news
Early-phase ATMP clinical trials see promising growth
Cell and Gene Therapy Catapult's (CGT Catapult) findings attest to the UK remaining “a highly attractive destination for clinical trials”.
List view / Grid view
Gene therapy
news
Long-term potential revealed for novel modifier gene therapy
The positive two-year findings from the Phase I/II gene therapy trial in retinitis pigmentosa reiterate the potential of mutation-agnostic therapies.
article
Potential blockbuster drugs to watch in 2025
Eleven drugs are poised to make waves in the pharmaceutical industry in 2025, according to the annual report.
whitepaper
Cell and Gene Therapy Containment
Overcoming Challenges in Cell and Gene Therapy Containment.
webinar
When Innovative Formulations Cloud Sterility Testing
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.
article
Quality from the start: designing a scalable AAV production platform
This Feature Partnership discusses AAVs and how manufacturers can overcome the key development challenges and create a scalable AAV production platform.
news
European manufacturing facility to strengthen gene therapy supply
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.
webinar
Monocyte activation test (MAT): Insight into examining regulations and markets prospects
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.
news
Gene therapy trial facilitates major vision improvement
Some of the rare disease participants experienced fast and sustained improvement in their eyesight during the clinical trial, data shows.
webinar
An alternative medium to support sterility testing using the Growth Direct® Rapid Sterility System
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.
article
Regulating therapies for rare diseases – recent approvals
This article summarises some of the recent notable drug approvals in the EU and US, including a gene therapy and an enzyme replacement therapy.
news
Pfizer receives EU haemophilia B gene therapy approval
The single-dose gene therapy was approved for certain patients with haemophilia B in the US and Canada earlier this year, marketed as BEQVEZ.
article
The route to enhanced gene therapies
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.
whitepaper
Application note: Automated nucleic acid amplification assay for mycoplasma detection in cell and gene therapy products
Ambili Menon, Pharma Quality Control, bioMérieux Inc. addresses the task of microbiological testing for advanced therapeutic medicinal products.
article
Drug approval roundup – May/June 2024
This article highlights some of the key regulatory approvals granted in May and June in the EU and US so far this year, including for oligonucleotide and biologic therapies.
