2019

GT Gain Therapeutics SA

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TARGET GENE

IDUA

TARGET ENZYME

Alpha-L-iduronidase

PRIMARY

STORAGE

Dermatan sulfate & heparan sulfate

SECONDARY

STORAGE

Proinflammatory cytokines (TNF-alpha and IL-1beta) and matrix metalloproteinases

EPIDEMIOLOGY

1 : 200-500,000

UNMET MEDICAL NEED

Neurological manifestations, ‘hard-to-treat’ organs (bone, heart valves, and cornea), inflammation, mortality and morbidity risk associated with HSCT, weekly 4 h infusions and risk of antibody development as well as drug-related adverse reactions with ERT

DEVELOPMENT

STAGE

Discovery

In vivo