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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.
Porosome Therapeutics has made a groundbreaking discovery in Alzheimer’s research by targeting the disease’s molecular causes, focusing on restoring secretory and metabolic functions. This approach could not only slow progression but potentially reverse early-stage pathology.
This article outlines the development of a new type of nanoparticle that can perform gene-editing in the lungs. Scientists from MIT and the University of Massachusetts Medical School have collaborated to create this Ribonucleic acid (RNA) delivery particles, which have the potential to revolutionise the treatment of lung diseases such…
Clarametyx says the FDA has accepted an IND application for first-in-human trials of CMTX-101, an antibody to treat bacterial pneumonia.
New research by the University of California, San Diego could provide a much simpler way to repair disease-causing mutations in RNA.
An experimental drug suggests that therapy for currently untreatable cases of cystic fibrosis is “clearly achievable”.
Researchers have developed a potential strategy to treat cystic fibrosis, using oligonucleotides to correct certain gene defects.
Scientists have developed an inhaled treatment for asthma that prevents excess mucus from building up in mice.
Engineered deubiquitinases combines a synthetic nanobody that recognises a protein that can rescue proteins tagged for destruction.
Researchers reveal the Vav3 protein is responsible for the formation of bacterial docking stations in the airway cells which promote the recurrent lung infections seen in Cystic Fibrosis patients.
An area where stem cell biology and medicine are combining effectively is the establishment of new cell therapies. However, current therapies are limited to a narrow set of cell types that can be isolated or created and expanded in vitro. Dr Owen Rackham discusses how utilising computational approaches will further…
Scientists have developed a new antimicrobial peptide therapy named D8 with high efficacy and low toxicity in mice which could be used to fight lung infections.
In an effort to address some of the more serious untreatable infections encountered by patients with cystic fibrosis, Calibr will collaborate with the Cystic Fibrosis Foundation on a two-year project.
A new process for producing organoids allows researchers to explore intestinal epithelium in isolation and could allow for the development of improved targeted treatments of diseases.
Gene editing using the CRISPR system has been established as the most powerful tool in the search for new drugs and is now being exploited for therapeutic purposes. Here, Pushpanathan Muthuirulan discusses the promises and wider opportunities of using CRISPR technology to open up the possibility of large-scale screening of…
