All Gene Therapy articles – Page 3
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NewsNew 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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NewsNasal 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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ArticleTackling 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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ArticleTransforming 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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ArticleCracking 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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NewsNew 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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ArticleAllele-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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ArticleBuilding 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.
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ArticleAI in drug discovery: faster, smarter, better
Claudia Zylberberg, Chair of Kosten Digital, reveals how AI is revolutionising cell and gene therapy, making drug discovery faster and more cost-effective.
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ArticleSynthetic promoters enhance gene therapy precision
Discover how Chromatin Bioscience’s synthetic promoters are transforming gene therapy by enhancing precision, boosting safety and improving manufacturing efficiency.
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WhitepaperGuide: Create DNA Template for In Vitro mRNA Synthesis
Learn how to simplify the entire DNA template generation workflow, streamlining mRNA therapeutic research.
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NewsReprogramming gut cells to treat short bowel syndrome
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.
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WhitepaperPoster: Lentiviral Gene Delivery Workflow
Use this lentiviral gene delivery workflow poster to understand how to optimize your workflow for successful viral transduction.
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ArticleGene silencing: a step forward for rare disease therapy
Gene silencing offers a promising approach to treating rare neurological diseases like H-ABC. With clinical trials on the horizon, find out how targeted therapies could bring real hope to patients and families.
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WebinarSolving the AOC puzzle: Strategies for chemistry, manufacturing and regulatory success
Watch our webinar with Abzena’s experts to explore practical solutions for overcoming chemistry, manufacturing and regulatory challenges in antibody-oligonucleotide conjugate (AOC) development, enabling progress for previously untreatable diseases.
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NewsAWS and ElevateBio join forces to advance gene editing
ElevateBio is teaming up with Amazon Web Services (AWS) to advance CRISPR gene editing using AI and cloud computing. This collaboration aims to accelerate drug discovery for genetic diseases, making next-generation therapies more efficient and accessible.
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NewsRare diseases, big advances: how research is powering a new era of treatments
This Friday, February 28, marks Rare Disease Day 2025, a global initiative raising awareness for the 300 million people living with rare conditions. Dr David Reynolds, CEO of LoQus23 Therapeutics, explores how advancements in rare disease research are driving the development of new treatments.
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NewsNew gene identified for treating malignant cardiac arrhythmias
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.
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NewsDNA repair genes drive Huntington's disease, UCLA study finds
A new UCLA study reveals that DNA mismatch repair genes play a crucial role in Huntington’s disease by driving neuronal damage and motor impairments. Targeting these genes, especially Msh3 and Pms1, could offer promising therapeutic avenues for the disease.
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ArticleEmpowering women in STEM: championing change in genetics
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.