VG2D Pharma

Cystic fibrosis (CF) is a genetic rare disease characterised by the dysfunction of the CFTR channel. This dysfunction causes the thickening of mucus  resulting in severe respiratory symptoms and other comorbidities that impact patients wellbeing and quality of life. 

The global population affected by CF is estimated at 162,428 patients (Guo et al., 2022). Despite near-universal diagnosis in industrialized countries and strong awareness in the medical community about the benefits of early CF treatment, only 12% of diagnosed patients worldwide receive the standard treatment, namely the triple therapy (Guo et al., 2022). This low uptake reflects the number of ineligible patients to this treatment either because of the type of genetic mutations or because they have tolerability issues. The high cost of the tritherapy (averaging $300k in the United States) also limits patients' access. This highlights the unmet needs in CF and emphasizes the importance of providing patients with an effective, well tolerated, and affordable treatment.

VG2D Pharma’s molecule addresses patients who are ineligible for existing CFTR modulators. These patients currently have to rely on symptomatic treatments only with no to very limited impact on their prognosis. Our technology offers a new multidimensional approach that tackles the root cause of the disease (CFTR dysfunction), while also addressing inflammation (that causes organs' degradation) and bone density loss, two major comorbidities of CF. With its first-in-class mechanism of action, VG2D Pharma’s molecule targets a broad range of genetic mutations unlike current treatments. Our patient-centric pricing strategy will increase access to treatment across all geographic regions. 

The VG2D Pharma team, composed of experienced professionals with deep expertise in CF, pharmaceuticals' launches and international healthcare ecosystems.

Our patented molecule was tested in POC in vivo and in vitro preclinical experiments confirming the triple efficacy with no major safety signals.

We are currently raising funds to move our programme to GLP tox studies and submit an orphan drug designation.

The CF market is estimated at over 20 billions USD in 2030 with a projected CAGR of 24%. Our target population is estimated at 24 883 patients.

Rather than directly compete with Vertex (the company behind the first line tritherapy), who dominates the market currently, our aim is to complement their offerings by targeting patients who do not respond well to the tritherapy or cannot tolerate this treatment, as well as those facing affordability/access challenges. Additionally, owing to its triple efficacy, the VG2D Pharma molecule is poised to mitigate issues associated with Vertex's tri-therapy, including drug-drug interactions, low adherence, complex dosing regimens, and high burden of the pill.