Approval Probability
TA Base Rate
Adjusted LOA
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MuSK-CAART · 1 trial · 1 indication
Incidence of adverse events (AEs), including dose-limiting toxicities (DLTs) and AEs that are related to MuSK-CAART.
| Arm | Type | Description |
|---|---|---|
| MuSK-CAART | EXPERIMENTAL | Cohort A: Infusion of MuSK-CAART at various dose levels with or without pre-treatment (6 groups planned). Cohort B: Infusion of MuSK-CAART at the dose regimen selected from Part A. |
| Name | Type | Description |
|---|---|---|
| MuSK-CAART | BIOLOGICAL | Intravenous infusion of MuSK-CAART at different doses. Subjects may also receive MuSK-CAART following pre-treatment with CY, or CY plus FLU. |
Inclusion Criteria: * Confirmed diagnosis of MuSK-type MG with at least 1 prior positive anti-MuSK antibody test. * History of a negative anti-AChR (acetylcholine receptor) antibody test. * Positive anti-MuSK antibody test at screening * MG severity Class I to IVa on the MGFA (Myasthenia Gravis Fou...
MuSK-CAART is an investigational cell therapy being developed for the treatment of MuSK Myasthenia Gravis, a neurological autoimmune disorder. It is designed to target and eliminate the autoreactive B cells that produce antibodies against the MuSK protein, potentially offering a targeted approach to managing this condition.
MuSK-CAART is being developed by Cabaletta Bio, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol CABA. The company is focused on developing cellular immunotherapies for autoimmune diseases, with MuSK-CAART being one of its lead investigational candidates.
MuSK-CAART is currently in Phase 1 clinical development. It has completed a Phase 1 open-label study evaluating the safety of various dosing regimens in patients with MuSK Myasthenia Gravis. The drug is investigational and has not yet been approved by regulatory authorities.
MuSK-CAART has been studied in one clinical trial, identified as NCT05451212. This was an open-label, uncontrolled Phase 1 study conducted in the United States, enrolling 7 adult patients with MuSK Myasthenia Gravis. The trial has been completed, and its primary focus was on evaluating the safety of different dosing regimens.
Yes, MuSK-CAART has received both Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration. These designations are intended to facilitate and expedite the development and review of drugs that treat serious conditions and fill an unmet medical need.