2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
1
Active Trials
142 recruiting
5
Rare Diseases
across 11 areas
0
News (30d)
Quiet
GEMMA Biotherapeutics is a company with 2 orphan drug designations across 5 rare diseases. Active clinical trials in 1 indication. 4 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 |
|---|---|---|
| GM1 gangliosidosis | non-replicating recombinant adeno-associated virus serotype hu68 vector, which contains human GLB1 transgene encoding the galactosidase beta 1 enzyme | Des.TrialAppr. |
| Krabbe disease | a non-replicating recombinant adeno-associated virus serotype hu68 vector, containing a transgene encoding the human galactosylceramidase (GALC) enzyme | Des.TrialAppr. |
| adult Krabbe disease | a non-replicating recombinant adeno-associated virus serotype hu68 vector, containing a transgene encoding the human galactosylceramidase (GALC) enzyme | Des.TrialAppr. |
| late-infantile/juvenile Krabbe disease | a non-replicating recombinant adeno-associated virus serotype hu68 vector, containing a transgene encoding the human galactosylceramidase (GALC) enzyme | Des.TrialAppr. |
| spinal muscular atrophy | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
4
affecting portfolio
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
4
affecting portfolio