2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
2
Active Trials
200 recruiting
3
Rare Diseases
across 6 areas
0
News (30d)
Quiet
ReCode Therapeutics, Inc. is a company with 2 orphan drug designations across 3 rare diseases. Active clinical trials in 2 indications. 1 active policy bill affects 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 |
|---|---|---|
| congenital heart disease | - | Des.TrialAppr. |
| cystic fibrosis | mRNA encoding full-length CFTR protein | Des.TrialAppr. |
| primary ciliary dyskinesia | DNAI1 mRNA encapsulated in a selective organ targeting lipid nanoparticle | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
9
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
1
affecting portfolio
0% of portfolio targets high unmet need diseases
9
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
1
affecting portfolio