rAAV1-CMV-GAA (study agent) Administration

Gene therapy

rAAV1-CMV-GAA (study agent) Administration is one of 28 small molecule programs targeting Pompe Disease. It is currently in development.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Pompe DiseaseActive, not recruiting

Pompe DiseasePhase 1/Phase 2

Active, not recruiting

Competition

Competitive Landscape

38 competitors in Pompe Disease

#DrugCompanyPhaseTrials

Rituxan

Roche

Approved

3 2

Trexall

Alnylam Pharmaceuticals

Approved

2 3

Asceniv

CSL Behring

Approved

1 4

Velcade

SShilpa Grover, MD, MPH

Approved

1 5

Salbutamol

GSK

Approved

1 6

Opfolda

Johnson & Johnson

Phase 3

3 7

BMN 333

BioMarin Pharmaceutical

Phase 3

2 8

Clenbuterol

UUniversity of Copenhagen

Phase 2

2 9

Proair HFA

GGlaxo Dba Glaxosmithkline

Phase 1

2 10

Benadryl

PParkedale

Phase 1

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

Clinical

Trials by Indication[1]

Pompe Disease1 trial

Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease

Completed·UUniversity of Florida·Phase 1/2·NCT00976352·9 enrolled

Sources

References

[1]
ClinicalTrials.gov — 1 trial record for rAAV1-CMV-GAA (study agent) Administration. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — rAAV1-CMV-GAA (study agent) Administration compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for rAAV1-CMV-GAA (study agent) Administration. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — University of Florida company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Gene therapy[2]
Company: UUniversity of Florida

rAAV1-CMV-GAA (study agent) Administration

Status by Indication[3][1]

Competitive Landscape

Trials by Indication[1]

Pompe Disease1 trial

References

Drug Info

Quick Facts

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