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Our Research Strategy

The vision of Cure VCP Disease is to make treatments available to patients affected by a pathogenic variant in the VCP/p97 gene.

Goal: An Ultra Rare Disease Strategy for Treatment Discovery & Clinical Trial Readiness

Our goal is to develop and apply a strategy to understand the gaps, remove the roadblocks, and fulfill the requirements needed for drug discovery and treatment development.

With limited financial resources and a small team, a four-pillar strategy is essential to building a pathway for treatments. 


Collected to accelerate drug discovery and research for academia and industry

Why a Toolbox?

To enable drug discovery, our team has assembled a pre-clinical toolbox to provide a standardized workflow and easy access to test molecules in relevant disease models at the in vitro and in vivo levels. Our pre-clinical toolbox may be used for research purposes and commercial purpose entities. 

If the tools listed do not meet your current research needs, any researcher may contact Cure VCP Disease directly and our organization may be able to facilitate collaborations.

Image by Hans Reniers

Learn more about our collection of tools by selecting below:

Cellular Models
Animal Models
Library of Research Resources


Our approach to in vitro models is to make standardized models available to all researchers and host biobanks with patient samples.


Our approach to in vivo models is to evaluate existing VCP models and explore funding and grant opportunities for new models.


The primary assay for VCP is the binding and enzymatic activity of VCP hexamers.​ The secondary assays are VCP functions at the cellular level.


Reference chemical compounds ready to store in 96 well-plates in-house

Clinical Trial Readiness

We collaborate with partners to power the understanding of the disease's progression, capture the patient's perspective, and establish clinical endpoints.

Master Protocol
- Learn More -

Master Protocol in Development:

  • Design a master protocol for clinical trials with the input of patients and clinicians.

  • Develop a clinical network in several locations

  • Establish a relationship with FDA advisors

Longitudinal Natural History Study

 - Learn More -

Nationwide Children’s Hospital Longitudinal Study:

  • 40 participants, two years of data collected

  • Functional measures, including strength & movement, cognitive.tests, and patient reported data

  • Remote and in-clinic assessments

  • Progression measured is sufficiently sufficient for endpoints

Emmes Video Scorecard:

  • 17 participants, 3 timepoints

  • Assessments complete

  • Creating custom VCP Video Scorecard


 - Learn More -

Current Biomarker Collaboration:

  • Analyze patient blood samples (from 0, 12, and 24 months) collected during the Longitudinal Study

  • Blood assays in UPLC/MS for VCP levels, FGF21, Neurofilament light chain, TDP43P

Potential Biomarker Collaboration:

  • Proteomics

  • Transcriptomics

Gene Editing Therapy

Small Molecules


Industry Interactions

  • VCP modulation 

  • TDP43 aggregation 

  • Stress granules assay 


Collaborative research agreement:

  • A joint effort between Cure VCP Disease and Genethon

  • PI: Isabelle Richard at Genethon

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Treatment Discovery

Our Collaborations Leverage Small Molecules & DNA/RNA Modulations.

Regulatory & Industry

We offer programs to drive awareness and support education for FDA, clinicians, and patients.

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Reach out to ARMELLE PINDON, Ph.D. using the form linked below to learn more about these opportunities.

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