3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
0
Active Trials
7
Rare Diseases
across 10 areas
0
News (30d)
Quiet
Amplo Biotechnology Inc. is a company with 3 orphan drug designations across 7 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 |
|---|---|---|
| Ullrich congenital muscular dystrophy 1A | Adeno Associated Virus Serotype 8 Encoding the Collagen Q Gene | Des.TrialAppr. |
| Ullrich congenital muscular dystrophy 1B | Adeno Associated Virus Serotype 8 Encoding the Collagen Q Gene | Des.TrialAppr. |
| collagen 6-related congenital muscular dystrophy | Adeno Associated Virus Serotype 8 Encoding the Collagen Q Gene | Des.TrialAppr. |
| congenital myasthenic syndrome | AAV9 gene therapy encoding the Dok7 protein | Des.TrialAppr. |
| congenital myasthenic syndrome 10 | Adeno-associated virus serotype 9 vector encoding the Dok7 protein | Des.TrialAppr. |
| fetal akinesia deformation sequence 3 | Adeno-associated virus serotype 9 vector encoding the Dok7 protein | Des.TrialAppr. |
| postsynaptic congenital myasthenic syndrome | Adeno-associated virus serotype 9 vector encoding the Dok7 protein | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/7
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/7
candidate diseases
0
avg importance: 0
0
affecting portfolio