Covalent Modifiers

NOVEMBER 26, 2023

While small molecule inhibitors for the G12C mutant have been successfully developed, allele-specific inhibition for other KRAS hotspot mutants remains challenging. Shokat Journal of the American Chemical Society 2022 144 (35), 15916-15921 DOI: 10.1021/jacs.2c05377

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Drug Target Review

JUNE 16, 2025

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. GPCR-targeting therapies such as DT-9046 represent a compelling opportunity in that respect, with major clinical and commercial potential.

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ASPET

OCTOBER 8, 2024

Tyrosine kinase inhibitors (TKIs) targeting the BCR-ABL fusion protein, such as imatinib (Gleevec), have revolutionized targeted cancer therapies. However, drug resistance and side effects, particularly those affecting hemostasis, continue to pose significant challenges for TKI therapies.

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Covalent Modifiers

AUGUST 14, 2024

Rullo Molecular Therapy 2024 DOI: [link] Proximity-induction of cell-cell interactions via small molecules represents an emerging field in basic and translational sciences. Lake, Galina Denisova,Joanne A. Hammill,Jonathan L. Bramson, Anthony F.

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Chemical Biology and Drug Design

MAY 10, 2025

Although it is currently only used in veterinary therapy, carprofen proves to be a molecule with a versatile therapeutic potential for human pharmacotherapy. Moreover, the molecule is a target in the drug discovery process for the development of new bioactive compounds.

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Drug Target Review

DECEMBER 4, 2024

Confo Therapeutics , led by CEO Dr Cedric Ververken, is at the forefront of developing innovative GPCR-targeted therapies using its proprietary ConfoBody ® platform. In addition to generating agonistic antibodies, Confo is also successfully applying the ConfoBody/ConfoChimer technology to drugging GPCR with small molecules.

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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

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DrugBaron

JULY 19, 2022

Despite the current hype around so called “advanced therapies”, which range from gene editing to cell therapies, and the inexorable advance of biologic therapeutics such as monoclonal antibodies, even in 2022 the majority of drugs in development and reaching patients are still small organic molecules.

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Small Molecule Drugs DNA Pharma Companies 100

New Drug Approvals

JULY 5, 2025

It is a small molecule with a dual mechanism of action, acting as both a complete estrogen receptor antagonist and a selective estrogen receptor degrader (SERD). Combination Therapy: Palazestrant is being evaluated in combination with other drugs like CDK4/6 inhibitors (e.g., ribociclib).

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Drug Target Review

JUNE 6, 2025

With an extensive background in small molecule chemistry, bioconjugation and protein science, these expert teams are leading efforts to place payload development at the centre of ADC innovation. The ADC itself is not necessarily a large molecule – it’s a small molecule with aspects of a large molecule, and vice versa.

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Covalent Modifiers

JUNE 24, 2024

Rullo, Molecular Therapy: Oncology , 2024 , 200842, [link] Proximity-induction of cell-cell interactions via small molecules represents an emerging field in basic and translational sciences. Lake, Galina Denisova, Joanne A. Hammill, Jonathan L. Bramson, Anthony F.

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PPD

NOVEMBER 11, 2024

As a result, biopharma and biotech companies working to bring their drug pipelines to the market require deep expertise from trusted partners to help deliver critical therapies for their patients. According to the United Nation’s 2024 World Population Prospects data, the global population of those 65 years old and over will grow from 0.8

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Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Patients and caregivers also assess the benefits offered by different therapies, weighing the progression-free survival with their off-target effects.

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Broad Institute

NOVEMBER 2, 2023

Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.

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ASPET

OCTOBER 12, 2023

The self-assembled structures can be small molecules, polymers, peptides, proteins, which can be utilized and functionalized to achieve tailored release and target specific cells, tissues, or organs. Significance Statement This review focuses on fundamentals and various drug delivery mechanisms based on SNs.

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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. . This includes therapies against the nuclear receptor ROR?t,

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Drug Target Review

JANUARY 4, 2024

What distinguishes Alder Therapeutics’ approach to regenerative cell therapy development from traditional methods, and how does it aim to reduce risks in the preclinical phase? Traditional approaches to regenerative cell therapy development are defined by several challenges. So, what makes these therapies unique?

