Vertex, CRISPR’s Casgevy Highlights Complex Path to Gene Therapy Profitability
        
            By Annalee Armstrong, 
                BioSpace
             | 09. 11. 2024
        
                    
                                    
                    
                                                                                                                                    
                                                                            
                              
    
  
  
    
  
          
  
      
    
            Complex gene therapies are starting to hit the market but all have faced the same reality: a tepid reception from the healthcare system and a cloudy path to profitability.
It can take about a year for a patient to go through the preparations needed to receive a gene therapy treatment, Jen Klarer, managing partner at The Dedham Group, told BioSpace. “I expected there to be a slow time from approval to treating the first patient.”
CRISPR Therapeutics and Vertex Pharmaceuticals are a great example. The companies made history last year when they achieved the first approval of a CRISPR-Cas9 gene editing therapy for sickle cell disease in December 2023. But when the companies reported 2024 second quarter earnings a few months later, the sobering—albeit expected—reality set in: no patients had been treated.
The fuller picture of Casgevy’s path to profitability is even more complicated. During Vertex’s second quarter earnings call, the company explained that 20 patients are “in the funnel” to receive Casgevy. That means that the patients have signed up and gone through the cell collection process. This...
 
       
 
  
 
    
    
  
   
                        
                                                                                
                 
                                                    
                            
                                  
    
  
  
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[OHSU News/Christine Torres Hicks]
On September 30th, a team of 21 scientists from Oregon Health & Science University (OHSU) published a significant paper in Nature Communications, with a scientifically accurate but, to many, somewhat abstruse headline:
Induction of experimental cell division to generate cells with reduced chromosome ploidy
The lead authors were Shoukhrat Mitalipov, recently described here as “a push-the-envelope biologist,” and his long-term colleague Paula Amato. (Recall that in July the pair had co-published with...
 
       
 
 
  
      
    
    
                
                        
                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                
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                                                           By Rob Stein,  NPR | 09.30.2025
                                                        
     
    
    
            Scientists have created human eggs containing genes from adult skin cells, a step that someday could help women who are infertile or gay couples have babies with their own genes but would also raise difficult ethical, social and legal issues...