5.1.1 CRISPR gene-editing technology has brought transformative capabilities to cell therapy, particularly in enhancing CAR-T cell production. One of CRISPR’s most valuable features is its ability to make multiple edits simultaneously, significantly increasing production efficiency. CRISPR can also be combined with viral vectors for genetic material delivery, enabling multiple modifications that enhance both the safety and performance of cell therapies. Such combinatorial approaches open new possibilities for treating complex diseases with multiple genetic factors. Additionally, gene-editing tools are being used to develop allogeneic therapies by removing genetic markers that trigger immune rejection.
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5.2.1 One of the primary goals in cell therapy is to develop off-the-shelf cell therapies that can evade immune rejection. Currently, encapsulating cells in biomaterials — such as zwitterionic or semipermeable hydrogels — is a common strategy to prevent the immune system from identifying donor cells as foreign. While effective to some degree, the survival of encapsulated cells is often limited by the foreign body response (FBR). Macrodevices for cell encapsulation provide better control and enable retrieval post-implantation, but their size can restrict nutrient and oxygen access, reducing cell viability.
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5.3.1 Translating developmental cell therapies into commercially viable products requires navigating numerous challenges, many of which are shared with gene therapies.[7] While some cell therapy candidates benefit from accelerated approval pathways, this often reduces the time available for developing processes, analytical methods, and formulations — an added challenge in this emerging field.[9]
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5.4.1 Even if 50–75 cell and gene therapies receive FDA approval by 2030, questions remain as to whether they will achieve commercial success. Safety issues with different viral vector gene delivery vehicles that have occurred in the past few years must also be addressed. Demonstration of long-term safety and efficacy must be achieved as well.
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5.5.1 For innovators whose cell or gene therapy achieves regulatory approval, commercial viability is the final hurdle to leap on the path to success. The costs involved in manufacturing these therapies are significant, and attributing value to potentially curative therapeutics is undeniably complex, requiring the balance of factors as diverse as reduced hospitalization, remission periods, patient-reported benefits, and even the cost of managing future comorbidities if the patient’s life is extended.[124]
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5.6.1 Artificial intelligence (AI) and machine learning (ML) have the potential to help overcome challenges in the cell and gene therapy field, including their complexity and heterogeneity with respect to therapeutic approaches, the supply chain, logistics, and so on. Companies looking to leverage these digital tools for cell and gene therapy development include Modulus Therapeutics, Outpace Bio, Serotiny, Dyno Therapeutics, and Patch Biosciences.[126]
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5.7.1 In cell therapy, synthetic biology allows for the creation of engineered regulatory circuits that establish new “input–output” relationships, enabling cells to respond to specific molecules or external cues to produce desired therapeutic effects. For example, synthetic circuits can control the level or timing of gene and protein expression in response to small molecule drugs. These circuits can also be designed with closed-loop sensing and response mechanisms, allowing cells to produce anti-inflammatory agents when inflammatory cytokines are detected.
5.7.2 Additionally, synthetic biology techniques can enhance CAR-T cell therapies. By incorporating engineered cell-surface receptors, scientists can control CAR-T cell activity, minimizing CRS and improving the specificity of tumor targeting, making CAR-T cells more effective for treating solid tumors.
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5.8.1 Despite being the established standard for delivering therapeutic DNA to cells both in vivo and ex vivo, viral vectors do have disadvantages, of which the complexity and cost of their manufacture is only one. Other notable disadvantages include concerns around toxicity and immune response; the latter being particularly problematic for AAV-based gene therapies that can currently only be administered once, since the patient’s immune system would fight a re-dose before it had time to exert any therapeutic effect. This can also exclude some patients from even a first dose using some AAV serotypes if they have had previous natural exposure to the virus.
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5.9.1 One of the ongoing challenges in the biomanufacturing industry is a serious labor shortage. The National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) is a public–private partnership that was founded in 2017 to study, highlight, and address the challenges in the industry.[129] In 2022, NIIMBL published a study in which they surveyed manufacturers across the U.S. and found the talent shortage to be ubiquitous.[130] While the shortage is felt across all levels of biomanufacturing organizations, it is striking that only 42% of job postings for manufacturing associates require a bachelor’s degree.[130] Traditionally, firms across industries will pay relocation fees for high-level employees such as managers and above, but relocation support for entry-level employees without a college degree is rare. Companies are left, then, to recruit from local talent for entry-level manufacturing associates jobs while they simultaneously compete nationally (or even globally) for more educated talent.
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5.10.1 In the last few years, the pace of innovation in cell and gene therapy manufacturing has been unremitting, bringing several possible solutions to the challenges associated with cost-effective manufacture of advanced therapy medicinal products (ATMPs) closer to clinical reality.
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