VERVE-101

Novartis's LEQVIO (inclisiran), the first FDA-approved siRNA therapy for LDL-C reduction, is projected to reach USD 2.2 billion in US market size by 2034, offering a novel mechanism with biannual dosing that enhances patient adherence.

Verve Therapeutics received FDA clearance to expand its VERVE-102 trial into the US, potentially offering a one-time base editing treatment for persistent high cholesterol conditions.

Verve Therapeutics anticipates initial data from the Heart-2 Phase 1b trial of VERVE-102, targeting PCSK9, in Q2 2025, including safety and efficacy data.

Researchers demonstrated successful mRNA delivery to CD34+ hematopoietic stem cells in rhesus monkeys using lipid nanoparticles (LNPs).

CRISPR-Cas9 gene editing has achieved a significant milestone with FDA approval of Casgevy for sickle cell disease and transfusion-dependent beta thalassemia.