Cure VCP Disease Showcases Progress at the RARE Drug Development Symposium

Cure VCP Disease recently participated in the Global Genes RARE Drug Development Symposium, hosted at Harvard Medical School in partnership with Boston Children’s Hospital. Following a competitive application process, we were one of two organizations selected to present a pitch highlighting the preclinical and clinical assets we have developed to de-risk investment in VCP disease. Our Director of Scientific Engagement and Collaborations, Dr. Zollie Yavarow, presented our pitch live to a panel of experts, termed “the dolphin tank.”

Zollie Yavarow, PhD, MA

The format was intentionally brief, with just a couple of slides in 7 minutes, designed as an elevator pitch of the organization’s strategy. For Cure VCP Disease, this was an opportunity to demonstrate the significant progress that has been made in both preclinical and clinical trial readiness and receive feedback from the distinguished panel.

Our presentation highlighted:

• Preclinical resources: tissue samples, datasets, and animal and cell models that are now in place to support VCP research.

• Clinical readiness: a completed 2-year natural history study, VCP family registry, genetic counseling and testing program, and researcher relationships to support future trials.

In addition to presenting what we’ve built, we’re inviting researchers, clinicians, and companies to use these resources. We want to pique curiosity, spark collaboration, and propose that investing in VCP disease could lead to meaningful scientific and therapeutic advances. The “ask” of our pitch is for partnership and for academics, clinicians, and companies to bring their expertise into this space.

The feedback underscored the importance of making these resources accessible and clearly defining the mechanisms for collaboration. This aligns directly with our ongoing efforts to reduce barriers to entry and encourage more research in VCP disease.

The pitch session was said to be the highlight of the conference by organizers and attendees. Following the presentation, many patient advocacy leaders and researchers connected to learn more about our approach and to discuss their own early-stage strategies. Zollie also participated in expert office hours, which provided additional opportunities to share insights and build connections across the rare disease community with conference attendees.

Looking ahead, our priority is to continue lowering the threshold for engagement by making VCP research as attractive and straightforward as possible. Progress in rare disease is built through small, continuous steps, by showing up in the right rooms, asking questions, sometimes making mistakes, and being brave enough to stand up and try again.

Cure VCP Disease remains committed to steady progress with the ultimate goal of delivering meaningful treatments to our community.

---

If you are interested in learning more, the full collection of

RDDS 2025 session recordings are now available on the

Global Genes YouTube channel.

---