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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 smallmolecules aimed at revolutionising cancer treatment. This is exciting because often resistance builds up to these other treatments.
All these features make GPCRs central to regulating many vital biological processes in the body, including immune responses and inflammation, and thus especially attractive for therapeutic intervention. GPCR-targeting therapies such as DT-9046 represent a compelling opportunity in that respect, with major clinical and commercial potential.
These therapies have broadened treatment options for patients to expand beyond the more traditional smallmolecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options. 3D rendering of Antibody Drug Conjugate Molecules.
The ability of smallmolecule lipophilic ligands to function as exogenous chaperones was examined using receptor-selective antagonists. Long term treatment leads to increases in receptor abundance and activity with no changes in mRNA supporting a role as pharmacological chaperones.
For example, transcriptomic processes are showing the potential to identify and track failures in gene expression and gene regulation of amyloid and tau-related biomarkers, understood as precursors to the onset of Alzheimers disease (AD). A biomarker is a measurable indicator of a biological process, disease state, or response to a treatment.
A New Treatment Option for a Debilitating Joint Tumor TGCT is a rare, debilitating condition that primarily affects young and middle-aged adults, often striking individuals in their prime working years. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational smallmolecule developed by Abbisko Therapeutics Co.,
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. . Our ambition is to develop life-changing treatments for patients.
Metabolism of 2022 FDA approved smallmolecule drugs part 2 Mixing it Up By Julia Shanu-Wilson In Part 1 of this topic we looked at metabolism of the smallmolecule drugs approved by the FDA in 2022 that were mediated by CYP3A4. Dermavant’s tapinarof is one such friend. 8 This is not the only point of interest.
BRAF and downstream mitogen-activated extracellular signal-regulated kinase (MEK) inhibitors are combined to treat BRAF-mutated melanoma, for example. Classically, rational drug combinations are designed to target two nodes in the same oncogenic signalling pathway.
This master regulator of the regulatory T cell (Treg) genome has opened new horizons in the fight against cancer, offering a precise and tailored solution to modulate the immune system’s response. Can you explain the role of steroid receptor coactivator-3 (SRC-3) in Tregs and how you are leveraging this discovery for immunotherapy?
These multifunctional smallmolecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. Similarly, PROTACs can target and degrade overexpressed proteins, offering a way to overcome drug resistance, a common issue in cancer treatment.
Additionally, treatment led to improvements in height-adjusted total kidney volume (htTKV) , an established clinical marker that correlates with disease progression in ADPKD. These modalities offer a new layer of precision in targeting diseases that are difficult to address with conventional smallmolecules or biologics.
Involved in various physiological processes, such as vision, taste, smell, immune response and neurotransmission, GPCRs are activated by various molecules including hormones, neurotransmitters and environmental stimuli, which trigger a cascade of cellular events that help regulate bodily functions.
Further study revealed that DR3penA may mitigate pulmonary fibrosis by negatively regulating the PI3K/AKT pathway and MAPK pathway. The peptide DR3penA can be further developed for the treatment of multiple fibrotic lung diseases.
Agonist antibodies of immune checkpoint regulators These represent a groundbreaking class of immunotherapeutic agents that mimic the natural function of endogenous ligands by binding to specific cell-surface receptors. This approach augments the strength of achieving immune homeostasis.
Drug discovery is a complex and vital field that continually seeks to identify new therapeutic targets and develop effective treatments. They found that although the targets themselves may not be easily druggable due to their disordered nature, their ability to condense can be modulated using smallmolecules.
Consequently, a GK activator may provide therapeutic treatment for NIDDM and associated complications, inter alia, hyperglycemia, dyslipidemia, insulin resistance syndrome, hyperinsulinemia, hypertension, and obesity. They regulate lipid metabolism subsequently enhancing the response of these tissues to the actions of insulin.
As part of Aptorum Group’s Acticule infectious disease platform, ALS-4 is a novel orally administered first-in-class smallmolecule that was developed based on an anti-virulence approach targeting Staphylococcus aureus including MRSA. About ALS-4.
ALS-4 is a novel smallmolecule adopting an anti-virulence (non-antibiotic) approach to address the growing unmet medical needs of infections caused by Staphylococcus aureus. Pending Health Canada’s approval, the Phase 1 trial is designed to test the safety, tolerability and pharmacokinetics of ALS-4 in healthy volunteers. About ALS-4.
As we delve into the intricate world of biosimilar market dynamics, we’ll explore how these complex molecules are reshaping treatment paradigms across diverse patient populations. Let’s explore how biosimilars are reshaping treatment paradigms across different therapeutic areas. from 2020 to 2025[1].
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. Nature Medicine, 28, 1656–1661 (2022) [link]
But, as is inevitable, there were some disputes about whether certain products should have transitioned, including several that resulted in litigation against FDA for continuing to regulate given products as drugs rather than biologics. Thus, the threat of competition is enough to confer standing.
Biological products have unlocked the potential for the management of several diseases such as cancers and autoimmune diseases for which treatment with smallmolecule, chemically synthesized drug molecules remain suboptimal. […].
In the case of urgent or immediate public interest, process validation may be conducted concurrently with manufacturing the commercial smallmolecule or biologic product to expedite product availability for patients.
The approval of remdesivir ( Veklury ) for the treatment of patients hospitalized with COVID-19 met this legal and scientific standard. The odds of improvement with the 10-day treatment group when compared to those receiving only standard of care were numerically favorable, but not statistically significantly different. Source: FDA.
This activity may be regulated by the K-loop, which is known to be important for KIF1A’s long-range movement. But we may find that we have a wealth of potential smallmolecule solutions for newly recognized disorders, just waiting to be rediscovered, and repositioned. Check out this #ScienceSaturday post to learn more.
SH ) submitted its pivotal phase III clinical trial application (IND) of national class I innovative drug Chiauranib to NMPA, for the treatment of Small Cell Lung Cancer (SCLC) as a single agent for the patients after 2nd-line systemic chemotherapy and recurrence afterwards. (Chipscreen Biosciences, Stock Symbol: 688321.SH
FDA Approves Klisyri (tirbanibulin) for the Treatment of Actinic Keratosis on the Face or Scalp. NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, today announced that the U.S. BUFFALO, N.Y.,
Therapies developed in recent decades have transformed the treatment of IBD, making hospitalisation and surgery less common. However, many patients respond poorly to corticosteroid treatment, or their immune system responds unfavourably to biological therapies, such as the development of autoimmune diseases.
Vatiquinone, developed from PTC’s Bio-e platform, is an investigational oral smallmolecule that inhibits 15-Lipoxygenase, a key enzyme that regulates oxidative stress and inflammation response pathways underpinning many neurological disease pathologies, including epilepsy. Peltz , Ph.D.,
Food and Drug Administration’s Office of Orphan Products Development has granted Orphan Drug Designation to Uttroside-B , a smallmolecule chemotherapeutic for the treatment of hepatocellular carcinoma (HCC), the most common form of liver cancer. (OTCQB: QBIO), announced today that the U.S.
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Metastasis is the most lethal attribute of cancer cells and clinical decisions regarding treatment are based largely upon the likelihood of developing metastases. 1 The research, supported by the National Institutes of Health, centres on a cellular chaperone protein called GRP78, responsible for regulating protein folding within cells.
Lessard created and advanced KIF1A.ORG’s Therapeutic Acceleration Program, facilitating the creation of patient-derived cell lines, mouse models, and drug screening platforms that are the bedrock of our search for treatments. Read more to learn about the factors involved in incentivizing investment in rare disease treatments.
1 Factor B is a positive regulator of the alternative complement pathway, where it activates C3 convertase and subsequently C5 convertase. 2 On December 6th, 2023, Iptacopan under the brand name Fabhalta was approved by the FDA for the treatment of adults with PNH. 1] It is taken by mouth. [1] Retrieved 6 September 2023. April 2019).
About VBL’s VB-600 Platform
VBL is conducting two parallel drug development programs that are exploring the potential of MOSPD2 (motile sperm domain-containing protein 2), a protein that VBL has identified as a key regulator of cell motility, as a therapeutic target for inflammatory diseases and cancer.
That can be a good thing: for example, when determining inherited traits and regulating critical cell functions. IRF5 is a genetically validated Transcription Factor that is important in a variety of diseases and has always been considered undruggable by smallmolecules because it doesn’t have an active site.
Smallmolecule candidate Anle138b targets disease modification for multiple system atrophy and other neurological disorders. With the introduction of small-molecule medication, we open a new chapter in the fight against neurodegenerative diseases and have the chance to improve the lives of millions of patients drastically.”.
Å resolution) 6 is now being targeted for smallmolecule inhibitor discovery and development, by exploiting emergent computational tools to identify potential candidate compounds in silico and then test these predicted inhibitors in in vitro biochemical assays. NIH R01 DA048153 (to D.P.S.), Epub 20230720. doi: 10.1128/jcm.01269-22.
The researchers regulated the abnormal immunological memory processes found in these patients. Treatment induced sustainable clinical responses and reduced systemic inflammation. Daratumumab already is approved for the treatment of multiple myeloma. SmallMolecule Inhibitors. Bioelectronic Platform.
Conjugation of smallmolecule drugs to glucuronic acid is catalysed by several UGTs to frequently form N – and O -glucuronides. As in this example, most N -glucuronides of smallmolecule drugs are innocuous and possess no pharmacological activity. A route to be avoided or a desired route of metabolism?
This acquisition gives AbbVie access to Syndesi’s portfolio of novel modulators of the synaptic vesicle protein 2A (SV2A), including its lead molecule SDI-118. ” The lead molecule, SDI-118, is a smallmolecule currently in Phase 1b studies, which is being evaluated to target nerve terminals to enhance synaptic efficiency. .
In this blog post, we outline typical elements of an IND-enabling package for a single ascending dose (SAD)/multiple ascending dose (MAD) study of an oral smallmolecule for a non-cancer indication with a maximum of 14 days of dosing.
Years of hard work, supported by the National Institutes of Health and the Cystic Fibrosis Foundation, painstakingly worked out the normal function of the protein that is altered in CF, called the cystic fibrosis transmembrane regulator (CFTR). Credit: Zhang & Chen, 2016, Cell 167, 1586–1597. About 30,000 Americans have CF.
AstraZeneca has developed a broad range of technologies, initially focused on smallmolecules and biologics and with a growing focus in precision medicine, genomics, oligonucleotides and epigenetics. 5% have US Food and Drug Administration -approved treatments. Over 7,000 rare diseases are known today, and only c.5%
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