4
Orphan Designations
0 approved
0
FDA Approvals
0
Active Trials
2
Rare Diseases
across 6 areas
0
News (30d)
Quiet
Homology Medicines, Inc. is a company with 4 orphan drug designations across 2 rare diseases. gene therapy candidates in 1 disease.
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 |
|---|---|---|
| metachromatic leukodystrophy | Adeno-associated virus serotype HSC15 expressing human arylsulfatase A | Des.TrialAppr. |
| phenylketonuria | Recombinant adeno-associated viral vector serotype HSC15, containing homology arms targeting the human phenylalanine hydroxylase genomic locus, and expressing human phenylalanine hydroxylaserecombinant adeno-associated viral vector serotype HSC expressing human phenylalanine hydroxylase | Des.TrialAppr. |
50% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
50% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
1/2
candidate diseases
0
avg importance: 0
0
affecting portfolio