Drug Target Review

NOVEMBER 1, 2023

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. Mediators of Inflammation. 2017;2017:1–15. Herr DR, Chew WS, Satish RL, Ong WY.

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Drug Discovery World

JANUARY 4, 2024

ANX1502 is a first-in-kind oral, selective small molecule inhibitor that targets the active form of C1s responsible for propagating classical pathway activation in association with C1q. The post Positive Phase I results for first-in-kind small molecule complement inhibitor appeared first on Drug Discovery World (DDW).

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Sygnature Discovery

NOVEMBER 29, 2023

The QN-302 Phase 1 clinical trial is a multi-site, open-label, dose-escalation and dose expansion, safety, pharmacodynamic and pharmacokinetic study of intravenous QN-302 in patients with advanced or metastatic solid tumors. A total of up to 36 patients will be enrolled in the dose escalation (Phase 1a) portion of the study. “The

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The Pharma Data

JULY 2, 2023

To this end, the team carefully evaluated hits from a screen of around ten thousand small molecule compounds and identified icFSP1 as a new in vivo effective drug. Although FSP1 has been considered as an attractive drug target for cancer therapy, in vivo efficacious FSP1 inhibitors have been lacking. Source link: [link]

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Metabolite Tales Blog

JANUARY 23, 2024

Metabolism of 2023 FDA Approved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. References [1] 2023 Novel Small Molecule FDA Drug Approvals. link] [11] Zheng et al.,

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Drug Discovery World

JULY 11, 2024

The funding will help advance Mission’s potential disease-modifying treatment for Parkinson’s disease (PD), MTX325. MTX325 is a potent, selective, small molecule brain-penetrant USP30 inhibitor, which is designed to protect dopamine-producing neurons by improving mitochondrial quality and function.

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Sygnature Discovery

NOVEMBER 29, 2023

The QN-302 Phase 1 clinical trial is a multi-site, open-label, dose-escalation and dose expansion, safety, pharmacodynamic and pharmacokinetic study of intravenous QN-302 in patients with advanced or metastatic solid tumors. A total of up to 36 patients will be enrolled in the dose escalation (Phase 1a) portion of the study. “The

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Molecular Design

FEBRUARY 7, 2023

> I’ll start this post by with reference to a disease that some of you many never have heard of. Chagas disease is caused by the very nasty T. cruzi parasite (not to be confused with the even nastier American politician) and is of particular interest in Latin America where the disease is endemic.

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Alta Sciences

JANUARY 31, 2024

Pharmacokinetics, Pharmacodynamics and Toxicokinetics Demystified pmjackson Wed, 01/31/2024 - 14:55 Understanding the effects of a drug, and how it interacts with the body, and vice versa, is critical to ensure it is safe for human use. This is where pharmacokinetic (PK), pharmacodynamic (PD) , and toxicokinetic (TK) analyses step in.

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The Pharma Data

FEBRUARY 25, 2022

The study met its primary goal by demonstrating pharmacokinetic equivalence in patients who switched multiple times between treatment with the two medicines. The root cause of many immunological diseases is immuno-inflammation, which requires specifically designed agents.

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Drug Target Review

JUNE 12, 2023

The current version of Chemistry42 uses over 40 generative models, including generative autoencoders and generative adversarial networks as well as both structure-based and ligand-based drug design to generate and optimise de novo small molecules.

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Drug Discovery World

SEPTEMBER 9, 2024

Vesper Bio has completed a Phase I study for its lead candidate, VES001, a potentially disease-modifying treatment for frontotemporal dementia patients with mutations in the progranulin gene (FTD(GRN)). Frontotemporal dementia is the most common cause of dementia in people under the age of 60.

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Drug Target Review

JULY 12, 2023

These assays provide insights into the molecular mechanisms of disease biology and drug response, enabling the characterisation of gene expression profiles and deviations in diseased cells. Importantly, transcriptomics information shows that certain aspects of a disease change the gene expression profile.

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The Pharma Data

OCTOBER 27, 2020

The company’s lead program is aimed at Friedrich’s Ataxia, a rare inherited genetic disease that causes cardiac and central nervous system dysfunction. AavantiBio has a unique opportunity to change the lives of those living with FA and other rare diseases,” Cumbo said. and China. said of B cells’ potential. “We

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The Pharma Data

APRIL 11, 2023

A focus of the presentation will be on the target engagement results and pharmacokinetics from its ongoing monotherapy dose-escalation study to explore a potential optimal dose and schedule to effectively inhibit AhR.

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The Pharma Data

AUGUST 21, 2020

As an expert in infectious diseases, Jennifer Leeds could see the storm brewing. The short-term goal of CARE is to provide a quick emergency response to the pandemic by testing existing drugs that were designed to treat other diseases against COVID-19. These will be screened against SARS-CoV-2.

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The Pharma Data

NOVEMBER 11, 2020

Adding a new building, which will house up to 100 biologists by the end of the year, will bring together all the key functions for cutting-edge and high-performance small molecule discovery and up to commercial development. On this basis, Evotec has built a broad and deep pipeline of approx.

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Drug Discovery World

OCTOBER 18, 2022

Enanta Pharmaceuticals, a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, has dosed the first subject in its Phase I clinical trial of EDP-323, a novel, oral L-protein inhibitor in development for the treatment of respiratory syncytial virus (RSV). .

