2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 4 areas
0
News (30d)
Quiet
Papillon Therapeutics Inc. is a company with 2 orphan drug designations across 3 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 |
|---|---|---|
| Danon disease | autologous human CD34+ hematopoietic stem and progenitor cells (HSPCs), derived from G-CSF/plerixafor mobilized peripheral blood stem cells (PBSCs) of patients with Danon disease (DD) | Des.TrialAppr. |
| Friedreich ataxia | autologous human CD34+ hematopoietic stem and progenitor cells isolated from mobilized peripheral blood stem cell of patients with Friedreich's ataxia, modified ex vivo using CRISPR/Cas9 technology to remove the GAA hyper-expansion mutation in the intron 1 of frataxin | Des.TrialAppr. |
| Friedreich ataxia 2 | autologous human CD34+ hematopoietic stem and progenitor cells isolated from mobilized peripheral blood stem cell of patients with Friedreich's ataxia, modified ex vivo using CRISPR/Cas9 technology to remove the GAA hyper-expansion mutation in the intron 1 of frataxin | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
7
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
7
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio