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AAV Gene Therapy Production

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uniQure Presents New Data Demonstrating Its Large-Scale Manufacturing Capabilities at the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting

~ Commercial Capacity for AAV Gene Therapy Production Significantly Enhanced by Highly Scalable 500 Liter Reactors ~

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Oct. 17, 2018 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today presented new data demonstrating its ability to manufacture gene therapies using a 500 liter single-use, stirred tank reactor. This presentation was featured at the European Society of Gene and Cell Therapy (ESGCT) 26st Annual Meeting in Lausanne, Switzerland.

uniQure produces its adeno associated virus (AAV)-based gene therapies in its state-of-the-art, Lexington-based manufacturing facility using a proprietary baculovirus expression vector system (BEVs). The company recently initiated the development of a 500 liter stirred tank reactor process that has the potential to significantly increase manufacturing capacity and enhance scalability. Analysis of drug substance produced using the 500 liter stirred tank reactor showed that overall process performance, biological activity, content and product purity were comparable to material produced by smaller scale, wave-based reactors.

“AAV is becoming a vector of choice for gene therapy applications, leading to increased demand to advance new therapies for larger patient populations,” stated Scott McMillan, Ph.D., uniQure’s chief operating officer. “We believe uniQure’s proprietary insect cell-based platform provides significant advantages in scaling production for commercial use. Based on our achievements to date, we believe uniQure has the capability to support the manufacturing needs across its expanding pipeline of gene therapy product candidates.”

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Het blijft lastig om aan deze asset "large scale manufacturing capabilities" een waarde toe te kennen.
Moet het geschaard worden in de categorie ASML met haar unieke productieproces, en dan alleen produceren voor ASML zelf?
Of een inhuurbaar productieproces voor externe bedrijven, een manufacturing uniek vanwege patenten?

Mijns inziens lijken de huidige beleggers nog geen waarde aan deze asset te hebben gegeven.
En toch geeft uniQure keer-op-keer zo hoog op van hun manufacturing capabilities.
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