Arthur Tzianabos, Homology Medicines CEO
Homology cleared to resume gene therapy trial for rare metabolic disease after FDA lifts hold
A few months after getting the stop sign from the FDA, Homology Medicines is now able to restart its Phase II trial of a gene therapy for the rare metabolic disease phenylketonuria, or PKU.
The FDA had shown concerns that Homology’s gene therapy, known as HMI-102, might lead to liver damage issues. In announcing the hold had been lifted, Homology said there were no hospitalizations required pertaining to the elevated liver function tests seen in the trial of adults.
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