Mucopolysaccharidosis Disorders Drug Development Pipeline Review 2018

This report provides an overview of the pipeline landscape for Mucopolysaccharidosis Disorders, a group of inherited lysosomal storage disorders. It provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for mucopolysaccharidosis I (MPS I) (Hurler syndrome), mucopolysaccharidosis II (MPS II) (Hunter syndrome) and mucopolysaccharidosis III (MPS III) (Sanfilippo syndrome), and features dormant and discontinued products.

MPS I is caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. There are 18 products in development for this indication.

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MPS II is a condition that affects many different parts of the body and occurs almost exclusively in males. Signs and symptoms include claw-like hands, protruding tongue, changing facial features, including thickening of the lips, tongue and nostrils and delayed development. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy. There are 15 products in development for this indication.

MPS III is caused by an absence or malfunctioning of GAGs. Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT). There are 18 products in development for this indication.

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Companies Mentioned:

Abeona Therapeutics Inc

AngioChem Inc

ArmaGen Inc

Axcentua Pharmaceuticals AB

BioMarin Pharmaceutical Inc

CRISPR Therapeutics

Denali Therapeutics Inc

Eloxx Pharmaceuticals Inc

GC Pharma

Table of Contents:

1 Table of Contents

2 Introduction 8

3 Therapeutics Development 9

4 Therapeutics Assessment 23

5 Companies Involved in Therapeutics Development 40

6 Dormant Projects 54

7 Discontinued Products 57

8 Product Development Milestones 58

9 Appendix 97

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Posted by on Monday May 20 2019, 6:45 AM EDT. All trademarks acknowledged. Filed under Business & Financial Services, Business Services, Diagnostics, Featured Press Release, Healthcare, Life Sciences, Pharmaceuticals. Comments and Trackbacks closed. Follow responses: RSS 2.0

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