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Transform your drug discovery pipeline with 3D cellular models
7 July 2025 | By Molecular Devices
Expert insights reveal how 3D cellular models are revolutionising preclinical testing and clinical translation.
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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 discovery shows how viral vectors release genes in therapy
Researchers at The University of Osaka have found how structural changes in the VP1 protein of AAV vectors trigger genome release - offering insights for improving the safety, efficiency and stability of gene therapy delivery systems.
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‘Delete-to-recruit’: new gene-editing method targets sickle cell
Researchers have developed a novel gene therapy approach that reactivates dormant genes by repositioning them closer to genetic switches called enhancers - showing promise for treating blood disorders like sickle cell disease.
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SB000: a safer path to anti-aging therapies
Shift Bioscience has announced new aging research, highlighting the discovery of SB000. This novel single-gene target reverses cellular aging without activating dangerous pluripotency pathways.
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New tRNA tech aims to rewrite rare disease treatment
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.
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Automated red blood cell exchange: bridging treatment gaps in sickle cell disease care
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…
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Astrocyte protein RTP801 linked to Alzheimer’s cognitive decline
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.
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New UCD centre targets the biggest bottleneck in rare research
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.
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Nasal spray gene therapy shows potential to treat lung diseases
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.
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Tackling complex manufacturing challenges in modern drug development
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.
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Transforming biotherapeutics delivery for local communities
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.
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Cracking the code to scalable cell and gene therapy
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.
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New enzyme tech offers hope for mitochondrial disease treatment
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.
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Allele-selective gene editing: a breakthrough in Huntington’s disease treatment
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.
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Building the requisite clinical development infrastructure for medical innovation to thrive
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.
