Exploring the Latest Research in Gene Therapy: Innovations and Insights
Gene therapy market is characterized as the disease treatment that occurs owing to the transfer of hereditary material into cells. It is broadly used to treat several medical indications such as neurological disorders, cancer, dystrophy, hepatological diseases, and various other indications. In this blog, readers will learn the latest research in gene therapy.
Increasing advances in the technology of gene-editing and genomics tools drive the market growth. In addition, according to a research report by Astute Analytica, the global gene therapy market is likely to increase at a compound annual growth rate (CAGR) of 24% over the projection period from 2023 to 2030.
Here are the innovations and research in the gene therapy:
Advancements in biotechnology: Novel cell and gene therapies are made possible by the rapidly developing field of genetic engineering. Therapies are made more precise and effective by instruments like viral vectors, CRISPR-Cas9 gene editing, and others. Positive conditions are being created by biotechnology’s increasing breakthroughs for these treatments.
Framework of supportive regulations: Regulatory bodies, like the European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA), are implementing several measures to accelerate the licensing procedure for gene and cell therapies. Additionally, this is promoting growth and investments in the gene therapy sector. Research and development efforts are more efficiently conducted when academic institutions, pharmaceutical corporations, and biotech enterprises collaborate and form partnerships.
Some of the regulations are:
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In November 2023, the Advanced Cell Therapy and Research Institute, Singapore (ACTRIS) and Agilent Technologies Inc. inked a Memorandum of Understanding (MOU) to work together on developing breakthroughs in gene therapy during the following three years.
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In October 2023, the Central Drugs Standard Control Organisation (CDSCO) approved Immuno ACT’s marketing authorization for the first humanized COVID-19 targeted Chimeric Antigen Receptor T cell (CAR-T cell) therapy product. It is intended for leukemia and relapsed/refractory B-cell lymphomas in India.
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In October 2023, The FDA in the United States has approved Novo Nordisk’s RivflozaTM (nedosiran) injection in dosages of 80 mg, 128 mg, or 160 mg. The goal of this once-monthly gene therapy is to reduce urine oxalate levels for adults with primary hyperoxaluria type 1 (PH1) with comparatively maintained kidney function, as well as children 9 years of age and older.
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In September 2023, The U.S. Food and Drug Administration (FDA) has received a Biologics Licence Application (BLA) from Abeona Therapeutics Inc. requesting approval of EB-101. This investigational autologous engineered cell therapy is a treatment for patients suffering from recessive dystrophic epidermolysis bullosa (RDEB).
Conclusion:
Latest discoveries are being discovered in the field of gene therapy at a very quick speed. Gene therapy has great promise for treating diseases that were previously incurable, and the development, regulatory, and investment communities are collaborating to bring this goal to reality.
In addition, innovative and novel technologies are being created daily to enhance the safety, effectiveness, and viability of gene therapy products. These comprise developments in stem cell research, new gene editing tools like CRISPR-Cas9, and enhancements in gene delivery systems like viral vectors and non-viral techniques.
