SMA Newsroom

Jul 20, 2023

Community statement: BIOCAD

Biocad, a Russian biotechnology company, shares a community statement on its gene therapy product, ANB-004, which is being developed to treat SMA.

It is designed to address the genetic root cause of SMA by inserting a working copy of the SMN1 gene into patients’ cells, with the aim of halting disease progression.

The trial of ANB-004, named Bluebell, is an open-label, non-comparative clinical study to evaluate the safety and efficacy of the product, after a single intravenous administration of escalating doses in children who live with SMA.

Find out more by reading the community statement.