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Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
Alltrna is redefining the future of rare disease treatment with engineered tRNA therapeutics that target genetic mutations - not just individual diseases. CEO Michelle Werner shares how this bold, mutation-driven approach could unlock universal treatments for thousands of patients long overlooked by traditional medicine.
Despite the promise of gene therapies, automated red blood cell exchange (aRBCX) remains an underutilised therapy in the management of sickle cell disease (SCD). In this article, Dr Aaron Haubner and Carly Newton of Terumo Blood and Cell Technologies, highlight the urgent need for partnerships and equitable access to this…
A new study from the University of Barcelona’s Institute of Neurosciences has discovered a crucial role for the RTP801 protein in astrocytes, potentially making way for future therapies aimed at slowing or reversing cognitive decline.
The world’s first translational research center dedicated to urea cycle disorders (UCDs) has been launched in Zürich, marking a significant moment in rare disease innovation.
Researchers at Mass General Brigham have developed a new gene therapy delivery system, AAV.CPP.16, that targets the lungs via nasal spray, which could offer a potential breakthrough in respiratory gene therapy.
Chief Scientific Officer at Solvias reveals how CROs are overcoming manufacturing challenges and harnessing AI to fast-track the development of groundbreaking cell and gene therapies.
Vitalant is transforming the path from preclinical drug discovery to patient care by enabling access to cutting-edge cell and gene therapies. With decades of expertise and a nationwide network, find out how they bridge the gap between innovative treatments and local communities, ensuring life-changing therapies reach patients efficiently.
Cell and gene therapy is rapidly transforming the treatment of complex diseases, yet scaling production efficiently remains a challenge. Dr Tia Harmon from PHC Corporation of North America explores the innovations that are optimising cell expansion to enhance the discovery of advanced therapies.
Japanese researchers have developed a new enzyme technology that can precisely alter the levels of mutated mitochondrial DNA in patient-derived stem cells, offering a promising new approach for treating mitochondrial disorders.
Huntington's disease remains a major challenge, but allele-selective gene editing offers new hope. By targeting only the mutant gene, this approach could provide a one-time, durable treatment. Life Edit’s Dr Amy Pooler reveals how this innovative therapy could reshape HD treatment.
Outdated clinical development systems are slowing breakthroughs like gene therapies and precision medicine. This article explores how a flexible, on-demand approach can accelerate drug development and bring innovations to patients faster.
Claudia Zylberberg, Chair of Kosten Digital, reveals how AI is revolutionising cell and gene therapy, making drug discovery faster and more cost-effective.
Discover how Chromatin Bioscience’s synthetic promoters are transforming gene therapy by enhancing precision, boosting safety and improving manufacturing efficiency.
Learn how to simplify the entire DNA template generation workflow, streamlining mRNA therapeutic research.
A preclinical study conducted by researchers at Weill Cornell Medicine have demonstrated a new gene-editing strategy to treat short bowel syndrome (SBS), a life-threatening condition in which patients lack a functional small intestine.
