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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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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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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Drug Target Review

JULY 3, 2025

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.

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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.

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The Pharma Data

JUNE 25, 2025

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.

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Drug Target Review

APRIL 7, 2025

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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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.

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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.

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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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Drug Target Review

JUNE 11, 2025

NGS has revolutionised genomic analysis, enabling the identification of disease-related genetic variants. This ability to correlate seemingly disparate data is crucial for tackling complex targets and discovering new treatments for diseases considered difficult to treat with conventional approaches.

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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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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.

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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.

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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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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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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.

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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?

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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.

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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.

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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.

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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.

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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.

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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.

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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?

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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.

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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.

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The Pharma Data

OCTOBER 27, 2021

Small molecule candidate Anle138b targets disease modification for multiple system atrophy and other neurological disorders. Anle138b targets pathological alpha-synuclein oligomers and is being evaluated in patients with neurodegenerative diseases for potential disease modification. Multiple System Atrophy.

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The Pharma Data

MAY 31, 2022

BIIB122 is an inhibitor of LRRK2, a potential novel target intended to impact the underlying biology and slow the progression of Parkinson’s disease Phase 2b LUMA to enroll approximately 640 participants with early-stage Parkinson’s disease; most advanced clinical study of a LRRK2 inhibitor. Denali Therapeutics Inc.

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The Pharma Data

DECEMBER 15, 2020

A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.

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The Pharma Data

JANUARY 5, 2021

Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. This is because many proteins do not have small-molecule binding sites. This is not the first deal for RNA therapeutics for Genentech.

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PerkinElmer

MAY 28, 2020

Time is of the essence when it comes to the development of a vaccine for the novel infectious disease in our current pandemic. The importance of pseudoviruses Because of their versatility and safety, tools such as pseudoviruses can be used by virologists as an alternative method to develop a vaccine for the novel viral disease.

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Sygnature Discovery

APRIL 18, 2023

Sygnature Discovery is pleased to have entered into a research collaboration agreement with the global healthcare group Daewoong Pharmaceutical to accelerate the discovery of a novel small molecule to target autoimmune disease.

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Broad Institute

SEPTEMBER 26, 2024

Yes, it changed me as a researcher: I gained the skills to overcome obstacles in science, plan and execute my experiments, and dive deep into the background of a project to build confidence in my research area. Additionally, astrocyte inflammation has been observed in patients with many neurodegenerative diseases.

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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.

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KIF1A

MAY 27, 2023

Dylan Verden of KIF1A.ORG summarize newly published KIF1A-related research and highlight progress in rare disease research and therapeutic development. Goodbye with Gratitude: Dr. Dominique Lessard It is with heavy hearts that this week we say goodbye to Dr. Dominique Lessard, Chief Science Officer of KIF1A.ORG. What’s a pre-print?

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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.

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