Genetic Engineering & Biotechnology News

NOV15 2017

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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Genetic Engineering & Biotechnology News | GENengnews.com | NOVEMBER 15, 2017 | 25 Ronald Rader Cell and gene therapies are relatively new ar- eas for commercial biopharmaceutical devel- opment. These sectors involve relatively new applications of both old technologies used for decades (e.g., bone marrow stem cell transplants), and many new technologies. These sectors also are populated by many new players, including startup companies— but also much more than the broader bio- pharmaceutical industry—these sectors cur- rently involve academic/nonprofit research organizations performing much R&D and even early product development. The overall situation with cell and gene therapies is rather similar to that of the bio- pharmaceutical industry about three decades ago, in the mid-1980s: just a few pioneering products entering the market; information about these industry sectors not yet existing or of poor quality; information being scat- tered and not readily retrieval; lack of good information slowing commercial progress (e.g., investors finding it difficult to justify in- vestments); and available manufacturing ca- pacity to handle projected commercial man- ufacturing insufficient for near-term needs. Plus, "capacity crunches" are getting worse as more products enter the market. The information resources currently avail- able concerning cell and gene therapies in commercially relevant development remain limited at best. This includes information on developers, technologies, manufacturing ac- tivities, and capabilities. There are wide varia- tions in reporting of the number and types of organizations actively developing products, and in the reported size and composition of these industry sectors. Identifying cell/gene therapy developers or products in commercial development is difficult—there are different organizations and products reported, with many new ones and many missing in each source. Part of this problem, is simply due to definitional issues that can confound report- ing about cellular and gene therapies. There is a lack of information on the pipeline, players involved, and organiza- tions—such as contract manufacturing or- ganizations (CMOs)—providing R&D and manufacturing services. While this is nor- mal for most new industry sectors, cell and gene therapies are not technically new. Gene therapies have been around and even in commercial development for decades now, but safety, inadequate technology and other problems have held back commercialization. Cellular therapies have been in use for de- cades, although these were not developed nor approved as products (in the United States). Instead, most of these types of thera- pies are considered medical procedures, such as bone marrow (stem cell) transplants. Those involved in cell therapy manufactur- ing are clearly concerned about how future commercialization will be undertaken. For example, In our global industry survey, 14th Annual Report on Biopharmaceutical Manufacturing Capacity and Production, we found that respondents are now begin- ning to see that cell therapy manufacturing is an merging concern, with 6.2% of global respondents indicating it is the single most important trend (Figure). While this overall percentage is relatively small, when consider- ing that the cell therapy segment represented in the study is relatively small, we can infer that manufacturing may be a top-of-mind concern for a great many of those who are involved in that segment. BioPlan preliminary estimates show there are about 150 cellular therapy and 150 gene therapy commercial developers that are targeting the U.S. and other major markets (with considerable overlap). But much as with the very earliest days of genetic engi- neering, many of these include universities and nonprofit research organizations in- volved in early R&D and trials. The great majority of the >1,000 ongoing cell/gene therapies clinical trials (another area with poor information and definition) primarily only have proof-of-principle–level evidence that a therapy approach has promise or is not obviously unsafe. Capacity Crunches May be Coming Since the mid-1980s, when the biophar- maceutical industry was just moving into an- tibody manufacturing, requiring large-scale manufacturing facilities, there have been sev- eral serious "capacity crunch" periods where dire shortages of capacity were projected for upcoming waves of mAb manufacture. The industry generally managed to avoid major problems (with some notable exceptions). Rapid construction of facilities, a steady increase in upstream titers, adoption of single- use systems, and other factors allowed indus- try to avoid major capacity crunches. But the cell and gene therapies industries are different, and it clearly appears to be on a trajectory for major capacity crunches in both R&D and commercial manufacturing. For some, capacity crunches for both cellular and gene therapies may already be here. The wait times for CMOs to start proj- ects now average ≥1.25 years, and are only increasing, with insufficient CMOs with technical competence entering the field. Fur- ther, new dedicated facilities have yet to be constructed, and staffing is likely to create a critical bottleneck. BioPlan Associates esti- mate that the current shortfall in worldwide cell/gene therapy may be about 5× current ca- pacity (based on cumulative bioreactor volu- metric capacity)—in other words, that much more CMO capacity would be used if avail- able. And within perhaps five years when current pipeline products reach clinical scale, unless significant changes occur, the required platforms and capacity will not be there. Further, the current cell and gene thera- pies tend to be super-orphan products, with limited market demand. But once more mainstream therapies enter the market, in- cluding diverse gene therapy viral-vector and allogeneic cell therapies—unless manufac- turing platforms are developed, along with single-use technologies, and the staffing for these facilities are ramped up, the demand for capacity may dramatically outstrip sup- ply. Many companies with projects now set for approvals may find production hurdles, and others may not yet be able to enter the fields due to lack of CMO access. Keep in mind that CMOs are a likely resource for avoiding the costs, complexities, and risks involved in developing the specialized in- Leading the Data Enabled World The first peer-reviewed journal to connect a world where the pace from research to implementation is ever accelerating The go-to platform for: ® Advances in new big data technologies ® Discussion of unique and innovative big data ideas ® Problems, challenges, and security issues in managing big data ® Sharing and dissemination of world-class data science expertise www.liebertpub.com/big See Cell and Gene Therapies on page 26 Cell and Gene Therapies: Industry Faces Potential Capacity Shortages Perspectives Bioprocessing Ronald Rader (info@bioplanassociates. com) is senior director of technical research at BioPlan Associates. Website: bioplanassociates.com. Cell and Gene Therapy Manufacturing May Be Facing a Major Capacity Crunch Figure. Single Most Important Biomanufacturing Trend or Operational Area, 2017 (Selected Responses) Source: BioPlan Associates 16.4% 12.3% 6.2% 11.0% Manufacturing Productivity/Efficiency Continuous Bioprocessing–Downstream Single-Use Systems: Implementation and Integration Cell Therapy In BioPlan's global industry survey, the company reported that 6.2% of respondents indicated that cell therapy manufacturing is the single most important biomanufacturing trend in 2017.

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