1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
2
Rare Diseases
across 3 areas
0
News (30d)
Quiet
Asklepios Biopharmaceutical, Inc. is a company with 1 orphan drug designation across 2 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 |
|---|---|---|
| autosomal recessive limb-girdle muscular dystrophy | adeno-associated virus serotype 9 (AAV9) vector containing a linear, self-complementary DNA genome encoding a codon-optimized, CpG motif-depleted, human fukutin-related protein (FKRP) gene under the control of a Syn100 promoter (scAAV9.Syn100.coHuFKRP) | Des.TrialAppr. |
| limb-girdle muscular dystrophy | adeno-associated virus serotype 9 (AAV9) vector containing a linear, self-complementary DNA genome encoding a codon-optimized, CpG motif-depleted, human fukutin-related protein (FKRP) gene under the control of a Syn100 promoter (scAAV9.Syn100.coHuFKRP) | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
0
affecting portfolio