All Rare & Genetic Disorders articles – Page 2
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ArticleThe predictive validity crisis: Pharma’s productivity paradox – Part II
Part II shows that the predictive validity crisis can be solved by rethinking how the industry chooses models, measures outcomes and integrates systems. Success stories from Vertex, Regeneron and AstraZeneca illustrate how aligning biology, measurement and strategy can reverse decades of declining productivity.
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NewsDNA repair secrets of naked mole-rats may guide new drug discovery
New research has demonstrated how tiny tweaks in a DNA-sensing enzyme may hold the key to the naked mole-rat’s extraordinary lifespan – offering insights that could one day inform therapies for aging and age-related diseases.
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ArticleEye movements as objective biomarkers: accelerating CNS drug development
Measuring disease progression remains one of the biggest hurdles in CNS drug development. Eye movements, now trackable with just a laptop and webcam, are emerging as a sensitive and scalable biomarker that could transform how trials are designed and therapies reach patients.
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NewsFibroblast mapping reveals potential universal drug targets
Scientists have mapped the diversity of fibroblasts and discovered how ‘rogue’ fibroblasts drive multiple diseases, revealing drug targets that could transform treatments across the body.
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NewsMapping lung disease: new atlas to accelerate drug discovery
Helmholtz Munich and Parse Biosciences have collaborated to create the world’s largest lung disease perturbation atlas – which could aid the discovery of new therapeutic targets and accelerate the development of future lung disease treatments.
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NewsRare disease drugs get boost from new NICE guidelines
NICE has raised the cost-effectiveness thresholds for ultra-rare disease drugs under its Highly Specialised Technology programme – a move welcomed by patient advocates and biotech leaders.
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NewsNew nanostructure makes CRISPR edits safer and three times faster
Northwestern University researchers have created DNA-coated nanoparticles that deliver CRISPR into cells three times more effectively while reducing toxicity. The advance could overcome one of the biggest barriers to gene-editing therapies.
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NewsThree powerful antibodies discovered with potential to treat mpox
Researchers at Mount Sinai have identified three antibodies that target mpox and prevent severe disease in vivo. The work positions A35-specific antibodies as candidates for therapeutic development.
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ArticleBeyond the hype: a veteran's honest assessment of AI in drug discovery - Part 2
Thibault Géoui explains why AI could finally help pharma overcome its productivity crisis and why the payoff won’t come as quickly as the optimists claim.
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NewsKidney organoids reveal hidden toxicities in AAV gene therapy
Stem cell-derived kidney organoids have revealed hidden toxicities in adeno-associated virus (AAV) gene therapy delivery – offering a powerful new way to improve the safety of future treatments.
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NewsNew CRISPR breakthrough could transform sickle cell treatment
Researchers at UNSW Sydney have unveiled a next-generation CRISPR tool that could lead to safer treatments for genetic disorders, including Sickle Cell.
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NewsFDA-approved asthma drug may protect against food allergy reactions
An existing asthma drug has been shown to almost completely prevent life-threatening allergic reactions to food in mice, after researchers found a previously unknown genetic pathway linked to anaphylaxis.
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NewsMast cells: new target for preventing meningitis and brain disease
Scientists at Washington University School of Medicine have discovered that mast cells – immune cells best known for triggering allergic reactions – also help protect the brain from bacterial and viral infections. This could have important implications for treating brain infections in the future.
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NewsNew 3D bioprinted lung tissue could transform respiratory research
UBC Okanagan researchers have developed a new 3D bio-printed lung model that closely mimics the complexity of human tissue – providing scientists with a powerful new tool for studying respiratory diseases.
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ArticleNGS is evolving: collaboration and tech lead the way
Next-generation sequencing (NGS) is advancing fast – and it’s not happening in isolation. Strategic partnerships and automation are streamlining workflows and reshaping what's possible in genomics research.
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NewsMeet the hemifusome: a new organelle with big impact
Scientists have discovered a previously unknown organelle inside human cells - the hemifusome - that could change our understanding of rare 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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ArticleHow GPCR-targeting therapies are advancing the fight against inflammatory disease
Discover how Domain Therapeutics is using its deep knowledge of GPCR biology to create novel therapeutics with the potential to combat inflammatory diseases such as atopic dermatitis, IBD and arthritis.
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NewsSB000: 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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ArticleAutomated 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 ...