2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
2
Rare Diseases
across 4 areas
0
News (30d)
Quiet
M6P Therapeutics is a company with 2 orphan drug designations across 2 rare diseases. 3 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 |
|---|---|---|
| Gaucher disease | AAV9 capsid encapsulating a bicistronic vector encoding for a unique combination of a recombinant human ?-glucocerebrosidase enzyme with the S1S3 variant of the N-acetylglucosamine-Phosphotransferase (S1S3 PTase) | Des.TrialAppr. |
| mucolipidosis | adeno-associated viral vector type 9 with S1S3 gene encoding S1S3 variant of N-acetylglucosamine-1-phosphotransferase | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
3
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/2
candidate diseases
0
avg importance: 0
3
affecting portfolio