2020年，我们曾探讨过欧洲为何仅有11种细胞和基因疗法获批。如今，四年多过去，这一数字已增至20种，几乎翻了一番。然而，这是否真的算得上是巨大的进步呢？反观美国市场，目前已有43种相关疗法获批。

UniQure's Glybera for lipoprotein lipase deficiency was approved a decade ago, but was pulled off the market a few years later on low demand and questions about its effectiveness. Orchard ...

The company secured approval in Europe for Glybera (alipogene tiparvovec) in 2012. The therapy was approved to treat the ultra-rare disease hereditary lipoprotein lipase deficiency (LPLD), and was ...

Earlier this year uniQure withdrew Glybera, the world’s first gene therapy product from Europe after having seen it used in just a handful of patients. Glybera had been priced at 1 million euro ...

The therapy uses an adeno-associated virus vector to carry the gene. Glybera (alipogene tiparvovec): uniQure Glybera (alipogene tiparvovec) uses an adeno-associated virus vector to transfer a copy of ...

Adeno-associated viruses (AAV) have emerged as the lead vector in clinical trials and form the basis for several approved gene therapies for human diseases, mainly owing to their ability to sustain ...

T. R. (2019). Recombinant adeno-associated virus gene therapy in light of Luxturna (and Zolgensma and Glybera): Where are we, and how did we get here? *Annual Review of Virology*, 6, 601-621 ...

This report describes the impact of glycosylation on the pharmacokinetics, pharmacodynamics, therapeutic activity, and production (biomanufacturing) of therapeutic proteins using several examples that ...