The Future of Novel Emerging Manufacturing Technologies
The spectrum of gene therapy with some specificity to viral vectors is very broad including both delivery vehicles developed for transient short-term and permanent long-term expression. Moreover, the types of vectors are represented by both RNA and DNA viruses with either single-stranded (ss) or double-stranded (ds) genomes. The approval and or under regulatory review of viral vector-based drugs are making progress in the cure and or treatment of certain ailments.
The field of gene therapy has seen some significant progress with nearly 3,000 clinical trials conducted by 2017. Not surprisingly, 64.6% of the trials relate to cancer therapy. Furthermore, 10.5% focus on monogenic diseases, 7.4% on infectious diseases, and 7.4% on cardiovascular diseases. Interestingly, nearly 70% of the trials have utilized viral vectors.1 Although, recent developments in gene manipulation methods, such as CRISPR, and more efficient delivery methods for nonviral vectors, viral vectors still remain attractive.
Based on Regerative Medicine & Rare Disease 2019 report (Alliance for Regenerative Medicine, ARM), there were 647 Clinical Trials in rare disease underway worldwide by the end of 2019, i.e.: Ph 1: 252, Ph 2: 353, and Ph 3 42.
It is clear that with the growth of this medicinal manufacturing segment of the industry with numerous products in development and or under review for approval by respective regulatory bodies and an expanding number of organizations involved in ATMPs (Cell and Gene Medicinal Products and Tissue Engineering) manufacture. The demand for streamlined manufacturing, better understood of regulatory requirement, knowledgeable operational and regulatory staff, and furthermore patient needs are key goals and an important vision for any drug product manufacturing organization to be striving to deliver the most effective drugs to the patients.
By establishing more consistent practices across manufacturing, logistics, compliance, and many other areas, the industry will be able to overcome some of the current growing pains it is experiencing.
Standardized and robust manufacturing platforms will need to be established, and a global network of facilities will be required to serve the patient globally at a local level at their origin. As well as fully optimized and robust process is needed, one that can be mass customized.
Cell and gene therapy innovators, manufacturers, providers, and their contract partners need to focus on manufacturability and process industrialization in the development cycle and avoid technical development bottlenecks. Developers need to be concerned with lean and agile manufacturing that can greatly benefit from working with regulatory, and operational excellence programs to bring about the most efficient and compliant manufacturing and process application specifically developed to deliver this type of product to the patient.
Support for the development and manufacture of safe and effective regenerative medicines and advanced therapies worldwide is required. The combination of therapeutic developers, major medical centers and research institutions, patients organizations, tool and technology providers, and regulatory enforcement body are key to the success of delivering, streamlining, and integrating of providing the patient with effective and safe drug product delivered by this segment of the industry.
As stated in: EudraLex - The Rules Governing Medicinal Products in the European Union, Volume IV, and Annex 2;
“Reasons for changes: Annex 2 of the GMP Guide has been revised as a consequence of the restructuring of the GMP Guide, new manufacturing technology and concepts, the increased breadth of biological medicinal products to include several new product types such as transgenic derived products and the Advanced Therapy Medicinal Products, (ATMPs) together with associated new legislation. The GMP guidance drawn up for the latter products is to meet the requirements of Article 5 of Regulation (EC) 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/20041 and to align with Part IV of Annex I to Directive 2001/83/EC, as introduced with Commission Directive 2009/120/EC of 14 September 2009 amending Directive 2001/83/EC of the European Parliament and of the Council on the Community code relating to medicinal products for human use as regards advanced therapy medicinal products.”2
Join us at the 2020 ISPE Biopharmaceutical Manufacturing Virtual Conference 1-2 June to hear leading experts discuss their success in this field of medicine. The virtual conference will provide practical examples and learnings about these novel emerging manufacturing technologies.
- 1. Beitelshees M., Hill A., Rostami P., Jones C.H., Pfefifer P.A. Pressing diseases that represent promising targets for gene therapy. Discov. Med. 2017;24:313–322. [PubMed] [Google Scholar]
- 2. Volume 4 EU guidelines for Good Manufacturing Practice for Medicinal Products for Human and Veterinary Use Annex 2 Manufacture of Biological active substances and Medicinal Products for Human Use.