The contract manufacturing industry is lagging behind in capacity for gene therapies at a time when gene therapies are poised for a wave of approvals, GlobalData analysis shows.
Contract Manufacturing Organisations (CMOs) are recognising the coming demand for gene therapies and have started to build production capacity, but compared with other biologic therapies such as monoclonal antibodies, there are far fewer gene therapy contract manufacturing sites worldwide: the GlobalData Contract Service Providers database shows 99 such facilities worldwide, owned by 73 companies.
Only six CMOs (including those with an excess capacity business model) have more than two gene therapy facilities worldwide. Hitachi Chemical Co. and Lonza have the most gene therapy facilities, with four each (Figure 1). Next by number of facilities are the Thermo Fisher Scientific (Waltham, MA, US) subsidiary Brammer Bio (Cambridge, MA, US, acquired in May 2019; B/POR, May 2019) and the Merck KGaA (Darmstadt, Germany) dedicated contract CMO subsidiary BioReliance (Rockville, MD, US), with three each. Notably, dedicated contract CMOs take the top four spots for number of gene therapy facilities. The excess capacity CMOs with the greatest number of gene therapy sites are Oxford BioMedica (Oxford, UK) and Biogen (Cambridge, MA, US).
Figure 1: Top gene therapy CMOs by number of gene therapy facilities
Four of these six top players are large-cap companies or subsidiaries of large-cap companies ($10–200bn), showing the high capital outlay and running costs needed for gene therapy manufacturing. The exceptions are Hitachi Chemical Co. (Tokyo, Japan; mid-cap: $2—10bn) and Oxford BioMedica (small-cap: $200m–$2bn).
A full three-quarters of gene therapy CMOs possess only one gene therapy facility, indicating this service offering is still niche and not considered a core service area by service providers. However, GlobalData anticipates demand for gene therapy manufacturing services will ramp up dramatically with a coming wave of gene therapy approvals.
Currently, two gene therapies are FDA-approved and marketed in the US: Spark Therapeutics’ (Philadelphia, PA, US) Luxturna and AveXis’s (Bannockburn, IL, US; a subsidiary of Novartis [Basel, Switzerland]) Zolgensma. The EMA has approved Luxturna and Orchard Therapeutics’ (London, UK) Strimvelis. The forthcoming GlobalData PharmSource report Gene Therapy Market Opportunity for CMOs – 2019 Edition, to be published in Q4, forecasts in detail a significant number of gene therapy approvals from the current clinical pipeline, as well as the accompanying manufacturing burden.
Sponsors of clinical-stage gene therapies are predominantly nano, micro, and small-cap companies, the GlobalData Drugs and Clinical Trial database show. These companies typically lack the capabilities to manufacture gene therapies in-house, amounting to a large pool of potential clients for those CMOs that are adequately equipped. (Fewer than a quarter of Phase I-III gene therapy trials between January 1, 2013 and May 18, 2019 were sponsored by private companies, more than half were sponsored by public companies, and the remainder were sponsored by institutions or multiple sponsors, according to the aforementioned GlobalData Gene Therapy report.)
Notably, for gene therapy manufacturing, dedicated contract facilities far outnumber excess capacity facilities by more than three to one. This is an indication that dedicated CMOs are willing to pick up the risk and cost of investing in gene therapy technology where gene therapy sponsors often are not. (Excluded from this analysis are facilities belonging to gene therapy marketing authorisation holders which manufacture solely proprietary products, and do not operate an excess capacity model; these facilities are not covered by the GlobalData Contract Service Provider database. Such facilities are rare, such as bluebird bio’s lentiviral vector facility in North Carolina, B/POR, April 2019.)
Cell & Gene Therapy Coverage on Pharmaceutical Technology supported by Cytiva.
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