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Novel gene-editing therapy shows promise for patients with transthyretin amyloid cardiomyopathy

Medical Xpress

A single IV infusion of NTLA-2001, a novel CRISPR/Cas9-based gene editing therapy, significantly reduced circulating transthyretin (TTR) protein levels in patients with ATTR amyloid cardiomyopathy, a progressive and fatal cause of heart failure, according to late-breaking research presented today at the American Heart Association's Scientific Sessions (..)

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Using CRISPR-Cas9 as an intervention to protect the heart from ischemia-reperfusion damage

Medical Xpress

A team of researchers at the University of Texas Southwestern Medical Center has found that it is possible in mice to protect the heart from ischemia-reperfusion using the CRISPR-Cas9 gene editing system.

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Vutrisiran Reduces Heart Risks for ATTR-CM Patients in Alnylam’s HELIOS-B Trial

XTalks

The data, presented at the European Society of Cardiology Congress 2024, suggest that vutrisiran could significantly reduce the risk of death and heart-related issues, marking a potential treatment breakthrough. Vutrisiran works by silencing the gene responsible for producing TTR, reducing both mutant and wild-type TTR proteins.

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Top 30 Pharma and Biotech Companies in 2023: Statistics and Trends

XTalks

Johnson & Johnson’s Top 5 Best-Selling Drugs of 2022: 1) Stelara (ustekinumab) Stelara is an immunosuppressant biologic therapy that blocks the IL-12 and IL-23 proteins that play a role in plaque psoriasis and Crohn’s disease. The monoclonal antibody targets CD20, a protein found on the surface of B lymphocytes (B cells).

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