news
Europe approves first gene therapy for treatment of haemophilia A
European approval of BioMarin’s ROCTAVIAN (valoctocogene roxaparvovec) one-time gene therapy represents a breakthrough in the treatment of adults with severe haemophilia A.
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Gene therapy
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Non-viral CRISPR could overcome cell and gene therapy bottlenecks
A new approach leveraging high-yield CRISPR could make it easier to re-engineer massive quantities of cells for therapeutic applications.
news
FDA approves Zynteglo gene therapy for beta-thalassemia patients
bluebird bio’s Zynteglo (betibeglogene autotemcel), a one-time treatment for transfusion-dependent beta-thalassemia, will roll out with a $2.8mn price tag per dose.
whitepaper
Whitepaper: Viral vectors as a gene delivery vehicle
Innovations in the use of viral vectors as a gene delivery vehicle are a key factor in the growth of the gene and cell therapy field.
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On-demand webinar: The advantages of cartridge technology
Why do pharmaceutical manufacturers still believe older methods are reliable for quality testing when there’s superior technology available on the market?
whitepaper
Whitepaper: Maintain product integrity in biologic clinical studies
Cold chain shipping challenges & root causes of common reference product sourcing problems.
whitepaper
ebook: Guidance on implementation of ARMMs
Many quality control professionals recognise that the need to modernise microbial detection methods is at an all-time high.
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On-demand webinar: Speed your biologic into phase 1-3 clinical trials
How shortening the timeline from development to clinic can be achieved through parallel processing versus using a task/time compression approach.
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Webinar: Comparator sourcing success
Collaboration between comparator sourcing specialists and clinical supply managers can improve inventory management and supply chain performance.
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Webinar: Responsibilities of specialised clinical supply experts
Learn the differences in areas of expertise and responsibilities between the Clinical Supply Manager, Project Manager and Financial Project Analyst.
whitepaper
Whitepaper: Bridging the cell and gene therapy gap
The current understanding among sponsors of the workflow from cell collection, to manufacturing, and to the clinical site for patient administration.
news
Final positive results of Zolgensma® trial for SMA published
After previous reports that nearly all spinal muscular atrophy (SMA) patients treated with Zolgensma® achieved key motor milestones, Novartis announces publication of full SPR1NT results.
whitepaper
Survey report: Direct-to-patient: industry perception and future trends
Understand the perceived importance of DTP to successful study execution.
whitepaper
Resources: Single use technologies – the ultimate future
Single use technologies is highly booming in biopharmaceutical sector due to its efficient use and benefits.
webinar
Digital versus digitised: common misconceptions about eConsent
6 June 2022 | By Medable
Learn about the common misconceptions surrounding eConsent, as well as the benefits they can realise through a digitally-enabled approach.
