Batten Disease Information | Amicus Therapeutics

About Batten Disease

Batten disease is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. In these diseases, a defect in a specific gene triggers a cascade of problems that interferes with a cell’s ability to recycle certain molecules. The disease has several forms with similar features and symptoms but vary in severity and age of onset. Each form is caused by a mutation in a different gene.

Most forms of Batten disease/NCLs usually begin during childhood.

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Batten Disease Clinical Trials

Batten CLN6 Gene Therapy: Phase 1/2 Study

More Information:
www.clinicaltrials.gov: NCT02725580

Batten CLN3 Gene Therapy: Phase 1/2 Study

More Information:
www.clinicaltrials.gov: NCT03770572

Preclinical Gene Therapy Program

Science and Technology

In 2018, Amicus Therapeutics acquired a gene therapy portfolio of ten clinical and preclinical stage adeno associated virus (AAV) programs in lysosomal disorders through a license agreement with The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital (NCH), including several forms of Batten disease. Active preclinical programs are underway for CLN -8, -3, and -1 Batten disease.