mRNA-3705 / Moderna - LARVOL DELTA

Improved therapeutic efficacy in two mouse models of methylmalonic acidemia (MMA) using a second-generation mRNA therapy. (PubMed, Mol Genet Metab) - "mRNA-3705 produced 2.1-3.4-fold higher levels of hepatic MMUT protein expression than the first-generation drug product mRNA-3704 when given at an identical dose level, which resulted in greater and more sustained reductions in plasma methylmalonic acid. The data presented herein provide comprehensive preclinical pharmacology to support the clinical development of mRNA-3705."

Journal • Preclinical • Genetic Disorders • Metabolic Disorders • Ophthalmology • Pediatrics

Characterizing the mechanism of action for mRNA therapeutics for the treatment of propionic acidemia, methylmalonic acidemia, and phenylketonuria. (PubMed, Nat Commun) - "These preclinical data were then used to develop translational PK/PD models, which were scaled allometrically to humans to predict starting doses for first-in-human clinical studies for each disease. The predicted first-in-human doses for mRNA-3927, mRNA-3705, and mRNA-3210 were determined to be 0.3, 0.1, and 0.4 mg/kg, respectively."

Journal • Genetic Disorders • Metabolic Disorders • Ophthalmology • Phenylketonuria • Rare Diseases

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 (clinicaltrials.gov) - P1/2 | N=63 | Recruiting | Sponsor: ModernaTX, Inc. | N=33 ➔ 63

Enrollment change • Genetic Disorders • Metabolic Disorders • Ophthalmology

A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia (clinicaltrials.gov) - P1/2 | N=63 | Recruiting | Sponsor: ModernaTX, Inc. | N=33 ➔ 63

Enrollment change • Genetic Disorders • Metabolic Disorders • Ophthalmology

A clinical study to evaluate mRNA-3705 in Patients with MMA (clinicaltrialsregister.eu) - P1/2 | N=33 | Sponsor: ModernaTX, Inc.

New P1/2 trial • Genetic Disorders • Metabolic Disorders • Ophthalmology

An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in the mRNA-3705-P101 Study (clinicaltrials.gov) - P1/2 | N=33 | Recruiting | Sponsor: ModernaTX, Inc.

New P1/2 trial • Genetic Disorders • Metabolic Disorders • Ophthalmology