Muscular Dystrophies

5
Pipeline Programs
3
Companies
5
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
2
0
2
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
267%
Cell Therapy
133%
+ 2 programs with unclassified modality

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Capricor Therapeutics
2 programs
1
1
DeramiocelPhase 3Cell Therapy1 trial
CAP-1002Phase 21 trial
Active Trials
NCT03406780Completed20Est. Mar 2020
NCT05126758Active Not Recruiting106Est. Dec 2026
BioMarin Pharmaceutical
2 programs
1
1
DrisapersenPhase 3RNA Therapeutic1 trial
DrisapersenPhase 2RNA Therapeutic1 trial
Active Trials
NCT01910649Terminated12Est. Sep 2016
NCT01803412Terminated53Est. Oct 2016
Edgewise Therapeutics
1 program
1
EDG-5506Phase 11 trial
Active Trials
NCT05492734Completed10Est. Aug 2022

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Capricor TherapeuticsDeramiocel
BioMarin PharmaceuticalDrisapersen
Capricor TherapeuticsCAP-1002
BioMarin PharmaceuticalDrisapersen
Edgewise TherapeuticsEDG-5506

Clinical Trials (5)

Total enrollment: 201 patients across 5 trials

NCT05126758Capricor TherapeuticsDeramiocel

A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Start: Jun 2022Est. completion: Dec 2026106 patients
Phase 3Active Not Recruiting
NCT01803412BioMarin PharmaceuticalDrisapersen

A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

Start: May 2013Est. completion: Oct 201653 patients
Phase 3Terminated
NCT03406780Capricor TherapeuticsCAP-1002

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

Start: Apr 2018Est. completion: Mar 202020 patients
Phase 2Completed
NCT01910649BioMarin PharmaceuticalDrisapersen

A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration

Start: Mar 2008Est. completion: Sep 201612 patients
Phase 2Terminated
NCT05492734Edgewise TherapeuticsEDG-5506

A Study to Assess the Feasibility of Non-invasive Dried Blood Sampling

Start: Aug 2022Est. completion: Aug 202210 patients
Phase 1Completed

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 late-stage (Phase 3) programs — potential near-term approvals
3 companies competing in this space