RP-L301

Phase 2Gene therapy

Rocket Pharmaceuticals

RP-L301 is one of 2 biologic programs targeting Pyruvate Kinase Deficiency. It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Pyruvate Kinase DeficiencyNot yet recruiting

Pyruvate Kinase DeficiencyPhase 2

Not yet recruiting

Competition

Competitive Landscape

1 competitor in Pyruvate Kinase Deficiency

#DrugCompanyPhaseTrials

Aqvesme

AAgios Pharmaceuticals, Inc.

Approved

2 2

RP-L301

Rocket Pharmaceuticals

Phase 2

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

Timeline

Expected Milestones

Q1 2029

Phase 2Not yet recruiting

Pyruvate Kinase Deficiency

NCT06422351

Clinical

Trials by Indication[1]

Pyruvate Kinase Deficiency2 trials

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Completed· Rocket Pharmaceuticals logo

Rocket Pharmaceuticals·Phase 1·NCT04105166·4 enrolled

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

Not yet recruiting· Rocket Pharmaceuticals logo

Rocket Pharmaceuticals·Phase 2·NCT06422351·10 enrolled

Sources

References

[1]
ClinicalTrials.gov — 2 trial records for RP-L301. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — RP-L301 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for RP-L301. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Rocket Pharmaceuticals company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Gene therapy[2]
Highest Phase: Phase 2[1]
Company: Rocket Pharmaceuticals

RP-L301

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2029

Trials by Indication[1]

Pyruvate Kinase Deficiency2 trials

References

Drug Info

Quick Facts

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