Complete List of Gene therapy product Medicines
                
                
                
                    
                    
                        Brand: CASGEVY | 
                        Strength: 4 M-13M CD34 cells/mL | 
                        Form: Solution For Injection
                    
                    
                    
                        Class: | Gene therapy product  
Use: | Treatment of beta-thalassemia in patients who require regular red blood cell transfusions and have a specific genetic mutation  
Adult dose: | Not applicable; pr...
                    
                    
                 
                
                
                    
                    
                        Brand: ZOLGENSMA | 
                        Strength: 2 x 10^13 vector genomes/mL | 
                        Form: Solution For Infusion
                    
                    
                    
                        Class:|Gene therapy product  
Use:|Treatment of spinal muscular atrophy (SMA) in pediatric patients less than 2 years of age with bi-allelic mutations in the SMN1 gene  
Adult dose:|Not applicable  
P...
                    
                    
                 
                
                
                    
                    
                        Brand: VYJUVEK | 
                        Strength: 5x10^9 PFU/ml | 
                        Form: kit
                    
                    
                    
                        Class: | Gene therapy product  
Use: | Treatment of adult patients with dystrophic epidermolysis bullosa (DEB) due to mutations in the COL7A1 gene  
Adult dose: | 5 x 10^9 PFU/ml administered as a sin...
                    
                    
                 
                
                
                    
                    
                        Brand: Elevidys | 
                        Strength: 10 ml | 
                        Form: Solution For Infusion
                    
                    
                    
                        Class: | Gene therapy product  
Use: | Treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the dystrophin gene  
Adult dose: | Not applicable; primarily indicated f...