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From the regulatory genome to novel treatments

Our innovative platform

Backed by decades of world class research

Nucleome decodes genetic variation within the human regulatory genome to find new ways to treat diseases. 

The regulatory genome makes up the majority of our DNA which does not encode for proteins and is largely uncharted. It acts like an instruction manual that defines cell type specificity, switching genes on and off at the right time and level. This allows for translation of the universal genome, present in human cells into multiple, differentiated cell types. 

The regulatory genome contains the majority of disease-linked genetic variants, but most have no function currently ascribed. Nucleome has the tools to unlock its potential and discover new drug targets along with associated biomarkers to develop therapeutic assets and treat diseases. This will lead to the discovery and development of better and safer drugs, guided by genetics. 

Our platform, based on decades of research from the University of Oxford, combines state of the art computational and experimental approaches invented to overcome the key challenges that hinder translation of the regulatory genome to novel treatments through the identification of:

The right variant

Nucleome’s machine learning model predicts disease-linked genetic variants that create and destroy functional elements in the regulatory genome

The right cell type

Nucleome’s machine learning model predicts the cell types affected by the genetic variants

The right gene

Nucleome’s 3D genome analysis methods experimentally link variants to genes

The right effect

Nucleome’s functional validation technology defines the effect of genetic variants on gene expression at scale in primary cell types

The right pathway

Nucleome’s assays are done at scale, allowing us to map disease affected pathways and understand how cell types are regulated in health and disease

Our 3D genome analysis methods offer significant benefits over other methods currently available:
Precision

Unprecedented accuracy
On average 256 base pairs, and our latest method, published in Nature, goes down to 1 base pair resolution

Confidence

Highest Sensitivity
Reproducible

Scalability

Highly Scalable
From single gene to every gene in the genome

Relevance

Small cell number requirement

Our research dramatically improves our understanding of how the regulatory genome drives disease and has the potential to discover novel high-quality targets guided by genetics across multiple diseases to build a robust pipeline of drug assets.

Pipeline

Nucleome is currently focused on lymphocytes and associated autoimmune-related diseases to discover first-in-class differentiated medicines that will shift the treatment landscape and improve patient outcomes in indications with high unmet medical need.

Partnerships

We are passionate about bringing transformative medicines to patients and developing precision medicines for complex diseases

Our technology enables us to discover novel targets central to disease with drug discovery hypothesis based on genetics, de-risk high value existing targets, repurpose existing drugs through new genetic and target insights, map disease-affected pathways and help design biomarker strategies for patient stratification. The technology is disease and modality agnostic.

Nucleome is backed by the venture arms of leading pharma companies, including J&J Innovation – JJDC, Inc., Pfizer, and Merck; among other top-tier investors.

If you are interested in partnering with Nucleome, please contact us here.