A Pioneering Company
Fundamentally transform therapies for neurodegenerative disorders by harnessing human genetics and gene regulatory networks
Rooted in Human Genetics
Neurodegenerative disorders have strong and compelling human genetic links. Addressing genetically linked mechanisms will lead to disease-modifying therapies.
Linking human genetics and gene expression networks identifies entirely unexploited targets for therapy development. A focus on small molecule drugs modulating key mediators of neurodegeneration will enhance accessibility and convenience for patients.
Targeted Patient Population
Applying the genetic background of patients will allow for a precision-medicine approach thereby improving health outcomes. A rational patient selection enables targeted clinical studies.
Our groundbreaking research addresses the root cause of
human neurodegenerative diseases.
Our Platform creates a rich Pipeline across Neurodegenerative Disorders
Our Alzheimer’s and Parkinson’s disease programs are the most advanced.
We have developed small molecule modulators of our novel, unexploited, highly druggable target. Our lead compounds restore APOE4-induced gene expression dysfunction and synaptic deficits. We are poised to rapidly move through lead optimization to IND-enabling studies.
Shared disease pathways and a common genetic mechanism in Alzheimer’s and Parkinson’s diseases allow us to link novel targets to clinical dysfunctions in Parkinson’s disease and to optimize our small molecules towards clinical candidates.
- Alzheimer’s Disease (APOE4)
- Parkinson’s Disease (SNCA A53T, LRRK2 G2019S)
- ALS/FTD (C9orf72 Repeat Expansion)
- Alzheimer’s Disease (Familial mutations)
Who We Are
Our team is made up of experienced leaders throughout the industry. Complimenting our highly skilled team is a global network of specialized collaborator companies. Together, we apply state-of-the-art technologies to execute our program with strong focus and great efficiency.
Anne Urfer-Buchwalder, PhD
Founder and President
Roman Urfer, PhD
Founder and Chief Executive Officer
Karoly Nikolich, PhD
Paolo Pevarello, PhD
Medicinal Chemistry Advisor
Therapies for Neurodegenerative Disorders by Harnessing Human Genetics and Gene Regulatory Networks