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Related groups Xavier lab Over the past two decades, large genetic studies have linked tens of thousands of DNA variants to thousands of human traits and diseases. Yet, correcting the effects of those variants to treat disease has been hampered in part by the lack of precise molecular tools to do so.
. #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.
sPLA 2 inhibitors have been developed for the treatment of inflammatory and other conditions such as cardiovascular disease, arteriosclerosis and rheumatoid arthritis. The three-dimensional structures presented in this review have been published by various research groups and have been deposited in the Protein Data Bank.
Global integrated drug discovery company, Sygnature Discovery and global healthcare group Daewoong Pharmaceutical, have entered into a research collaboration agreement to accelerate the discovery of a novel smallmolecule to target autoimmune disease.
Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.
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.
In recent years, a novel approach known as condensate biology has emerged, revolutionising the way researchers think about drug discovery and development. He now leads research and development (R&D) at Dewpoint. Dewpoint’s breakthrough was the realisation that certain condensates are associated with human diseases.
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. . The agreement encompasses Lead Pharma’s entire pipeline.
a new Italian incubator dedicated to the discovery and development of smallmolecule-based treatments for neurodegenerative diseases, announced today that it has adopted Collaborative Drug Discovery’s CDD Vault as its platform of choice for managing drug discovery data.
Novel smallmolecule 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 smallmolecule compounds directed against the GPR75 target with the potential to treat obesity and related co-morbidities.
These therapies have broadened treatment options for patients to expand beyond the more traditional smallmolecule drug alternatives. In practice, some patients may be left managing a syndrome associated with the therapy along with the disease. 3D rendering of Antibody Drug Conjugate Molecules.
R&D collaboration building on Lead Pharma’s expertise in the discovery, design and optimization of smallmolecule treatments.
” About Immune Mediated Diseases. A wide range of human diseases is driven by deregulated immune function.
OSS, Netherlands , Nov.
OSS, Netherlands , Nov.
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.
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. PACE allows researchers to rapidly evolve new proteins that have useful features.
A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with smallmolecules. The efficiency of exon 7 inclusion in the mature SMN2 mRNA varies significantly among patients and poor inclusion efficiency correlates with higher disease severity.
Cardiovascular disease (CVD) remains one of leading causes of death worldwide. Here we design an assay to screen a compound library of smallmolecules inhibitors. Four compounds were identified as potential smallmolecule inhibitors one of which, BM-8372, demonstrated significant effect in platelet aggregation assays.
Shortly after finishing my studies, I landed my first job in industry working on cell biology research for several disease indications. At the time, research and leadership roles in biotech/pharma industry were predominately held by men and there were not as many opportunities for career development and mentorship from women leaders.
Approximately three percent of the global population — 240 million people — experience autoantibody diseases, which occur when one’s own body attacks critical organs and tissues. This can create an abnormal immune response that attacks the cells of our bodies and contributes to the development of autoantibody diseases.
Researchers would sift through natural extracts , hoping to stumble upon a compound that exhibited beneficial effects. 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.
This is a game-changer, especially in the fight against cancer and other complex diseases. These multifunctional smallmolecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. This can potentially correct the root causes of some diseases at the genetic level.
By Makenzie Kohler October 17, 2023 Credit: Allison Colorado, Broad Communications Amy Deik is a research scientist and lab manager in the Metabolomics Platform. She’d always had an interest in genetics research and moved to Boston in 2002 to work for a genetics-focused startup. What do you do as a research scientist at Broad?
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 smallmolecules.
How does morADC technology combine the properties of smallmolecules 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.
recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Smallmolecule GLP1s? Rise of the best-in-class biologics plays: Are you looking to in-license or invest in “de-risked” biology that is already in the clinic? Join the club. Additional trials (e.g.,
Interstitial lung diseases (ILDs) are a group of restrictive lung diseases characterized by interstitial inflammation and pulmonary fibrosis. Our previous research showed that the novel and low-toxicity peptide DR3penA had a strong antifibrotic effect on a bleomycin-induced murine model.
Advances in research and development and the utilisation of combination treatment strategies are revolutionising the haematology care landscape, but more must be done to address patients’ individual needs. 2 It is this complexity that necessitates powerful, targeted combination therapies.
Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize.
researching DNA photoproducts related to skin cancer. Her interest in research landed her a job at Genentech, where she focused on ADCs—and learned other skills critical to her career. Remember my post-doc research? In her smallmolecule DMPK research, proteomics also served to identify reactive metabolites (e.g.,
Preclinical research demonstrated that removing SRC-3 from Tregs resulted in long-lasting tumour eradication and prevented growth of new tumours. Could you provide more information about CoRegen’s SRC small-molecule inhibitor (SMI) and SRC small-molecule enhancer (SMs) within your portfolio?
Indeed, recognition of the role of SGK-1 in regulation of cell survival and proliferation has led to introduction of a number of small-molecule inhibitors for some types of cancer. Significance Statement Since the discovery of SGK-1, extensive research has unraveled its role in cancer biology and, thus, its therapeutic targeting.
2 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 1-5 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. He is a researcher at University of Oldenburg, Germany.
Antibody-drug conjugates (ADCs) have been a groundbreaking approach to cancer treatment with their ability to deliver cytotoxic drugs directly to diseased cells while sparing healthy tissues. The research community pressed on, turning to novel compounds that were substantially more potent than those used in the first generation.
In the quest to combat and eradicate diseases, scientists and researchers are increasingly turning to the immense potential of artificial intelligence (AI) as a tool. It was here that the Impact Medicine Fund was raised as a concept around wellness for those with disease, with the ambition of unlocking some of this vast potential.
Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare diseaseresearch and therapeutic development. This would allow a single gene therapy to replace an area of KIF1A that includes multiple disease-causing mutations, and treat multiple mutations at once.
(Headquarters: Cambridge, UK, “Wren”) today announced that the companies have entered into an exclusive research collaboration agreement aiming to advance the discovery of novel smallmolecules that target ?-synuclein synuclein, which is associated with the onset and progression of these diseases. The accumulation of ?-synuclein
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 smallmolecule to target autoimmune disease.
Abstract The smallmolecule, isatin, is a well-known reversible inhibitor of the monoamine oxidase (MAO) enzymes with IC 50 values of 12.3 This study investigated the SARs of MAO inhibition by isatin analogues and discovered five compounds with IC 50 < 1 μM. μM for MAO-A and MAO-B, respectively.
Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists.
We are now committed to pursuing other CGRP-mediated diseases through advancing novel investigational agents such as HTL0022562 into human studies.”
TOKYO and CAMBRIDGE, England , Dec. Vlad Coric , M.D.,
Applying our technology has enabled us to develop a new smallmolecule, 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 smallmolecule binds to the bromodomain of CBP/p300 in a highly specific manner.
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 smallmolecules against predicted future variants of SARS-CoV-2 and other related human viruses. References. Hilgenfeld, Febs J. 2014), 281, 4085-4096. Pillaiyar, et al, J.
Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare diseaseresearch and therapeutic development. Using similar logic, researchers measured the movement of KIF1A homodimers (a pair of healthy KIF1A motors, or a pair of mutant KIF1A motors).
Smallmolecule 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.
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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