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:
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CFTR: is the main gene whereby its mutation directly causes cystic fibrosis;
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AQ1, AQ3: aquaporins are a family of regulated proteins that form ion;
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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;
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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:
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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;
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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:
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Due to its status of an orphan disease, a Fast Track route is available to apply to, for faster regulatory approval;
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Previous results by the Oxford Consortium study have demonstrated promising results, which, we believe, can be significantly improved;
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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)
Comments
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Favorable initiatives taken by regional and national nonprofit organizations is one of the key factors propelling growth;
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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;
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This drug only accounts for 4–5% cases of cystic fibrosis and there are still no medicines for most CF cases;
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Even here, the cost of Ivacaftor is $311,000 per year, roughly similar to the price of other drugs for rare diseases;
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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
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:
The patent data is available at the link: https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2019235970