Defunct Company
Total Trials
12
As Lead Sponsor
9
As Collaborator
3
Total Enrollment
145,292
NCT02122952
Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
Phase: Phase 1
Role: Lead Sponsor
Start: May 5, 2014
Completion: Dec 15, 2017
NCT03421977
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
Phase: N/A
Start: Sep 21, 2017
Completion: Dec 2, 2030
NCT03306277
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Phase: Phase 3
Start: Oct 24, 2017
Completion: Nov 12, 2019
NCT03381729
Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
Start: Dec 14, 2017
Completion: Nov 18, 2021
NCT03505099
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
Start: Apr 2, 2018
Completion: Jun 15, 2021
NCT03461289
Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Start: Aug 16, 2018
Completion: Sep 11, 2020
NCT03720418
Study of OXB-102 (AXO-Lenti-PD) in Patients With Bilateral, Idiopathic Parkinson's Disease
Phase: Phase 1/2
Start: Oct 17, 2018
Completion: Apr 12, 2022
NCT03837184
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
Start: May 31, 2019
Completion: Jun 29, 2021
NCT03952637
A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis
Role: Collaborator
Start: Aug 19, 2019
Completion: Jan 1, 2028
NCT04042025
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Start: Feb 10, 2020
Completion: Dec 31, 2035
NCT05368038
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Start: May 10, 2021
Completion: Aug 31, 2029
NCT05481164
Newborn Screening for Spinal Muscular Atrophy
Start: Mar 8, 2022
Completion: Mar 7, 2025