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Drug Discovery World

JULY 8, 2024

While mRNA vaccines and RNA interference therapies have garnered much attention, a new class of RNA therapeutics known as aptamers is gaining momentum, particularly to address acute diseases. By silencing transthyretin (TTR) mRNA, patisiran decreases the production of TTR protein, the molecule responsible for causing the disease.

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Drug Discovery World

FEBRUARY 22, 2024

Libra Therapeutics is a San Diego-based biotech focused on developing novel disease-modifying therapeutics, which restore the cellular balance that is disrupted in neurodegenerative diseases. This article is sponsored by Collaborative Drug Discovery.

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The Pharma Data

AUGUST 29, 2020

Seven of ten patients discontinued eculizumab and remained on LNP023 as monotherapy, retaining hemoglobin (Hb) levels with no changes in biomarkers of disease activity and with no signs or symptoms of breakthrough hemolysis . Peffault de Latour.

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The Pharma Data

DECEMBER 20, 2020

Pending Health Canada’s approval, the Phase 1 trial is designed to test the safety, tolerability and pharmacokinetics of ALS-4 in healthy volunteers. ALS-4 is a novel small molecule adopting an anti-virulence (non-antibiotic) approach to address the growing unmet medical needs of infections caused by Staphylococcus aureus.

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Drug Discovery World

NOVEMBER 3, 2022

VRG50635 is a small molecule inhibitor of PIKfyve, a novel therapeutic target for ALS discovered by CONVERGETM, Verge’s all-in-human, AI-powered platform. . Through the evaluation of more than 11.4 The CONVERGETM platform starts with, and integrates human data and human model systems throughout discovery and development.

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The Pharma Data

JANUARY 19, 2021

The secondary objective is to assess the pharmacokinetic profile of SAD and MAD of ALS-4 administered orally to healthy subjects. The primary objective of the trial is to evaluate the safety and tolerability of SAD and MAD of ALS-4 administered orally to healthy subjects. About ALS-4.

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LifeSciVC

APRIL 24, 2024

Target validation : Do we believe the target(s) plays a central role in disease biology and that modulation will modify disease? There is nothing more exciting than digging into a new target and trying to develop a thesis on whether modulation may be impactful in disease. Is the functionality of mutations known?

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The Pharma Data

NOVEMBER 17, 2020

The study demonstrated favorable proof-of-concept for LYT-100’s tolerability and pharmacokinetic (PK) profile, which will also enable twice-a-day (BID) dosing of LYT-100 in future studies. IPF, unclassifiable interstitial lung diseases (uILDs), Long COVID respiratory complications and related sequelae) and lymphedema.

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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

NOVEMBER 4, 2020

Published Findings on Alopecia Areata Burden of Disease in Journal of Investigative Dermatology. Concert Pharmaceuticals is a clinical stage biopharmaceutical company that is developing small molecule drugs that it discovered through the application of its DCE Platform® (deuterated chemical entity platform).

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The Pharma Data

JANUARY 19, 2021

a privately-held neuron regeneration therapeutics company, today announced the publication of results of their novel pharmacotherapy: NNI-362 to allosterically stimulate neural regeneration in human cultures and in vivo models of aging and disease. There is a great need for a disease-modifying treatment for the 5.3 CLARKSVILLE, Md.,

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Drug Discovery World

APRIL 12, 2023

Testing new modality drugs poses additional challenges The drug development pipeline is no longer predominantly composed of small molecule drugs. The importance of recapitulating metabolic disease phenotypes for toxicology studies Disease pathophysiology can also change how the liver reacts to a drug.

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Drug Discovery World

JANUARY 20, 2023

Testing new modality drugs poses additional challenges The drug development pipeline is no longer predominantly composed of small molecule drugs. The importance of recapitulating metabolic disease phenotypes for toxicology studies Disease pathophysiology can also change how the liver reacts to a drug.

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Drug Discovery World

DECEMBER 13, 2023

Custom antibodies can be developed for pharmacokinetic assays and biodistribution assays, supporting therapeutic development by giving researchers data about the behaviour of their novel therapeutic in vivo. Caplacizumab, for the treatment of a rare blood clotting disease, achieved FDA approval in 2019 2.

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Drug Target Review

OCTOBER 4, 2023

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. Groundbreaking strategies like proteolysis-targeting chimeric molecules (PROTACs) are also being explored.

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The Pharma Data

NOVEMBER 11, 2020

As an orally bioavailable small molecule with broad antiviral activity, SLV213 could be a valuable treatment to meet today’s urgent need to fight COVID-19 as well other life-threatening infectious diseases, such as Chagas disease, Ebola virus disease, and Nipah virus infection.”. About SLV213.

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Metabolite Tales Blog

DECEMBER 13, 2023

Conjugation of small molecule drugs to glucuronic acid is catalysed by several UGTs to frequently form N – and O -glucuronides. As in this example, most N -glucuronides of small molecule drugs are innocuous and possess no pharmacological activity. J Pharm Biomed Anal. Sundararaghavan VL, Sindhwani P, Hinds TD Jr.

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Agency IQ

JUNE 9, 2023

However, this guideline is primarily intended to provide recommendations on evaluating the immune system’s response to small molecule drugs. The guidance contains recommendations for small molecule drugs, as well as any biological products that fall within CDER’s purview.

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Alta Sciences

APRIL 17, 2024

Gene Therapy Gene therapy is currently considered for diseases, often rare, that have no other cures. We have conducted over 130 in-life studies in the last five years (over 95% conducted in NHPs), including IND-enabling studies for rare diseases and CRISPR gene editing therapeutics in NHPs.

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