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MGTA-456 · 1 trial · 1 indication
Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.
| Arm | Type | Description |
|---|---|---|
| MGTA-456 | EXPERIMENTAL | MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation. |
| Name | Type | Description |
|---|---|---|
| MGTA-456 | DRUG | Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456. |
Inclusion Criteria: * Age \< 2.5 years with Hurler syndrome, age 2-17 years with cerebral adrenoleukodystrophy (cALD), age \< 16 years with metachromatic leukodystrophy (MLD) and age ≤ 10 years with globoid cell leukodystrophy (GLD) (also referred to as Krabbe) * Cord blood grafts require genetic t...
MGTA-456 is an investigational small molecule being studied for the treatment of inherited metabolic disorders (IMD). It is designed for patients undergoing hematopoietic stem cell transplantation (HSCT). The drug is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
MGTA-456 is a small molecule, but its specific molecular target has not been disclosed. It is being investigated for its potential role in improving outcomes for patients with inherited metabolic disorders undergoing hematopoietic stem cell transplantation.
MGTA-456 is being developed by Dianthus Therapeutics, Inc., a biopharmaceutical company traded on the stock exchange under the ticker symbol DNTH. The company is conducting clinical research to evaluate the drug's safety and efficacy in patients with inherited metabolic disorders.
MGTA-456 is in Phase 2 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory authorities. The drug is being studied for the treatment of inherited metabolic disorders in patients undergoing hematopoietic stem cell transplantation.
MGTA-456 has been studied in a Phase 2 clinical trial with the identifier NCT03406962. This trial, titled 'MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT),' was completed and enrolled 8 participants in the United States. The trial was uncontrolled and not blinded.
MGTA-456 is the primary name for this investigational drug. No alternative names have been reported for this asset. It is being developed by Dianthus Therapeutics, Inc. for the treatment of inherited metabolic disorders.