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Wherein JNK3 is specifically expressed in brain and emerges as therapeutic target, especially for neurodegenerative diseases. However, developing JNK3 selective inhibitors as chemical probes to investigate its therapeutic potential in diseases remains challenging.
Modulating PPIs with smallmolecules has gained increasing attention in drug discovery, particularly targeting the 14-3-3 protein family, which interacts with several hundred client proteins and plays a central role in cellular networks.
Experimental drug NU-9 -- a smallmolecule compound approved by the U.S. Food and Drug Administration (FDA) for clinical trials for the treatment of amyotrophic lateral sclerosis (ALS) -- improves neuron health in animal models of Alzheimer's disease, according to a new study.
Related groups Xavier lab Over the past two decades, large genetic studies have linked tens of thousands of DNA variants to thousands of human traits and diseases. Yet, correcting the effects of those variants to treat disease has been hampered in part by the lack of precise molecular tools to do so.
2025 , 147 , 25 , 21512–21525 [link] Targeted protein degradation (TPD) has emerged as a promising therapeutic strategy for treating various diseases. However, current smallmolecule degraders predominantly rely on a limited set of E3 ubiquitin ligases, such as CRBN and VHL, which restricts their applications.
a new Italian incubator dedicated to the discovery and development of smallmolecule-based treatments for neurodegenerative diseases, announced today that it has adopted Collaborative Drug Discovery’s CDD Vault as its platform of choice for managing drug discovery data.
Whether enhanced mitochondrial quality control through inhibition of USP30 can protect dopaminergic neurons is currently being explored in a clinical trial for Parkinsons disease. However, the molecular basis for specific inhibition of USP30 by smallmolecules has remained elusive.
. #2: MRTX1133 (Mirati Therapeutics/Array BioPharma, 21%) The 2nd place runner-up is MRTX1133 , the non-covalent KRAS(G12D) inhibitor and is the first reported inhibitor specific for G12D, which makes up as much as 33% of all KRAS mutations and carries a 2 to 3-fold higher disease burden than G12C. LP0200 has completed a Ph.
Antibodies have become the most prevalent class of therapeutics, primarily due to their ability to target specific antigens, enabling them to treat a wide range of diseases, from cancer to autoimmune disorders. As a result… Source
Targeting GPCRs to fight inflammatory diseases GPCR-targeting drugs are well known therapies for a range of disease types, including cardio-metabolic, central nervous system and endocrinological disorders. It also holds potential for neuroinflammatory conditions such as certain types of migraine.
This review highlights recent advancements in smallmolecule inhibitors targeting Cdc2-like kinase 4 (Clk4), emphasizing their potential in treating cancers and neurodegenerative diseases. This review highlights recent advancements in Clk4 inhibitors, covering both natural, and synthetic compounds.
Currently, the FDA has granted approval for the use of the smallmolecule inhibitor Ivosidenib (AG-120) in the treatment of IDH1-mutated AML and cholangiocarcinoma. Although AG-120 has benefited patients clinically, drug resistance has gradually emerged and has become a major problem in the treatment of mutant IDH1 (mIDH1) diseases.
3c01835 Building on recent advances in peptide science, medicinal chemists have developed a hybrid class of bioconjugates, called peptide–drug conjugates, that demonstrate improved efficacy compared to peptides and smallmolecules independently. Moore Journal of Medicinal Chemistry 2024 DOI: 10.1021/acs.jmedchem.3c01835
sPLA 2 inhibitors have been developed for the treatment of inflammatory and other conditions such as cardiovascular disease, arteriosclerosis and rheumatoid arthritis. Due to their roles in inflammation, the sPLA 2 enzymes are of much medicinal interest.
Thomas Kodadek Angewandte Chemie International Edition 2024 e202316726 [link] Many of the highest priority targets in a wide range of disease states are difficult-to-drug proteins. This has engendered interest in strategies to increase the potency of a given protein inhibitor by routes other than further improvement in gross affinity.
Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.
We have integrated structural and quantitative proteomics with biochemical assays to decipher the mode of action of covalent USP30 inhibition by a smallmolecule containing a cyanopyrrolidine reactive group, USP30-I-1. The inhibitor demonstrated high potency and selectivity for endogenous USP30 in neuroblastoma cells.
G protein-coupled receptors (GPCRs) represent a cornerstone of modern drug discovery due to their crucial role in regulating human physiology and their involvement in numerous diseases. Many of these receptors are key targets for addressing obesity and metabolic disorders.
In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop smallmolecule therapeutics to target RNA processing.
Mutations that impair nuclear protein association with chromatin are implicated in numerous diseases. Covalent ligands are a promising strategy to pharmacologically target nuclear proteins, such as transcription factors, which lack ordered small-molecule binding pockets. TurboID (His-TID) construct, with chemoproteomics.
