Add a new, 100 million California facility to the growing gene therapy infrastructure.

Merck KGaA announced they are opening a second gene therapy and viral vector factory in Carlsbad, California. The new center will be just one point in the global supply chain Big Pharma is rapidly erecting to keep manufacturing capacity for the new technology at pace with clinical development. Over the past year, Novartis, PTC Therapeutics, Pfizer and Vertex each announced or opened new facilities in Switzerland, North Carolina and New Jersey that will help build gene therapy.

In November,Reutersreported that 11 drugmakers had set aside $2 billion for the manufacturing effort. They were led by Novartis, the giant behind the second FDA-approved gene therapy in Zolgensma which planned to spend $500 million, and Pfizer, which has yet to get a gene therapy approved but will spend $600 million. Catalent and Thermo Fisher, meanwhile, each spent over a billion dollars acquiring companies involved in gene therapy or viral vector manufacturing.

Merck KGaA last shored up its gene therapy manufacturing in 2016 the year before the first gene therapy was approved in the US when they expanded their original Carlsbad facility from 44,000 to 65,000 feet. At 140,000 feet, the new facility will be more than double the size of its older neighbor. Gene therapies and the viral vectors used to deliver them will be cooked up in 1,000 liter bioreactors metal vats roughly the size of 260 gallons of milk. Merck has not said when it is scheduled for completion.

Although Merck KGaA lists no gene therapies in its most recent pipeline, the German drugmaker uses its facilities to help biotechs focused on the emerging modality to manufacture their products. The list of approved gene therapies can still be counted on one hand, but hundreds are now in clinical trials.

Manufacturing emerged as a pressing bottleneck almost as soon as Spark Therapeutics got Luxturna, a treatment for a form of inherited blindness, approved as the US first gene therapy. In an op-ed in STAT last year, Sparks head of technical operations, Diane Blumenthal, described the process of building in Philadelphia one of the first in-house gene therapy manufacturing facilities. With regulators increasingly open to approving the treatments quickly, she encouraged other companies to invest in manufacturing in advance of even knowing if the therapy works, particularly because gene therapy requires customization in ways few other modalities do.

And she warned the problem will only get more pressing.

There isnt a gene therapy manufacturing playbook yet to guide the development of gene therapies, Blumenthal wrote. Manufacturing a gene therapy is only half the battle. The other half is making enough of it, doing that as efficiently as possible, and getting it to the patients who need it. These challenges become even more urgent to tackle as the industry shifts to the next chapter in gene therapy development, from treatments made in small batches for small patient populations to bigger volumes for larger rare-disease populations and commercial scale.

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Merck KGaA adds to gene therapy manufacturing boom with 100M facility in California - Endpoints News