About
Ex-vivo autologous hematopoietic stem cell gene therapy
Areas of Focus
Categories
Gene Therapy, RNA & Peptide TherapyBoston & New England
Clinical Trials (15)
View allTotal Trials
15
As Lead Sponsor
5
As Collaborator
10
Total Enrollment
119,089
By Phase
PHASE3: 2PHASE1/PHASE2: 8PHASE2: 1
By Status
ACTIVE_NOT_RECRUITING: 6COMPLETED: 5UNKNOWN: 2ENROLLING_BY_INVITATION: 1RECRUITING: 1
Clinical Trials Pipeline15 trials
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Phase 1/2
Phase 2/3
N/A
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Today
Hover over trial bars to see details
Recent Trials
NCT06149403Lead Sponsor
A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
PHASE3Active, not recruiting
41 participants
Started: Dec 11, 2023 · Completed: Mar 31, 2031
NCT05354219Collaborator
Validation and Reliability of Iris Cameras in Mucopolysaccharidoses
N/AUnknown
60 participants
Started: Mar 8, 2023 · Completed: Sep 8, 2023
NCT05687474Collaborator
Baby Detect : Genomic Newborn Screening
N/ACompleted
6,824 participants
Started: Sep 1, 2022 · Completed: Jun 2, 2025
NCT04283227Lead Sponsor
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
PHASE3Active, not recruiting
6 participants
Started: Jan 17, 2022 · Completed: Mar 31, 2031
NCT05368038Collaborator
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
N/AEnrolling by invitation
100,000 participants
Started: May 10, 2021 · Completed: Aug 31, 2029
Locations (1)
Headquarters
Boston, MA, United States
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