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Mucopolysaccharidosis (Morquio Syndrome) Therapeutics Pipeline Analysis, 2017 - Clinical Trials & Results, Patent and Other Developments
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The prevalence of mucopolysaccharidosis varies based on its types. According to the Orphanet, one person in a population of 100,000 is affected with mucopolysaccharidosis type I or Hurler syndrome. According to the Journal of Applied Genetics, a survey was conducted between 1970 to 2010, which found that the prevalence of mucopolysaccharidosis type III was 0.86 out of 100,000 live births. Additionally, the prevalence in Polish population was estimated to be 1.81 out of 100,000 individuals. The growth in the pipeline of mucopolysaccharidosis therapeutics is attributed to increasing technical advancements in molecular biology and genetics. Gene therapies, cell therapies, immunotherapies and microRNA are the first line of treatment of mucopolysaccharidosis. The funding from rare disease organizations is also one of the factors, which is expected to increase the collaboration between companies, associations and institutes for the development of therapies for the treatment of mucopolysaccharidosis.
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In May 2017, the gene therapy, SB-913 from Sangamo Therapeutics Inc., was granted a rare pediatric disease designation by USFDA. In January 2017, another gene therapy from Sangamo Therapeutics Inc., SB-318, was designated as orphan drug by the USFDA.
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Some of the companies having a pipeline of Mucopolysaccharidosis therapeutics include, Ultragenyx Pharmaceutical Inc., Shire plc, BioMarin Pharmaceutical Inc., ArmaGen Inc., Genzyme Inc., JCR Pharmaceuticals Co., Ltd., Green Cross Corporation, Alexion Pharmaceuticals Inc., Concert Pharmaceuticals Inc.
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