BioMarin’s PKU Gene Therapy Clinical Hold To Last ‘Several Quarters’
Hemophilia A Candidate Unaffected
BMN 307 for phenylketonuria was put on a clinical hold in September due to liver cancer observed in mice and was expected to lift in Q1 of 2022, but the US FDA has requested additional preclinical studies.
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Gene Therapy: AAV Doses Should Not Be Subject To Fixed Upper Limit, US FDA Panel Says
Lack of reference standards inhibits ability to make comparisons between products or set caps for total vector genome dose or total capsid dose, advisory committee says; panelists suggest other approaches to prevent and mitigate toxicities, including running longer-term animal studies and investigating the efficacy of immunosuppression prophylaxis and treatment strategies.