Recent news that the US Food and Drug Administration (FDA) will ask cell and gene therapy (CGT) developers to provide more consistent evidence with their submissions has resulted in drug makers delaying development plans — and working on new ways to address the agency’s concerns
New genetically-validated drug targets are found at accelerated speed using population scale biobank genotype-phenotype datasets after a foundational partnership is formed between Paradigm4 and Alnylam.
Over the past 5 decades or so, the growing understanding that RNA can influence protein function through routes other than direct translation has opened the prospect of discovering small molecules for tackling diseases in novel ways.
