Archives: 2019-05-22

New International Commission Launched on Clinical Use of Heritable Human Genome Editing

An international commission has been convened by the U.S. National Academy of Medicine (NAM), the U.S. National Academy of Sciences (NAS), and the Royal Society of the U.K., with the participation of science and medical academies around the world, to develop a framework for scientists, clinicians, and regulatory authorities to consider when assessing potential clinical

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Establishing Manufacturing Controls: A Hurdle for the Cell and Gene Therapy Industry

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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Millions of Europeans at risk of chronic digestive diseases, new report reveals

Source  United European Gastroenterology Poor nutritional choices, including a high intake of ultra-processed foods and trans-fats, are putting millions of Europeans at an increased risk of a range of chronic digestive diseases, including digestive cancers, wheat related disorders and functional GI disorders, as well as obesity, a new report shows. Nutrition and Chronic Digestive Diseases,

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New tools for new treatments

Source Nature From antibiotics and organoids to CRISPR, improved biomedical methods and apparatus are enabling new therapies. Organoids Mini organs stand in for test models The term ‘organoid’ didn’t exist a decade ago. Now it’s a buzzword in biomedical science for a three-dimensional multi-cellular structure or ‘mini organ’. Thanks to advances with pluripotent stem cells

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A Technology Roadmap For Today’s Gene Therapy Manufacturing Challenges

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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Could AI Make Gene Editing More Accurate?

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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