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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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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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…
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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.
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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.
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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.
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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.
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Evaluating novel gene therapies in a whole human liver
Use of the whole liver could revolutionise the development of viral vectors, providing more effective treatments for inherited diseases.
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How fusion proteins drive paediatric cancer
Researchers discover that fusion proteins and a gene regulatory protein complex interact through disordered domains.
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Women in Stem with Dr Amber D. Van Laar
Dr Amber Van Laar shares her remarkable journey in STEM. From a childhood fascination with science and medicine to a pivotal role as VP Clinical Development, AskBio. In this interview, she explores the profound impact of her early exposure to neuro-oncology, the challenges faced as a physician-scientist, and the pursuit…
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Gamma delta T cells: a rising star in cancer therapy
Gamma delta (γδ) T cells comprise a distinct and powerful subpopulation of T cells, bridging innate and adaptive immunity, that holds great promise for treating cancer. IN8Bio co-founder and CEO William Ho and COO Kate Rochlin discuss what makes γδ T cells unique, how preclinical research suggests their antitumour activity…
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A promising new option for male contraception
Scientists have targeted the SMRT-retinoic acid receptor interaction, which could provide a reversible and non-hormonal male contraceptive method.
