US FDA CBER Chief Calls For Industry-Academia Collaboration In Developing Treatments For Ultra-Rare Diseases
The head of the US FDA’s Center for Biologics Evaluation and Research said that new approaches are needed to facilitate product development for ultra-rare diseases. The FDA also announces the creation of a new CBER Advanced Technologies Team to promote the adoption of innovative manufacturing technologies for cell and gene therapy products.
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US FDA investigators are more likely to cite cell and gene therapy manufacturers for facilities and production deficiencies, agency says, while industry reports difficulties complying with drug GMP requirements that are not always applicable to cell and gene therapies.
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.