Gene therapies hold promise for treating a wide range of diseases. Innovative companies such as bluebird bio and BioMarin are at the forefront of clinical development. Yet, firms face numerous challenges, in all phases, from pre-clinical through Phase 3. Furthermore, what is sometimes underappreciated is the fact that even when gene therapies obtain marketing authorization, they’re still often hampered by substantial delays between their date of approval and the time they launch.
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