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Mgta-456

Inborn Errors Of Metabolism

Explore 12 assets in Inborn Errors Of Metabolism
Floatz Rating
B57/100
Confidence
Indicative
v0.2
Sponsor
Magenta Therapeutics, Inc.
Modality
UNKNOWN
Development Phase
Phase 2
Status
Dormant
Evidence ledger · v0.2

Clinical Evidence

Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
42Moderate confidence
Detailed axis rationale is planned and will be published soon.
TrialPhaseStatusNPrimary endpointReadout
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
Completed

Competitive Position

Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95Moderate confidence
Detailed axis rationale is planned and will be published soon.

Same indication · Inborn Errors Of Metabolism

AssetSponsorPhaseRating
Mgta-456 (this asset)Magenta Therapeutics, Inc.P2B · 57
Nexiguran ZiclumeranIntellia TherapeuticsP3B
ThiotepaUniversity of California, San FranciscoP2CCC
HydroxyureaPaul SzabolcsP2CCC
BusulfanumMemorial Sloan Kettering Cancer CenterP2CCC
Cald Hr-DMasonic Cancer Center, University of MinnesotaP2CCC
AlemtuzumabPaul SzabolcsP2CCC
Hpc, Cord BloodMemorial Sloan Kettering Cancer CenterP2CCC
Cyclosporin AMemorial Sloan Kettering Cancer CenterP2CCC

+7 more in the Inborn Errors Of Metabolism cohort

Other indications for Mgta-456

IndicationSponsorPhaseRating
Acute Lymphoblastic LeukemiaP2CCC · 53
Acute Leukemia Of Ambiguous LineageP2CCC · 53
Chronic Myeloid LeukemiaP2CCC · 53

Scientific Foundation

Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR

Planned for methodology v0.2.

Development Feasibility

How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR

Planned for methodology v0.2.

Commercial Opportunity

Commercial prize: addressable population, unmet need, and the value case for the indication.
NR

Planned for methodology v0.2.

IP & Exclusivity

Exclusivity position, covering patent protection and freedom-to-operate runway.
NR

Planned for methodology v0.2.

Manufacturing & Supply

Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR

Planned for methodology v0.2.

Related assets

Citation

Floatz Terminal. Mgta-456 in Inborn Errors Of Metabolism. Methodology v0.2.
Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026.
Accessed July 14, 2026.
https://terminal.floatz.ai/assets/mgta-456-inborn-errors-of-metabolism

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