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DrugBank

JULY 24, 2024

These multifunctional small molecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed.

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New Drug Approvals

JULY 7, 2025

Food and Drug Administration (FDA) and the Center for Drug Evaluation (CDE) of NMPA granted pimicotinib breakthrough therapy designation (BTD) for the treatment of tenosynovial giant cell tumor patients that are not amenable to surgery in January 2023 and July 2022, respectively. [7] 6] The U.S. 15 : 53–66. doi : 10.2147/OTT.S345878.

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Drug Target Review

JUNE 2, 2025

Bioconjugates are rapidly emerging as a key frontier in targeted therapies, particularly through Antibody-Drug Conjugates (ADCs) , which combine antibodies with cytotoxic drugs to deliver more precise and effective treatments. Very often, you have a payload that is extremely toxic, and then you require specially trained people to handle it.

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Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. In some cases, cell therapy can be curative for the patient.

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ASPET

SEPTEMBER 7, 2023

This recently approved enzyme replacement therapy (ERT) was glycoengineered to maximize CIMPR binding through high-affinity interactions with ~7 bis-M6P moieties. 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

Broad Institute

OCTOBER 4, 2023

By Allessandra DiCorato October 4, 2023 Credit: AbbVie The new small molecule inhibitor (green) sits inside the PTPN2 protein, where acidic sites are marked in red and basic sites are marked in blue. A new small-molecule drug candidate being tested in an early-stage clinical trial aims to improve patient responses to immunotherapy.

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Alta Sciences

JULY 3, 2025

Because this transformation can significantly improve the stability, solubility, and overall performance of pharmaceutical compounds, especially when it comes to small molecules. Why does that matter? Beyond the science, what’s exciting is how this technology opens doors. The response? Ready to advance your next project?

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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. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.

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Covalent Modifiers

AUGUST 14, 2023

Acquired cysteines are both driver mutations and sites targeted by precision therapies. However, despite their ubiquity, nearly all acquired cysteines remain uncharacterized.

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The Pharma Data

JUNE 12, 2025

This stands in contrast to traditional small-molecule inhibitors, which typically aim to block a protein’s activity or binding. Bristol Myers Squibb’s platform includes a range of small-molecule degraders, which exploit the cell’s own disposal mechanisms — typically by sending the target protein to the proteasome for destruction.

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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. It is somewhat like how PROTACs have changed the perspective on what is a tractable therapeutic target.

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Drug Target Review

OCTOBER 6, 2023

This interview explores how CoRegen’s innovative techniques are not only transforming cancer treatment but also hold promise for a wide range of medical applications, all while minimising the side effects often associated with traditional therapies. What are the key benefits of your approach?

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Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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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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DrugBank

MAY 15, 2024

One approach is to look beyond the traditional drug molecule. Researchers are experimenting with biologics—larger biological molecules that can do things small molecules can't, like targeting larger, more complex structures on cell surfaces or even inside cells.

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LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. Small molecule GLP1s?

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Drug Target Review

OCTOBER 11, 2023

1,2 Many, however, do have dysfunctional proteins at their core, and for these diseases, a new option is being explored: autophagy-boosting therapies. Gene therapy, of course, also holds great promise. Autophagy boosters, in contrast, are conventional small molecule drugs.

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Drug Target Review

JULY 3, 2025

New approach methodologies data In April, the US Food and Drug Administration (FDA) announced a plan to replace animal testing in the development of monoclonal antibodies and other therapies with validated “human-relevant” methods, including AI-based computational models evaluating toxicity, cellular lines and organoid toxicity.

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Drug Target Review

NOVEMBER 6, 2023

2 It is this complexity that necessitates powerful, targeted combination therapies. 4, 5 More recently, new combination regimens have emerged that incorporate targeted therapies to treat a variety of blood cancers, including multiple myeloma (MM), chronic lymphocytic leukaemia (CLL) and acute myelogenous leukaemia (AML).

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Drug Target Review

JUNE 11, 2025

Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments.

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