The active genes in the body have to be copied, or transcribed into RNA molecules, which are then processed in a variety of ...

Wave Life Sciences shows promising data in genetic mutation treatments, with GSK collaborations and strong financial footing.

While most exon-skipping tools target only one sequence, SPLICER edits both the starting and ending sequences. As a result, the targeted exons are skipped over more efficiently, Miskalis said.

The drug – formerly known as SRP-5051 – is a more potent version of Exondys 51 (eteplirsen), an exon-skipping therapy that can be used to treat around 13% of the population with the muscle ...

“With current exon-skipping techniques, sometimes not all of the exon gets skipped, so there’s still part of the sequence we don’t want expressed. In the cookbook analogy, it’s like trying ...

Limited (“HUTCHMED”) (Nasdaq/AIM: HCM; HKEX: 13) today announces that the supplemental New Drug Application for ORPATHYS ® (savolitinib) has been granted approval by the China National Medical ...

Limited (“HUTCHMED”) (Nasdaq/AIM: HCM; HKEX: 13) today announces that the supplemental New Drug Application for ORPATHYS® (savolitinib) has been granted approval by the China National Medical Products ...

Advancing INLIGHT clinical study with novel, long-acting, muscle-sparing approach for obesity using Wave’s best-in-class GalNAc-siRNA ...

SPLICER uses a gene editing approach called exon skipping, which is of particular interest for health conditions caused by mutations that produce misfolded or toxic proteins, such as Duchenne’s ...

Chinese biopharma Hutchmed today announced that the supplemental New Drug Application for Orpathys (savolitinib) has been ...

SPLICER uses a gene editing approach called exon skipping, which is of particular interest for health conditions caused by mutations that produce misfolded or toxic proteins, such as Duchenne's ...