Lysosomal Storage Disorders Pipeline, 2017

The deficiency of lysosomal enzymes cause lysosomal storage disorders (LSDs). It occurs due to mutations of genes that encode proteins, enzyme and related cofactors. There are approximately 50 types of LSDs. LSDs such as Gaucher disease, Fabry disease, Niemann-Pick disease, Pompe disease, Hunter syndrome, Mucopolysaccharidosis type III, Metachromatic leukodystrophy, Tay-Sachs disease and Batten disease affect different parts of body including brain, heart, skeleton, skin and central nervous system.

The LSDs pipeline has more than 70 drugs. In pipeline analysis, drugs are analyzed based on route of administration and molecule type. The pipeline is also analyzed based on monotherapy and combination therapy, and different clinical phases including Phase III, Phase II, Phase I and Preclinical stage.

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EGT-301 is under development by Esteve Quimica, S.A. in collaboration with University of Barcelona for the treatment of Hunter syndrome. The therapeutic candidate consists of an adeno-associated viral vector of serotype 9 (AAV9) containing the human Iduronate-2-sulfatase (I2S) transgene.  EGT-301 is designed to restore I2S functional deficiency. Preclinical study is also known as animal study. It is done before testing a drug in people to find out the toxicity profile of the drug. Preclinical study is of two types, including In vitro and In vivo. There are total 22 drugs in Preclinical stage.

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Pipeline analysis provides description about the key companies which are developing LSDs drugs. Some of the key players actively involved in the research and development are BioMarin Pharmaceutical, Inc., Ultragenyx Pharmaceutical Inc., Orphazyme ApS, Sanofi, Belrose Pharma, Inc., Fate Therapeutics, Inc., Amicus Therapeutics and Esteve Quimica, S.A.

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