About
Gene Therapy
Areas of Focus
Categories
Gene Therapy, RNA & Peptide TherapyBoston & New England
Clinical Trials (5)
View allTotal Trials
5
As Lead Sponsor
3
As Collaborator
2
Total Enrollment
171
By Phase
PHASE1: 2NA: 2PHASE1/PHASE2: 1
By Status
RECRUITING: 2COMPLETED: 2ACTIVE_NOT_RECRUITING: 1
Clinical Trials Pipeline5 trials
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Phase 1/2
Phase 2/3
N/A
2015
2016
2017
2018
2019
2020
2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
Today
Hover over trial bars to see details
Recent Trials
NCT06555419Collaborator
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
PHASE1Recruiting
58 participants
Started: Jan 16, 2025 · Completed: May 15, 2026
NCT05866419Lead Sponsor
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
NARecruiting
90 participants
Started: Nov 27, 2023 · Completed: Mar 31, 2027
NCT03770572Lead Sponsor
Gene Therapy for Children With CLN3 Batten Disease
PHASE1/PHASE2Active, not recruiting
7 participants
Started: Nov 13, 2018 · Completed: Sep 30, 2029
NCT03178032Collaborator
Oncolytic Adenovirus, DNX-2401, for Naive Diffuse Intrinsic Pontine Gliomas
PHASE1Completed
12 participants
Started: May 26, 2017 · Completed: Jan 31, 2021
NCT02651337Lead Sponsor
Quantitative Characterization of Safe Irrigation for Ventricular Shunt Catheters
NACompleted
4 participants
Started: Apr 1, 2016 · Completed: Jun 14, 2016
Locations (1)
Headquarters
Lowell, MA, United States
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