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ProQR and Lilly collaborate to develop RNA therapeutic
ProQR and Lilly announced a collaboration to combat genetic disorders in the liver and nervous system using Axiomer® RNA editing platform.
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Genomics
![Researcher looking at an experiment [Credit: Unsplash].](https://www.europeanpharmaceuticalreview.com/wp-content/uploads/Bell-advanced-therapies-feature-300x278.jpg)
![Researcher looking at an experiment [Credit: Unsplash].](https://www.europeanpharmaceuticalreview.com/wp-content/uploads/Bell-advanced-therapies-feature.jpg)
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UK at the forefront of advanced therapies
Advanced therapies are a ground-breaking new class of medicines that use gene therapy, cell therapy or tissue engineering to treat disease and injury. Rachel Bell, Trainee Patent Attorney at Marks & Clerk, and Ceri Roberts, Scientific Training Manager – Cellular and Molecular Therapies at NHS Blood and Transplant, discuss some…
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Gene therapies set to revolutionise cardiovascular treatments
Gene therapies could revolutionise management of cardiovascular diseases such as heart failure, according to a GlobalData report.
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Genomic sequencing is a potential key to precision medicine for cancer
A new US study demonstrated that comprehensive genomic sequencing is crucial for the development of precision medicine for cancer patients.
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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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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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Big pharma collaborates on free resource linking genetic variants to human disease
The collaborative work has produced the largest browsable resource linking rare protein-coding genetic variants to human health and disease that will accelerate targeted drug development.
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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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Vaccine slows loss of insulin production in type 1 diabetes genetic subgroup
In a Phase II trial, injecting GAD-alum into the lymph nodes of newly diagnosed type 1 diabetes patients with a genetic variant slowed insulin production degradation.
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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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Gene therapy successfully treats rare immunodeficiency in paediatric patients
In three Phase I/II trials an experimental gene therapy caused all but two patients with severe combined immunodeficiency to acquire and retain robust immune function for more than two years.
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Which is the best in vitro diagnostic test method?
In this article, Liz Thorn, Senior Consultant, Diagnostics at Team Consulting, explores the various in vitro diagnostics (IVDs) techniques available and where they are best applied in the context of influenza and COVID-19.
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Machine learning used to predict most effective cancer drugs
According to a new study, Drug Ranking Using Machine Learning (DRUML) can accurately rank cancer therapies by efficacy across a range of cancer types.
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bluebird bio releases new data on its gene therapy for cerebral adrenoleukodystrophy
Data from nearly seven years of follow up suggests one-time treatment with eli-cel may durably stabilise cerebral adrenoleukodystrophy disease progression.