(PharmaNewsWire.Com, January 10, 2017 ) "Gene Therapies: A Diverse Range of Technologies with a Promising Long-Term Outlook"" discusses all gene therapies under the broad criteria of genetic material introduced to the cell for a therapeutic purpose. It includes not only the insertion of a gene into the cell for expression, but also gene silencing with RNA interference (RNAi) and antisense RNA, aptamers (DNA or RNA polymers which bind to a protein target), and oncolytic viruses.
Gene therapies have been in development in humans for 25 years, and a number of products have begun to enter the pharmaceutical market. However, due to various challenges and clinical trial setbacks, progress in developing this technology and achieving suitability for commercial usage has been slow. Gene therapies only account for a marginal market share and many efficacy and safety concerns remain unaddressed. However, the pipeline is robust; 906 pipeline gene therapies are currently in development. Most are at an early stage of development, with 76% at the Discovery or Preclinical stage. However, there are currently 23 gene therapy programs in Phase III development and two at the Pre-registration stage. This indicates that although gene therapies are beginning to reach the market after two decades of research in humans, the majority remain in relatively early development.
This CBR report provides a comprehensive view of the clinical, R&D, commercial and competitive landscape of Gene Therapy, and assesses key developments in delivery vector technology, and challenges and advances associated with the production of such vectors.
Despite 25 years of clinical research, only a few gene therapies of all types have reached the market globally, and none have achieved strong clinical or commercial success
Table of Content
Gene Therapy Overview Types of Gene Therapy Types of Intervention Types of Vector Currently Approved Gene Therapies Glybera (alipogene tiparvovec) Kynamro (mipomersen) Macugen (pegaptanib) Vitravene (fomivirsen) Gendicine (rAd-p53) Oncorine (rAd5-H101) Neovasculgen (Pl-VEGF165) Gene Therapy Production Strategies Production of Viral Vectors Case Study: Challenges in the Manufacture of AAV Vectors Challenges to Gene Therapy Development Gene Therapy Pipeline Gene Therapy Pipeline by Therapy Area and Stage of Development Gene Therapy Pipeline by Intervention and Vector Type Company Positioning Companies by Therapy Area Companies by Stage of Development Companies by Intervention Type Companies by Vector Type Conclusion
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