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

The baby boy with a rare urea cycle disorder received the bespoke therapy within a few months of birth and is doing well, ...

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

"We are unique in our ability to deliver this innovative treatment in such a short timeline," said Mark Wetzel, VP/GM mRNA CDMO Services at Aldevron. "This CRISPR therapy was made under ...

An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care ...

"We are unique in our ability to deliver this innovative treatment in such a short timeline," said Mark Wetzel, VP/GM mRNA CDMO Services at Aldevron. "This CRISPR therapy was made under exceptional ...

"With mRNA-4106, we sought to design an inclusive therapy that encodes for antigens commonly shared across patients and tumor types. We are thrilled to be able to bring this medicine to trial ...

Treatment seems to have been effective, but it is not clear whether such bespoke therapies can be widely applied.

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 Phase 1 trial (ClinicalTrials.gov identifier: NCT06880549) will evaluate the safety, pharmacodynamics, immunogenicity and preliminary efficacy of mRNA-4106 administered alone and in combination ...

One such strategy is to “consider psilocybin-assisted therapy,” referring to the compound found in psychedelic mushrooms. She details her thinking on the subject in a 750-word passage.