Pablo Corral: Can One Infusion Lower LDL for Years
Pablo Corra, Pharmacology Professor at FASTA University, Past President of Argentine Lipid Society, shared a post on LinkedIn about a recent article by Børge G. Nordestgaard et al., published in NEJM, adding:
“A glimpse into the future of LDL-C lowering: one infusion, potentially years of effect.
VERVE-102 uses in vivo base editing of PCSK9 aiming to reproduce the lifelong protection seen in carriers of PCSK9 loss-of-function variants.
Early Phase 1 results are striking:
- 35 patients with HeFH and/or premature CAD
- Single IV infusion – no chronic dosing
- At the highest dose (1.0 mg/kg):
PCSK9 decreased 88%
LDL-C decreased 62%
Absolute LDL-C reduction: −78 mg/dL
- LDL-C fell from 128 to 51 mg/dL on average
- Reductions appeared sustained, with grater than or equal to 1-year follow-up available in 15 patients
- No dose-limiting toxicity or deaths; infusion reactions were generally mild/moderate, with transient ALT elevations reported.
Still Phase 1. Small numbers.
Long-term safety – including off-target editing – and cardiovascular outcomes remain unknown, with 15-year follow-up planned.
But the paradigm is provocative:
From ‘lower LDL-C for life’ to potentially ‘one treatment for lifelong LDL-C lowering.'”
Title: A New Approach to Lowering LDL Cholesterol
Author: Børge G. Nordestgaard
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