Source RAPS Addressing manufacturing controls for the cell and gene therapy industry, this article discusses criticality of establishing Chemistry Manufacturing Controls (CMC) Readiness, Critical Quality Attributes (CQAs) and Critical Process Parameters (CPPs) for cell and gene therapy products. The author suggests manufacturers need sound drug development and manufacturing facility plans covering a product’s lifecycle and

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In Nov. 2018, The National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) published technology roadmaps addressing needs and gaps in three key product areas: gene therapy, antibody-dug conjugates, and vaccines. The roadmaps were developed with the collaborative input of industry, academic, and government experts. This piece highlights technology opportunities for gene therapy manufacturing. The NIIMBL

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Machine learning algorithms predict the repairs made to DNA after Cas9 cuts. The papersM.W. Shen et al., “Predictable and precise template-free CRISPR editing of pathogenic variants,” Nature, 563:646–51, 2018. F. Allen et al., “Predicting the mutations generated by repair of Cas9-induced double-strand breaks,” Nat Biotechnol, 37:64–72, 2019. During gene editing with CRISPR technology, the Cas9 scissors that

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