VCP Research Programs
The research tools and programs of Cure VCP Disease are centered around the four pillars of our research strategy and our vision of making treatments available to patients affected by a pathogenic variant in the VCP/p97 gene.
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
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Master Protocol in Development:
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Design a master protocol for clinical trials with the input of patients and clinicians.
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Develop a clinical network in several locations
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Establish a relationship with FDA advisors
Longitudinal Natural History Study
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Nationwide Children’s Hospital Longitudinal Study:
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40 participants, two years of data collected
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Functional measures, including strength & movement, cognitive.tests, and patient reported data
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Remote and in-clinic assessments
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Progression measured is sufficiently sufficient for endpoints
Emmes Video Scorecard:
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17 participants, 3 timepoints
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Assessments complete
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Creating custom VCP Video Scorecard
Biomarkers
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Current Biomarker Collaboration:
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Analyze patient blood samples (from 0, 12, and 24 months) collected during the Longitudinal Study
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Blood assays in UPLC/MS for VCP levels, FGF21, Neurofilament light chain, TDP43P
Potential Biomarker Collaboration:
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Proteomics
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Transcriptomics
Gene Editing Therapy
Small Molecules
SMALL MOLECULES
NCATS Collaboration on phenotypic screening using muscle organoids
Industry Interactions
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VCP modulation
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TDP43 aggregation
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Stress granules assay
GENE EDITING THERAPY
Prime gene editing with Genethon:
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A joint effort between Cure VCP Disease and Genethon
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PI: Isabelle Richard at Genethon
Point gene editing with Nationwide:
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Young Investigator Eleonora D’Ambrosio
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.
CONNECT WITH US
Contact Armelle Pindon, Ph.D. to learn more about our research programs and collaboration opportunities.