Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment.

The company has launched a competition to offer grants of up to $100,000 to multiple projects that can uncover "new insights about the role of the PCSK9 protein/gene in health and disease", with a ...

Familial hypercholesterolemia results from gene mutations approximately halving ... decreased transintestinal cholesterol excretion. PCSK9, which mediates post-translational destruction of LDL ...

PubMed and Scopus databases were searched to retrieve clinical trials on the efficacy and/or safety of novel LDL-C-lowering agents including PCSK9 inhibitors, mipomersen (Kynmaro®), lomitapide ...

Oct. 28, 2024 — Using state-of-the-art technology, researchers have identified several specific steps needed for CRISPR to become active and perform its gene editing function. These preclinical ...

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

As an alternative to the systemic delivery of immune checkpoint inhibitors, here the authors develop an in vivo gene-edit strategy using a conductive hydrogel-based electroporation system to ...

The employee received an aggregate of 5,330 restricted stock units (RSUs). The RSUs will vest in equal annual installments on the first four anniversaries of January 1, 2025, subject to such ...

Nov. 26, 2024 — A genetic predisposition to having lower insulin production and less healthy fat distribution are major causes of early-onset type 2 diabetes in ...

Q4 2024 Earnings Call Transcript November 26, 2024 Arrowhead Pharmaceuticals, Inc. misses on earnings expectations. Reported ...