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In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contractmanufacturing organisations (CMOs). The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder.
The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.
Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. The evolution of NGS technologies has made it possible to sequence thousands of genes with a suspected geneticdisease predisposition, in a very short period of time and at minimal costs.
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