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Dr Maria Escolar, Chief Medical Officer, Forge Biologics, explores the advantages of the company’s novel AAV gene therapy for Krabbe disease, which could help to overcome some of the immune and safety challenges the gene therapy sector is facing.
This report is dedicated to exploring some of the latest innovations in the cell and gene therapy space, from development and quality control, through to manufacturing and clinical trials.
13 May 2024 | By European Pharmaceutical Review, sponsored by MasterControl
During this virtual panel, industry experts will discuss adoption of continuous manufacturing in the pharmaceutical industry.
A Phase I/II trial has shown that a CRISPR-based gene editing therapy can be safely delivered to the retina and provide clinically meaningful outcomes.
Under a new initiative, manufacturers could increase productivity via innovative technologies, enabling faster patient access to new medicines.
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.
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.
The new members of the Cell and Gene Therapy Catapult’s board hold expertise across therapy development, digitalisation and leadership.
European Pharmaceutical Review Issue 2 includes articles on cell and gene therapy, from supply chain and manufacturing to microbial testing.
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.
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.
Greater use of CRISPR-based therapies in clinical trials is expected to drive further advancements in precision medicine, GlobalData states.
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.
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.
Here Thibault Jonckheere, CEO, Exothera discusses how continuous RNA production can set a new benchmark in the pharmaceutical industry.
