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Cystic Fibrosis

Overview & Proposed Solution

Our solution is based on a combination of different genes, rather than just one:

We aim at creating a gene therapy DNA vector VTvaf17 carrying CFTR, AQ1 and AQ3 therapeutic genes for the treatment of Cystic Fibrosis:

  1. CFTR: is the main gene whereby its mutation directly causes cystic fibrosis;

  2. AQ1, AQ3: aquaporins are a family of regulated proteins that form ion;

  3. channels allowing water to penetrate certain membranes. A close relationship of aquaporins 1 and 3 with the cystic fibrosis transmembrane conductance regulator has been shown;

  4. The genetic constructions described above have been separately filed for patent and the respective strains have been deposited in an international depository.

Oxford Consortium study showed:

  • That monthly application of the pGM169/GL67A gene therapy formulation was associated with a significant, albeit modest, benefit compared with placebo after 1 year, indicating a stabilization of lung function in the treatment group;

  • We believe that adding further supportive genes in combination, as well as optimizing for frequency and duration of administration can further improve the result.

Further Advantages:

  • Due to its status of an orphan disease, a Fast Track route is available to apply to, for faster regulatory approval;

  • Previous results by the Oxford Consortium study have demonstrated promising results, which, we believe, can be significantly improved;

  • Due to its status as an orphan disease, government funded support for patients is more widely available.

Market Trends & Projections

Market Size Growth Forecast (2017 – 2025)



  • Favorable initiatives taken by regional and national nonprofit organizations is one of the key factors propelling growth;

  • As a market comparable: Ivacaftor was developed by Vertex Pharmaceuticals as the first drug to treat the underlying cause of CF, however not through a gene therapy, but chemically;

  • This drug only accounts for 4–5% cases of cystic fibrosis and there are still no medicines for most CF cases;

  • Even here, the cost of Ivacaftor is $311,000 per year, roughly similar to the price of other drugs for rare diseases;

  • Therefore, we believe that our estimate of price in the table below is extremely conservative; however, even with those assumptions significant sales figures can be achieved.

Projections of Sales in the first 5 Years

cf 5y.png

Assumptions and Rate Definitions:
Incidence Rate: Average percentage of disease occurrence across the population.

Diagnosed Population: Percentage of the population with a correctly diagnosed disease.
Patients seeking new therapy: Diagnosed patients that are actively searching for alternative remedies for their illness; and are able and willing to undergo a new treatment.

Compliance rate: Out of the patients, who are actively seeking an alternative therapy, it is the percentage that actually make the switch and select a new therapy.

Patent information:

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