3
Orphan Designations
0 approved
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 7 areas
0
News (30d)
Quiet
AVROBIO, Inc. is a company with 3 orphan drug designations across 3 rare diseases. 3 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 |
|---|---|---|
| Fabry disease | autologous CD34+ cell- enriched population transduced with lentiviral vector encoding the codon-optimized human alpha-galactosidase A complementary deoxyribonucleic acid sequence. | Des.TrialAppr. |
| Gaucher disease | autologous CD34+ cell enriched hematopoietic stem cells genetically modified ex vivo with a lentiviral vector to contain codon-optimized complementary deoxyribonucleic acid that encodes human beta-glucocerebrosidase | Des.TrialAppr. |
| Hurler syndrome | ex vivo, lentiviral vector (LV)-mediated, genetically modified autologous cell therapy intended for the stable provision of functional human iduronate?2? sulfatase (IDS) enzyme to subjects with Hunter syndrome | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
4
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
3
affecting portfolio
0% of portfolio targets high unmet need diseases
4
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
3
affecting portfolio