30 人 · 首次发布于 2025 年 7 月 8 日

Voretigene neparvovec(Luxturna)

Fas Gene therapy (Retinal dystrophy)

AAV-RPE65

RETINAL CONSULTANTS

United States

RETINAL CARE INC.

United States

RETINAL RESEARCH INSTITUTE, LLC

United States

Study Charts rAAV Dose Costs by Indication and Identifies Cost-Cutting Strategies

Variant Secures EMA Endorsement for VAR002 Gene Therapy Targeting Inherited Retinal Dystrophies

Real-World Study Confirms Long-Term Efficacy of Luxturna Gene Therapy for Inherited Retinal Disease