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Here, Dr Jinpeng Li at WuXi AppTec reveals the analytical challenges that are hindering advancement of oligonucleotide therapeutics, medicines which have demonstrated clinical promise in the gene therapy space, and discusses promising analytical innovations.
The committee’s approval of the one-time gene therapy means the healthcare inequalities for patients with this genetic blood disorder can begin to be re-addressed.
Cell and Gene Therapy Catapult's (CGT Catapult) findings attest to the UK remaining “a highly attractive destination for clinical trials”.
The positive two-year findings from the Phase I/II gene therapy trial in retinitis pigmentosa reiterate the potential of mutation-agnostic therapies.
Eleven drugs are poised to make waves in the pharmaceutical industry in 2025, according to the annual report.
Overcoming Challenges in Cell and Gene Therapy Containment.
30 October 2024 | By Charles River
Webinar presented by Miriam Guest, Senior Principal Scientific Advisor at Charles River, to explore how ATP-bioluminescence outperforms traditional sterility testing in complex matrices.
This Feature Partnership discusses AAVs and how manufacturers can overcome the key development challenges and create a scalable AAV production platform.
The new manufacturing facility in Finland will aid global supply of the first FDA-approved intravesical non-replicating gene therapy for adults with a common cancer.
9 September 2024 | By FUJIFILM Wako
Watch this webinar to learn about the current regulations on the monocyte activation test (MAT) and the new type of MAT entering the markets.
Some of the rare disease participants experienced fast and sustained improvement in their eyesight during the clinical trial, data shows.
4 September 2024 | By Rapid Micro Biosystems
Watch on demand to learn how a new single medium, Rapid Sterility Medium (RSM), performs as well as or better for the growth promotion of test microorganisms compared to compendial sterility test media.
This article summarises some of the recent notable drug approvals in the EU and US, including a gene therapy and an enzyme replacement therapy.
The single-dose gene therapy was approved for certain patients with haemophilia B in the US and Canada earlier this year, marketed as BEQVEZ.
In this article, Roger Palframan, Head of US Research at UCB, delves into the potential of gene therapy and which modalities have the most promise, what the industry should prioritise to advance the field, as well as what is needed to develop the workforce.
