Date: 2016-10-07
Type of information: Publication of results in a medical journal
phase: preclinical
Announcement: publication of results in the Journal of Neuroscience
Company: Abeona Therapeutics (USA - NY)
Product: ABO-201 (AAV-CLN3)
Action
mechanism: gene therapy. ABO-201 (AAV-CLN3) is an AAV-based gene therapy which has shown promising preclinical efficacy in delivery of a normal copy of the defective CLN3 gene to cells of the central nervous system with the aim of reversing the effects of the genetic errors that cause for juvenile neuronal ceroid lipofuscinosis (JNCL) (also known as juvenile Batten disease).
Disease: Batten disease
Therapeutic area: Rare diseases - Genetic diseases - Neurological diseases - Neurodegenerative diseases
Country:
Trial details:
Latest
news: * On October 7, 2016, Abeona Therapeutics announced that preclinical data supporting clinical trials for ABO-201 (AAV-CLN3), the AAV-based single intravenous gene therapy program for juvenile Batten disease, (juvenile neuronal ceroid lipofuscinosis, JNCL), were published in the September issue of the Journal of Neuroscience. Researchers concluded that a single intravenous injection "led to widespread virus biodistribution in the brain, spinal cord, and eye" that was capable of "improving motor function, attenuating microglial and astrocyte activation, and reducing lysosomal pathology, all hallmarks of JNCL" at an age when significant lysosomal pathology had already manifested.
