Release Date: 2024-02-12

Licensing Processes for Gene Therapy Products: Approved and Pending Clinical Trials in the World and Turkey

Buket Gungor (Author), Coskun Silan (Author)

Release Date: 2024-02-12

Gene therapy is a medical technology that aims to treat diseases by alteration, insertion or correction of genes. Gene therapy offers potential in treating many genetic diseases such as cystic fibrosis, blood cancers and neurological disorders. Preclinical processes include laboratory research, vector selection, gene editing studies and animal experiments. Clinical research phases consist of four [...]

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    Work TypeBook Chapter
    Published inGene Therapy
    First Page153
    Last Page187
    DOIhttps://doi.org/10.69860/nobel.9786053358824.10
    Page Count35
    Copyright HolderNobel Tıp Kitabevleri
    Licensehttps://nobelpub.com/publish-with-us/copyright-and-licensing

    Gene therapy is a medical technology that aims to treat diseases by alteration, insertion or correction of genes. Gene therapy offers potential in treating many genetic diseases such as cystic fibrosis, blood cancers and neurological disorders. Preclinical processes include laboratory research, vector selection, gene editing studies and animal experiments. Clinical research phases consist of four phases: Phase I, safety and tolerability; Phase II, clinical efficacy and best dosages; Phase III, efficacy, safety and availability; Phase IV evaluates long-term effects and rare side effects.
    Gene therapy products undergo rigorous clinical research and approval processes. Regulatory bodies make decisions about the safety and effectiveness of products. Approval of gene therapy products is subject to different regulations around the world. . These processes may differ in countries such as the USA, China, the European Union and Türkiye. As of 2023, 6590 clinical trials have been conducted in the field of gene therapy in Turkey, the majority of which are in Phase I and Phase I/II stages. The first approved gene therapy product was launched in 1998, and rapid advances have been made in this field in recent years. In this section, information and examples about clinical research on gene therapies are given. Information about the registration processes of gene therapy products and approved products was presented.

    Buket Gungor (Author)
    Çanakkale Onsekiz Mart University
    https://orcid.org/0000-0002-5802-1635
    3Assistant Professor Buket Güngör graduated from Trakya University Faculty of Medicine in 2008. In 2009, she received her specialization in Pharmacology from Canakkale Onsekiz Mart University Faculty of Medicine. She worked at Turkish Medicines and Medical Devices Agency Clinical Research Department between 2014-2017. She worked at Antalya Provincial Health Directorate and same time she completed master’s degree in clinical drug research Dokuz Eylül University Institute of Health Sciences. Since 2022, she has been working at from Canakkale Onsekiz Mart University Faculty of Medicine until today. She has studies on clinical trials.

    Coskun Silan (Author)
    Professor, Çanakkale Onsekiz Mart University
    https://orcid.org/0000-0002-8352-6571
    3Professor Coşkun SILAN graduated from Ege University Faculty of Medicine in 1989. In 1994, he received his specialization in Pharmacology from İstanbul University Faculty of Medicine. He worked at Abant İzzet Baysal University Faculty of Medicine 2000-2008. Since 2008, he has been working at from Canakkale Onsekiz Mart University Faculty of Medicine until today. He has studies on Pharmaceutical Toxicology, Pharmaceutical Chemistry, Life Sciences, Biotechnology and Biomaterials.

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