About
Gene Therapy
Areas of Focus
Categories
Gene Therapy, RNA & Peptide TherapyNJ, NY, PA
Clinical Trials (3)
View allTotal Trials
3
As Lead Sponsor
3
As Collaborator
0
Total Enrollment
110
By Phase
PHASE1/PHASE2: 3
By Status
NOT_YET_RECRUITING: 1ACTIVE_NOT_RECRUITING: 1COMPLETED: 1
Clinical Trials Pipeline3 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
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Recent Trials
NCT07070999Lead Sponsor
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
PHASE1/PHASE2Not yet recruiting
22 participants
Started: Oct 31, 2025 · Completed: Apr 30, 2029
NCT04713475Lead Sponsor
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
PHASE1/PHASE2Active, not recruiting
26 participants
Started: Mar 17, 2021 · Completed: Feb 28, 2029
NCT00034255Lead Sponsor
Single Dose and 34-Day Tolerance Study of INGAP Peptide in Insulin Deficient Patients
PHASE1/PHASE2Completed
62 participants
Started: Dec 31, 2001
Locations (1)
Headquarters
Philadelphia, PA, United States
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