Riccardo Menandro: How Lipid Nanoparticles Are Shaping the Future of Rare Disease and Hemophilia Treatment
Riccardo Menandro, Pharmaceutical Scientific Reporter at Maven Pharma, shared on LinkedIn:
”Most conversations about lipid nanoparticles focus on the science.
This one focuses on the decisions.
LNP technology has moved from research platform to clinical mainstream faster than almost any other delivery system in recent memory.
Patisiran demonstrated hepatic gene silencing at therapeutic scale.
The COVID-19 vaccines demonstrated industrial scalability.
The pipeline now spans oncology, rare disease, hemophilia, and in vivo CRISPR editing.
But for organizations building or evaluating LNP-based programs, the scientific opportunity is only part of the picture.
Formulation expertise, IP position, tissue targeting strategy, and regulatory engagement are the variables that determine whether a program reaches the clinic on a competitive timeline, or doesn’t.
This white paper examines all four, with a focus on the decisions that matter most at the development planning stage.
Full document attached.”
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