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Funded Studies

Exploiting a Novel Mechanism for Gene Regulation to Develop Innovative, Targeted Therapies for Parkinson’s Disease

Study Rationale: Neurodegenerative disorders such as Parkinson’s disease (PD) and Alzheimer’s disease (AD) are associated with a variety of disease-causing mutations and genetic variants. In addition to producing disease pathology, these genetic lesions can alter the regulation of other genes in their chromosomal vicinity. We contend that this collateral dysregulation of additional genes also contributes to the disease, and we have discovered compounds that improve the function of these target genes. In this study we will assess whether enhanced analogs of these compounds can improve the observed movement and cognitive deficits in preclinical animal models of PD.

Hypothesis: Our hypothesis is that restoring the activity of genes that are dysregulated in the presence of PD-associated variants will ameliorate forgetfulness, improve concentration and address other cognitive and movement symptoms in PD.

Study Design: In this project, we will enhance our test compounds so that they can be administered by mouth and remain stable in the body long enough to reach their site of action in the brain. We will achieve this improvement by modifying aspects of the compounds’ chemical structure and measuring the effects on their activity and stability. These newly designed compounds will then be administered in preclinical models of PD to assess whether they improve the symptoms observed in these models, including deficits in movement and cognition.

Impact on Diagnosis/Treatment of Parkinson’s disease: Based on our understanding of how these compounds work, we believe that our drug candidates have the potential to improve the movement-related symptoms and memory deficits in people with PD.

Next Steps for Development: We will conduct safety testing of our most advanced compounds in preclinical models to evaluate whether the intended dose levels are safe to be administered to humans. These findings will enable first-in-humans Phase 1 clinical studies in which these compounds will be given to healthy subjects.


  • Roman Urfer, PhD

    San Mateo, CA United States

  • Anne Urfer-Buchwalder, PhD

    San Mateo, CA United States

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