Regenxbio will not move forward with the development of RGX-181 and RGX-381, gene therapy candidates for the treatment of late infantile Batten disease and its ocular (eye) manifestations. Clinical studies were ongoing in Brazil and the U.K. The few patients who have already received a one-time dose of…

A child with late infantile Batten disease treated with the gene therapy RGX-181 experienced a more than 80% reduction in seizure frequency over the following six months, without any serious side effects. That’s according to data from a single-patient study that were announced by RGX-181’s developer, Regenxbio, in…

Regenxbio plans to provide updates on its RGX-181 and RGX-381 gene therapy candidates for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease, in the second half of this year. The company is currently reviewing information requests from…

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 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…