Exciting Progress in Treating Rare Metabolic Disorders

Moderna is working on therapy for rare aciduria

Great news for those affected by rare metabolic disorders! Moderna’s new investigational treatment for methylmalonic acidemia (MMA), known as mRNA-3705, has been selected by the U.S. FDA for the START pilot program. This initiative aims to accelerate the development of promising therapies for rare diseases. MMA is a severe condition caused by an enzyme deficiency leading to toxic acid buildup in the body. Moderna’s innovative mRNA-3705 works by instructing the body to produce the missing enzyme, potentially offering a groundbreaking treatment for this life-threatening disorder.

For more details, read the full announcement here.

Promising results show that mRNA treatments are safe for Propionic Acidemia

Propionic acidemia (PA) is a rare and severe inherited metabolic disorder caused by a deficiency in the liver enzyme propionyl-coenzyme A carboxylase (PCC). This deficiency leads to the accumulation of toxic compounds in the body, resulting in life-threatening metabolic decompensation events (MDEs) and various multisystemic complications, including neurological issues, heart problems, and growth retardation. Traditionally, there have been no effective therapies targeting the root cause of PA.

However, a groundbreaking first-in-human phase 1/2 clinical trial for mRNA-3927, a novel therapeutic mRNA drug developed by Moderna, has shown promising results. This investigational therapy is designed to restore the deficient enzyme activity in patients with PA by encoding for the PCCA and PCCB subunit proteins of the PCC enzyme.

Key Findings from the Study

1. Restored Enzyme Activity: The trial demonstrated that mRNA-3927 successfully restored enzyme activity in patients, addressing the core issue of the disease.
2. Safety and Tolerability: The therapy was well tolerated with no dose-limiting toxicities observed. While some participants experienced treatment-emergent adverse events (TEAEs), these were mostly mild and related to the initial doses.
3. Clinical Benefits: There was a significant reduction in the frequency of MDEs. Participants who received higher doses of mRNA-3927 experienced no MDEs, highlighting the dose-dependent efficacy of the treatment.
4. Long-Term Potential: The study’s interim data indicate that mRNA-3927 could offer substantial clinical benefits for patients with PA, providing a much-needed therapeutic option where none previously existed.

This innovative approach using mRNA therapy not only holds promise for PA but also paves the way for treating other metabolic disorders. By leveraging mRNA technology, researchers can potentially correct enzyme deficiencies at their source, offering new hope for patients with rare and debilitating conditions.

For more details, read the full study here.

Watch a quick video here.