Target biology
Define the PCSK9-related function an intervention would need to influence.
Cardiovascular / Cholesterol
New research directions for cholesterol control
A new cholesterol approach needs a clear reason for patients and development teams to care. We are exploring research questions around PCSK9, a target involved in the body's handling of LDL cholesterol.
Early research · Target biology and development strategy
The opportunity
Start with the benefit a new option would need to offer
Effective cholesterol treatments raise the bar for new development. A compelling approach would need to fit a meaningful use case: reaching the intended LDL response, supporting practical long-term use or addressing a specific development constraint. Our PCSK9 research direction begins with that question of fit.
Our research direction
Connect an established target with a focused research question
PCSK9 influences the availability of receptors that help clear LDL cholesterol from the blood. That biology provides a starting point for exploring additional intervention approaches. We want to connect target engagement with the relevant functional response, and choose comparisons that can distinguish an interesting molecular idea from a direction worth developing.
Useful comparisons
Choose measurements and existing benchmarks that address the proposed development benefit.
Practical fit
Bring treatment burden, exposure and the intended use into the research question early.
Where we could connect
Shape a program around a cardiovascular development decision
We welcome conversations with lipid researchers, drug-discovery teams and cardiovascular development partners. Bring the constraint you want to resolve and the evidence your team would need next. Together, we could define a bounded evaluation with a relevant comparison before considering a broader program.
Explore BioTwin capabilities ↗Questions worth asking
Before we begin
Why explore PCSK9 when effective approaches already exist?
The target's established role gives a new direction a useful starting point and demanding comparators. Further work should be justified by a specific potential benefit, rather than novelty alone.
Does engaging PCSK9 establish a cardiovascular benefit?
Target engagement is an early step. Its relationship to LDL response needs appropriate functional evidence, and any claim about cardiovascular outcomes needs evidence for the particular intervention. Results from existing medicines do not establish the performance of this research direction.
Is the aim to replace current cholesterol medicines?
That is not a predefined role. The intended use and place alongside existing options would depend on the proposed approach and its evidence. A first discussion can help identify which development setting is worth investigating.
Can we assess a fit without sharing confidential chemistry?
Yes. Begin with the target, the broad development constraint and the comparison your team would find useful. Access to detailed research, permitted use and intellectual-property terms can be agreed before sensitive material is exchanged.
External scientific context
Published background for the research question.
Start a conversation
What would you want a new cholesterol approach to achieve?
Bring your objective, the question you want to resolve and a little non-confidential context. We can discuss the fit and a useful next step.