3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
0
Active Trials
130 recruiting
2
Rare Diseases
across 1 areas
0
News (30d)
Quiet
IPS Heart is a company with 3 orphan drug designations across 2 rare diseases. 5 active policy bills affect their portfolio.
Disease-by-disease pipeline progression from orphan designation through clinical trials to FDA approval.
Source: FDA Orphan Drug Designations, ClinicalTrials.gov, Drugs@FDA
| Disease | Drug(s) | Stage |
|---|---|---|
| Becker muscular dystrophy | human induced pluripotent stem cell derived myogenic progenitor cells | Des.TrialAppr. |
| Duchenne muscular dystrophy | Human induced pluripotent stem cell derived cardiac progenitor cellsHuman induced pluripotent stem cell derived myogenic progenitor cells (GIVI-MPC) | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
25
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
5
affecting portfolio
0% of portfolio targets high unmet need diseases
25
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
5
affecting portfolio