whitepaper
One HEK of a viral vector to develop
Viral vector manufacturing presents many challenges related to HEK cell line development, fluid transfer and product viability in the cold chain.
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Gene therapy
article
Modifier gene therapy – clinical development and manufacturing considerations
Expanding on his earlier podcast discussion with EPR, Dr Arun Upadhyay, Chief Scientific Officer and Head of Research & Development at Ocugen, discusses the company’s promising modifier gene therapy candidates for ophthalmic disorders.
article
Fresh or frozen? Navigating the cryopreservation dilemma for CGT
Cell and gene therapy development grapples with a paradox: fresh starting material is often equated with higher-quality drug products, but cryopreservation is crucial for scale-up. In fact, every approved autologous cell-based therapy relies on freezing cells. Reconciling these truths can be key for the success of any commercial therapy based on…
news
Oligonucleotide ALS therapy approved in EU
The European Commission (EC) has granted Biogen its third rare disease treatment approval in the European Union.
article
Pioneering AAV gene therapy for Krabbe disease
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.
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Pharma Horizons: Cell and Gene Therapy
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.
webinar
Pharmaceutical continuous manufacturing: what’s next for industry?
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.
news
“Landmark” genetic blindness CRISPR trial outcomes released
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.
news
Enhancing medicine manufacturing through digitalisation
Under a new initiative, manufacturers could increase productivity via innovative technologies, enabling faster patient access to new medicines.
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.
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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.
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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.
