4
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
4
Rare Diseases
across 9 areas
0
News (30d)
Quiet
Neurogene Inc. is a company with 4 orphan drug designations across 4 rare diseases. 2 active policy bills affect 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 |
|---|---|---|
| Krabbe disease | Recombinant serotype 9 adeno-associated virus encoding a codon-optimized human galactosylceramidase (GALC) transgene (hGALCopt2) | Des.TrialAppr. |
| Rett syndrome | Recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence, complementary miRNA binding sites and a full-length human methyl cytosine binding protein 2 (MECP2) | Des.TrialAppr. |
| neuronal ceroid lipofuscinosis 5 | Adeno-associated virus serotype 9 vector with engineered transgene encoding the human CLN5 gene | Des.TrialAppr. |
| neuronal ceroid lipofuscinosis 7 | An adeno-associated virus serotype 9 (AAV9) vector with engineered transgene encoding the human CLN7/MFSD8 gene for expression of active human major facilitator superfamily domain containing 8 | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
2
affecting portfolio
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
2
affecting portfolio