Gene Therapy

2 Drugs classified under this drug class

Gene Therapy. delandistrogene moxeparvovec 1.33 X 10^13.
Vial. Sol. for inf. 1.33 x 10¹³ vector genomes/ml  10-70 vials X 10 ml
For single-dose IV infusion only. Recommended dose is 1.33 × 10¹⁴ vector genomes per kilogram (vg/kg) of BW (or 10 mL/kg BW) for pts weighing less than 70 kg or 9.31 × 10¹⁵ vg total fixed dose for pts weighing 70 kg or greater. See full prescription info.
Tmt. of ambulatory ped. pts aged 4 to under 8 years with Duchenne musc. dystrophy (DMD) with a confirmed mutation in the DMD gene.
C/I: Hypersens. 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.

Gene Therapy. eladocagene exuparvovec 5.6 X10^11 vg/ml.
Solution for infusion 1X 0.5 ml
 1.8 × 10¹¹ vg delivered as four 0.08 mL (0.45 × 10¹¹ vg) infusions ( 2 per putamen)
Tmt. of pts aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L-amino acid decarboxylase (AADC) defic. with a sev. phenotype.
C/I:  hypersens.