Danon Disease
2
Pipeline Programs
1
Companies
4
Clinical Trials
1 recruiting
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
1 companies ranked by most advanced pipeline stage
Rocket PharmaceuticalsNEW YORK, NY
4 programs1
1
RP-A501Phase 21 trial
RP-A501Phase 11 trial
Heart TransplantN/A1 trial
The Natural History of Danon DiseaseN/A1 trial
Active Trials
+1 more trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Rocket PharmaceuticalsRP-A501
Rocket PharmaceuticalsRP-A501
Rocket PharmaceuticalsHeart Transplant
Rocket PharmaceuticalsThe Natural History of Danon Disease
Clinical Trials (4)
Total enrollment: 280 patients across 4 trials
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Start: Sep 2023Est. completion: Apr 203214 patients
Phase 2Recruiting
Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B
Start: Apr 2019Est. completion: Mar 20257 patients
Phase 1Unknown
Natural History of Danon Disease
Start: Sep 2022Est. completion: Oct 202359 patients
N/ACompleted
The Natural History of Danon Disease
Start: Nov 2018Est. completion: Nov 2024200 patients
N/AUnknown
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
1 actively recruiting trials targeting 280 patients
1 companies competing in this space