podcasts
Overcoming obstacles in personalised medicine
Tune in to this episode to learn how overcoming the key challenges in personalised medicine could transform the future of healthcare.
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Gene Therapy
Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
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New insights into the role of viral capsids in gene therapy safety
Viral capsid assembly and quality are key factors in gene therapy success and safety. Here, Dr Chelsea Pratt explores how cutting-edge tools are enabling scientists to tackle these challenges and improve patient outcomes.
news
Blood-brain barrier breakthrough for ALS and Alzheimer’s disease
A new blood-brain barrier-crossing platform enables the delivery of therapeutic molecules to the brain, offering potential treatments for ALS and Alzheimer’s disease.
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Women in STEM with Veerle d’Haenens
We had the privilege of talking to Veerle d’Haenens, General Manager, Global Therapy Innovations at Terumo Blood and Cell Technologies. Her successful career has been driven by her creativity, expertise and passion, and she advises women who are aspiring to STEM roles to always think big.
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Cell and gene therapy development moves into cardiac indications
The success of CAR-Ts in oncology has stoked enthusiasm for developing comparable curative therapies in other disease areas. CellProthera’s Chief Scientific Officer, Ibon Garitaonandia, explains the potential and progress for CGTs in cardiology, where disease-modifying therapies are largely non-existent.
podcasts
Scaling genetic medicine with tRNA
Tune into this episode which explores how engineered tRNAs could treat thousands of rare diseases and cancers that share the same mutation.
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Overcoming operational challenges in CGT manufacturing with AI
In this Q&A, Dalip Sethi, scientific lead for Terumo Blood and Cell Technologies, Cell Therapy Technologies and Innovation portfolio, discusses the integration of AI into CGT manufacturing processes to enhance operational efficiency and accelerate treatment access. He also elucidates the strategies that are being considered to overcome hurdles when implementing…
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The rising demand for lentiviral vectors for in vivo gene therapy
Fuelled by advances in rare disease treatments and vaccination efforts, Natalia Elizalde, CBO at VIVEbiotech, discusses how the market demand for lentiviral vectors is evolving, the new therapeutic areas emerging as potential targets for in vivo gene therapy and the latest technological advancements in the development and delivery of in…
news
Spinal muscular atrophy: searching for a cure
Organoids with SMA-pathology uncovered key findings about the disease, which could be utilised to develop new therapeutic options.
news
Prime editing corrects the CFTR gene mutation
The approach precisely and durably corrects the CFTR mutation in human lung cells, which could lead to superior treatments.
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Novel version of CRISPR gene-editing protein engineered
Researchers have developed a new, compact EbCas12a variant that can be packaged into an all-in-one AAV system with its crRNA.
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Enhancing gene therapy with Circio
In this Q&A, Erik Wiklund, CEO of Circio, explains the key findings of their circVec circular RNA platform technology, why they chose AAV-based gene therapy for AATD as the lead programme, and their plans for the future to enhance the potency and reduce the cost of current gold-standard gene therapy.
news
Gene editing for HSV-1 shows significant reduction of viral load
The experimental therapy eliminated 90 percent of HSV-1 after facial infection and 97 percent of HSV-1 after genital infection.
whitepaper
Beyond the lab: cell and gene therapy
Download this report now to access exclusive content on innovative cancer treatments, regenerative medicine, gene therapy, and expert insights from leading professionals in the field.
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Investigating new treatments for polycystic kidney disease
Organoid models enabled the researchers to study the effectiveness of eukaryotic ribosomal selective glycoside drugs on PKD cyst formation.