1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 8 areas
0
News (30d)
Quiet
Hubble Therapeutics is a company with 1 orphan drug designation across 3 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 |
|---|---|---|
| PMM2-congenital disorder of glycosylation | Adeno-associated viral 2/5 (AAV2/5) vector expressing the human KCNJ13 gene | Des.TrialAppr. |
| inherited retinal dystrophy | Adeno-associated viral 2/5 (AAV2/5) vector expressing the human KCNJ13 gene | Des.TrialAppr. |
| retinal dystrophies primarily involving Bruch's membrane | Adeno-associated viral 2/5 (AAV2/5) vector expressing the human KCNJ13 gene | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
6
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
6
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio