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:
CFTR: is the main gene whereby its mutation directly causes cystic fibrosis;
AQ1, AQ3: aquaporins are a family of regulated proteins that form ion;
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;
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.
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
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.
The patent data is available at the link: https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2019235970