One-time gene therapy could “transform” Huntington’s treatment landscape
uniQure’s positive topline data “are the most convincing in the field to date” and indicate the gene therapy’s potential disease-modifying effects.
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uniQure’s positive topline data “are the most convincing in the field to date” and indicate the gene therapy’s potential disease-modifying effects.
Advances cell therapy and gene therapy candidates, with both showing neurorestorative potential against the disease.
This in-depth focus features articles on cell and gene therapy in Europe and enhancing drug delivery with cost-effective solutions.
EPR Issue 3 includes articles on microbiology, downstream processing, manufacturing, drug formulation and more.
2 September 2025 | By FUJIFILM Wako Europe GmbH
Join this webinar to find out how RT-rt PCR is revolutionising sterility testing and discover a unique approach that provides reliable one-day results and enhanced sensitivity and detection capabilities.
Will expand the company’s CAR T-cell therapy footprint and gain access next generation in vivo technology.
The German pharma company’s BioXcellence arm will supply the drug substance for AnGes’ peripheral arterial disease treatment Collategene.
Further to its potential in Stargardt disease, if approved, the one-time gene therapy could become standard of care for retinitis pigmentosa, alongside other retinal degenerative diseases.
CBER head makes surprise comeback just weeks after his abrupt resignation.
11 August 2025 | By Entegris
This webinar explores the design and implementation of pre use post sterilisation integrity test assemblies (PUPSIT) in sterile filtration processes to ensure regulatory compliance while minimising operational risk.
But the impact of George Tidmarsh’s CBER appointment after Vinay Prasad’s abrupt resignation remains to be seen.
The landmark regulation establishes a future where highly personalised treatments are part of routine care, says MHRA’s Chief Executive.
The study represents the first time the injectable gene therapy has been evaluated in teenagers and adults.
In this article, Stacey Ramsey, Senior Manager – Microbial Applications Lab, Charles River Laboratories, discusses the modernisation of rapid microbiological methods (RMMs) and the potential of ATP-bioluminescence when testing cell-based products.
2 July 2025 | By MasterControl
This webinar explores innovative, purpose-built AI solutions that elevate compliance and efficiency in quality and manufacturing operations.