DELANDISTROGENE MOXEPARVOVEC-ROKL

12.1 Mechanism of Action ELEVIDYS is the recombinant gene therapy product that is comprised of a non-replicating, recombinant, adeno-associated virus (AAV) serotype rh74 (AAVrh74) capsid and a single-stranded DNA expression cassette flanked by inverted terminal repeats (ITRs) derived from AAV2. The cassette contains: 1) an MHCK7 gene regulatory component comprising a creatine kinase 7 promoter and an α-myosin heavy chain enhancer, and 2) the DNA transgene encoding the engineered micro-dystrophin protein. Vector/Capsid: Clinical and nonclinical studies have demonstrated AAVrh74 serotype transduction in skeletal muscle cells. Additionally, in nonclinical studies, AAVrh74 serotype transduction has been demonstrated in cardiac and diaphragm muscle cells. Promoter: The MHCK7 promoter/enhancer drives transgene expression and has been shown in animal models to drive transgenic micro-dystrophin protein expression predominantly in skeletal muscle (including diaphragm) and cardiac muscle. In cli

Contraindications

• • 4 CONTRAINDICATIONS ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9
• • including a deletion of any portion or the entirety of these exons
• • in the DMD gene [see Warnings and Precautions ( 5.5 )] . ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9
• • including a deletion of any portion or the entirety of these exons
• • in the DMD gene. ( 4 )