Covalent Modifiers

JANUARY 29, 2024

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 small molecules independently. Moore Journal of Medicinal Chemistry 2024 DOI: 10.1021/acs.jmedchem.3c01835

Small Molecule 173

Small Molecule Drugs Science Disease 173

Chemical Biology and Drug Design

JANUARY 29, 2024

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.

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Small Molecule Immune Response Treatment Disease 100

Drug Hunter

AUGUST 15, 2023

LRRK2 has been a hotly pursued drug target for Parkinson’s Disease based on human genetics.

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Small Molecule Trials Treatment Disease 229

Covalent Modifiers

JANUARY 13, 2024

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.

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Small Molecule International Disease Drugs 195

Drug Target Review

OCTOBER 11, 2023

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.

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Disease Treatment Small Molecule Therapies 113

Covalent Modifiers

JULY 25, 2024

We have integrated structural and quantitative proteomics with biochemical assays to decipher the mode of action of covalent USP30 inhibition by a small molecule containing a cyanopyrrolidine reactive group, USP30-I-1. The inhibitor demonstrated high potency and selectivity for endogenous USP30 in neuroblastoma cells.

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Biochemical Assays Small Molecule Treatment Disease 182

Drug Target Review

JUNE 15, 2023

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 small molecule therapeutics to target RNA processing.

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RNA Small Molecule Disease Assay Development 96

Covalent Modifiers

DECEMBER 11, 2023

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.

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Small Molecule DNA Regulations Disease 162

The Pharma Data

NOVEMBER 17, 2020

Lead Pharma has entered into a collaboration and license agreement with Roche , hoping to lead the way in the development of oral small molecules for immune-mediated diseases like rheumatoid arthritis, psoriasis and inflammatory bowel diseases. . The agreement encompasses Lead Pharma’s entire pipeline.

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Small Molecule Disease Immune Response Licensing 52

Drug Target Review

JULY 13, 2023

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.

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Disease Small Molecule Cell Biology Drugs 98

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule 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.

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Small Molecule Drugs Clinical Pharmacology Trials 145

Dark Matter Blog

AUGUST 27, 2020

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. The efficiency of exon 7 inclusion in the mature SMN2 mRNA varies significantly among patients and poor inclusion efficiency correlates with higher disease severity.

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Small Molecule RNA Clinical Trials Drugs 52

The Pharma Data

JULY 28, 2021

Novel small molecule 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 small molecule compounds directed against the GPR75 target with the potential to treat obesity and related co-morbidities.

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Small Molecule Research Science Disease 52

Metabolite Tales Blog

JANUARY 23, 2024

Metabolism of 2023 FDA Approved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. References [1] 2023 Novel Small Molecule FDA Drug Approvals. Blood 129(13): 1823-1830.

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Small Molecule FDA Approval FDA Pharmacokinetics 52

Drug Discovery Today

APRIL 20, 2023

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 small molecule to target autoimmune disease.

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Small Molecule Research Drugs Pharmaceuticals 113

Metabolite Tales Blog

JANUARY 26, 2023

Metabolism of 2022 FDA approved small molecule drugs – Part 1 Does CYP3A4 still rule? By Julia Shanu-Wilson It won’t come as much surprise to learn that of the 17 small molecules* 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.

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Small Molecule FDA Approval FDA Drugs 52

The Pharma Data

NOVEMBER 15, 2020

R&D collaboration building on Lead Pharma’s expertise in the discovery, design and optimization of small molecule treatments. ” About Immune Mediated Diseases. A wide range of human diseases is driven by deregulated immune function. OSS, Netherlands , Nov. OSS, Netherlands , Nov.

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Small Molecule Licensing Licensing Disease 52

ASPET

JULY 19, 2024

Cardiovascular disease (CVD) remains one of leading causes of death worldwide. Here we design an assay to screen a compound library of small molecules inhibitors. Four compounds were identified as potential small molecule inhibitors one of which, BM-8372, demonstrated significant effect in platelet aggregation assays.

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Small Molecule Disease Research 100

Covalent Modifiers

APRIL 28, 2023

Food and Drug Administration (FDA) approved small molecules. 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.

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Small Molecule FDA Approval FDA Disease 130

Broad Institute

MARCH 14, 2024

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.

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Targeted Protein Degradation Small Molecule Medical Schools Science 141

Drug Target Review

JULY 7, 2023

Approximately three percent of the global population — 240 million people — experience autoantibody diseases, which occur when one’s own body attacks critical organs and tissues. This can create an abnormal immune response that attacks the cells of our bodies and contributes to the development of autoantibody diseases.

Disease 98

Disease Immune Response Therapies Treatment 98

Drug Target Review

JULY 23, 2024

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.

