2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
2
Active Trials
200 recruiting
5
Rare Diseases
across 11 areas
0
News (30d)
Quiet
PYC Therapeutics, LLC is a company with 2 orphan drug designations across 5 rare diseases. Active clinical trials in 2 indications.
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 |
|---|---|---|
| Behr syndrome | peptide-oligonucleotide conjugate that targets OPA1 mRNA | Des.TrialAppr. |
| PMM2-congenital disorder of glycosylation | peptide-oligonucleotide conjugate that targets CNOT3 mRNA | Des.TrialAppr. |
| inherited retinal dystrophy | peptide-oligonucleotide conjugate that targets CNOT3 mRNA | Des.TrialAppr. |
| mitochondrial disease | - | Des.TrialAppr. |
| retinal dystrophies primarily involving Bruch's membrane | peptide-oligonucleotide conjugate that targets CNOT3 mRNA | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
6
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
6
overlap in 2+ diseases
0/5
candidate diseases
0
avg importance: 0
0
affecting portfolio