CDX-6417

CDX-6417 is a gene therapy candidate under development by Codexis for Pompe disease, a rare lysosomal storage disorder. It is part of Codexis's gene therapy pipeline, as referenced in their 2021-2022 biotherapeutics pipeline summary. The therapy likely utilizes an engineered gene to address the enzyme deficiency characteristic of Pompe disease, but the specific molecular mechanism or gene construct has not been publicly detailed[3]. Developmental information points to CDX-6417 as an investigational gene therapy aimed at restoring the function or activity of acid alpha-glucosidase enzyme which is deficient in Pompe disease.