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Gene Therapy Patient Pipeline Builds, But Slowly, In Q2

 
• By Jessica Merrill

Bluebird, Vertex and Sarepta are starting to see some commercial gene therapy traction and expect momentum to pick up in the second half, while hemophilia remains a challenging area. 

gene therapy
gene therapies slowly picked up momentum in Q2 • Source: Shutterstock

Some gene therapy manufacturers, like bluebird bio, Vertex Pharmaceuticals Incorporated and Sarepta Therapeutics, Inc., are beginning to see a ramp up in their commercial businesses and reported an expanding pipeline of patients beginning the process to start treatment in the second quarter. But the number of patients treated with new gene therapies remains limited and companies focused on hemophilia, including CSL Limited and BioMarin Pharmaceutical Inc., have continued to experience slow uptake.

Key Takeaways
  • With several gene therapy launches under way, manufacturers are gaining valuable commercial experience.
  • Sarepta stands out as an early commercial success with Elevidys for DMD, while...

Of the handful of companies that have launched new gene therapies during the last two years, Sarepta stands out for its early commercial win with Elevidys (delandistrogene moxeparvovec), a gene...

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