Genetic Modification Therapies – Clinical Applications & Technology Platforms
Genetic modification therapies are changing the landscape of medicine. These advanced therapies have the unprecedented ability to offer a one-time cure for severe, debilitating diseases that were previously untreatable. BCC Research found that this industry is segmented by technology platform, delivery method (viral versus nonviral), and clinical application. This specialization enables companies to build significant barriers to entry and competitive advantage.
Genetic modification therapy incorporates a functional gene into a cell-based therapy. The genetic modification generally occurs outside the body, but the resulting genetic change to the patient's DNA is permanent. The active payload comprise of the gene encoding for production of the therapeutic protein and gene controls that regulates production of the therapeutic gene. The vector, which may be either non-viral or viral, delivers the gene and gene control payload to the cells which are to be genetically modified. The most common cell types include T cells, and HSCs. Genetic modification has brought a milestone into medicine. Genetic modification is very crucial in treating severe diseases.
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Strong patent and licensing arrangements also contribute to these competitive positions. Manufacturing to meet the large-scale demands of the industry will play an important role in the future. The interest level in corporate partnerships has been enhanced by recent clinical successes and regulatory approvals for genetic modification therapy candidates. Industry alliances have been centered on several objectives, including access to genetic modification therapy technologies by large pharma/biotech, access to delivery technologies, and entry into new therapeutic markets.
Key factors for meeting revenue forecasts include continuing demonstration of genetic modification therapy efficacy and safety in clinical settings, migration to earlier-line settings in cancer, and success in efforts to standardize and scale up the manufacture of viral delivery vectors. The following sections examine the main genetic modification therapy platform technologies: gene therapies, genetically modified cell therapies, RNA therapies, and gene editing.
A rich late-stage pipeline of gene therapies is being developed for a wide range of disease categories. In the next 5 years, gene therapy is poised to exert a significant influence on genetic modification therapies. Gene therapies seek to permanently replace defective genes in a cell’s DNA. This can restore cellular function and has the potential to totally eliminate a disease. Monogenic diseases, for which a specific disease-causing gene can be identified, are well-suited for gene therapies.
To be successful, gene therapy candidates must meet important clinical criteria: 1) the pathogenic mechanism should be clear and addressable, 2) the target organ must be accessible to the gene therapy, and 3) there must be a compelling, unmet medical need. Luxturna, which is the first gene therapy to be approved by the FDA, fully meets these criteria. Luxturna is indicated for biallelic RPE65-mediated inherited retinal disorders. In this indication, mutations in the RPE65 gene is the clear and addressable pathogenic mechanism. The target organ (the eye) is easily accessible to the gene therapy, which uses an AAV2 viral vector to carry the correct version of this gene. Finally, there is a clear and pressing medical need for therapies that can restore functional vision in children and adults who have this disorder.
References:
https://www.theinsightpartners.com/reports/genetic-modification-therapies-market/
https://drug-dev.com/genetic-modification-therapies-clinical-applications-technology-platforms/