Cystic fibrosis (CF) is a monogenic disease caused by pathogenic mutations in the CFTR gene on chromosome 7. Affecting multiple organs, its clinical manifestations vary, impacting the respiratory, reproductive, and gastrointestinal systems, as well as the mental health of patients. Despite significant progress, the management of CF still presents several challenges.
CF is the most common autosomal recessive disease among individuals of North European descent.
Despite important advancements thanks to CFTR modulators, the treatment of CF still has major unmet needs. Firstly, challenges related to access to treatment are a key problem for patients with certain genetic mutations not addressed by current drugs and for patients who cannot access treatment because of pricing. Secondly, in view of the recency of treatments (and tritherapy in particular), the long-term quality of life during the prolonged life expectancy is still to be further documented. Similarly, more long term safety and efficacy data collection in real life clinical practice is still needed.
VG2D Pharma is committed to addressing these challenges. Our pipeline features a novel molecule with a unique mechanism of action offering significant benefits.
Our molecules have been engineered to address types II to VI mutations, our family of leads broadens the scope of effective treatment.
A simplified regimen of one oral tablet enhances adherence and improves patient lifestyles.
Through efficient production, we aim to expand treatment accessibility and reduce financial burdens.
We have finalized our preclinical proof-of-concept package and lead optimization. Here’s our current progress.
VG2D Pharma is gearing up for clinical trials. Stay tuned for more updates.
Dive deeper into the scientific foundations and research that inform our understanding of cystic fibrosis and our innovative approach at VG2D Pharma.
Our commitment to transparency and knowledge-sharing is rooted in science. To learn more, please consult the trusted sources below.
1. Collins FS (1992) Science 256(5058):774‐779.
2. Kerem et al. (1989) Science 245(4922):1073‐1080.
3. Bear et al. (1992) Cell 68(4):809‐818.
4. Kerem E. (2005) Pediatr Pulmonol. 40(3):183‐196.
5. De Boeck K., Amaral MD. (2016) Lancet Resp Med. 4(8):662-74.
6. Mckone et coll. (2003) Lancet 361(9370):1671‐1676.
7. Chen et al. (2021) Animal Model Exp Med. 4(3):220-232.
8. Fondation de la fibrose kystique, Comprendre les changements dans l’espérance de vie. Consulté le 8 juillet 2023.
9. McKone et al. (2021) Eur Respir J. 58(3):2002288.
10. Allen et al. (2023) Nature Communication 4:693.
11. Purkayastha et al. (2023) Journal of Cystic Fibrosis 22(3): 478-483.
12. Arooj et a’. (2023) BMJ Open Respir Res. 10(1):e001590.
