Mucopolysaccharidosis (MPS) Treatment Market Witnesses Research and Development of New Therapies

 

Mucopolysaccharidosis refers to a group of inherited conditions that are characterized by the inability of the body to properly breakdown mucopolysaccharides (long chains of sugar molecules that are found throughout the body). Seven distinct clinical types and numerous subtypes of the mucopolysaccharidoses have been identified. Hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy with a recombinant form of human IDUA administered intravenously are current disease modifying therapies for MPS.

The mucopolysaccharidosis (MPS) treatment market is witnessing R&D of new therapies. For instance, in February 2021, Orchard Therapeutics, a gene therapy company, announced new data from several of its hematopoietic stem cell (HSC) gene therapies in development for neurodegenerative disorders at the 17th Annual WORLDSymposium. The presentation included interim data on multiple clinical outcomes for OTL-203 in MPS-I, encouraging preliminary biomarker data for OTL-201 in MPS-IIIA and natural history data in MLD supporting future patient identification and market access.

Physical symptoms of MPS generally include coarse facial features, dwarfism, dysplasia, and other skeletal irregularities. Coarse facial features include, a flat nasal bridge, thick lips, and enlarged mouth and tongue. Mucopolysaccharidosis type 1 (MPS 1), a rare lysosomal storage disease belonging to the group of mucopolysaccharidosis, is characterized by enlarged head, lips, cheeks, tongue, and nose; enlarged vocal cords, resulting in a deep voice; and frequent upper respiratory infections. In December 2020, Sigilon Therapeutics, Inc., one of the major players in the mucopolysaccharidosis (MPS) treatment market, received the U.S. Food and Drug Administration’s Orphan Drug designation to SIG-005 for the treatment of mucopolysaccharidosis type I (MPS-1).

In December 2020, Regenxbio, another player in the mucopolysaccharidosis (MPS) treatment market, dosed  announced the first patient with RGX-111, an investigational one-time gene therapy designed to deliver the a-l-iduronidase (IDUA) gene directly to the central nervous system (CNS) using the NAV AAV9 vector, in a phase I/II trial for the treatment of Mucopolysaccharidosis Type I (MPS I). Similarly, in November 2020, Paradigm Biopharmaceuticals Ltd. initiated a Phase II clinical trial of subcutaneous injectable Pentosan Polysulphate Sodium (iPPS), in patients with the ultra-rare orphan disease Mucopolysaccharidosis Type 1 (MPS-1).

However, limitations of various therapies is expected to hinder growth of the mucopolysaccharidosis (MPS) treatment market. For instance, intravenous enzyme therapy does not treat the central nervous system manifestations of MPS I, whereas HSCT can be associated with clinically significant morbidity and mortality.