3
Orphan Designations
0 approved
0
FDA Approvals
0
Active Trials
191 recruiting
5
Rare Diseases
across 14 areas
0
News (30d)
Quiet
Sigilon Therapeutics, Inc. is a company with 3 orphan drug designations across 5 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 |
|---|---|---|
| Fabry disease | Cultured human retinal pigment epithelial cells (ARPE-19) genetically modified with a non-viral vector to express Human alpha-galactosidase A (h alpha-Gal A), encapsulated within two-layer modified alginate spheres | Des.TrialAppr. |
| alpha thalassemia-X-linked intellectual disability syndrome | Cultured human retinal pigment epithelial cells (ARPE-19) genetically modified with a non-viral vector to express Human alpha-galactosidase A (h alpha-Gal A), encapsulated within two-layer modified alginate spheres | Des.TrialAppr. |
| alpha-thalassemia-myelodysplastic syndrome | Cultured human retinal pigment epithelial cells (ARPE-19) genetically modified with a non-viral vector to express Human alpha-galactosidase A (h alpha-Gal A), encapsulated within two-layer modified alginate spheres | Des.TrialAppr. |
| hemophilia A | Cultured human retinal pigment epithelial cell line (ARPE-19) genetically modified with a non-viral vector to express B domain deleted human factor VIII protein, encapsulated within two-layer modified alginate spheres | Des.TrialAppr. |
| mucopolysaccharidosis type 1 | Cultured human retinal pigment epithelial cells (ARPE-19) genetically modified with a non-viral vector to express human native alpha-L-iduronidase enzyme (hIDUA), encapsulated within two-layer modified alginate spheres | Des.TrialAppr. |
33% of portfolio targets high unmet need diseases
6
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio
33% of portfolio targets high unmet need diseases
6
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio