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Drug Discovery Today

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https://www.readbyqxmd.com/read/30610920/acceleration-of-rare-disease-therapeutic-development-a-case-study-of-agil-aadc
#1
Sonya Das, Samuel Huang, Andrew W Lo
Rare-disease drug development is both scientifically and commercially challenging. This case study highlights Agilis Biotherapeutics (Agilis), a small private biotechnology company that has developed the most clinically advanced adeno-associated virus (AAV) gene therapy for the brain. In an international collaboration led by Agilis with National Taiwan University (NTU) Hospital and the Therapeutics for Rare and Neglected Diseases (TRND) program of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health, Agilis' gene therapy for aromatic L-amino acid decarboxylase deficiency (AADC), AGIL-AADC, was granted biologics license application (BLA)-ready status by the FDA in 2018 only 18 months after being licensed from NTU by Agilis...
January 2, 2019: Drug Discovery Today
https://www.readbyqxmd.com/read/30583089/the-use-of-fast-photochemical-oxidation-of-proteins-coupled-with-mass-spectrometry-in-protein-therapeutics-discovery-and-development
#2
REVIEW
Jing Li, Guodong Chen
Structural analysis of protein therapeutics is a challenging task owing to complexities associated with large molecular size and 3D structures. Recent advances in fast photochemical oxidation of proteins (FPOP) coupled with mass spectrometry (MS) have provided a means to characterize higher order structure (HOS) of protein therapeutics in solution. In this review, the utility of FPOP-MS in protein therapeutics discovery and development is presented, focusing on the principles and applications of epitope mapping for target-protein-protein-therapeutics interactions and biopharmaceutical HOS comparison studies...
December 21, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30579785/hurdles-in-gene-therapy-regulatory-approval-a-retrospective-analysis-of-european-marketing-authorization-applications
#3
REVIEW
Marta Alexandra Bogalho Rodrigues de Carvalho, Ana Paula Mecheiro Almeida Martins Silvestre Correia, Bruno Miguel Nogueira Sepodes
Gene therapy medicinal products (GTMPs) are innovative and promising treatment strategies. In Europe, the Committee for Advanced Therapies (CAT) is responsible for making marketing authorization recommendations to the Committee of Human Medicinal Products (CHMP). In the current study, we identified major objections, issues, or concerns raised during the Marketing Authorization Application (MAA) process for products resulting from the interaction of both committees between 2008 and 2017. During the first few years following CAT establishment, quality issues were often identified as major deficiencies, whereas issues at the nonclinical level appeared to be less frequent...
December 20, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30572117/post-translational-modification-of-eskape-pathogens-as-a-potential-target-in-drug-discovery
#4
REVIEW
Vishvanath Tiwari
ESKAPE pathogens are gaining clinical importance owing to their high pervasiveness and increasing resistance to various antimicrobials. These bacteria have several post-translational modifications (PTMs) that destabilize or divert host cell pathways. Prevalent PTMs of ESKAPE pathogens include addition of chemical groups (acetylation, phosphorylation, methylation and hydroxylation) or complex molecules (AMPylation, ADP-ribosylation, glycosylation and isoprenylation), covalently linked small proteins [ubiquitylation, ubiquitin-like proteins (UBL) conjugation and small ubiquitin-like modifier (SUMO)] or modification of amino acid side-chains (eliminylation and deamidation)...
December 17, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30562586/an-approach-towards-enhancement-of-a-screening-library-the-next-generation-library-initiative-ngli-at-bayer-against-all-odds
#5
Markus Follmann, Hans Briem, Andreas Steinmeyer, Alexander Hillisch, Monika H Schmitt, Helmut Haning, Heinrich Meier
Pharmaceutical companies often refer to 'screening their library' when performing high-throughput screening (HTS) on a corporate compound collection to identify lead structures for small-molecule drug discovery programs. Characteristics of such a library, including the size, chemical space covered, and physicochemical properties, often determine the success of a screening campaign. Therefore, strategies to maintain and enhance the overall quality of screening collections are crucial to stay competitive and to cope with the 'novelty erosion' that is observed gradually...
December 15, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30562585/modified-cells-as-potential-ocular-drug-delivery-systems
#6
REVIEW
Tatiana Tennikova, Arto Urtti
Drug delivery to ocular targets is problematic, especially in retinal disease treatment. Therefore, targeted drug delivery, prolonged drug action, and minimally invasive treatments are needed. In this review, we describe cell technologies for drug delivery. These technologies are based on genetic engineering and nongenetic-based approaches for cell modification. In principle, cell technologies enable targeted delivery, long drug action, and minimally invasive administration, but they have only been sparsely studied for ocular drug delivery...
