Programs & Pipeline
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. The first symptom is usually progressive vision loss in previously healthy children followed by personality changes, behavioral problems and slow learning. Seizures commonly appear within 2-4 years of vision loss. Seizures and psychosis can appear at any time during the course of disease. Progressive loss of motor functions and eventually, those affected become wheelchair-bound, are bedridden, and die prematurely.
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
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.