AccurEdit Therapeutics today announced that its innovative in vivo gene editing product, ART001, has obtained Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA).

The LNP-mRNA modality is a novel approach that uses ... The first successful attempt at mRNA in vivo expression occurred the following year. Over the next few years, cellular and humoural immunity ...

“Research conducted at Penn demonstrates the tremendous promise of harnessing mRNA and targeted LNP delivery to train a patient’s body to make CAR-T cells in vivo, potentially creating new ...

EVM14 is the first messenger RNA (mRNA) therapeutic vaccine developed internally by the company to gain this approval. Its ...

In Prime Medicine’s initial in vivo data, LNP delivery of Prime Editors targeting the Pi*Z (E324K) mutation demonstrated up to 72% precise correction of the SERPINA1 gene in the hepatocytes of ...