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 ...

The baby boy received the bespoke therapy within a few months of birth and is doing well, according to a presentation at ASGCT.

Treated with an individualized gene-editing therapy that corrects mutations directly on the genome, a patient shows ...

Even with novel technologies like gene therapy, gene editing, or mRNA, each must address a specific ... and phenylketonuria ...

in Chicago April 25-30 and the American Society of Gene and Cell Therapy (ASGCT) in New Orleans May 13-17. The proof-of-concept studies demonstrate that Strand’s programmable mRNA genetic ...