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PBGM01

Biologic

GGemma Biotherapeutics

PBGM01 is one of 8 biologic programs targeting GM1 gangliosidosis. It is currently in development.

1

Indications

1

Total Trials

1

Active Trials

7

Competitors

Pipeline

Status by Indication[3][1]

Indication

GM1 GangliosidosisActive, not recruiting

GM1 GangliosidosisPhase 1/Phase 2

Active, not recruiting

Competition

Competitive Landscape

9 competitors in GM1 Gangliosidosis

#DrugCompanyPhaseTrials

Busulfex

Akero Therapeutics

Cytoxan

BBaxter Healthcare Corporation

Medrol

Rituxan

Rapamune

PPrism Pharmaceuticals

Rayos

RRoxane Laboratories

AZ-3102

PBGM01

GGemma Biotherapeutics

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q1 2029

PreclinicalActive, not recruiting

GM1 Gangliosidosis

NCT04713475

Clinical

Trials by Indication[1]

GM1 gangliosidosis1 trial

Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Pediatric Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 Gangliosidosis

Active, not recruiting·GGemma Biotherapeutics·Preclinical·NCT04713475·26 enrolled

Sources

References

[1]
ClinicalTrials.gov — 1 trial record for PBGM01. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — PBGM01 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for PBGM01. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Gemma Biotherapeutics company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Biologic[2]
Company: GGemma Biotherapeutics

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