Moderna, Inc., a clinical-stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, announced today that the first patient had been enrolled in the Phase 1/2 study evaluating the safety and tolerability of mRNA-3705, its investigational mRNA therapeutic for methylmalonic acidemia (MMA), which is administered via intravenous infusion in patients with isolated MMA due to MUT deficiency. mRNA-3705 is Moderna’s second rare disease program to enter clinical studies

Source: Moderna, Inc.

Methylmalonic acidemia or aciduria (MMA) is a genetically heterogeneous group of disorders caused by faulty amino acid (isoleucine, methionine, threonine, or valine) metabolism odd-chain fatty acids, or cholesterol esters. The accumulation of methylmalonic acid in all body fluids and tissues is a biochemical characteristic of MMA. Most MMA patients present with clinical signs and symptoms within the first few days or weeks of life. The overall prognosis is generally poor, with intermittent life-threatening acute metabolic decompensations precipitated by catabolic factors and significant long-term squeal, including neurologic and renal impairment.

• Estimates of incidence in Western populations range from 1:48,000 to 1:61,000 births for MMA and from 1:50,000 to 1:500,000 deliveries for PA. The overall incidence is believed to be ~ 1: 50,000 for isolated MMA and ~ 1:100’000 to 150,000 for PA.

However, the current Methylmalonic Acidemia (MMA) treatment market share, market uptake, attribute analysis concerning the most potential emerging therapies (mRNA-3705, hLB-001, BBP-671, and HST5040) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Methylmalonic Acidemia (MMA) treatment. The key companies in the advanced development stage are ModernaTX, Inc., LogicBio Therapeutics, Inc., CoA Therapeutics, Inc., and HemoShear Therapeutics targeting MMA.

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook report on Methylmalonic Acidemia (MMA) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2020 to 2030.

Thelansis specializes in pharmaceutical market research and market Insight Report Company, published reports across the therapeutic area, including rare / ultra-rare and mainstream indication. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.

Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the primary focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making

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