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.
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Gene Therapy Reviews Return To US FDA Advisory Committee Schedule
The upcoming review of two novel gene therapies from bluebird bio marks will be the first time the US FDA’s CTGTAC has met for a genetic product review in five years. That is a surprisingly long gap – and one that probably will not be repeated.
BioMarin’s PKU Gene Therapy Clinical Hold To Last ‘Several Quarters’
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.
Broader Horizons For Advanced Biotech In 2022: New Targets, Settings For IO, Cell And Gene Therapy
US FDA’s user fee goal calendar for 2022 already contains 45 applications for novel agents featuring a wide array of biologics, from cell therapy in cancer and hematology to bispecifics and new immuno-oncology targets that could move the field beyond PD-1/L1.