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mRNA-3705

Phase 1

Methylmalonic Acidemia | RNA therapy | Rare Disease |Moderna, Inc.|Trials Updated: Dec 23, 2025

mRNA-3705 development status

Highest phase Phase 1
Registered trials 2 across 1 sponsor since May 2021

mRNA-3705 target and mechanism

ModalityRNA therapy

Also known as modified mRNA encoding human, methylmalonyl-coenzyme A mutase

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment56

FDA Designations

No designations recorded

mRNA-3705 clinical trials

mRNA-3705 · 1 trial · 1 indication

Phase 1 1
NCT05295433An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705Methylmalonic Acidemia
RECRUITING56 Analytics
PHASE1RECRUITING
An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705
Methylmalonic AcidemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Drug-Related TEAEs, Unrelated TEAEs, Adverse Events of Special Interests (AESIs), Serious Adverse Events (SAEs) and TEAEs Leading to Discontinuation
Baseline up to follow-up period (up to 6 years + 6 months)

Secondary Endpoints

Percent Change in Plasma Methylmalonic Acid and 2-Methylcitric Acid (2-MC) Levels (Primary Biomarkers) From Baseline up to 18 Months
Baseline, Year 6 (Treatment Period) + Month 6 (Follow up)
Pre- and Postdose Human Methylmalonyl-Coenzyme A Mutase (hMUT) mRNA and SM-86 Levels
Baseline up to 6 years
Change From Baseline in Pretreatment and Post-treatment Annualized Metabolic Decompensation Events (MDEs) Rate
Baseline, Year 6 (Treatment Period) + Month 6 (Follow up)
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
mRNA-3705EXPERIMENTALParticipants will receive mRNA-3705 at the same dose levels at the same dosing interval (every 2 weeks \[Q2W\], or every 3 weeks \[Q3W\]) last received in the clinical study of mRNA-3705 in which they initially participated, unless the Sponsor recommends modification.

Interventions

NameTypeDescription
mRNA-3705DRUGA sterile liquid for injection
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Eligibility Criteria

Age Range1 Year to N/A
SexALL
Healthy VolunteersNo
Study Sites12

Inclusion Criteria: * Completed the assigned dose regimen treatment time period in other clinical studies of mRNA-3705 or is eligible for early transition to this study because they missed more than 3 consecutive doses of study drug due to coronavirus disease 2019 (COVID-19) vaccination during Stud...

Countries:United StatesCanadaFranceNetherlandsSpainUnited Kingdom
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Frequently asked questions about mRNA-3705

What is mRNA-3705?

mRNA-3705 is an investigational RNA therapy developed by Moderna, Inc. for methylmalonic acidemia, a rare inherited metabolic disorder. It is in Phase 1 clinical development and is being studied in an extension trial evaluating long-term safety and clinical activity in participants previously enrolled in other mRNA-3705 studies.

What is mRNA-3705 used for in methylmalonic acidemia?

mRNA-3705 is being developed as a treatment for methylmalonic acidemia, a rare disease in which the body cannot properly process certain amino acids and fats. It is investigational and has not been approved for any use. The ongoing Phase 1 extension study is evaluating its long-term safety and clinical activity.

Who makes mRNA-3705?

mRNA-3705 is developed by Moderna, Inc., which trades on the Nasdaq under the ticker MRNA. Moderna is the sponsor of the clinical program studying the therapy in methylmalonic acidemia.

What phase is mRNA-3705 in?

mRNA-3705 is in Phase 1 clinical development. It is an investigational therapy and is not approved by the FDA. The program consists of one active, recruiting Phase 1 extension study with a planned enrollment of 56 participants.

What clinical trials is mRNA-3705 in?

mRNA-3705 is being studied in the Phase 1 extension trial NCT05295433, titled an extension study to evaluate the long-term safety and clinical activity of mRNA-3705 in participants previously enrolled in other clinical studies of mRNA-3705. The trial is recruiting in the United States, Canada, France, Netherlands, Spain, and the United Kingdom.