AT-GTX-501

Unassigned

New Medicines

CLN6 Batten disease (syndrome) - first-line

Information

Advanced therapy medicinal product (ATMP)
Amicus Therapeutics
Amicus Therapeutics

Development and Regulatory status

None
None
Phase II Clinical Trials

Category

Gene therapy
Batten disease is caused by a mutation in the CLN3 gene at gene locus 16p12.1. Of the nine clinical variants (CLN1-CLN9), six have been genetically identified (as well as the CLN3 gene, these include CLN1, CLN2, CLN5, CLN6 and CLN8). It occurs in about 1 to 5 cases per 100,000 generally. There is often rapid deterioration of vision and a slower, but progressive deterioration of intellect. Seizures and psychosis develop later, and there may be features of Parkinson disease [1].
CLN6 Batten disease (syndrome) - first-line
Intrathecal