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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.

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Master Protocol
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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

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Longitudinal Natural History Study

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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

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Emmes Video Scorecard:

  • 17 participants, 3 timepoints

  • Assessments complete

  • Creating custom VCP Video Scorecard

Biomarkers

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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

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Potential Biomarker Collaboration:

  • Proteomics

  • Transcriptomics

Gene Editing Therapy

Small Molecules

SMALL MOLECULES

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NCATS Collaboration on phenotypic screening using muscle organoids

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Industry Interactions

  • VCP modulation 

  • TDP43 aggregation 

  • Stress granules assay 

GENE EDITING THERAPY

Prime gene editing with Genethon:

  • A joint effort between Cure VCP Disease and Genethon

  • PI: Isabelle Richard at Genethon

Point gene editing with Nationwide:​

  • Young Investigator Eleonora D’Ambrosio

Image by D koi

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.

Image by Sincerely Media
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