An exclusive license agreement has been signed between SATT Nord and the startup VG2D Pharma for the development of a new approach to treating cystic fibrosis, a rare genetic disease that affects approximately 162,500 patients worldwide.
Cystic fibrosis is a rare hereditary genetic disorder that primarily affects the lungs, but also the digestive and reproductive systems. It is caused by an autosomal recessive mutation in the gene encoding the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) protein, carried by chromosome 7. This protein, which is present in most tissues, but especially in the membrane of the respiratory and digestive mucous membranes, functions as a channel allowing the exchange of chloride ions between the inside and outside of the cell. When this gene mutates, the channel malfunctions. This alteration results in thickening of the mucus covering the mucous membranes due to a decrease in the amount of water on their surface and, as a result, repeated infections and inflammation that destroys organs.
Despite near-universal diagnosis in industrialized countries and strong awareness among the medical community of the benefits of early treatment for cystic fibrosis, only 12% of patients diagnosed worldwide were receiving the standard treatment—triple therapy with CFTR modulators—in 2022.
This low usage rate can be explained by the ineligibility of certain patients for this treatment (due to uncoverage of mutations, efficacy or tolerance issues) as well as the high cost of this treatment (averaging $300,000 in the United States). This observation reflects the unmet needs in the treatment of cystic fibrosis and highlights the importance of providing patients with treatment that is effective and well-tolerated, but also affordable.
Based on research conducted by Stéphane Gérard, professor at the Faculty of Pharmacy of the University of Reims Champagne-Ardenne and researcher at the Institute of Molecular Chemistry of Reims (ICMR/UMR CNRS 7312), and Frédéric Velard, Research Engineer at the Biomaterials and Inflammation in Bone Sites (BIOS) unit at URCA, in collaboration with the COBRA laboratory (University of Rouen/CNRS) and INSERM, the MucoPyf project focuses on the development of a new treatment for cystic fibrosis.
The originality of the invention lies in the molecules' ability to restore the function of the defective CFTR channel, combined with an anti-inflammatory effect.
MucoPyf treats all mutation classes except one, has an anti-inflammatory effect, is effective against bone loss associated with cystic fibrosis (a major comorbidity for nearly 50% of patients), and has better long-term tolerance.
"The VG2D Pharma adventure is the result of SATT Nord's initial support for the MucoPyF project. The fruit of exciting exchanges with Dr. Velard and the intense work of a consortium bringing together colleagues from different disciplines, this project enabled us to highlight the promising activity of a hit that we were able to optimize as part of a maturation program. Our goal of bringing therapeutic innovation to as many people as possible has guided our daily work ever since, with the help of Dr. Wild, who joined the team." Stéphane Gérard, Professor of Pharmacy, ICMR, Co-founder and CSO of VG2D Pharma.
"This project began 10 years ago, during scientific discussions focused on other health issues. Testing the products resulting from Professor Gérard's research in the context of correcting the CFTR function defect found in cystic fibrosis is undoubtedly one of the best ideas of my career. Our shared desire to bring our discoveries to patients did the rest." Frédéric Velard, IgR URBIOS, Co-founder and CSO VG2D Pharma.
This project was identified by SATT Nord in June 2016 and was the subject of a support program (scientific developments aimed at consolidating the biological effect of the molecules via in vitro, in vivo, and pharmacotoxicology tests, management of intellectual property aspects, implementation of a market and receptivity study, and the search for a CEO for the future startup VG2D Pharma, in the person of Ms. Imane Wild) financed by SATT Nord for a total amount of €445,000.
"During the development of the MucoPyf project, certain molecules demonstrated unexpected properties, which led to a new project that is also being supported by SATT Nord in a new application." François-Xavier Denimal, Head of the Health Business Unit.
In November 2024, the MucoPyf project was sublicensed to the startup VG2D Pharma, based in Reims.
VG2D Pharma is characterized by itsin-depth expertisein cystic fibrosis (CF), its market, and international healthcare ecosystems, particularlyorphan drugs. It received the I-Lab award in 2024 and, most recently, the BFTE grant awarded by Bpifrance, and has begun its search for seed funding in the amount of €400,000.
It is currently preparing for clinical trials scheduled for 2026/2027 and is in the process of raising funds from private investors.
"Equitable access to treatments for life-threatening diseases is at the heart of our mission. VG2D Pharma aspires to become a leader in the treatment of cystic fibrosis by offering patients an innovative therapeutic alternative, paving the way for a new paradigm in the management of the disease through multidimensional efficacy, while promoting broader access to treatment on a global scale." Imane Wild, CEO VG2D Pharma