2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
2
Rare Diseases
across 3 areas
1
News (30d)
Camp4 Therapeutics is a company with 2 orphan drug designations across 2 rare diseases. 1 news article in the past 30 days.
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 |
|---|---|---|
| Dravet syndrome | synthetic, single stranded, fully phosphorothioated 2¿-OMethyl-RNA and DNA mixmer oligonucleotide-based compound targeted against natural antisense transcripts (NATs) | Des.TrialAppr. |
| urea cycle disorder | single strand 20-mer antisense oligonucleotide (ASO) targeting a CPS1 regulatory RNA with a triantennary N-acetylgalactosamine (GalNAc) ligand attached at the 3' end | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/2
candidate diseases
1
avg importance: 40
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/2
candidate diseases
1
avg importance: 40
0
affecting portfolio