Moderna’s mRNA-3705: Investigational Therapeutic for Methylmalonic Acidemia (MMA)
Moderna Therapeutics, a biotechnology company pioneering the development of mRNA-based medicines, is advancing a novel therapeutic targeting methylmalonic acidemia (MMA) due to methylmalonic-CoA mutase (MUT) deficiency. The investigational therapeutic is known as mRNA-3705, which is designed to instruct the body to restore the missing or dysfunctional proteins that cause MMA.
What is MMA?
Methylmalonic acidemia (MMA) is an inherited metabolic disorder affecting the body’s ability to process certain proteins and fats. The condition is most commonly caused by a deficiency in the mitochondrial enzyme MUT, which is responsible for breaking down certain amino acids and fatty acids. When this enzyme is deficient, toxic acids accumulate in the body, leading to metabolic crises and progression into multi-organ disease.
The symptoms of MMA can range from mild to severe and usually present in infancy or early childhood. They may include poor feeding, vomiting, dehydration, lethargy, developmental delay, seizures, and coma. Currently, liver or combined liver and kidney transplants are the only effective treatments available for MMA.
Moderna’s mRNA-3705: An Investigational Therapeutic for MMA
mRNA-3705 is a promising new treatment for MMA under investigation in a Phase 1/2 Landmark study. The adaptive, open-label study is designed to evaluate the safety and tolerability of the investigational therapeutic administered via intravenous infusion in patients one year and older with isolated MMA due to methylmalonyl-CoA mutase (hMUT) deficiency.
Moderna’s mRNA-3705 consists of mRNA encoding human MUT, encapsulated within the company’s proprietary lipid nanoparticle. The mRNA is designed to instruct the body’s own cells to produce functional MUT enzyme, which can then break down the toxic acids that accumulate in MMA patients.
The START Pilot Program: Accelerating the Development of Novel Treatments for Rare Diseases
In September 2023, the U.S. Food and Drug Administration (FDA) initiated the START pilot program to accelerate the development of novel treatments addressing unmet medical needs in rare diseases. The program aims to expedite the clinical development of novel therapies by providing rapid, ad hoc FDA interactions that support study design, patient population, and statistical methods beyond standard formal meetings.
The START pilot program will initially select up to seven novel treatments, including three by the Center for Drug Evaluation and Research and four by the Center for Biologics Evaluation and Research. Moderna’s mRNA-3705 was one of the selected programs, which is expected to enhance communication with the FDA and accelerate the development process as the company prepares for a pivotal study initiation in 2024.
Conclusion
MMA is a rare and debilitating metabolic disorder that severely impacts patients’ quality of life. Currently, there are limited treatments available, and liver or combined liver and kidney transplants remain the only effective therapies. Moderna’s mRNA-3705 is a new investigational therapeutic designed to target MMA by instructing the body to restore the missing or dysfunctional MUT enzyme. The potential approval of mRNA-3705 could offer patients and families new hope for a better quality of life and improved outcomes.
Originally Post From https://www.tradingview.com/news/benzinga:ba1344a1d094b:0-fda-selects-moderna-s-rare-disease-candidate-for-accelerated-development-program/
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