The requirements for a multimodal facility can be complex and variable. Timeframe of launch and capacity demand.At-scale manufacturing operations and flow.Particular products successfully licensed.This highlights yet another of the challenges for sponsors of new C> products: The facility and suite design must be flexible to support many existing future unknowns, including the following: The two sources cited for Tables 1 and 2, while contemporary to each other, show a slightly contrasting view of the current VV landscape. AAV vectors are commonly associated with in-vivo gene therapies AdV vectors show promise for vaccine applications including oncolytic virotherapy and LV vectors are commonly associated with such ex-vivo approaches as CAR-T cell therapy. The most successful vectors to date have been adeno-associated virus (AAV), adenovirus (AdV), and lentivirus (LV). Each vector modality presents distinct values in the current range of therapeutic entities, clinical indications, cells to be modified, and evolving manufacturing methods. Tables 1 and 2 exemplify an aspect of this diversity in only the most popular current VV methods. The traditional, rigid facility design approach associated with the well-defined processes of classic products are not meeting the needs of the C> manufacturing field. This diverse landscape and process-specific supply-chain issues are driving the need for highly flexible facilities that may run multiple products and/or production modes. “Viral Vector Platforms Within the Gene Therapy Landscape.” Signal Transduction and Targeted Therapy 6, no. “Gene-Therapy Innovation: Unlocking the Promise of Viral Vectors.” McKinsey & Company website. Table 2: Summary of viral vectors currently being used in clinical trials (c. Table 1: Viral vectors by type in percent of worldwide assets (c. For such reasons, a number of viral vector (VV) systems are currently in place, with many other gene-vector systems in development. This can be challenging for many reasons: it can be difficult to estimate the size and number of polynucleotides to transfer, the efficiency of the vector in the particular cells addressed, the scale of production required, and whether a patient’s immune system will respond to vector particles as a microorganism. In many C> processes, success is dependent on the ability to efficiently deliver new genetic material to the target cells. This article discusses types of facilities and design considerations for C>. For all these reasons, C> have unique needs or require special considerations in manufacturing suites beyond those for classic products. C> is still a relatively young field and therefore continually evolving, which has resulted in diverse research pipelines, entity types, manufacturing technologies, clinical trials, and commercial scale facility designs. These considerations include processing safety (e.g., levels of biological, chemical, and solvent handling safety), multiple scaled-out batches, and the requirement for end-to-end aseptic processing. This can require special manufacturing considerations less common in well-established biopharmaceuticals, such as enzymes or monoclonal antibodies (mAbs). C sponsors currently address numerous emerging pharmaceutical entities, as well as manufacturing platforms, modes, and scale. ![]() Cell and gene therapies are part of advanced therapy medicinal products (ATMPs) and offer great potential for regenerative medicine, including ways to treat and cure a variety of acquired and inherited diseases.
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