2
Orphan Designations
0 approved, 2 designated
0
FDA Approvals
2
Active Trials
200 recruiting
4
Rare Diseases
across 8 areas
0
News (30d)
Quiet
Dyne Therapeutics, Inc. is a company with 2 orphan drug designations across 4 rare diseases. Active clinical trials in 2 indications. 5 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 |
|---|---|---|
| Duchenne muscular dystrophy | a Fab drug targeting the human TfR1 and conjugated to a PMO designed to bind to DMD exon 51 | Des.TrialAppr. |
| familial partial lipodystrophy, Dunnigan type | Human transferrin 1 receptor (TfR1) targeting humanized IgG 1 kappa fragment antibody conjugated to an antisense oligonucleotide (ASO) | Des.TrialAppr. |
| multiple system atrophy | - | Des.TrialAppr. |
| muscular dystrophy | - | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
39
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
5
affecting portfolio
0% of portfolio targets high unmet need diseases
39
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
5
affecting portfolio