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New targeted gene therapy could reverse neurological diseases
Researchers have developed a novel gene therapy that could help treat children with incurable genetic and neurodegenerative diseases.
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Gene therapy
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Pharmacovigilance Risk Assessment Committee (PRAC) meeting highlights, July 2021
The Pharmacovigilance Risk Assessment Committee (PRAC) provided safety warnings on COVID-19 vaccines, and found no evidence linking Zynteglo to blood cancer.
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WHO publishes recommendations on human genome editing
To promote the safe and ethical use of genome editing for the treatment of disease and genetic disorders WHO has published the first global recommendations.
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Brochure: Welcome to bioprocessing – pathways for pioneers
The journey from large molecule to approved biotherapeutic is long, costly, complex and fraught with risk – yet rewards for success are substantial.
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Zolgensma® meaningfully improves spinal muscular atrophy in patients
Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene therapy achieved motor milestones never observed in the natural history of spinal muscular atrophy.
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beti-cel gene therapy demonstrates durable efficacy in beta thalassaemia patients
The betibeglogene autotemcel (beti-cel) gene therapy caused paediatric and adult beta thalassaemia patients to achieve transfusion independence lasting over two years.
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New consortium to develop PAT for cell and gene therapies
The Cell and Gene Therapy Catapult’s new consortium aims to accelerate therapeutic development by developing process analytical technologies (PAT) for cell and gene therapy manufacturing.
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On-demand webinar: Celsis® for sterility – product spotlight
A rapid sterility test method that can be validated as quickly and confidently as it provides test results.
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On-demand webinar: Contamination control strategy essentials
This webinar presents the fundamentals of a contamination control strategy including forms of contamination, as well as cleaning and disinfection.
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Application note: Resilient plastics in the Cold Chain
Pictorial that explains how Aramus TM single-layer fluoropolymer bag can withstand gamma irradiation and cold-chain temperatures down to -196°C.
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First patient treated with potentially curative HIV cell and gene therapy
The first-in-human study will evaluate the safety and efficacy AGT103-T, a cell and gene therapy product for HIV, in up to six participants.
webinar
Variable pathlength spectroscopy for dilution-free concentration measurement in GMP environments
27 May 2021 | By Repligen
Watch this on-demand webinar, where we reviewed VPT and how it can be used in various applications, from protein therapeutics to gene therapy.
news
Industry hustling to ensure supply of recombinant vector COVID-19 vaccines
A new report suggests pharma is working hard to combat worldwide viral vectors shortage that could threaten the COVID-19 vaccine rollout.
news
EMA’s human medicines committee (CHMP) meeting highlights, May 2021
In its latest meeting the CHMP recommended eight medications for approval, 17 medicines for indication extension and concluded reviews of two COVID-19 treatments.
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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.
