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mRNA-3705

Phase 1

Methylmalonic Acidemia | Small molecule | Rare Disease |Moderna, Inc.|Last Updated: Dec 23, 2025

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment56

FDA Designations

No designations recorded

Clinical trial landscape

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 used for?

mRNA-3705 is an investigational therapy being developed for the treatment of methylmalonic acidemia, a rare inherited metabolic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who makes mRNA-3705?

mRNA-3705 is being developed by Moderna, Inc., a biotechnology company traded on the Nasdaq under the ticker symbol MRNA. The company is conducting clinical trials to evaluate the safety and clinical activity of this investigational therapy.

What phase is mRNA-3705 in?

mRNA-3705 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The ongoing Phase 1 trial is recruiting participants to evaluate long-term safety and clinical activity.

What clinical trials is mRNA-3705 in?

mRNA-3705 is being studied in one clinical trial with the identifier NCT05295433. This is a Phase 1 extension study evaluating long-term safety and clinical activity in participants previously enrolled in other clinical studies of mRNA-3705. The trial is recruiting up to 56 participants with methylmalonic acidemia.

Is mRNA-3705 the same as methylmalonic acidemia treatment?

No, mRNA-3705 is not the same as methylmalonic acidemia. Methylmalonic acidemia is the disease condition that mRNA-3705 is being developed to treat. mRNA-3705 is an investigational drug candidate, not a disease.