Imperial College Health Partners (ICHP)

ID 383

This open-label, first-in-human, phase 1b study is designed to determine the safety profile of VERVE-101 administered to patients with HeFH, ASCVD, and uncontrolled LDL-C.

The outcome of this study will have many potential long-term and short-term benefits to patients: In the short-term patients will benefit from 24/7 supervised medical attention. Patients will learn more about ASCVD and awareness of the disease will be raised within the population. Clinicians will also benefit from learning more about the innovative techniques used in base-editing technology. Version 4.0 – April 2023 Page 4 of 23 In the long-term, unlike current medications that require lifelong adherence, VERVE-101 aims to permanently edit the PCSK9 gene, potentially eliminating the need for ongoing medication. There will be improved cholesterol control in patients which will allow for better cardiovascular health outcomes. Lower cholesterol levels could also significantly reduce the risk of developing HeFH-related complications like heart disease, stroke, and peripheral arterial disease. Finally, VERVE-101 could potentially improve patients' overall well-being and quality of life by simplifying their treatments and decreasing any side effects from long term medications.

18/01/2024

TRE