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

Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A

计划入组 7 人 · 首次发布于 2020 年 6 月 5 日

arylsulfatase A gene therapy

TTR gene editing therapy (Trucode Gene)

dysferlin gene therapy (Atamyo)

CELL AND GENE THERAPY CATAPULT

United Kingdom

JIUYUAN GENE

Triple-Gene LLC

United States

Apertura Gene Therapy Launches Open Aperture Program to Democratize Access to CNS-Targeting AAV Capsid

Apertura Gene Therapy Partners with Rett Syndrome Research Trust to Advance Blood-Brain Barrier-Crossing Gene Therapy Platform

Apertura Gene Therapy Licenses Blood-Brain Barrier-Penetrant AAV Capsid to Multiple Partners for CNS Treatments