AVTX-803 is a therapeutic dose of L-fucose currently in development for the treatment of leukocyte adhesion deficiency type II (LAD II), also known as SLC35C1-CDG.
CDG represent a large group of rare inherited diseases affecting glycosylation, the process of adding sugar-building blocks (glycans) to proteins or lipids. CDG are typically present from infancy and can affect any part of the body, with symptoms that range from mild to potentially life-threatening. Individual CDG are caused by mutations in a specific gene.
LAD II is caused by loss of function mutations in the SLC35C1 gene encoding a GDP-fucose transporter found in the Golgi apparatus, where it acts as the donor substrate for protein fucosylation. Currently, there is no approved product for the treatment of LAD II.
AVTX-803 is an oral formulation of L-fucose that replenishes critical metabolic intermediates, reduced or absent due to a genetic variant, to support glycoprotein synthesis, maintenance, and function.