December 15, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30557651/quality-by-design-approach-as-a-systematic-tool-for-the-development-of-nanopharmaceutical-products
#7
REVIEW
Sarwar Beg, Mahfoozur Rahman, Kanchan Kohli
In recent decades, extensive emphasis has been given on developing nanopharmaceutical products for improving the therapeutic performance of drugs, resulting in an increasing number of product approvals by drug regulatory agencies. Although nanopharmaceuticals are subjected to the same regulatory pathways as conventional pharmaceutical products, their biopharmaceutical characteristics at the nanoscale make them vulnerable to high variability in quality. Positive effects on drug quality assurance have resulted from adopting systematic quality-by-design (QbD) principles, boosting pharmaceutical manufacturing with improvements in the quality, safety, and efficacy of drugs...
December 14, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30529762/tgf%C3%AE-and-bmprii-signalling-pathways-in-the-pathogenesis-of-pulmonary-arterial-hypertension
#8
REVIEW
Birger Tielemans, Marion Delcroix, Catharina Belge, Rozenn Quarck
Pulmonary arterial hypertension (PAH) is a severe condition characterised by remodelling of precapillary pulmonary arteries sometimes associated with mutations in the bone morphogenetic protein receptor type 2 (BMPR2) gene. Even in the absence of BMPR2 mutations, increased transforming growth factor (TGF)β receptor signalling and decreased BMPRII signalling have been shown to contribute to PAH pathogenesis. In this Keynote, we review the potential mechanisms by which the imbalance of BMP/TGFβ signalling contributes to endothelial dysfunction, vascular remodelling, inflammation and disordered angiogenesis in PAH...
December 7, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30529076/corrigendum-to-drug-metabolism-and-pharmacokinetic-strategies-for-oligonucleotide-and-mrna-based-drug-development-drug-discovery-today-23-2018-1733-1745
#9
Shalini Andersson, Madeleine Antonsson, Marie Elebring, Rasmus Jansson-Löfmark, Lars Weidolf
No abstract text is available yet for this article.
December 6, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30529326/efficient-drug-discovery-by-rational-lead-hybridization-based-on-crystallographic-overlay
#10
REVIEW
Shuo Zhang, Jian Zhang, Ping Gao, Lin Sun, Yuning Song, Dongwei Kang, Xinyong Liu, Peng Zhan
In this review, we provide an overview of recent applications of crystallographic overlay-based molecular structure hybridization of lead compounds as a rational strategy for efficient drug discovery, with selected examples, and briefly discuss its advantages compared with other ligand-based methodologies.
December 4, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30521936/behavior-experience-skill-and-training-a-four-pillar-framework-and-tool-for-career-development-in-science
#11
EDITORIAL
Rebecca Ashkenazy
No abstract text is available yet for this article.
December 3, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30521935/pharmaceutical-cocrystals-from-serendipity-to-design-to-application
#12
REVIEW
Oisin N Kavanagh, Denise M Croker, Gavin M Walker, Michael J Zaworotko
The field of pharmaceutical cocrystals has reached a tipping point, particularly because cocrystals can improve the physicochemical properties of drugs without compromising their therapeutic benefit. Accounts of cocrystal investigations in the literature started in earnest in 2003 and patent applications soon followed. The frequency of both has steadily accelerated, demonstrating an enhanced understanding of the design, characterisation, and manufacture of cocrystals and heightened interest from industry. Indeed, there were four new product approvals from 2014 to 2017 and more are in the pipeline...
December 3, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30513339/the-drug-repurposing-landscape-from-2012-to-2017-evolution-challenges-and-possible-solutions
#13
REVIEW
Prasanthi Polamreddy, Nandu Gattu
As the name suggests, drug repurposing is a strategy to identify new therapeutic uses for marketed drugs, discontinued and/or shelved drugs, and drug candidates currently in clinical development. Although not a recent concept, drug repurposing has gained momentum over the past few years and several drugs have been successfully repurposed. Here, we summarize the drug repurposing landscape from 2012 to 2017, with a major focus on repurposed drugs, collaborative opportunities, and funding opportunities specific to drug repurposing projects...
December 1, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30508640/essential-oils-from-prevention-to-treatment-of-skin-cancer
#14
REVIEW
P S Pavithra, Alka Mehta, Rama S Verma
The increasing incidence of cutaneous malignancies signifies the need for multiple treatment options. Several available reviews have emphasized the potential role of various botanical extracts and naturally occurring compounds as anti-skin-cancer agents. Few studies relate to the role of chemoprevention and therapeutic activity of essential oils (EOs) and EO components. The present review summarizes an overview of chemopreventive, anti-melanoma and anti-nonmelanoma activities of EOs from various plants and EO components in in vitro and in vivo models with special emphasis on skin cancer...
