Programs & Pipeline ❯ Batten Disease
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.
Batten Disease Clinical Trials
Batten CLN6 Gene Therapy: Phase 1/2 Study
Batten CLN3 Gene Therapy: Phase 1/2 Study
Preclinical Gene Therapy Program
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 -3 and -1 Batten disease.