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.
You may also be interested in...
While other gene therapy companies are scaling back plans or struggling to raise cash, Regenxbio is benefitting from AbbVie’s partnership in wet AMD.
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.
FDA seeks advice on screening patients potentially at higher risk for liver and other injuries, strategies to implement before or after gene therapy administration to head off or mitigate potential adverse events, and whether upper limits should be set for the total vector genome dose and total capsid dose.