1
Orphan Designations
0 approved
0
FDA Approvals
1
Active Trials
200 recruiting
4
Rare Diseases
across 7 areas
0
News (30d)
Quiet
Voyager Therapeutics is a company with 1 orphan drug designation across 4 rare diseases. Active clinical trials in 1 indication. 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 |
|---|---|---|
| Alzheimer disease | - | Des.TrialAppr. |
| Huntington disease | recombinant adeno-associated virus, serotype 1, containing a transgene that encodes a microRNA targeting huntingtin messenger RNA | Des.TrialAppr. |
| Huntington disease-like 1 | recombinant adeno-associated virus, serotype 1, containing a transgene that encodes a microRNA targeting huntingtin messenger RNA | Des.TrialAppr. |
| Huntington disease-like 2 | recombinant adeno-associated virus, serotype 1, containing a transgene that encodes a microRNA targeting huntingtin messenger RNA | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
21
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
2
affecting portfolio
0% of portfolio targets high unmet need diseases
21
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
2
affecting portfolio