Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

计划入组 5 人 · 首次发布于 2019 年 12 月 17 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

A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy

计划入组 1 人 · 首次发布于 2025 年 5 月 21 日

SGSH gene therapy (AAV9, mucopolysaccharidosis IIIA)

OTL-201

AAV9-SGSH

JIUYUAN GENE

Triple-Gene LLC

United States

MOMOTARO-GENE INC.

Japan

Ultragenyx's UX111 Gene Therapy Shows Promise in Sanfilippo Syndrome Type A

Orchard Therapeutics Receives Innovation Passport for Gene Therapy OTL-201 Targeting Rare Sanfilippo Syndrome

FDA Issues Complete Response Letter for Ultragenyx's UX111 Gene Therapy for Sanfilippo Syndrome Type A