From https://www.tctmd.com/news/gene-editing-therapy-safely-lowers-pcsk9-ldl-cholesterol-phase-i-heart-2
Title : Gene-Editing Therapy Safely Lowers PCSK9, LDL Cholesterol in Phase I: Heart-2
Though there’s still a long way to go before a clinical application ...
By Todd Neale , June 2026
A single infusion of a gene-editing therapy ... which targets PCSK9 production in the liver, shows promise for lowering LDL cholesterol (by 62%) in patients with heterozygous familial hypercholesterolemia (FH) and/or premature coronary disease, an interim analysis of a study shows...
the key to reducing cardiovascular events is to keep the LDL as low as possible for as long as possible ...
current treatment model relies on ... daily pills, intermittent injections ... efficacy is limited by high discontinuation rates ...
This new therapy ... uses in vivo base editing to inactivate the PCSK9 gene and reduce production of the PCSK9 protein.
This interim analysis of the study is an important first step to demonstrating that such a one-time therapy to durably lower LDL may be possible ...
Daniel Soffer, MD (University of Pennsylvania) ... said that use of this therapy is “super exciting mainly because it’s a very clever way to inhibit PCSK9, but also because it’s super cool to think about gene editing ..." ...
Title : Gene-Editing Therapy Safely Lowers PCSK9, LDL Cholesterol in Phase I: Heart-2
Though there’s still a long way to go before a clinical application ...
By Todd Neale , June 2026
A single infusion of a gene-editing therapy ... which targets PCSK9 production in the liver, shows promise for lowering LDL cholesterol (by 62%) in patients with heterozygous familial hypercholesterolemia (FH) and/or premature coronary disease, an interim analysis of a study shows...
the key to reducing cardiovascular events is to keep the LDL as low as possible for as long as possible ...
current treatment model relies on ... daily pills, intermittent injections ... efficacy is limited by high discontinuation rates ...
This new therapy ... uses in vivo base editing to inactivate the PCSK9 gene and reduce production of the PCSK9 protein.
This interim analysis of the study is an important first step to demonstrating that such a one-time therapy to durably lower LDL may be possible ...
Daniel Soffer, MD (University of Pennsylvania) ... said that use of this therapy is “super exciting mainly because it’s a very clever way to inhibit PCSK9, but also because it’s super cool to think about gene editing ..." ...
