When Will Gene Therapy Come to the U.S.?
        
            By Susan Young, 
                MIT Technology Review
             | 09. 30. 2013
        
                    
                                    
                    
                                                                                                                                    
                                                                            
                              
    
  
  
    
  
          
  
      
    
            Though many gene therapies have been tested in patients around the world in hopes of curing hereditary diseases, few governments have approved their sale, and none has been approved in the United States. That could change in coming years as several therapies enter advanced trials.
A big step forward already came in November 2012, when the European Medicines Agency gave the Dutch biotech startup UniQure permission to sell its treatment. That approval came as a relief to many in the field, who had been waiting for a break in the clouds hanging over the technology since failed and fatal trials in the 1990s. “You see a resurgence in terms of investors, and in truth, a number of problems have been solved,” says Katherine High, a medical researcher at Children’s Hospital of Philadelphia, who is overseeing a late-stage clinical trial for a different gene therapy.
Still, experts say it is likely to be a few years before a treatment is approved in the U.S. With its European approval in hand, UniQure may have good chance of also getting the first U.S...
 
       
 
  
 
    
    
  
   
                        
                                                                                
                 
                                                    
                            
                                  
    
  
  
      Related Articles
    
  
          
  
  
  
  
  
  
      
            
                  
  
      
    
    
                
                        
                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                
                                                           By Megan Molteni and Anil Oza,  STAT | 10.07.2025
                                                        
     
    
    
            For two years, a panel of scientific experts, clinicians, and patient advocates had been hammering out ways to increase community engagement in National Institutes of Health-funded science. When they presented their road map to the NIH Director Jay Bhattacharya last...
 
       
 
 
  
      
    
    
                
                        
                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                
                                                           By Shoumita Dasgupta,  STAT | 10.03.2025
                                                        
     
    
    
            
President Trump and health secretary Robert F. Kennedy Jr. have characterized the rise in autism diagnoses in recent years as an epidemic requiring emergency intervention.
This approach is factually wrong: The broadening definition of autism and the improvement in diagnosis...
 
       
 
 
  
      
    
    
                
                        
                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                
                                                           By Abby McCloskey,  The Dallas Morning News | 10.10.2025
                                                        
     
    
    
            We Texans like to do things our way — leave some hide on the fence rather than stay corralled, as goes a line in Wallace O. Chariton’s Texas dictionary This Dog’ll Hunt. Lately, I’ve been wondering what this ethos...
 
       
 
 
  
      
    
    
                
                        
                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                
                                                           By Émile P. Torres,  Truthdig | 10.17.2025
                                                        
     
    
    
            The Internet philosopher Eliezer Yudkowsky has been predicting the end of the world for decades. In 1996, he confidently declared that the singularity — the moment at which computers become more “intelligent” than humanity — would happen in 2021, though...