5
Orphan Designations
0 approved, 5 designated
0
FDA Approvals
1
Active Trials
200 recruiting
10
Rare Diseases
across 13 areas
0
News (30d)
Quiet
Cabaletta Bio, Inc. is a company with 5 orphan drug designations across 10 rare diseases. Active clinical trials in 1 indication. gene therapy candidates in 1 disease.
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 |
|---|---|---|
| acquired idiopathic inflammatory myopathy | autologous anti-CD19 chimeric antibody receptor T cells designed to deplete CD19+ B cells | Des.TrialAppr. |
| congenital myasthenic syndrome 9 | autologous Muscle-Specific tyrosine Kinase Autoantibody Receptor T cells (MuSK-CAART) | Des.TrialAppr. |
| creatine phosphokinase, elevated serum | autologous anti-CD19 chimeric antibody receptor T cells designed to deplete CD19+ B cells | Des.TrialAppr. |
| hypokalemic alkalosis, familial, with specific renal tubulopathy | autologous Muscle-Specific tyrosine Kinase Autoantibody Receptor T cells (MuSK-CAART) | Des.TrialAppr. |
| idiopathic inflammatory myopathy | autologous anti-CD19 chimeric antibody receptor T cells designed to deplete CD19+ B cells | Des.TrialAppr. |
| intellectual disability-muscle weakness-short stature-facial dysmorphism syndrome | autologous Muscle-Specific tyrosine Kinase Autoantibody Receptor T cells (MuSK-CAART) | Des.TrialAppr. |
| lupus erythematosus | - | Des.TrialAppr. |
| myasthenia gravis | resecabtagene autoleucel | Des.TrialAppr. |
| pemphigus vulgaris | Autologous Desmoglein 3 (DSG3) Chimeric Autoantibody Receptor-directed (CAAR) T cells | Des.TrialAppr. |
| systemic sclerosis | autologous T cells transduced with lentivirus to express a fully human anti-CD19 single chain variable fragment | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
14
overlap in 2+ diseases
1/10
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
14
overlap in 2+ diseases
1/10
candidate diseases
0
avg importance: 0
0
affecting portfolio