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mRNA-3745

Phase 1

Glycogen Storage Disease | RNA therapy | Metabolic |Moderna, Inc.|Trials Updated: Dec 15, 2025

mRNA-3745 development status

Highest phase Phase 1
Registered trials 1 across 1 sponsor since Oct 2021

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment15

FDA Designations

No designations recorded

mRNA-3745 clinical trials

mRNA-3745 · 1 trial · 1 indication

Phase 1 1
NCT05095727A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)Glycogen Storage Disease
ACTIVE NOT_RECRUITING15 Analytics
PHASE1ACTIVE NOT_RECRUITING
A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
Glycogen Storage DiseaseUnlock trial analytics

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 to 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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Frequently asked questions about mRNA-3745

What is mRNA-3745?

mRNA-3745 is an investigational RNA therapy being developed by Moderna, Inc. for glycogen storage disease. It is designed as a messenger RNA-based treatment and is currently in Phase 1 clinical development. The program is focused on glycogen storage disease type 1a, a metabolic condition.

What is mRNA-3745 used for in glycogen storage disease?

mRNA-3745 is being studied for the treatment of glycogen storage disease type 1a (GSD1a), a metabolic disorder. It is an investigational therapy and has not been approved for any indication. The Phase 1 trial enrolls adult and pediatric participants with GSD1a.

Who makes mRNA-3745?

mRNA-3745 is being developed by Moderna, Inc., which trades on the Nasdaq under the ticker MRNA. Moderna is the sponsor of the clinical program evaluating the therapy in glycogen storage disease type 1a.

What phase is mRNA-3745 in?

mRNA-3745 is in Phase 1 clinical development. It is an investigational therapy and has not been approved by the FDA or any other regulatory agency. The single Phase 1 trial is currently active but not recruiting participants.

What clinical trials is mRNA-3745 in?

mRNA-3745 is being evaluated in one Phase 1 trial, NCT05095727, titled A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a). The trial has an estimated enrollment of 15 participants and is active but not recruiting. It is conducted in the United States, Canada, France, Netherlands, Poland, and Spain.

What does mRNA-3745 target?

mRNA-3745 is an RNA therapy, specifically a messenger RNA-based treatment. It is designed to address the underlying metabolic defect in glycogen storage disease type 1a. The therapy is being developed by Moderna, Inc. and is in Phase 1 testing.