3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
1
Active Trials
200 recruiting
3
Rare Diseases
across 3 areas
0
News (30d)
Quiet
Ractigen Therapeutics is a company with 3 orphan drug designations across 3 rare diseases. Active clinical trials in 1 indication. 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 | saRNA oligonucleotide designed to target and upregulate the expression of human utrophin gene (UTRN) | Des.TrialAppr. |
| Duchenne muscular dystrophy | saRNA oligonucleotide designed to target and upregulate the expression of human utrophin gene (UTRN) | Des.TrialAppr. |
| amyotrophic lateral sclerosis | a synthetic double-stranded siRNA oligonucleotide targeting FUS mRNASynthetic double-stranded siRNA oligonucleotide targeting SOD1 mRNA | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
32
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
5
affecting portfolio
0% of portfolio targets high unmet need diseases
32
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
5
affecting portfolio