Verve Cleared on NDA for Gene Editing Med to Durably Lower Cholesterol After Single Dose

  Verve Therapeutics, a clinical-stage company developing a new class of genetic medicines for cardiovascular disease, today announced the clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for VERVE-102 for the treatment of patients living with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD). VERVE-102 is a novel, investigational in vivo base editing medicine designed to be a single-course treatment that inactivates the PCSK9 gene in the liver to durably lower blood low-density lipoprotein cholesterol (LDL-C).

“The IND clearance from the U.S. FDA represents an important step in our journey to advance a new class of in vivo gene editing medicines for people worldwide living with cardiovascular disease,” said Sekar Kathiresan, M.D., co-founder and chief executive officer of Verve Therapeutics. “There are multiple cholesterol lowering medicines currently available that can lower LDL-C at a single time point; however, time on treatment for these medicines remains low. This is a significant problem as heart attack risk reduction tracks most closely with cumulative cholesterol reduction, a function of both the magnitude of cholesterol reduction and time on treatment. To address this unmet need, Verve’s medicines are designed to deliver lifelong cholesterol lowering after a single course of treatment and, consequently, drive more meaningful efficacy. With our ongoing Heart-2 clinical trial for VERVE-102 progressing internationally, we are excited to begin activating trial sites in the U.S. as we expect it to play a key role in our continued clinical development.”

https://finance.yahoo.com/news/verve-therapeutics-announces-clearance-investigational-110000116.html