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With over 1,000 skin diseases lacking approved treatments, a search-and-develop model is changing how new therapies are sourced and developed. Chief Scientific Officer, Jacob Pontoppidan Thyssen, outlines the strategy behind it.
Most gene therapies rely on static DNA promoters to control gene activity, but nature uses far more sophisticated tools. Dr Matthew Dale explores how harnessing RNA-level control could enable treatments that sense and respond in real time, offering unprecedented precision and safety.
Oral peptide-based drugs are set to revolutionise the pharmaceutical industry, overcoming the long-standing challenge of poor bioavailability. Santosh Kulkarni reveals how new breakthroughs in drug discovery and delivery offer the potential for more convenient, effective treatments for a range of conditions – without the need for injections.
Scientists have created a new class of ultra-stable chiral molecules – a discovery that could lead to more precise drug design by preventing potentially harmful molecular “flipping” over time
QTX153, a novel HDAC6 inhibitor, has shown significant symptom reversal in preclinical models of Rett syndrome. This represents progress toward a therapy for a condition with no approved options.
A study from Weill Cornell Medicine shows that pre-treating pancreatic islet cells with a small molecule cocktail significantly improves survival after transplantation in type 1 diabetes models. The approach could help make donor cells go further and transplants more efficient.
MitoRx Therapeutics has announced new preclinical data for its small molecule Myo4, showing restored insulin sensitivity and enhanced fat loss with muscle preservation in an obesity model - offering a potential alternative to GLP-1-based therapies.
A practicing surgeon turned biotech CEO is developing a novel pain medicine that could sidestep the failures of both opioids and paracetamol.
Forget the buzzwords - SLAS 2025 showed what’s genuinely driving progress in drug discovery: usable AI, collaborative platforms and tools that solve real problems.
Novartis has teamed up with PTC Therapeutics to develop PTC518, a groundbreaking treatment that targets the cause of Huntington's disease, offering new hope for patients.
Insilico Medicine’s AI-designed drug ISM5939 has received FDA IND clearance for treating solid tumours. By targeting ENPP1, it enhances anti-tumour immunity and is set to begin Phase I trials soon.
Small molecules are essential for developing effective medicines, but their structural complexity and material behaviour present difficulties. In this article, Chris Brown, Senior Chemist, Onyx Scientific Ltd, discusses how the right technologies and approaches can enhance drug development and mitigate risk from initial phase to commercial launch.
Researchers screened over 2,000 small molecules and have now more than doubled the known compounds reported to induce trained immunity.
PVP-037, identified through a small molecule screen, induces a more durable and broader immune response to vaccines.
In this episode, we discuss how new insights into stem cell biology are leading to novel therapeutics in regenerative medicine beyond cell therapy.
