All Advanced therapies manufacturing articles – Page 5
-
NewsPfizer 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.
-
ArticleThe 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.
-
News‘Accidental’ media switch leads to CAR T therapy discovery
The investigators hope that their new approach could lead to large-scale manufacturing of the CAR T cells for blood cancer patients.
-
WhitepaperApplication 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.
-
ArticleDrug 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.
-
WhitepaperOne 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.
-
ArticleModifier 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.
-
ArticleAdvancing targeted treatments for multiple myeloma
Edmond Chan, Senior Director, EMEA Therapeutic Area Lead, Haemato-Oncology, Johnson & Johnson Innovative Medicines, offers insight into the promising evidence of cell therapies and biologic-based treatments for patients with multiple myeloma.
-
ArticleFresh 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 ...
-
NewsOligonucleotide ALS therapy approved in EU
The European Commission (EC) has granted Biogen its third rare disease treatment approval in the European Union.
-
ArticlePioneering 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.
-
ReportPharma 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.
-
NewsImproving stem cell transplantation success in high-risk blood cancers
The new medicine is expected to enable over 99 percent of people from a wide range of racial/ethnic groups to find a donor, research says.
-
ArticleProgressing personalised cell therapies in oncology
Dr Imran Khan, PhD, Vice President, Medical Affairs, Hematology, Johnson & Johnson Innovative Medicine discusses the major benefits and current challenges of CAR-T cell therapies, as well as the potential of the company’s novel BCMA-targeted treatment for multiple myeloma.
-
NewsCell therapy could help curb progression of heart failure
New clinical trial data demonstrates the potential for a stem cell treatment as a cardiac regenerative therapy.
-
NewsAddressing drug delivery challenges of emerging drug modalities
The new company will work to develop solutions to overcome issues within new therapeutic modalities, in areas such as formulation, manufacturing and supply chain.
-
WebinarPharmaceutical continuous manufacturing: what’s next for industry?
During this virtual panel, industry experts will discuss adoption of continuous manufacturing in the pharmaceutical industry.
-
NewsChallenges and opportunities in the CGT supply chain
This article explores the unique logistics and supply chain challenges of cell and gene therapy development and the future potential of this innovative sector.
-
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.
-
NewsEnhancing medicine manufacturing through digitalisation
Under a new initiative, manufacturers could increase productivity via innovative technologies, enabling faster patient access to new medicines.