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Can FDA's Breakthrough Program Finally Midwife Gene Therapy?

 
• By Bridget Silverman

The often-troubled field is showing promise, and the extra resources that breakthrough designation will bring to two candidates could be what gene therapy needs to move from the theoretical to the marketable.

DNA

The field of gene therapy is heating up again, with candidates advancing toward late-stage trials and eventual submission to FDA. The extra resources and attention provided by FDA's breakthrough therapy program could help nurture the whole field through the inevitable regulatory challenges that the novel and highly complex products will bring.

Spark Therapeutics Inc. is at the forefront of gene therapy breakthrough designations (BTDs), with two of the five BTDs...

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