Homology joins BioMarin in halting gene therapy studies for rare metabolic disease
It was a tough week for companies, patients and families hoping a gene therapy for the rare metabolic disease phenylketonuria is on its way.
On Friday afternoon, after BioMarin had already announced its program would be on hold for at least “several quarters,” Homology Medicines disclosed that the FDA had also halted trials for its phenylketonuria gene therapy.
The timing partly reflects the greater scrutiny gene therapy has come under at the FDA over the last year. But despite similarities between the two programs, the agency’s concerns appear to be unrelated, reflecting distinct safety concerns with the technology.
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