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Smallmolecule drugs make up most of the drugs we take conveniently as pills, including painkillers like ibuprofen (Advil), antibiotics like penicillin and amoxicillin, or cholesterol-lowering drugs like atorvastatin (Lipitor). The smallmolecules drugs of today look nothing like the molecules of the 1970s.
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.
ABSTRACT Drug targeting strategies, such as peptidedrug conjugates (PDCs), have arisen to combat the issue of off-target toxicity that is commonly associated with chemotherapeutic smallmolecule drugs.
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.
Under a collaboration with NextRNA Therapeutics, Bayer will access the biotech’s platform to target long, non-coding RNA interactions with smallmolecule drugs.
Herein, we use afatinib as a lead to undertake a structure-based drug design approach, aided by mass-spectrometry and x-ray crystallography, to develop DHC-156, a smallmolecule that more selectively binds brachyury and downmodulates it as potently as afatinib.
Researchers have succeeded in restoring lost brain function in mouse models of stroke using smallmolecules that in the future could potentially be developed into a stroke recovery therapy.
These therapies have broadened treatment options for patients to expand beyond the more traditional smallmolecule drug alternatives. Patients and caregivers also assess the benefits offered by different therapies, weighing the progression-free survival with their off-target effects.
The most common adverse effects are hyperphosphatemia caused by FGFR1 inhibition and diarrhea due to FGFR4 inhibition, as current therapies are not selective among the FGFRs.
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.
The self-assembled structures can be smallmolecules, 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.
While smallmolecule 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
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?
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. . This includes therapies against the nuclear receptor ROR?t,
A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with smallmolecules. Using gene therapy, their approach was beautifully straightforward – “Missing an important gene? But we have not been the only people on this mission. We will replace it.”
Precision medicine developer Kura Oncology is positioned to make a meaningful difference in the treatment of cancer in the next few years with its two, wholly-owned, smallmolecule compounds. As with any innovative therapy, “It takes multiple iterations and time to get things right,” he pointed out. “We Photo courtesy of Kura.
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.
2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates. These can include anti-tubulin compounds, potentially reducing side effects and improving quality of life.
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.
By Allessandra DiCorato October 4, 2023 Credit: AbbVie The new smallmolecule 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.
These multifunctional smallmolecules 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.
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. It is somewhat like how PROTACs have changed the perspective on what is a tractable therapeutic target.
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?
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.
Rullo Molecular Therapy 2024 DOI: [link] Proximity-induction of cell-cell interactions via smallmolecules represents an emerging field in basic and translational sciences. Lake, Galina Denisova,Joanne A. Hammill,Jonathan L. Bramson, Anthony F.
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 smallmolecules.
We developed a selective potent smallmolecule pan AMPK activator, compound 1 , and tested its ability to increase AMPK activity and preserve kidney function during ischemia/reperfusion injury in rats. Significance Statement No approved pharmacological therapies currently exist for acute kidney injury.
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 smallmolecule drugs.
One approach is to look beyond the traditional drug molecule. Researchers are experimenting with biologics—larger biological molecules that can do things smallmolecules can't, like targeting larger, more complex structures on cell surfaces or even inside cells.
Groundbreaking strategies like proteolysis-targeting chimeric molecules (PROTACs) are also being explored. 6 Combining the effect of payloads with different mechanisms of action – an approach that revolutionised smallmolecule chemotherapy – also holds the promise of enhanced therapeutic activity for ADCs.
This smallmolecule reactivates the apoptosis cascade in tumor cells while sparing healthy cells in animal models. Apoptosis, or programmed cell death, is a natural end process for all cells. Trueline Therapeutics also tested the compound in a canine model and did not detect any markers of cardiac toxicity.
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. Smallmolecule GLP1s?
New approach may yield modified messenger RNAs for treating a wide range of conditions By Ari Navetta September 23, 2024 Breadcrumb Home New approach may yield modified messenger RNAs for treating a wide range of conditions The LEGO framework could help scientists create mRNA therapies that last longer and require smaller doses.
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).
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
This laid the groundwork for my career in drug discovery using novel modalities like smallmolecule inhibitors, antibodies, RNA interference (RNAi) and, currently, smallmolecule protein degraders – which are opportunities to change how we think about medicines.
Rare Roundup KRIBB develops new gene therapy candidate for hereditary spastic paraplegia This week we’re flipping the format and starting with our Rare Roundup, after families contacted us with this article about a gene therapy candidate for hereditary spastic paraplegia.
How significant are the results in terms of advancing TCR-T cell therapy and AMP immunotherapy for the treatment of solid tumours? We have already applied this approach to several important tumour targets where enhancements in TCR-T therapy may lead to important gains in clinical benefit, including mutated KRAS, NY-ESO, and HPV E7 antigens.
“Manufacturing cells is not like manufacturing smallmolecules,” Brian Culley, CEO of Lineage Cell Therapeutics , told BioSpace. For cell therapy products to mature into ‘real’ products that deliver on the promises of 10 years ago, they must be scalable – which drives affordability – and they must solve their purity issues.”.
Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists.
We look forward to working on this new collaboration with Biohaven, a world leader in the clinical development of CGRP-targeted therapies.”
TOKYO and CAMBRIDGE, England , Dec. Vlad Coric , M.D.,
Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. This blog will outline three characteristics of a successful nonclinical program to support entry into clinical trials for a cell or gene therapy product.
Designing an adequate nonclinical program to support the safety of cell or gene therapy products is not always straightforward. The cell or gene therapy should be pharmacologically active in the animal model. Note that there is no strict requirement of toxicity studies in two species, which is often needed for smallmolecule programs.
The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.
Rullo, Molecular Therapy: Oncology , 2024 , 200842, [link] Proximity-induction of cell-cell interactions via smallmolecules represents an emerging field in basic and translational sciences. Lake, Galina Denisova, Joanne A. Hammill, Jonathan L. Bramson, Anthony F.
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