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Developments in Biotechnology Platforms Leading to Increasing Immuno-oncology Applications
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This research service investigates technologies that propel the development of new targeted immunotherapies, particularly those that deliver therapeutic DNA and proteins. The top 3 growth opportunities focus on enhanced gene delivery systems for greater precision, the support of such systems through novel biotech platforms, and the affordability of precision medicine/targeted immunotherapies through modular, decentralized manufacturing systems.Regarding technology, nonviral gene delivery and nucleic acid delivery offer significant advantages in terms of safety, particularly from immunogenicity and carcinogenicity. Not using viral vectors also makes the regulatory pathway and the biomanufacturing process more affordable because no additional precautions are needed; this, in fact, makes the price tag lower. The study touches on the advantages and limitations of viral and nonviral gene delivery systems for targeted immunotherapies. Safety of administration without immunogenicity remains as the most relevant advantage. Liposomes have no replication risk and are less immunogenic than viruses. Other benefits are: • a practically unlimited transgene size; • the possibility of repeated administration;• a cost-affordable model; and• an easy manner to produce them in large amounts.The plurality of gene delivery systems is broadly benchmarked in terms of transfection efficiency, precision in cancer immunotherapy, and capability to pass the membrane barrier.As for remarkable innovation in biotech platforms, the research highlights gene addition as a novel approach that uses a delivery system to insert new genes directly into cells. The addition of a functional gene can take place either outside (ex vivo) or inside (in vivo) the body and can be used in cancer immunotherapy through CAR T-cell technology.Gene delivery strategies are based on a comprehensive suite of clinically validated technologies, including electroporation, liposomes, nanoparticles, and nonviral and viral delivery modalities. Electroporation is best suited for efficient delivery to blood cells and immune cells ex vivo. Lipid nanoparticles (LNP) are better indicated for in vivo delivery to the liver and potentially other organs. Adenoassociated vectors are typically used for in vivo delivery to the eye and central nervous system (CNS).As for optimal manufacturing process, closed-ended DNA (ceDNA) vectors exhibit a linear and continuous structure, which can be used for insertion of a transgene into a gene safe harbor (GSH) in the genome. For gene delivery, cell-targeted lipid nanoparticle (ctLNP) designed to avoid activation of the immune system upon initial dose can be used, while capsid-free approaches can be utilized to facilitate manufacturing.This research profiles a remarkable number of companies succeeding in the targeted immunotherapy space. A competitive analysis of the top three innovators, based on their IP activity and patent innovation focus, is included. Funding and investment, including leading deals and investors focused on the technologies empowering targeted immunotherapies, are also covered.Key Points Discussed:1. Top innovations in biotechnology platforms focused on targeted immunotherapies2. Emerging technologies in gene and nucleic acids direct delivery systems3. Outstanding companies succeeding the targeted immunotherapy space 4. Industry landscape and patent filing trends in the industry5. Major deals and investors focused on developing novel technologies for targeted immunotherapies6. Growth opportunities in the targeted immunotherapy area7. Best practices for risk management during the development of new technologies that enhance targeted immunotherapies
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