BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU ...
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Alltrna plans to tackle rare diseases using tRNA-based therapiesEven with novel technologies like gene therapy, gene editing, or mRNA, each must address a specific gene or disease ... methylmalonic acidaemia (MMA) and phenylketonuria (PKU). One of the interesting ...
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