Collaboration is first step in understanding if CRISPR-Cas technology can correct mutations in the VCP gene
(Sept. 14, 2023, Warner Robins, GA) – Cure VCP Disease, Inc., is excited to announce a collaboration with Genethon as part of its preclinical efforts to speed the path to a treatment or a cure for VCP disease, also referred to as multisystem proteinopathy-1. As a leading global gene therapy organization focused on the design and development of gene therapies for rare diseases and the sole patient advocacy organization supporting VCP disease patients worldwide, this partnership will see Genethon and Cure VCP Disease utilize CRISPR-Cas technology and gene editing to attempt to correct prevalent mutations in the VCP gene and restore function in patient cells.
“Our partnership with Genethon is a testament to our commitment to accelerating the path to potential treatments for VCP disease. As an ultra-rare disease patient organization, we aren’t just hoping for a breakthrough, we’re actively working towards it,” said Nathan Peck, CEO of Cure VCP Disease, Inc.
The initial phase of the program will focus on variants present in two-thirds of the patient population, with genetic variations located in the region of amino acids 155 up to 164. The aim in this phase is to establish a proof of concept: can this technology reach the specific target to restore proper functionality in reprogrammed motor neurons and muscle cells?
"Cure VCP Disease Inc. is deploying a two-pronged strategy to work towards a treatment and cure for this life-changing, ultra-rare disease: the first avenue is traditional drug discovery (small molecules), and the second is gene editing and RNA silencing (nucleic acid intervention),” said Armelle Pindon, Ph.D., Chief Scientific Officer at Cure VCP Disease, Inc. "Our community's engagement has enabled us to kickstart this vital step in our search a treatment and cure."
The Cure VCP Disease collaboration with Genethon will be led by the team of Dr. Isabelle Richard, Research Director at the CNRS and co-founder of Atamyo Therapeutics. Dr. Richard has been instrumental in gene identification in Limb-Girdle Muscular Dystrophy (LGMD) and has successfully developed several therapies. Genethon, part of the AFMTelethon, is a non-profit organization that brings together experts in gene therapy, muscle disorders, stem cells, organoids, and cell therapy. Genethon's successful track record includes gene therapy products already on the market for rare neuromuscular diseases.
For more information about this groundbreaking collaboration and the ongoing work of Cure VCP Disease, Inc. and Genethon, please visit www.curevcp.org and www.genethon.com.