All articles tagged with #drug discovery
USC researchers identified selective inhibitors of the brain inflammation enzyme cPLA2 that may lower Alzheimer’s risk, especially for APOE4 carriers; the compounds cross the blood-brain barrier, showed activity in brain cells and mouse models, and represent a promising but early-stage drug discovery path with safety and feasibility in humans to be determined.
Two AI-agent systems described in Nature—Google DeepMind's Co-Scientist and FutureHouse's Robin—use teams of AI to generate hypotheses, pick experiments, and analyze data to speed up early drug-discovery work. In initial tests they proposed plausible repurposing candidates for AML and dry AMD, with humans carrying out the experiments and validating results; while promising, most candidates require further validation in broader use.
Nature editors argue that AI agents can dramatically speed up research and drug discovery (as shown with Robin and Co-Scientist) but cannot replace humans: humans frame problems, guide experiments, validate results, and guard against AI errors, making human oversight and ethical considerations crucial as science increasingly relies on AI co-scientists.
Isomorphic Labs closed a $2.1 billion Series B, cementing its leadership in AI-driven drug discovery and prompting questions about how the funds will be deployed, pipeline progress, and the broader implications for biotech and AI investment.
The piece marks 25 years since click chemistry’s birth—reactions that join molecules with high specificity and minimal by-products—showing how this efficiency-driven approach transformed pharma, biology, and engineering and earned Sharpless a share of the 2022 Nobel Prize in Chemistry.
OpenAI launches GPT-Rosalind, a life sciences-focused AI model designed to accelerate research in genomics and biochemistry by synthesizing evidence and generating hypotheses, while keeping humans in the loop; it features enterprise-grade security and is being rolled out in a research preview to trusted customers (e.g., Amgen, Moderna, Allen Institute, Thermo Fisher) to speed health outcomes and drug discovery without replacing scientists, amid concerns about misuse.
Researchers at the Medical University of South Carolina are pursuing a treatment for familial hypercholesterolemia by reducing apoB, the backbone of LDL particles, using human iPSC-derived liver cells to screen about 130,000 compounds. A lead class lowered apoB release and cellular lipid levels in human cells and in humanized mice, bypassing the LDL receptor and potentially helping patients with defective receptors. Further work will clarify the mechanism, safety, and how such drugs could complement existing therapies like statins.
Deep underground Lechuguilla Cave hosts ancient, isolated bacteria that resist most natural antibiotics, suggesting antibiotic resistance predates humans and could inspire new drugs while offering clues on how pathogens might evolve resistance to upcoming therapies.
AI is speeding up drug discovery by screening millions of compounds to find new antibiotics against drug-resistant bacteria such as Neisseria gonorrhoeae and MRSA, and by identifying potential Parkinson's therapies through machine learning. The technology is also repurposing existing drugs for rare diseases and creating virtual disease models to test treatments, potentially slashing development time and costs—though validation, data access, and the long path to clinical use remain significant hurdles.
QDX's Extreme-scale Electronic Structure System (EXESS) can run large-molecule quantum chemistry calculations thousands of times faster on conventional hardware by fragmentation and parallelization, enabling drug discovery and materials research to move from weeks or months to minutes, with free access for approved research projects.
Researchers identified two brain receptors (SST1 and SST4) that regulate neprilysin, the enzyme that degrades amyloid beta. Activating both receptors in mice raised neprilysin levels, reduced amyloid buildup, and improved memory-related behavior, suggesting a potential for safe, affordable oral Alzheimer’s treatments that enhance the brain’s own plaque-cleaning system; however, findings are preclinical and require human studies.
An open-source AI named MOSAIC clusters about one million patent reactions and trains thousands of specialist models to predict reaction conditions and draft complete, executable lab procedures, enabling researchers to synthesize 35 novel compounds with potential pharmaceutical, agrochemical, or cosmetic uses without further data trawling.
UCLA researchers overturned Bredt's rule by creating cage-shaped cubene and quadricyclene with highly distorted double bonds, revealing bond orders around 1.5 and expanding 3D-molecule building blocks for future drug design.
OpenAI CFO Sarah Friar floated a value-sharing model at Davos, suggesting OpenAI could license discoveries made with its technology or take a stake in the profits from AI-enabled breakthroughs (such as new drugs), signaling a potential shift toward revenue sharing as it pursues new income beyond subscriptions.
UC Davis researchers developed a light-driven method that combines amino acids with tryptamine and, after UV exposure, reshapes them into new molecules that activate the serotonin 5-HT2A receptor and may offer psychedelic-like brain benefits without altering perception. In mice, the leading compound fully activated 5-HT2A but did not induce hallucinogenic behaviors (head-twitch responses), leading researchers to pursue further studies on how other serotonin receptors might modulate or suppress perceptual effects.