2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
1
Active Trials
200 recruiting
3
Rare Diseases
across 5 areas
0
News (30d)
Quiet
Yoltech Therapeutics Co., Ltd is a company with 2 orphan drug designations across 3 rare diseases. Active clinical trials in 1 indication.
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 |
|---|---|---|
| alpha 1-antitrypsin deficiency | lipid nanoparticles containing base editor CRISPR-Cas9 messenger RNA (mRNA) and a single guide RNA (sgRNA) targeting SERPINA1 gene | Des.TrialAppr. |
| amyloidosis | - | Des.TrialAppr. |
| primary hyperoxaluria | lipid nanoparticles containing CRISPR/Cas12HF mRNA and a CRISPR RNA (crRNA) targeting the HAO1 gene | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio