Epidermolysis Bullosa
Overview & Proposed Solution
Our approach to Epidermolysis Bullosa (EB) targets multiple genes rather than relying on a single therapeutic vector. Specifically, we are developing a VTvaf17-based gene therapy vector carrying KRT5, KRT14, COL7A1, and LAMB3 to address the most common EB subtypes:
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Epidermolysis Bullosa Simplex (EBS) — often associated with mutations in KRT5 and KRT14
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Junctional EB (JEB) — frequently linked to LAMB3 mutations
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Dystrophic EB (DEB) — commonly caused by defects in COL7A1
By enabling robust expression of these critical genes, our therapy aims to restore the structural integrity of skin layers, mitigating blister formation and promoting effective wound healing. Each construct is patented separately, with corresponding bacterial strains deposited in an international repository.
General Information
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Epidermolysis Bullosa (EB) comprises a group of rare genetic conditions affecting the skin and related tissues.
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EB is commonly inherited from one or both parents who either have the disease or carry a mutated gene.
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Patients experience extreme skin fragility, leading to blisters and chronic, hard-to-heal wounds.
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EB’s rarity, limited data, and absence of approved therapies have so far prevented a definitive cure.
Comments
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First recently conducted clinical trials of gene therapy for EB have shown promising results for this therapy providing effective treatment;
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Abeona Therapeutics, Fibrocell Science and Krystal Biotech are some of the players that are currently in the process of clinical trials;
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Improvements in gene delivery and in preventing immune reactions are seen among the biggest challenges that lie ahead during further therapeutic development.
Additional Advantages
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Orphan Disease Status
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Opens opportunities for Fast Track regulatory approval.
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Often supported by government funding specifically allocated for rare diseases.
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Gene Therapy Validation
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Early clinical trials demonstrate the potential to correct both the molecular defects and clinical symptoms of EB.
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Multi-Gene Strategy
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Combining KRT5, KRT14, COL7A1, and LAMB3 addresses multiple EB subtypes simultaneously, aiming for broader and more effective patient outcomes.
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By integrating these four key genes into our proven VTvaf17 vector, we aim to develop a transformative gene therapy solution for the diverse spectrum of Epidermolysis Bullosa
Patent information: