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. I went on to complete my MBA and PhD at The Institute of Cancer Research (ICR) in drug development.

Treatment

BioPharma Drive: Drug Pricing

JULY 8, 2025

Love Employee via Getty Images For decades now, scientists have been experimenting with a new way to target and destroy proteins linked to disease. It may also be a more efficient way to shut down known disease targets. Drugmakers are working to coopt this process with targeted therapies.

Targeted Protein Degradation

Drug Target Review

JUNE 16, 2025

Targeted drug delivery and therapeutic development MSI offers significant advantages for those developing small molecule therapeutics. Multimodal Imaging Distribution Assessment of a Liposomal Antibiotic in an Infectious Disease Model. For instance, Cheng et al. Bioanalysis 2019, 11 (11), 1099–1116. Release 2022, 352 , 199–210.

Drugs

Drug Target Review

APRIL 7, 2025

The challenge of GPCR drug discovery G protein-coupled receptors (GPCRs) are one of the most desirable and challenging target classes in drug discovery, as their mutation can lead to a wide range of diseases such as cancer, cardiovascular disorders and neurological conditions.

Fragment Screening

Drug Target Review

JULY 18, 2025

This opens the door for a disease-modifying approach both as a monotherapy and in combination, expanding treatment options and offering new hope to millions of patients.” Whether we are working with small molecules or larger biologics, our platform enables a precise design of innovative drug candidates with highly differentiated profiles.”

Medical Chemistry

Drug Target Review

DECEMBER 4, 2024

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.

Small Molecule

The Pharma Data

JUNE 24, 2025

The extended agreement will support joint research efforts targeting critical therapeutic areas, including oncology, cardiovascular and renal diseases, neurology, rare diseases, and immunology. This progress is driven by the dedicated efforts and strong collaborations among innovators of science and business.

Pharma R&D

The Pharma Data

JUNE 9, 2025

Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co., A New Chapter in TGCT Management If approved, pimicotinib would become the first systemic therapy for TGCT in China , potentially redefining the treatment landscape for this rare but impactful disease.

Treatment

Sygnature Discovery

APRIL 7, 2025

Introducing a novel payload -focused approach to next generation ADC development NewPath ADC offers integrated solutions for antibody drug conjugates, designed by highly skilled multidisciplinary teams covering medicinal chemistry and protein sciences, as well as in vitro and in vivo assessment (including DMPK). Big ADC problem?

Small Molecule

sptlabtech

DECEMBER 6, 2024

3D models better replicate the in vivo conditions that are critical for studying biological processes and disease progression, whilst providing an alternative to animal-based studies. Additionally, the ability to use patient-derived cells in 3D models has opened the door to personalized medicine approaches.

Small Molecule

LifeSciVC

JULY 31, 2025

there is the potential to unearth a completely new finding for a target that completely alters the paradigm for a disease. Harnessing our Smart Allostery platform, we have developed the first small molecule inhibitors of the CBM complex to demonstrate binding the signalosome and locking it into an inactive conformation.

Small Molecule

PPD

APRIL 14, 2025

Last year, an American Heart Association presidential advisory for the first time formally identified the strong connections between cardiovascular disease (CVD), kidney disease, Type 2 diabetes and obesity as reason to define cardiovascular-kidney-metabolic (CKM) syndrome.

Clinical Trials

Science Daily: Pharmacology News

APRIL 8, 2025

Experimental drug NU-9 -- a small molecule 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.

Small Molecule

Drug Hunter

APRIL 20, 2023

. #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.

Small Molecule

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

Science Daily: Pharmacology News

MARCH 26, 2025

A small molecule shaped like a lasso may be a powerful tool in the fight against infectious diseases.

Small Molecule

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.

RNA

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.

Disease

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.

Small Molecule

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.

Small Molecule

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. The new study appears in Science. Paper cited Mercer JAM, et al. March 14, 2024.

Targeted Protein Degradation

Drug Target Review

JULY 23, 2024

I studied Natural Sciences at the University of Cambridge, where I was exposed to a range of disciplines from chemistry to physiology, specialising in my last year in biochemistry. Shortly after finishing my studies, I landed my first job in industry working on cell biology research for several disease indications.

Small Molecule

DrugBank

MAY 15, 2024

This method was more about serendipity than science. 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. One approach is to look beyond the traditional drug molecule.

Small Molecule

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.

Targeted Protein Degradation

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.

Small Molecule

DrugBank

OCTOBER 17, 2024

Data science has emerged as an innovative tool in the biopharmaceutical industry, leveraging the power of machine learning and artificial intelligence to drive innovation and efficiency across the entire drug development lifecycle. These complex molecules require precise engineering to ensure optimal efficacy and safety.

Drug Development

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).

Small Molecule

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.

Small Molecule

DrugBank

MAY 22, 2024

These tough nuts to crack in medical science—biological targets known to play roles in diseases but resistant to traditional drug design—are now seeing new strategies that shift the paradigm from "undruggable" to "druggable." " The Challenge of Selectivity  Why are some targets so elusive?

Small Molecule

Broad Institute

OCTOBER 17, 2023

That interest in science led her to a weekend science course and a field trip to a plant research laboratory, where she saw scientists hybridizing roses and genetically modifying tomatoes. A lot of what we do at the Metabolomics Platform is look for early indicators of disease and use metabolomics to monitor disease progression.

Research

LifeSciVC

JULY 6, 2023

Although these results cannot prove that there is no possibility of finding a high-affinity small molecule binder of NBD1, they are discouraging…” It turns out the small molecules they were looking for are not only possible – they were ready for a dedicated team to push them ahead.

Small Molecule

Drug Target Review

SEPTEMBER 25, 2024

Applying our technology has enabled us to develop a new small molecule, TT125-802, and to assign a new role to the epigenetic regulator CBP/p300 as a novel master regulator of non-oncogene resistance. This orally available small molecule binds to the bromodomain of CBP/p300 in a highly specific manner.

Small Molecule

The Pharma Data

NOVEMBER 19, 2020

The Company is also aiming to leverage the highly conserved structure of the SARS-CoV-2 M pro protease as a basis for the design of novel oral small molecules against predicted future variants of SARS-CoV-2 and other related human viruses. Zhang, et al, Science (2020) DOI: 10.1126/science.abb3405. References. Hilgenfeld, Febs J.

Small Molecule

KIF1A

JULY 22, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. A suppressor mutant means the disease model worm shows movement defects, however the suppressor shows better movement. Endpoints are measurements of a disease.

Small Molecule

KIF1A

JULY 1, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. In science, aligning mathematical models with experimental observations improves our understanding of the underlying biology. What’s a pre-print? . What’s a pre-print?

Small Molecule

NIH Director's Blog: Drug Development

OCTOBER 30, 2018

Now comes word of another absolutely incredible use of cryo-EM: determining with great ease and exquisite precision the structure of the smaller organic chemical compounds, or “small molecules,” that play such key roles in biological exploration and drug development. Also analyzed were eight less-familiar small molecules.

Small Molecule

DrugBank

JANUARY 9, 2025

This lack of selectivity often results in significant side effects, including nausea and limited efficacy, particularly in advanced or metastatic disease. This targeted therapy is a small molecule inhibitor of the BCR-ABL tyrosine kinase, the oncogenic driver of CML. However, targeted therapies have limitations.

Therapies