Sponsor pipeline

Gemma Biotherapeutics clinical trial pipeline

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Data updated daily from ClinicalTrials.gov, last refresh 2026-10-16

Active trials by phase

Phase 1/2
2

Active trials

NCT IDTitleConditionStatusUpdated
Phase 1/2
NCT07070999 Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular… Spinal Muscular Atrophy Type I Recruiting 2026-06-29
NCT04713475 Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1… GM1 Gangliosidosis Active, Not Recruiting 2026-06-30

Disease areas by active trials

Spinal Muscular Atrophy (1)

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