All Rare & Genetic Disorders articles – Page 2
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NewsBlocking the P2X4 receptor: a potential pathway to new therapies
Researchers have discovered how to inhibit the P2X4 receptor, a key protein linked to chronic pain, inflammation and certain cancers.
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NewsAutomated lung organoids to speed up new drug development
Scientists have developed an automated method to grow lung organoids, with the hope of speeding up drug testing, reducing reliance on animal models and helping to develop new personalised treatments.
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NewsBacterial traces discovered within gliomas and brain metastases
Scientists have discovered active bacterial traces inside brain tumours, overturning assumptions about the brain’s sterility and opening up new possibilities for understanding – and potentially treating – gliomas and brain metastases.
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NewsNew framework enhances reliability of virtual cell models
Shift Bioscience have announced new research revealing that AI-driven virtual cell models perform far better than previously thought when assessed with correctly calibrated metrics.
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ArticleRecombinant KLK1: the next step in stroke and preeclampsia treatment
With its lead candidate DM199, DiaMedica Therapeutics is advancing a recombinant form of KLK1 to restore blood flow, improve endothelial function and address unmet needs in the treatment of stroke and preeclampsia.
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ArticleMeet WRPRFa: the precision peptide changing how we study pain
A newly developed peptide, WRPRFa, is giving scientists a clearer view of how the acid-sensing ion channel (ASIC3) drives pain signalling.
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NewsExisting nerve pain drugs may help stop bone cancer spread
Scientists have discovered that two existing pain medications – bupivacaine and rimegepant – may not only relieve the severe pain caused by osteosarcoma but also slow the disease’s growth.
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NewsTRF1 protein identified as potential obesity drug target
Researchers have discovered that removing a telomere-protecting protein, TRF1, makes mice leaner and metabolically healthier without shortening their telomeres, potentially leading to new methods for tackling obesity and age-related diseases.
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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.