We're Excited To Share How You are Helping to Drive Science
Several months ago we shared our scientific agenda for 2023 and beyond. Today we wanted to share with you some updates on recent developments as well as upcoming events! Our mission is to find new treatments for VCP disease. That's where your donations are making a difference.
With your help, we have built a toolbox so that our partners can easily screen for new treatments for VCP disease.
In January, Cure VCP Disease started the process to create patient-derived IPSCs (stem cells) to assist all interested researchers.
Blood was donated from two patients and transformed into models that can be used to screen for potential treatments and for scientific discovery.
We are excited to report that these IPSCs are now reprogrammed into motor neurons and myoblasts that are now available to all our partners, industry and research to use.
Several biotech companies and universities will be using them. They will also be used to launch the Cure VCP Disease gene editing program (CRISPR/Cas correction). For more information, please reach out to Armelle Pindon, our Chief Scientific Officer, or visit our Research page.