ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9

计划入组 6 人 · 首次发布于 2022 年 2 月 4 日

Global FKRP Registry

计划入组 1,000 人 · 首次发布于 2019 年 6 月 28 日

A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease

计划入组 31 人 · 首次发布于 2025 年 6 月 10 日

GTFP-100

TTR gene editing therapy (Trucode Gene)

CYBB gene editing therapy (Trucode Gene)

JIUYUAN GENE

Triple-Gene LLC

United States

MOMOTARO-GENE INC.

Japan

Cure Rare Disease Advances LGMDR9 Gene Therapy Following Positive FDA Pre-IND Meeting

AskBio's AB-1003 Receives FDA Rare Pediatric Disease and Orphan Drug Designations for LGMD2I/R9

AskBio's AB-1003 Receives FDA Designations for LGMD2I/R9 Treatment