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Epidermolysis Bullosa

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 KRT5 and KRT14 therapeutic genes for a treatment of Epidermolysis Bullosa Simplex (of basal  subtype, localized, herpetiform and other generalized forms). Previous research results have shown that insufficient expression of proteins encoded by these genes are associated with the development of a spectrum of
    diseases, including, but not limited to, connective tissue diseases, wound healing, inherited and acquired pathological processes, such as epidermolysis bullosa;

  • Therefore we believe that genetic constructs that provide expression of proteins encoded by a combination of those genes can be used to develop drugs for the treatment of EB and damaged skin tissue;

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

General Information

  • Epidermolysis Bullosa (EB) is a category of rare genetic disorders involving the skin and skin-like tissues;

  • EB is generally inherited from one or both parents, who have been diagnosed with EB or carry as mutated EB gene;

  • People suffering from EB have fragile skin which causes blisters that can lead to wounds, which are difficult to heal;

  • Low prevalence, lack of data, and no available medications make EB a non-curable disease.

Further Advantages:

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

  • First clinical trials of gene therapy have demonstrated that those therapies have the potential to correct the molecular and clinical phenotype of patients with EB;

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

Market Trends & Projections

Market Size Growth Forecast (2018 – 2026)

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Comments

  • First recently conducted clinical trials of gene therapy for EB have shown promising results for this therapy providing effective treatment;

  • Abeona Therapeutics, Fibrocell Science and Krystal Biotech are some of the players that are currently in the process of clinical trials;

  • Improvements in gene delivery and in preventing immune reactions are seen among the biggest challenges that lie ahead during further therapeutic development.

Projections of Sales in the first 5 Years

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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=WO2020139152

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