AAV Vector Consortia Address Quality Assays, Endpoints
Two outside groups aim to help CBER on gene therapy questions from ‘N of one’ to markers for hemophilia.
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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.
Experience and data from the study, which will use the same vector platform to deliver different transgenes for different diseases, could feed into the US FDA’s efforts to establish a public-private partnership to enable manufacturing of gene therapies for ultra-rare diseases.
Robert Califf’s nomination to return as FDA commissioner will raise protests about a ‘revolving door’ pathway between industry and agency. But, in this case, it may be more apt to talk about how Califf’s time outside FDA led to evolving views on the role of big data in drug regulation.