AT-GTX-502
CLN3 Batten disease (Juvenile)
PreclinicalIND-enabling
Key Facts
Indication
CLN3 Batten disease (Juvenile)
Phase
Preclinical
Status
IND-enabling
Company
About Amicus Therapeutics
Amicus Therapeutics is a pioneer in the development of novel treatments for rare metabolic diseases, particularly lysosomal storage disorders. The company has successfully commercialized Galafold for Fabry disease and is advancing a next-generation pipeline that includes enzyme replacement therapies (ERTs) like Pombiliti + Opfolda for Pompe disease and gene therapies for conditions such as CLN6 Batten disease. Their strategy combines scientific innovation in protein engineering and gene therapy with a global commercial footprint to address significant unmet medical needs.
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