Alice Melao,  —

FDA Grants PLX-100 Orphan Drug Designation to Treat Batten Disease

Polaryx Therapeutics’ investigative drug PLX-100 has been granted orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of neuronal ceroid lipofuscinosis (NCL), also known as Batten disease. This follows the previous announcement of ODD status granted to PLX-200, another drug being tested…

CLN5 Protein Loss in Batten Disease Led to Fewer Functional Neurons in Mouse Study

Researchers studying the loss of the CLN5 protein, which causes Batten disease, found that it leads to the reduced production of new fully functional neurons. It also increased neuronal inflammation in mouse models of the neurodegenerative disorder. The study, “Loss of CLN5 causes altered neurogenesis in a childhood neurodegenerative…

ABO-201 Improves Inflammation, Movement in Mice with Juvenile Batten Disease, Study Shows

Abeona Therapeutics‘ ABO-201 reduced nerve inflammation and improved movement in mice with juvenile Batten disease (JBD), according to a preclinical trial study. Scientists at Abeona designed ABO-201 to overcome the effects of the CLN3 genetic mutation that is responsible for the disease. A single injection led to the correct form of the gene being delivered…