3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
0
Active Trials
9
Rare Diseases
across 16 areas
0
News (30d)
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MeiraGTx UK II Ltd is a company with 3 orphan drug designations across 9 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 |
|---|---|---|
| Bardet-Biedl syndrome | recombinant, replication incompetent adeno-associated virus viral vector platform with a serotype 8 capsid and a cDNA encoding the human BBS10 gene (AAV8-RK-BBS10) | Des.TrialAppr. |
| Bardet-Biedl syndrome 12 | recombinant, replication incompetent adeno-associated virus viral vector platform with a serotype 8 capsid and a cDNA encoding the human BBS10 gene (AAV8-RK-BBS10) | Des.TrialAppr. |
| Bardet-Biedl syndrome 2 | recombinant, replication incompetent adeno-associated virus viral vector platform with a serotype 8 capsid and a cDNA encoding the human BBS10 gene (AAV8-RK-BBS10) | Des.TrialAppr. |
| Leber congenital amaurosis 13 | Adeno-associated virus serotype 5 containing the human RDH12 gene | Des.TrialAppr. |
| PMM2-congenital disorder of glycosylation | adenovirus associated viral vector serotype 8 containing the human AIPL1 gene | Des.TrialAppr. |
| RDH12-related dominant retinopathy | Adeno-associated virus serotype 5 containing the human RDH12 gene | Des.TrialAppr. |
| RDH12-related recessive retinopathy | Adeno-associated virus serotype 5 containing the human RDH12 gene | Des.TrialAppr. |
| inherited retinal dystrophy | adenovirus associated viral vector serotype 8 containing the human AIPL1 gene | Des.TrialAppr. |
| retinal dystrophies primarily involving Bruch's membrane | adenovirus associated viral vector serotype 8 containing the human AIPL1 gene | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
7
overlap in 2+ diseases
0/9
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
7
overlap in 2+ diseases
0/9
candidate diseases
0
avg importance: 0
0
affecting portfolio