José Lopes, PhD,  managing science editor—

The U.S. Food and Drug Administration (FDA) accepted Abeona Therapeutics’ investigational new drug application for its one-time gene therapy candidate ABO-202 for children with infantile Batten disease, also known as CLN1 disease. The FDA’s positive decision clears the company to start a global Phase 1/2 trial,…

Treatment with Brineura (cerliponase alfa) reduces decline in motor and language function in children with ceroid lipofuscinosis type 2 (CLN2), a subtype of Batten disease, new results from an extension Phase 1/2 trial show. The findings are in, “Study of Intraventricular Cerliponase Alfa for CLN2 Disease,” published…

Gene therapy is able to reduce toxic protein aggregates in a zebrafish model of one form of adult-onset Batten disease, a new study from China shows. The study, “Gene Therapy of Adult Neuronal Ceroid Lipofuscinoses with CRISPR/Cas9 in Zebrafish,” appeared in the journal Human Gene Therapy. The study’s…

Stem cell models of lysosomal storage disorders (LSDs), including Batten disease (BD), could significantly add to knowledge of disease biology and help develop improved therapies, a new review highlights. The study, titled “Induced pluripotent stem cell models…

Lopid (gemfibrozil), an FDA-approved lipid-lowering drug, was found to improve life expectancy and motor function in mice with late infantile neuronal ceroid lipofuscinosis (late infantile NCL, or LINCL), which belongs to a group of disorders collectively referred to as Batten disease. The study, “Gemfibrozil, Food and Drug Administration-approved lipid-lowering drug,…