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.
Of the handful of companies that have launched new gene therapies during the last two years, Sarepta stands out for...
