1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
3
Active Trials
200 recruiting
4
Rare Diseases
across 6 areas
0
News (30d)
Quiet
Shanghai BDgene Co., Ltd. is a company with 1 orphan drug designation across 4 rare diseases. Active clinical trials in 3 indications.
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 |
|---|---|---|
| Huntington disease | Virus-like particle containing Cas9/gRNA ribonucleoprotein targeting the human HTT gene | Des.TrialAppr. |
| Huntington disease-like 1 | Virus-like particle containing Cas9/gRNA ribonucleoprotein targeting the human HTT gene | Des.TrialAppr. |
| Huntington disease-like 2 | Virus-like particle containing Cas9/gRNA ribonucleoprotein targeting the human HTT gene | Des.TrialAppr. |
| thalassemia | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
15
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
15
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio