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
Stoke Therapeutics, Inc. is a company with 2 orphan drug designations across 3 rare diseases. Active clinical trials in 2 indications. 2 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 |
|---|---|---|
| Dravet syndrome | 18-mer antisense oligonucleotide complementary to SCN1A mRNA | Des.TrialAppr. |
| autosomal dominant optic atrophy | 18?mer antisense oligonucleotide complementary to OPA1 pre-mRNA | Des.TrialAppr. |
| epilepsy | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
6
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
2
affecting portfolio
0% of portfolio targets high unmet need diseases
6
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
2
affecting portfolio