Status
Study
Lead sponsor
Phase
Enrol
US states
Start
Active, closed
Insmed Gene Therapy LLC
Phase 1
12
Arkansas, California, Georgia, New York, Ohio, Tennessee, Virginia
2025-07-22
Active, closed
Hoffmann-La Roche
Phase 2
30
Arkansas, California, Georgia, Michigan, Texas, Virginia
2025-04-04
Recruiting
Solid Biosciences Inc.
Phase 1/2
60
Arkansas, California, Georgia, Illinois, Missouri, Ohio, Oregon, Pennsylvania
2024-05-06
Active, closed
NS Pharma, Inc.
Phase 2
20
Colorado, Georgia, Illinois, Kansas, Massachusetts, New York, Ohio, Pennsylvania
2024-02-22
Active, closed
Avidity Biosciences, Inc.
Phase 2
39
Arkansas, California, Georgia, Massachusetts, Minnesota, North Carolina, Ohio, Texas
2024-01-22
Active, closed
REGENXBIO Inc.
Phase 2/3
65
Arkansas, California, Colorado, Florida, Georgia, Illinois, Iowa, Kansas
2023-01-04
Active, closed
REGENXBIO Inc.
Observational
200
Georgia
2022-12-20
Active, closed
Edgewise Therapeutics, Inc.
Phase 2
76
Arkansas, California, Colorado, Florida, Georgia, Iowa, Kansas, Maryland
2022-10-24
Recruiting
Wave Life Sciences USA, Inc.
Phase 1/2
26
Arkansas, Georgia
2021-09-28
Recruiting
Italfarmaco
Phase 2/3
206
California, Connecticut, Florida, Georgia, Iowa, Missouri, Oregon, Pennsylvania
2017-10-24