November 30, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30503804/contemporary-medicinal-chemistry-strategies-for-the-discovery-of-selective-butyrylcholinesterase-inhibitors
#15
REVIEW
Lanlan Jing, Gaochan Wu, Dongwei Kang, Zhongxia Zhou, Yuning Song, Xinyong Liu, Peng Zhan
Butyrylcholinesterase (BChE) is considered a promising drug target for the treatment of moderate to severe Alzheimer's disease (AD). Here, we review medicinal-chemistry strategies that are currently available for the discovery of selective BChE inhibitors.
November 29, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30503803/recent-advances-in-pharmaceutical-dosage-forms-and-devices-using-additive-manufacturing-technologies
#16
REVIEW
Christos I Gioumouxouzis, Christina Karavasili, Dimitrios G Fatouros
The era of 'one-size-fits-all' treatment approaches is becoming history for pharmaceutical manufacturing with the future encountering a revolution in drug development through the introduction of additive manufacturing technologies. The innovative elements of this disruptive technology will affect all shareholders of the pharmaceutical chain from the industrial sector to the dispensing facilities and, ultimately, the patient end-user. In this review, we provide an overview of the most recent advances in dosage forms and devices using additive manufacturing technologies, along with the regulatory landscape framing the development and safety requirements for 3D-printed drug products before entering the pharmaceutical market...
November 29, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30502513/computational-modeling-for-formulation-design
#17
REVIEW
Chetan Hasmukh Mehta, Reema Narayan, Usha Y Nayak
Formulation design is an important phase in the development of new drug s. However, this process at an experimental level requires exhaustive experimental work. Excipient selection, prediction of solubility, encapsulation efficiency, release patterns, drug absorption, stability, and mechanism of nanoparticle formation are some of the essential steps in formulation design. The use of various computational tools, including quantitative structure-activity relationships (QSARs), molecular modeling, molecular mechanics, discrete element modeling, finite element method, computational fluid dynamics, and physiologically based pharmacokinetics (PBPK) modeling, help in the identification of drug product inadequacies and to recommend avenues for understanding complex formulation design in less time with lower investment...
November 28, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30496853/characterization-of-circulating-tumor-cells-as-a-reflection-of-the-tumor-heterogeneity-myth-or-reality
#18
REVIEW
Hannah Brown, Marta Tellez-Gabriel, Pierre-François Cartron, François Vallette, Marie-Francoise Heymann, Dominique Heymann
The current main goal of diagnostic medicine is to detect crucial events in 'infinitely' small samples. The key question now is how to determine whether the rare cell events isolated and characterized from these samples reliably reflect the disease and heterogeneity of the tumor. In this review, we provide a short overview of the most recent methods developed for the isolation and characterization of rare cell events in clinical practice, with a specific focus on circulating tumor cells. We discuss the biological value to studying these cells at the single cell level and how these rare cell events can reflect tumor heterogeneity...
November 26, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30481556/phosphinic-acids-current-status-and-potential-for-drug-discovery
#19
REVIEW
Moaz M Abdou, Paul M O'Neill, Eric Amigues, Magdalini Matziari
Phosphinic acid derivatives exhibit diverse biological activities and a high degree of structural diversity, rendering them a versatile tool in the development of new medicinal agents. Pronounced recent progress, coupled with previous research findings, highlights the impact of this moiety in medicinal chemistry. Here, we highlight the most important breakthroughs made with phosphinates with a range of pharmacological activities against many diseases, including anti-inflammatory, anti-Alzheimer, antiparasitic, antihepatitis, antiproliferative, anti-influenza, anti-HIV, antimalarial, and antimicrobial agents...
November 24, 2018: Drug Discovery Today
https://www.readbyqxmd.com/read/30476550/meeting-the-discovery-challenge-of-drug-resistant-infections-progress-and-focusing-resources
#20
REVIEW
Gordon Dougan, Chris Dowson, John Overington
Following multiple warnings from governments and health organisations, there has been renewed investment, led by the public sector, in the discovery of novel antimicrobials to meet the challenge of rising levels of drug-resistant infection, particularly in the case of resistance to antibiotics. Initiatives have also been announced to support and enable the antibiotic discovery process. In January 2018, the Medicines Discovery Catapult, UK, hosted a symposium: Next Generation Antibiotics Discovery, to consider the latest initiatives and any remaining challenges to inform and guide the international research community and better focus resources to yield a novel class of antibiotic...
November 23, 2018: Drug Discovery Today
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