Paul Nioi

Paul Nioi

Company: Alnylam Pharmaceuticals

Job title: Senior Director, Translational Research


Dr. Paul Nioi joined Alnylam in March 2018 and leads the biomarker and human genetics groups as well as a team of research scientists advancing new programs through discovery and development. His biomarker team focuses on ‘omics approaches to profile levels of large numbers of analytes from human clinical trial samples.  These efforts led to the recent discovery of neurofilament light chain as a biomarker of polyneuropathy in hereditary transthyretin amyloidosis.  In addition, Paul has built the human genetics capability at Alnylam and his team are currently mining genotype-phenotype data, from hundreds of thousands of individuals, to identify new drug targets. Paul also leads a team of research scientists that are responsible for advancing new liver and CNS RNAi therapeutics for a variety of diseases.

Prior to Alnylam, Paul spent 15 years in the biotech and pharmaceutical industry including tenures at deCODE genetics, Amgen and Schering-Plough.

Paul obtained his academic training at the University of Edinburgh (BSc) and the University of Dundee (PhD).



Panel Discussion: Identification of Rare Disease Mechanisms Through Proteomics Analysis 11:00 am

Maximizing information gained from precious samples Linking genetics to actionable protein biomarkers Multi-omics and the future of understanding disease Opportunities presented by proteomic analysis specific to rare disease, current and future trendsRead more

day: Day One

Neurofilament Light Chain (NfL) as a Potential Biomarker in Hereditary Transthyretin-Mediated (hATTR) Amyloidosis 9:00 am

Hereditary transthyretin amyloidosis is a progressive and fatal disease caused by mutations in the TTR gene that can lead to both cardiomyopathy and polyneuropathy. No blood based biomarkers exist that allow diagnosis, disease progression or response to therapy to be easily monitored. We conducted an unbiased proteomic analysis of blood samples from the APOLLO Phase…Read more

day: Day One

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