10 Best Gene Therapy Stocks to Buy Right Now

5) Verve Therapeutics, Inc. (NASDAQ:VERV)

Number of Hedge Fund Holders: 25

Verve Therapeutics, Inc. (NASDAQ:VERV) is a clinical-stage genetic medicines company, which is engaged in developing gene-editing medicines for patients to treat cardiovascular diseases in the US.

Verve Therapeutics, Inc. (NASDAQ:VERV) continues to actively develop next-generation PCSK9 therapies, focusing on offering innovative solutions for patients with high cholesterol. Its approach involves using gene editing technology to target PCSK9, a protein that is critical in regulating cholesterol levels in the blood.

The development of PCSK9 therapies is the focal point for Verve Therapeutics, Inc. (NASDAQ:VERV), with 2 distinct generations of treatments in the pipeline. The first-generation PCSK9 therapy saw setbacks, which led to a pause in enrollment because of concerns about ALT elevation and thrombocytopenia. These issues were believed to be related to the lipid nanoparticle (LNP) delivery system rather than the gene editor itself.

As a result, Verve Therapeutics, Inc. (NASDAQ:VERV) transitioned its focus to the next-generation PCSK9 therapy. This updated version incorporates improvements like GalNAc for targeted delivery and uses learnings from indirectly validated LNP technology. The next-gen therapy remains focused on enhancing liver tropism, potentially addressing the safety concerns observed in the first-generation treatment.

Verve Therapeutics, Inc. (NASDAQ:VERV)’s gene editing approach to PCSK9 inhibition can revolutionize treatment compliance in cardiovascular disease management. Traditional PCSK9 inhibitors tend to require regular injections, leading to poor adherence over time. Verve Therapeutics, Inc. (NASDAQ:VERV)’s one-time treatment is expected to eliminate the need for ongoing administration, which can improve patient compliance significantly.