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Cystic Fibrosis

Overview & Proposed Solution

Our approach targets Cystic Fibrosis (CF) using a combination of three genes rather than relying on a single therapeutic target:

  • CFTR: The primary gene implicated in CF; mutations directly lead to disease manifestation.

  • AQ1 & AQ3 (Aquaporins): These regulated proteins form ion channels to facilitate water transport across membranes. Research indicates a close relationship between aquaporins and the CFTR gene.

We have filed patents for these gene constructs, and the corresponding strains have been deposited in an international repository. Our aim is to incorporate them into a single gene therapy vector, VTvaf17, specifically designed for CF treatment.

Supporting Evidence: Oxford Consortium Study:

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A pivotal study showed that monthly applications of a pGM169/GL67A gene therapy formulation yielded modest yet statistically significant improvements in lung function over a one-year period, effectively stabilizing the disease. We believe that integrating additional supportive genes and optimizing dosing frequency can significantly enhance these results.

Comments

  • 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.

Additional Advantages

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  1. Orphan Disease Status

    • CF qualifies for Fast Track regulatory pathways, potentially expediting approval.

    • Government-backed patient support programs are often more accessible for orphan diseases.

  2. Established Proof of Concept

    • Previous gene therapy trials, such as the Oxford Consortium study, demonstrated promising outcomes, serving as a foundation for further innovation.

  3. Improved Efficacy Through Combination

    • By targeting CFTR and aquaporins simultaneously, our strategy aims to maximize therapeutic impact while maintaining safety.

 

By leveraging multiple therapeutic genes, the VTvaf17 vector platform, and existing clinical evidence, we aim to advance a more effective and efficient gene therapy for cystic fibrosis.

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

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