Suzhou GenAssist’s GEN6050X Gains FDA Orphan Drug Status for Duchenne Muscular Dystrophy

China-based Suzhou GenAssist Therapeutic Co., Ltd. announced that its in vivo base editing therapy, GEN6050X, has been granted Orphan Drug Designation (ODD) by the US FDA for the treatment of Duchenne Muscular Dystrophy (DMD).

Therapy Overview

GEN6050X is an intravenous cytosine base editor designed for DMD patients amenable to exon 50 skipping. The therapy consists of ss.AAV9.oTAM, which expresses an oTAM base editor driven by a muscle-specific promoter, and ss.AAV9.hE50-sgRNA, carrying three copies of sgRNA and a human ACTG1 gene under a separate muscle-specific promoter.

Clinical Progress

Following FDA clearance of its IND application in March this year, GenAssist is currently preparing to initiate Phase I clinical trials in the US.-Fineline Info & Tech