Monosaccharide substrate replacement therapy for an ultra-rare congenital disorder of glycosylation


Targeting the Glycosylation Defects Associated with Congenital Disorder of Glycosylation IIc

CERC-803 is an ultra-pure, oral formulation of L-fucose currently in development for the treatment of Leukocyte Adhesion Deficiency Type II (LADII), also known as SLC35C1-CDG.

of CERC-803

  • High purity and quality
  • Ease of use and administration
  • Documented efficacy and safety in published literature
  • Orphan Drug and Rare Pediatric Disease designation
  • Potential for insurer reimbursement

About CDG-IIc

Currently there is no approved product for the treatment CDG-IIc.

CDG-IIc 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.

We believe that substrate replacement with CERC-803 in patients with CDG-IIc is expected to reverse the hypofucosylation defects found in this patient population.