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Novo Nordisk investing in rare disease with major new manufacturing site
A total of DKK 8.5 billion will fund the new production facility in Odense, Denmark, which is set to facilitate the manufacture of medicines for rare diseases.
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Gene therapy: a radical pharmaceutical revolution
Karen Pinachyan, Head of Medical Affairs Europe at CSL Behring provides an overview of past and present gene therapy development, plus the progress of clinical trials and key challenges in the sector.
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The evolution of AAVs in cell and gene therapy
AAV development for cell and gene therapy in 2023 is being impacted by manufacturing and regulation challenges, however advancing technologies offer opportunity, according to leaders in the field.
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£12 million given to Sheffield Biomedical Research Centre
The UK’s Sheffield Biomedical Research Centre has been awarded £12 million to help accelerate R&D of vaccines and treat conditions like HIV.
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Summer 2022 haemophilia trial update
Results from several haemophilia trials have been presented at the International Society on Thrombosis and Haemostasis (ISTH) Annual Congress 2022, here EPR summarises some of the key developments.
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EMA’s human medicines committee (CHMP) May 2022 meeting highlights
The latest meeting saw EMA’s human medicines committee (CHMP) recommend the approval of nine new medicines, refuse two biosimilar medications and recommend extensions of therapeutic indication for six medicines.
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Challenges in downstream purification of advanced therapies
John Liddell, Chief Technologist at the Centre for Process Innovation (CPI), writes about the challenges associated with gene therapy products, which constitute a major portion of the overall cell and gene therapy market.
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Five marketing strategies for novel therapeutics
Long before the advent of Coronavirus, next-generation therapies were making headlines, but the successful development of COVID-19 vaccines, including mRNA vaccines, has now shone a spotlight on next-generation therapies. This offers a boost to the long-term growth projections for the advanced therapy medicinal products (ATMPs) sector. But although ATMPs hold…
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Curative cell and gene therapies and healthcare system disruption
In this article, experts from Charles River Associates discuss how the introduction of cell and gene therapies will impact upon various aspects of the healthcare system, from provision of care to delivery and supply, and pricing and market access.
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Plasma-derived therapies require a mastery of advanced bioproduction techniques
Plasma-derived therapies and medicines provide life-changing treatments for patients around the world who suffer from serious, and often rare, diseases that affect the immune system or result from non-functioning proteins in blood plasma. Carole Resman, Executive Vice President Production at LFB, describes the complex and highly regulated manufacturing process of…
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Concizumab haemophilia trials paused over safety concerns
The occurrence of non-fatal thrombotic events in Phase III study participants has called into question the safety of concizumab.
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Sanofi to acquire haemophilia specialists Bioverativ for $11.6bn
Sanofi and Bioverativ Inc., have entered into a definitive agreement under which Sanofi will acquire all of the outstanding shares of Bioverativ...
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Microcapsules to enhance the efficiency of genome-editing
Researchers have found that polymer and hybrid silica-coated microcapsules are efficient in genome-editing...
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Shire on Haemophilia: How to treat a bleeding disorder
We caught up with Naghmana Bajwa, MD, Global Medical Affairs Haematology Lead at Shire to find out the latest in haemophilia disease research...
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UCL Technology Fund invests £1m in gene therapy company, Freeline
20 June 2016 | By Victoria White, Digital Content Producer
Freeline is developing and commercialising gene therapies for bleeding and other life threatening or debilitating disorders...
