Patricia Inácio, PhD,  science writer—

Abeona Therapeutics’ one-time gene therapy candidate ABO-202 has been granted fast track designation by the U.S. Food and Drug Administration (FDA) for treating children with infantile Batten disease, also known as CLN1 disease. A fast track designation by the FDA is intended to accelerate the review of new compounds that potentially could fill…

The U.S. Food and Drug Administration has granted rare pediatric disease designation to Regenxbio’s one-time gene therapy RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease. Regenxbio plans to submit an investigational new drug (IND) to the FDA…

The activation of a recycling pathway, a process called autophagy, which leads to large storage vesicles in muscle cells, is a feature of all forms of juvenile Batten disease, a small study suggests. The study, “Autophagic vacuolar myopathy is a common feature of CLN3 disease,” was published in the …

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Regenxbio’s one-time gene therapy RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease. Regenxbio expects to submit an Investigational New Drug (IND) application to the FDA…

Regenxbio is developing a novel, one-time gene therapy, called RGX-181, for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease. The company expects to submit an investigational new drug (IND) application to the U.S. Food…

Husbandry conditions of mouse models of CLN3-Batten disease may influence research outcomes, such as potential disease biomarkers, a new study reports. Researchers highlight the need to standardize the environment where animals are maintained to identify robust biomarkers that might help develop therapeutics for Batten disease subtypes. The study,…

The expression of two genes associated with neuronal ceroid lipofuscinosis, better known as Batten disease, was restricted to certain cells of the central nervous system in a fruit fly research model. Namely, these genes are mainly expressed in cells that form the blood brain barrier — a membrane that protects the…

Long-term follow-up of two siblings with Batten disease unveiled symptoms of slowly progressive motor deterioration, persistent photosensitivity, and seizures without dementia, according to a case report. The case report, “Long-term follow-up of two siblings with adult-onset neuronal ceroid lipofuscinosis, Kufs type A,” was published in the journal Epileptic Disorders.

Loss of a protein called CLN7 causes levels of multiple soluble lysosomal proteins to drop, contributing to the development and progression of Batten disease, a mouse model shows. The study, “Loss of CLN7 results in depletion of soluble lysosomal proteins and impaired mTOR reactivation,” was published in the journal…

The U.S. Food and Drug Administration (FDA) has granted a rare pediatric disease designation to Abeona Therapeutics’ investigational ABO-202 program being developed for Batten disease caused by mutations in the CLN1 gene, also known as infantile neuronal ceroid lipofuscinosis. ABO-202 is an adeno-associated virus (AAV)-based gene therapy (AAV-CLN1) where weakened…