3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
3
Active Trials
200 recruiting
7
Rare Diseases
across 12 areas
0
News (30d)
Quiet
4D Molecular Therapeutics, Inc. is a company with 3 orphan drug designations across 7 rare diseases. Active clinical trials in 3 indications. 1 active policy bill affects 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 | Adeno-Associated Virus Serotype 2 capsid variant (4D-C102) carrying a transgene encoding a codon-optimized human alpha-galactosidase A gene | Des.TrialAppr. |
| cardiovascular disorder | - | Des.TrialAppr. |
| choroideremia | adeno-associated virus (AAV2) capsid variant carrying a transgene encoding a codon-optimized human choroideremia (CHM) gene | Des.TrialAppr. |
| choroideremia hypopituitarism | adeno-associated virus (AAV2) capsid variant carrying a transgene encoding a codon-optimized human choroideremia (CHM) gene | Des.TrialAppr. |
| choroideremia-deafness-obesity syndrome | adeno-associated virus (AAV2) capsid variant carrying a transgene encoding a codon-optimized human choroideremia (CHM) gene | Des.TrialAppr. |
| congenital heart disease | - | Des.TrialAppr. |
| cystic fibrosis | 4D-A101.CMV173-CFTRDeltaR | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
28
overlap in 2+ diseases
0/7
candidate diseases
0
avg importance: 0
1
affecting portfolio
0% of portfolio targets high unmet need diseases
28
overlap in 2+ diseases
0/7
candidate diseases
0
avg importance: 0
1
affecting portfolio