“We want to offer an alternative to the 78% of patients with cystic fibrosis who do not currently benefit from the standard triple therapy.” Researchers at the University of Reims Champagne-Ardenne (URCA) Frédéric Velard and Stéphane Gérard are indeed working on another solution, a drug aimed at extending the life expectancy of people suffering from this rare genetic disease. Their start-up, VG2D Pharma, based in Reims and managed by Imane Wild, has just been rewarded by the national i-Lab competition. This bonus grants them aid of 375,000 euros which will allow them to continue the research and development of this therapeutic solution, by recruiting two research engineers for each of their specialty (biology and chemistry).
Life expectancy under triple therapy at 49 years
No treatment currently provides a cure for this rare genetic disease that is cystic fibrosis. But patients were literally able to breathe better thanks to triple therapy launched in France in 2021. It helps extend the life expectancy of some 52% of patients treated in France to around 49 years. “Perhaps 70 years in the coming decade,” indicate Doctor Frédéric Velard, specialist in cystic fibrosis, and Professor Stéphane Gérard, expert in medicinal chemistry and drug design. Triple therapy, which costs on average 300,000 euros per year per patient, is covered by the French health system. The fate of thousands of other cystic fibrosis patients around the world is very different. “A 2022 study estimates that only 22% of these patients benefit from triple therapy worldwide,” indicate the two researchers. “Our slogan is to put therapeutic innovation at the service of as many people as possible.”
Research phases to refine the understanding of the biological phenomenon are still necessary before moving on to a phase of testing on humans — Photo: VG2D Pharma
A promising innovation
How did these two researchers manage to detect a solution? By working on the CFTR protein, the mutation of which is responsible for cystic fibrosis. During their research, Frédéric Velard and Stéphane Gérard discovered the corrective action of a family of molecules on the function of this CFTR protein. “When this mutation occurs, the protein no longer functions correctly. Our approach aims to help it do its job, when it is not normally able to do so,” explains Frédéric Velard, who has been working for more than 12 years on the disease. This discovery led to the filing of a patent two years later.
A billion euros to develop a drug
Concretely, to obtain a drug, “it takes around 12 years and a billion euros of funds,” explains Stéphane Gérard. “We’re about 6 years in and we still have a ton of funds to raise.” This is why, in January 2024, after two years of reflection, the two researchers decided to create a start-up to facilitate and accelerate financial research. Supported by the Innovact incubator and the URCA, which granted them a status allowing them, alongside their functions, to be co-inventors and to develop the start-up’s activity, the duo also welcomed as as CEO a doctor in neuroscience, Imane Wild, based in Cardiff. “She has business skills and experience in Big Pharma on the issue of marketing which complements us very well.”
Hope for first clinical phases in 2027
At this stage, laboratory work takes place in vitro and animal tests have been carried out. Research phases to refine the understanding of the biological phenomenon are still necessary before moving on to a phase of testing on humans. Both researchers are optimistic. “We hope to envisage the first clinical phases in 2027 and, ideally, a marketing authorization in 2031-2032.”
To achieve these objectives, VG2D Pharma is seeking to raise between 1 and 1.5 million euros from 2025. The support of i-Lab is valuable to them, especially as it allows them to maintain territorial roots by generating employment. “We start with a relatively modest fundraising, but we have to give credibility to the approach, generate confidence,” explains Stéphane Gérard. “It’s a long-term development, but when you believe in your project, you have to persevere.”
