Gene-editing heart disease treatment developed from Penn CRISPR research is being advanced by Eli Lilly.
Media News Source reports on a groundbreaking approach in the fight against heart disease, spearheaded by Kiran Musunuru, a prominent cardiologist at the University of Pennsylvania. Musunuru, who also cofounded Verve Therapeutics, is pioneering the use of CRISPR technology to potentially eliminate the need for lifelong statin medications and prevent heart attacks through a single, two-hour infusion.
The innovative therapy focuses on using CRISPR, a cutting-edge gene-editing tool that enables precise alterations to DNA. By targeting a specific liver gene known for producing low-density lipoprotein (LDL) cholesterol—often labeled as “bad” cholesterol—the treatment aims to substantially lower cholesterol levels, thereby reducing the risk of cardiovascular events. This approach has captured the attention of major pharmaceutical companies, as evidenced by Eli Lilly’s announcement in June regarding a planned acquisition of Verve for up to .3 billion.
Musunuru’s work is particularly notable given the growing prevalence of heart disease, which remains one of the leading causes of death globally. He posits that if such a therapy could be administered before a heart attack occurs, it would fundamentally shift the paradigms of preventive care. Current medications, like statins, often flounder in terms of long-term adherence, especially after an acute event, which underscores the advantage of a one-time gene-editing treatment.
The method involves embedding CRISPR components into lipid nanoparticles, which are then infused into the bloodstream. These nanoparticles are also equipped with messenger RNA (mRNA) that acts as a guide, directing the CRISPR components to their specific genetic target within the liver. Once there, they effectively switch off the gene responsible for high LDL cholesterol levels.
In clinical studies, early results have shown promise, including a significant decrease in LDL levels for individuals with severe cholesterol issues, with one patient’s bad cholesterol level dropping by 60% and remaining low over a two-year period.
The implications of this research extend beyond mere cholesterol management; it opens the possibility of a versatile application of CRISPR in various medical contexts, including the prevention of several genetic disorders and even cancers associated with BRCA gene mutations. Current research seeks to explore targeted interventions that could inhibit cancer development in high-risk individuals by directly altering genetic predispositions.
As the healthcare landscape shifts towards more personalized and efficient treatment modalities, Musunuru and his team continue to work towards advancing the potential of gene editing technologies in both preventive and therapeutic settings. However, it is important to note that while the promise of these therapies is significant, rigorous clinical testing and regulatory approvals are still necessary before these innovations can be widely implemented.
