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GEMMA Biotherapeutics

HQ: Philadelphia, PA source · gemmabiotx.com ↗

Clinical Phase 2

2 open jobs · last seen Oct 23, 2026 · 3 sources

Location: Philadelphia, PA · source · Oct 14, 2026

Profile checked Oct 10, 2026

Keywords: GM1 Gangliosidosis Type I GM1 Gangliosidosis Type 2 GM1 Gangliosidosis Beta-Galactosidase-1 (GLB1) Deficiency Globoid Cell Leukodystrophy Spinal Muscular Atrophy Type I PBGM01 PBKR03 GB221

2Open jobs3Trials (company is sponsor)

Hiring

TitleLocation (from posting)Employer postedFirst seen
Director, FinancePhiladelphia, PA, USSep 23, 2026Oct 11, 2026
Vice President, FinancePhiladelphia, PA, USSep 21, 2026Oct 11, 2026

See all 2 jobs

Timeline

  1. Trial start Phase 1/Phase 2: Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1 source ↗
  2. Trial start Phase 1/Phase 2: Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease source ↗
  3. Trial start Phase 1/Phase 2: Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis source ↗

Upcoming milestones

Estimated primary completion per ClinicalTrials.gov. Dates move.

Clinical trials (lead sponsor) 3

RecordTitlePhaseStatusStartPrimary completion
NCT04713475Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 GangliosidosisPhase 1/Phase 2Active, not recruitingMar 17, 20212028-02 (estimated)
NCT04771416Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe DiseasePhase 1/Phase 2SuspendedFeb 24, 20222027-01 (estimated)
NCT07070999Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1Phase 1/Phase 2RecruitingJan 6, 20262028-12 (estimated)

Open jobs 2

TitleLocation (from posting, not HQ)Employer postedFirst seenLevel
Director, FinancePhiladelphia, PA, USSep 23, 2026Oct 11, 2026Senior
Vice President, FinancePhiladelphia, PA, USSep 21, 2026Oct 11, 2026Executive

Level (Entry/Mid/Senior/Executive) is estimated from the job title and required years of experience. Descriptions are short excerpts (about the first 60 words) of the posting plus skills picked out of it; use Read full posting for the complete text.

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LinkedIn page linked from the company's own site: source · seen 2026-10-18

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