Lead Pharma has entered into a collaboration and license agreement with Roche , hoping to lead the way in the development of oral smallmolecules for immune-mediated diseases like rheumatoid arthritis, psoriasis and inflammatory bowel diseases. . The agreement encompasses Lead Pharma’s entire pipeline.
In this article, we will delve into the world of condensate biology and explore the groundbreaking research projects undertaken to discover the potential they hold for diseases such as amyotrophic lateral sclerosis ( ALS ) and colorectal cancer. Klein notes the importance of Dewpoint’s disease-agnostic approach.
Introduction Therapeutic antibodies have proven to be indispensable medicines for addressing the most debilitating diseases. Agonist antibodies in immunological diseases Agonist antibodies targeting immune checkpoint pathways are increasingly recognised and investigated for their potential in immunological diseases.
These therapies have broadened treatment options for patients to expand beyond the more traditional smallmolecule drug alternatives. In practice, some patients may be left managing a syndrome associated with the therapy along with the disease. 3D rendering of Antibody Drug Conjugate Molecules.
Novel smallmolecule drug candidates will target GPR75 to potentially address obesity and related co-morbidities. AstraZeneca has entered into a collaboration with Regeneron to research, develop and commercialise smallmolecule compounds directed against the GPR75 target with the potential to treat obesity and related co-morbidities.
Metabolism of 2023 FDA Approved SmallMolecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 smallmolecules out of a total of 55 new drugs [1]. References [1] 2023 Novel SmallMolecule FDA Drug Approvals. Blood 129(13): 1823-1830.
Global integrated drug discovery company, Sygnature Discovery and global healthcare group Daewoong Pharmaceutical, have entered into a research collaboration agreement to accelerate the discovery of a novel smallmolecule to target autoimmune disease.
Metabolism of 2022 FDA approved smallmolecule drugs – Part 1 Does CYP3A4 still rule? By Julia Shanu-Wilson It won’t come as much surprise to learn that of the 17 smallmolecules* approved by the FDA in 2022, CYP3A4 was the major player in drug metabolism. References Iversen et al., Curr Drug Metab. 20(4): 254-265.
This is a game-changer, especially in the fight against cancer and other complex diseases. These multifunctional smallmolecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. This can potentially correct the root causes of some diseases at the genetic level.
Food and Drug Administration (FDA) approved smallmolecules. CysDB is publicly available at [link] and features measures of identification for 62,888 cysteines (24% of the cysteinome), as well as annotations of functionality, druggability, disease relevance, genetic variation, and structural features.
A number of studies have shown that blocking the CSF-1R signaling pathway could effectively modulate and change macrophage functions, and potentially treat many macrophage-dependent human diseases. [1] Abstract LB-288: A highly selective smallmolecule CSF-1R inhibitor demonstrates strong immunomodulatory activity in syngeneic models.
Today, we're able to identify and target specific molecules involved in disease processes—a method that's much more like using a sniper rifle than throwing darts blindfolded. Among these targets are proteins, receptors, and enzymes that are fundamental to disease mechanisms.
Expanding Targeted Protein Degradation in Hematologic Malignancies Targeted protein degradation is a powerful therapeutic approach designed to destroy disease-driving proteins within cells, thereby inhibiting their ability to contribute to disease progression.
Shortly after finishing my studies, I landed my first job in industry working on cell biology research for several disease indications. TPD engages the body’s natural protein recycling system by selectively eliminating disease-causing proteins, ultimately addressing the root cause of disease.
Related groups Liu lab Choudhary lab Fischer lab Researchers studying the role of proteins in health and disease use experimental tools that inactivate proteins, destroy them, or prevent them from being made in cells. Liu is also a Howard Hughes Medical Institute investigator and a professor at Harvard University.
Novartis Finalizes Acquisition of Regulus Therapeutics, Strengthening Its Renal Disease Portfolio with Promising ADPKD Therapy Novartis AG, a leading global pharmaceutical company, has officially completed its acquisition of Regulus Therapeutics Inc. , a biotechnology firm known for its expertise in microRNA-targeting therapies.
Given macro healthcare influences (eg, economic uncertainty, environmental changes) and the numerous available treatments for major diseases, drug developers may need to reassess their therapeutic strategies. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.
How does morADC technology combine the properties of smallmolecules and monoclonal antibodies to enhance anti-aggregation effects for CNS applications? The morADC are able to cross the blood-brain barrier more efficiently and offer higher potency than individual parent molecules.
This article compiles recent high-profile clinical readouts and related news with smallmolecules of general interest and structures where they are available. Another THR-Beta Positive Readout in N ASH request a trial You don’t have time to read everything, but you can’t afford to fall behind.
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Monitoring biomarkers can help assess changes in a disease, its level of expression, or the extent of its progression.
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