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The last year has seen intense investment activity into raising cell and gene therapy manufacturing capacity.
The successes of winning regulatory approval from FDA and further approvals in Europe have sparked an increase in the further development of new cell and gene therapies, many of which aim to treat difficult-to-treat or previously untreated diseases. As a result of a growing clinical pipeline for these therapies, manufacturing capacity on the market is tight. Biopharmaceutical companies, contract manufacturing organizations (CMOs), and contract development and manufacturing organizations (CDMOs) have turned an interested eye toward boosting manufacturing capacity. A summary of some of the manufacturing investments made during 2019 and up to the present in the cell and gene therapy fields follows.
Fujifilm.In January 2019, Fujifilm announced that it will add current good manufacturing practices (CGMP) aseptic filling of recombinant proteins to its existing fill/finish services at its Fujifilm Diosynth Biotechnologies (FDB) facility at College Station, TX, by March 2021 (1). The services will also apply to gene therapy fill/finish. The company previously announced that it would start offering gene therapy fill/finish services. Fujifilm followed this up with a $55-million investment to establish a new gene therapy innovation center adjacent to its FDB CDMO’s existing gene therapy manufacturing facility at College Station (2). The new 60,000-ft2 innovation center will house state-of-the-art upstream, downstream, and analytical development technologies, the company announced in November 2019. The facility will be operational in autumn 2021.
GE Healthcare.GE Healthcare Life Sciences commercially launched a KUBio box in October 2019 intended to accelerate the production of viral-vector-based gene therapies and increase capacity in the viral vector space (3). The new KUBio box features a Germfree biosafety level 2 modular bioprocessing environment and is equipped with the FlexFactory single-use biomanufacturing platform, which has been tailored specifically for the production of viral vectors, according to the company.
Hitachi Chemical.Hitachi Chemical Advanced Therapeutics Solutions (HCATS), a subsidiary of Hitachi Chemical and the North America representative of Hitachi Chemical’s Regenerative Medicine Business Sector, opened a new commercial-scale cell and gene therapy manufacturing facility in Allendale, NJ, in late January 2020 (4). The new facility houses six classified environment rooms and has the capacity to add more rooms that can be specifically configured according to need. The facility includes manufacturing and development labs as well as quality control and microbiology labs.
Nationwide Children’s Hospital.Nationwide Children’s Hospital, based in Columbus, OH, in conjunction with key partners created an affiliate company named Andelyn Biosciences in January 2020 to manufacture gene therapy products. The new company is scheduled to begin operations in 2023 (5). The new startup will establish the first commercial-scale CGMP clinical manufacturing facility in Ohio devoted to gene therapies, according to Nationwide Children’s Hospital. The new company is the result of the hospital’s clinical manufacturing success and work with gene therapy via its Abigail Wexner Research Institute (AWRI). AWRI has operated a small-scale CGMP gene therapy manufacturing facility on-site for several years focused on early-stage gene therapy products for Phase I and II clinical trials. Beginning in summer 2020, the current facility will manufacture products compliant with Phase III clinical trial regulations while the new commercial-scale manufacturing site under Andelyn Biosciences is being built and validated.
Novartis.Novartis opened its new cell and gene therapies manufacturing facility in Stein, Switzerland, in November 2019 following on the heels of the company’s first successfully completed clinical production of a cell and gene therapy batch at Stein in September 2019 (6). The new facility houses manufacturing areas for novel chimeric antigen receptor T cell (CAR-T) cell therapies in addition to hosting production of innovative, difficult-to-manufacture solid dosage forms.
Pfizer.In August 2019, Pfizer announced that it is investing $500 million to construct a new gene therapy manufacturing facility in Sanford, NC (7). The facility is expected to support Pfizer’s ongoing investment in gene therapy R&D. The facility would increase Pfizer’s ability to supply both clinical- and commercial-scale quantities of gene therapies, specifically, highly specialized, potentially one-time gene therapies that use custom-made recombinant adeno-associated virus (rAAV) vectors.
The Discovery Labs.The Discovery Labs, an MLP Venture company and provider of CGMP manufacturing, turnkey laboratory solutions, critical materials, and office space, formed a new CDMO and specialty investment company in January 2020 in partnership with Deerfield Management, a healthcare investment management firm. Under the new CDMO, named The Center for Breakthrough Medicines, The Discovery Labs and Deerfield will invest $1.1 billion to build a manufacturing facility for cell and gene therapies in King of Prussia, PA, which is expected to provide instant capacity to the market (8). The new CDMO will provide manufacturing from preclinical- through to commercial-scale of cell therapies, gene therapies, and component raw materials. It will also offer process development, plasmid DNA, viral vectors, cell banking, cell processing, and support testing capabilities, all housed under one roof. Renovations are also underway at the King of Prussia site to construct a total of 86 plasmid, viral-vector production, universal cell processing, CGMP testing, process development, and cell banking suites.
Thermo Fisher Scientific.Thermo Fisher Scientific opened its new $90-million viral vector CDMO site in Lexington, MA, in December 2019 (9). The 50,000-ft2 facility will support development, testing, and manufacture of viral vectors for gene and cell therapies. The new facility adds much-needed capacity for viral vector development and manufacturing, which has been a bottleneck area for biotech companies, according to Thermo Fisher, which has noted that the demand for new gene therapies has outpaced capacity. The investment is expected to accelerate commercialization of new cell and gene therapies by providing a range of services-from drug development through clinical trials to commercial manufacturing.
1. Fujifilm, “Fujifilm Announces the Extension of Its Fill Finish Services to Include Recombinant Protein Aseptic Filling Market,” Press Release, Jan. 15, 2019.
2. Fujifim Diosynth Biotechnologies, “Fujifilm Diosynth Biotechnologies Announces $55-Million USD Investment to Expand Gene Therapy Development Capabilities,” Press Release, Nov. 14, 2019.
3. GE Healthcare, “New KUBio Box for Viral Vectors Boosts Gene Therapy Manufacturing,” Press Release, Oct. 28, 2019.
4. PCT, “Hitachi Chemical Opens New Cell and Gene Therapy Manufacturing Facility,” Press Release, Jan. 30, 2020.
5. Nation Wide Children’s Hospital, “Nationwide Children’s Hospital Announces Plans for Andelyn Biosciences,” Press Release, Jan. 13, 2020.
6. Novartis, “Novartis Opens Facility for Innovative Cell and Gene Therapies in Switzerland,” Press Release, Nov. 28, 2019.
7. Pfizer, “Pfizer Invests Half Billion Dollars to Advance State-of-the-Art Gene Therapy Facility in Sanford, North Carolina,” Press Release, Aug. 21, 2019.
8. The Discovery Labs, “The Center for Breakthrough Medicines is Building the World’s Largest Cell and Gene Therapy Contract Development and Manufacturing Organization (CDMO) to Launch in King of Prussia, PA,” Press Release, Jan. 22, 2020.
9. Thermo Fisher Scientific, “Thermo Fisher Scientific Opens $90-Million Viral Vector Manufacturing Site in Massachusetts,” Press Release, Dec. 4, 2019.
Vol. 44, No. 3
When referring to this article, please cite it as F. Mirasol, “Industry Pushes Forward with Cell and Gene Therapies,” Pharmaceutical Technology 44 (3) 2020.