3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
0
Active Trials
2
Rare Diseases
across 4 areas
0
News (30d)
Quiet
Beacon Therapeutics is a company with 3 orphan drug designations across 2 rare diseases.
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 |
|---|---|---|
| achromatopsia | recombinant adeno-associated virus vector expressing the cyclic nucleotide gated channel alpha subunit (CNGA3)recombinant adeno-associated virus vector expressing the cyclic nucleotide gated channel beta subunit (rAAV-CNGB3) | Des.TrialAppr. |
| retinitis pigmentosa | recombinant adeno-associated virus vector expressing the retinitis pigmentosa GTPase regulator | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio