news
Enhancing medicine manufacturing through digitalisation
Under a new initiative, manufacturers could increase productivity via innovative technologies, enabling faster patient access to new medicines.
List view / Grid view
Gene therapy
podcasts
EPR Podcast 24 – Developing modifier gene therapy – Ocugen
In this podcast, Dr Neena Haider, Faculty at Harvard Medical School and Dr Arun Upadhyay, Chief Scientific Officer at Ocugen, discuss the development of modifier gene therapy for ocular diseases.
news
Novel haemophilia B gene therapy approved
Pfizer has attained its first approval for a gene therapy from the US Food and Drug Administration (FDA), which is indicated to treat the rare blood disorder haemophilia B.
news
CGT Catapult appoints new Non-Executive Directors
The new members of the Cell and Gene Therapy Catapult’s board hold expertise across therapy development, digitalisation and leadership.
article
European Pharmaceutical Review Issue 2 2024
European Pharmaceutical Review Issue 2 includes articles on cell and gene therapy, from supply chain and manufacturing to microbial testing.
article
Supporting female leaders to advance cell and gene therapies
At the 2024 Advanced Therapies conference, panellists discussed the lack of women in leadership positions within the cell and gene therapy space and what could be contributing to this.
news
AAV gene therapy demonstrates positive activity in Duchenne muscular dystrophy
Initial trial results show the gene therapy enabled up to 85 percent of muscle fibres expressing microdystrophin in boys with Duchenne muscular dystrophy eight weeks post-injection.
news
CRISPR technologies fuelling haematological innovations
Greater use of CRISPR-based therapies in clinical trials is expected to drive further advancements in precision medicine, GlobalData states.
article
Championing UK ATMP clinical trials
In this Q&A, Jacqueline Barry, Chief Clinical Officer for Cell and Gene Therapy Catapult, elucidates on how the Advanced Therapy Treatment Centre (ATTC) Network can strengthen UK ATMP clinical trials.
news
One-time gene therapy could improve wet AMD
A Phase I/IIa trial suggests that a subretinal gene therapy for wet age-related macular degeneration (wet AMD) could reduce the need for patients to receive anti-VEGF injections.
video
Moving towards continuous batch RNA production
Here Thibault Jonckheere, CEO, Exothera discusses how continuous RNA production can set a new benchmark in the pharmaceutical industry.
webinar
Comprehensive evaluation of Growth Direct® Rapid Sterility for microorganism detection
22 March 2024 | By Rapid Micro Biosystems
Discover the extensive panel testing done to evaluate the new, innovative sterility testing platform, Growth Direct® Rapid Sterility.
news
FDA approves innovative gene therapy for MLD
Following US FDA approval of Lenmeldy™ (atidarsagene autotemcel) for early-onset metachromatic leukodystrophy (MLD), the US wholesale acquisition cost of the gene therapy has been set to $4.25 million.
article
ebook: A practical guide to single-use filtration in biopharma
This ebook explains how single-use filtration can support safer, more efficient biopharmaceutical production. Discover more on different filtration stages and unveil key characterisations, like pore size and membrane material, empowering tailored selection to specific biomolecules and processes.
article
Pharma Horizons: Environmental Monitoring
This report is dedicated to exploring some of the latest advances in pharmaceutical environmental monitoring, covering topics from contamination control and cleanroom validation through to emerging technologies for bacterial endotoxin testing.
