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mRNA-3745

Phase 1

Glycogen Storage Disease | Small molecule | Metabolic |Moderna, Inc.|Last Updated: Dec 15, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment15
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05095727A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)PHASE1 ACTIVE NOT_RECRUITING 15 — — Jun 1, 2022Nov 30, 2026Dec 15, 202516 United States, Canada +4
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Study Endpoints
Primary Endpoints
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and TEAEs Leading to Treatment Discontinuation
Day 1 up to approximately 3.5 years
Secondary Endpoints
Number of Participants Not Experiencing Hypoglycemia During Fasting Challenges
Baseline through up to Week 32
Change From Baseline of Area Under the Effect Curve (AUEC) of Blood Glucose and Lactate During Fasting Challenges
Baseline through up to Week 32
Change From Baseline in Time to Hypoglycemia During Fasting Challenges
Baseline through up to Week 32
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
SAD: mRNA-3745EXPERIMENTALParticipants will receive a single intravenous (IV) dose of mRNA-3745 on Day 1 in an inpatient setting. Participants that are/have been enrolled in the study and receive an administration of mRNA-3745 may also enroll in one of the MAD cohorts. The first MAD dose must occur at least 21 days after the SAD dose.
MAD: mRNA-3745EXPERIMENTALParticipants will receive multiple IV doses of mRNA-3745 in an inpatient setting. Participants will have the option to continue treatment in the OLE.
Interventions
NameTypeDescription
mRNA-3745DRUGSterile frozen liquid dispersion for injection
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Eligibility Criteria
Age Range6 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites16

Inclusion Criteria: * Documented GSD1a with confirmation of biallelic gene encoding glucose-6-phosphatase-α (G6PC) mutations by genetic testing. * Absence of hospitalization for hypoglycemia in the 4 weeks prior to Screening Exclusion Criteria: * Solid organ transplant * Received gene therapy for...

Countries:United StatesCanadaFranceNetherlandsPolandSpain
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05095727primaryCompletionDate: changed
LOWMay 24, 2026NCT05095727studyFirstPostDate: changed