Can FDA's Breakthrough Program Finally Midwife Gene Therapy?
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.
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Two manufacturing control assays have taken longer to develop than previously thought, putting back completion of a BLA submission to early 2017 for US company Spark Therapeutics, while further progress has been made in the company’s early-stage hemophilia gene therapy program.
A death was reported in the company's early-stage gene therapy trial. It has not been determined yet whether the death was related to the drug and what the next steps for the company are.
Keeping Track: Novel Orphan Drugs Amondys 45, Nulibry Clear US FDA; First-Line NSCLC Claim For Libtayo
The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker