Verve Therapeutics

Verve Therapeutics, led by CEO Sekar Kathiresan, will present at the 43rd Annual J.P. Morgan Healthcare Conference. The company focuses on genetic medicines for cardiovascular disease, aiming to transform treatment with gene editing. Their programs target genes to lower LDL-C, addressing ASCVD and familial hypercholesterolemia.

PCSK9 inhibitors, targeting cholesterol levels, are gaining traction with market growth anticipated from 2020-2034. Key companies include Innovent Biologics, Amgen, and AstraZeneca, with therapies like Tafolecimab and Evolocumab. The market is driven by increasing prevalence of hypercholesterolemia and cardiovascular diseases, with significant advancements in pipeline products.

Verve Therapeutics granted a new employee 5,330 restricted stock units (RSUs) on Nov. 29, 2024, as part of its 2024 Inducement Stock Incentive Plan.

Cerba Research shared recent cell and gene therapy developments at the 31st ESGCT Congress, highlighting advancements in genome editing for cholesterol management and hearing restoration in children. CAR T-cell therapies for autoimmune diseases and innovations in treatment monitoring were also discussed, emphasizing the rapid integration of technology and AI in gene therapy development.

Beam Therapeutics acknowledges a patient death in a Phase 1/2 trial for sickle cell disease, attributing it to pre-conditioning, not the base-editing treatment. AstraZeneca, UT, and LIVESTRONG report successful therapeutic genome editing in mouse liver via lipid nanoparticles. Korean researchers find base and prime editors produce fewer large DNA deletions than Cas9 nucleases. YolTech demonstrates effective mRNA delivery to bone marrow cells for blood disorder treatment. Japanese scientists develop a curative strategy for congenital purpura fulminans using engineered APC. US-France team identifies key host factors enhancing rotavirus vaccine production. Allogene Therapeutics shares positive Phase 1 data for renal cell carcinoma treatment. Cellectis presents strategies to enhance CAR T-cell efficacy in solid tumors. Caszyme and Integra Therapeutics enter a licensing agreement for safer gene therapies. Industry updates include financial results and clinical trial progress from various companies. CRISPR genome engineering symposium to feature latest advancements. CRISPR gene editing can integrate mitochondrial DNA into the nuclear genome, but exonucleases can mitigate this. HuidaGene Therapeutics' CRISPR-Cas13 RNA-editing therapy for macular degeneration is accepted by the FDA.

A single dose of CRISPR-based NTLA-2002 reduced angioedema attacks by 75-77% in patients with hereditary angioedema, with 80% of patients in the 50 mg group experiencing complete response.

Cardiology gene therapy and cell therapy progress includes Rocket Pharmaceuticals completing enrollment for Danon disease gene therapy RP-A501, LX2006 improving Friedreich Ataxia cardiomyopathy biomarkers, Nexcella dosing first patient in light chain amyloidosis CAR-T trial, Verve Therapeutics starting phase 1b trial for VERVE-102, and Sardocor receiving fast track designation for heart failure gene therapy SRD-001.

Precision medicine uses genetic, environmental, and lifestyle data to tailor treatments, with AI, CRISPR-Cas9, and mRNA as key trends. AI accelerates genomic analysis and drug discovery, CRISPR-Cas9 offers precise gene editing, and mRNA vaccines expand beyond COVID-19, driving personalized healthcare.

CRISPR technology, including CRISPR-Cas9, CRISPR-Cas12, and CRISPR-Cas13, is revolutionizing genome editing. FDA approved the first CRISPR-Cas9 drug, Casgevy®, for sickle cell disease and beta thalassemia. Other applications include treating urinary tract infections, hereditary transthyretin amyloidosis, hereditary angioedema, cardiovascular diseases, type 1 diabetes, systemic lupus erythematosus, HIV, and blood cancers. Challenges include cost, regulatory standards, and ethical considerations.