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Scaling up cell therapy is no easy feat. PluriCDMO's Andy Lewin reveals the critical factors for success and how they are helping companies bring life-saving therapies to market.
Researchers have identified a small gene, SCN10a-short, that could enable gene therapy to treat malignant cardiac arrhythmias. This discovery offers the potential for a one-time treatment, reducing the reliance on lifelong medication and invasive procedures.
An advocate for genetic medicines and the enormous potential they hold, Leah Sabin at Regeneron Genetic Medicines reveals how passion and curiosity can forge the path to a rewarding career in STEM.
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.
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…
Researchers utilised AI to identify genes that reprogramme GBM cancer cells into dendritic cells within the tumour.
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.
The experimental therapy eliminated 90 percent of HSV-1 after facial infection and 97 percent of HSV-1 after genital infection.
Use of the whole liver could revolutionise the development of viral vectors, providing more effective treatments for inherited diseases.
The new study suggests that this gene therapy method may combat ARVC in both early and more advanced stages of the condition.
