3
Orphan Designations
0
FDA Approvals
7
Rare Diseases
1
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| Duchenne muscular dystrophy | delpacibart zotadirsen | Orphan Designation | - |
| facioscapulohumeral muscular dystrophy | An antibody oligonucleotide conjugate, comprised of a human transferrin receptor 1 targeting, effector function null, humanized IgG1 antibody, a MCC maleimide linker, and a double-stranded siRNA oligonucleotide targeting DUX4 | Orphan Designation | - |
| facioscapulohumeral muscular dystrophy 1 | An antibody oligonucleotide conjugate, comprised of a human transferrin receptor 1 targeting, effector function null, humanized IgG1 antibody, a MCC maleimide linker, and a double-stranded siRNA oligonucleotide targeting DUX4 | Orphan Designation | - |
| facioscapulohumeral muscular dystrophy 2 | An antibody oligonucleotide conjugate, comprised of a human transferrin receptor 1 targeting, effector function null, humanized IgG1 antibody, a MCC maleimide linker, and a double-stranded siRNA oligonucleotide targeting DUX4 | Orphan Designation | - |
| juvenile-onset Steinert myotonic dystrophy | an antibody oligonucleotide conjugate (AOC), comprised of a human transferrin receptor 1 (TfR1) targeting, effector function null, humanized IgG1 antibody (AV01mAb) conjugated to one small interfering RNA (siRNA). | Orphan Designation | - |
| late-onset Steinert myotonic dystrophy | an antibody oligonucleotide conjugate (AOC), comprised of a human transferrin receptor 1 (TfR1) targeting, effector function null, humanized IgG1 antibody (AV01mAb) conjugated to one small interfering RNA (siRNA). | Orphan Designation | - |
| myotonic dystrophy type 1 | an antibody oligonucleotide conjugate (AOC), comprised of a human transferrin receptor 1 (TfR1) targeting, effector function null, humanized IgG1 antibody (AV01mAb) conjugated to one small interfering RNA (siRNA). | Orphan Designation | - |