Pyruvate Kinase Deficiency

2
Pipeline Programs
2
Companies
3
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
1
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Rocket Pharmaceuticals
2 programs
1
1
RP-L301Phase 21 trial
RP-L301Phase 11 trial
Active Trials
NCT04105166Completed4Est. Jun 2025
NCT06422351Not Yet Recruiting10Est. Jan 2029
C
CENTOGENEGermany - Rostock
1 program
Pyruvate Kinase Deficiency Epidemiological Study (PIECE)N/A1 trial
Active Trials
NCT03866590Completed75Est. May 2021

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
Rocket PharmaceuticalsRP-L301
Rocket PharmaceuticalsRP-L301
CENTOGENEPyruvate Kinase Deficiency Epidemiological Study (PIECE)

Clinical Trials (3)

Total enrollment: 89 patients across 3 trials

NCT06422351Rocket PharmaceuticalsRP-L301

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

Start: Aug 2024Est. completion: Jan 202910 patients
Phase 2Not Yet Recruiting
NCT04105166Rocket PharmaceuticalsRP-L301

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Start: Jul 2020Est. completion: Jun 20254 patients
Phase 1Completed
NCT03866590CENTOGENEPyruvate Kinase Deficiency Epidemiological Study (PIECE)

Pyruvate Kinase Deficiency Epidemiological Study (PIECE)

Start: Jan 2020Est. completion: May 202175 patients
N/ACompleted

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

2 companies competing in this space