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Small Molecule Targeted Protein Degradation Science Disease 116

DrugBank

MAY 15, 2024

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.

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Small Molecule Molecular Biology Drugs Disease 81

DrugBank

JULY 24, 2024

This is a game-changer, especially in the fight against cancer and other complex diseases. These multifunctional small molecules 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.

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Small Molecule Drugs Clinical Trials Therapies 82

Drug Target Review

SEPTEMBER 11, 2024

How does morADC technology combine the properties of small molecules 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.

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Small Molecule Disease Therapies Drugs 52

Drug Hunter

JUNE 26, 2023

This article compiles recent high-profile clinical readouts and related news with small molecules 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.

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Small Molecule RNA Trials Treatment 130

Drug Target Review

JULY 6, 2023

TPD is a rapidly evolving therapeutic modality to degrade a disease-causing proteins, thereby eliminating their function specifically. 1 TPD is expected to challenge undruggable proteins, which are highly difficult to target by conventional small molecules.

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Targeted Protein Degradation Small Molecule Clinical Trials Science 98

ASPET

SEPTEMBER 16, 2024

Mitochondrial dysfunction is a hallmark of many genetic neurodegenerative diseases, but therapeutic options to reverse mitochondrial dysfunction are limited. Significance Statement Mitochondrial dysfunction is widespread and broadly contributory in neurodegeneration, but difficult to target therapeutically.

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Small Molecule Pharmacokinetics Engineering Disease 100

LifeSciVC

JANUARY 16, 2024

recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Small molecule GLP1s? Kidney disease: outcomes in CKD are likely to benefit from weight loss as well, with the semaglutide Phase 3 FLOW trial stopped early last October after an interim efficacy read (full data expected this year).

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Small Molecule Trials Therapies Disease 138

ASPET

DECEMBER 21, 2023

Interstitial lung diseases (ILDs) are a group of restrictive lung diseases characterized by interstitial inflammation and pulmonary fibrosis. The peptide DR3penA can be further developed for the treatment of multiple fibrotic lung diseases.

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Biochemical Assays Small Molecule Disease Regulations 100

ASPET

JUNE 7, 2024

Acute Kidney Injury (AKI) is characterized by an abrupt decline in kidney function and has been associated with excess risks of death, kidney disease progression, and cardiovascular events.

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Small Molecule Therapies Disease 100

ASPET

SEPTEMBER 7, 2023

Pompe disease is a rare glycogen storage disorder caused by a deficiency in the lysosomal enzyme acid α-glucosidase, which leads to muscle weakness, cardiac and respiratory failure, and early mortality. Avalglucosidase alfa has received marketing authorization in several countries for infantile-onset and/or late-onset Pompe disease.

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Small Molecule Therapies Disease Treatment 100

Drug Target Review

NOVEMBER 25, 2024

As the CEO of iOnctura, an innovative oncology biopharmaceutical company she co-founded in 2017, Catherine has played a key role in advancing the development of highly targeted small molecules aimed at revolutionising cancer treatment. This suited my personality and constant curiosity more than academic research.

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Treatment Small Molecule Clinical Trials Trials 52

Chemical Biology and Drug Design

JULY 27, 2023

Abstract The small molecule, isatin, is a well-known reversible inhibitor of the monoamine oxidase (MAO) enzymes with IC 50 values of 12.3 This study investigated the SARs of MAO inhibition by isatin analogues and discovered five compounds with IC 50  < 1 μM. μM for MAO-A and MAO-B, respectively.

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Small Molecule Treatment Disease Drugs 100

Drug Target Review

OCTOBER 6, 2023

While our focus is on SRC-3 and cancer, it’s just the tip of the iceberg, as the same SRC-3 mechanism can improve non-malignant disease too; including SRC stimulators for myocardial infarction, heart failure and stroke. For what indications can this approach be used? For what indications can this approach be used?

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Small Molecule Engineering Protein Expression Therapies 111

ASPET

SEPTEMBER 28, 2023

Indeed, recognition of the role of SGK-1 in regulation of cell survival and proliferation has led to introduction of a number of small-molecule inhibitors for some types of cancer. The family of serum-glucocorticoid-regulated kinase (SGK) consists of three paralogs- SGK-1, SGK-2 and SGK-3 with SGK-1 being the better studied.

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Regulations Small Molecule Disease Research 100

Drug Target Review

NOVEMBER 6, 2023

Advances in disease understanding and combination strategies in haematology We now know that blood cancers are characterised by molecular and phenotypic heterogeneity. These regimens frequently include one or two small molecule inhibitors and immuno-oncology (IO) therapies, such as monoclonal antibodies and T-cell engagers.

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Small Molecule Therapies Treatment Disease 111