Floatz Rating
CCC48/100
Confidence
Indicative
Sponsor
Gemma Biotherapeutics
Modality
UNKNOWN
Development Phase
Phase 1/2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
33Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04713475CT.gov Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis | — | Active Not Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · GM1 Gangliosidosis
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Pbgm01 (this asset) | Gemma Biotherapeutics | P1/2 | CCC · 48 |
| Cyclophosphamide | Masonic Cancer Center, University of Minnesota | P2/3 | BB |
| Busulfanum | Masonic Cancer Center, University of Minnesota | P2/3 | B |
| az-3102 | Azafaros B.V. | P3 | B |
| Anti-Human T-Lymphocyte Immunoglobulin, Rabbit | Masonic Cancer Center, University of Minnesota | P2/3 | B |
| Hydroxyurea | Masonic Cancer Center, University of Minnesota | P2 | B |
| Clofarabine | Masonic Cancer Center, University of Minnesota | P2 | B |
| Melphalan | Masonic Cancer Center, University of Minnesota | P2 | B |
| Alemtuzumab | Masonic Cancer Center, University of Minnesota | P2 | B |
+1 more in the GM1 Gangliosidosis cohort
Other indications for Pbgm01
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| GM1 Gangliosidosis Type 1 | — | P1/2 | NR |
| GM1 Gangliosidosis Type 2 | — | P1/2 | NR |
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
Citation
Floatz Terminal. Pbgm01 in GM1 Gangliosidosis. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/pbgm01-gm1-gangliosidosis
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