Pharmaceutical Technology

JULY 29, 2022

In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs). The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. David Bumcrot PhD, CSO, CAMP4 Therapeutics.

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Pharmaceutical Technology

JANUARY 19, 2023

The US Food and Drug Administration (FDA) has granted Fast Track designation for Avidity Biosciences’ AOC 1020 to treat facioscapulohumeral muscular dystrophy (FSHD). AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4.

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Drug Discovery World

AUGUST 21, 2023

SBIR fast-track grant from the NIH to apply its proprietary Multiplexed Assays for the Rational Modulation Of Transcription Factors (MARMOT) platform to their deep learning AI model and accelerate the development of transcription factor drugs. Talus Bio was also awarded a $2.0M The post $4.3

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BioSpace

JULY 11, 2022

Epic Bio aims to transform genetic medicine by developing a new class of drugs that target the epigenome to alter gene expression.

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Drug Discovery World

MARCH 2, 2023

A new paper has solved the 20-year mystery of how epigenetic modifications act as traffic lights to control gene expression and could ultimately speed up the development of a new class of epigenetic cancer drugs. Epigenetics is still largely unexplored and referred to as the ‘dark matter’ of the genome.

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Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What are the challenges of drug discovery in this area? How do they work?

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pharmaphorum

OCTOBER 12, 2021

A data-mining study conducted by researchers in the US has found that an already-approved diuretic drug could have potential as a treatment for some patients with Alzheimer’s disease. The post Data dive finds cheap diuretic could be Alzheimer’s drug appeared first on.

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pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. The exact mechanism depends on the disease in question.

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Drug Discovery World

OCTOBER 10, 2023

Over recent months, there have been a number of ground-breaking discoveries in drug discovery for mental health conditions. The article in the journal Psychopharmacology focuses on new data showing that ACD856, the lead clinical drug candidate in the NeuroRestore platform, exhibits antidepressant effects in various preclinical models.

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Drug Discovery World

FEBRUARY 16, 2024

The top stories: Dual-action treatment for fibrotic disease secures orphan drug status AM1476, a first-in-class, peripheral-acting serotonin receptor antagonist, has been granted US and EU Orphan Drug Designation as an anti-fibrotic treatment for systemic sclerosis.

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Scienmag

DECEMBER 22, 2020

Blocking gene expression in mitochondria in mice stops cancer cells from growing Credit: Hauke S. Hillen A newly developed compound starves cancer cells by attacking their “power plants” – the so-called mitochondria. The new compound prevents the genetic information within mitochondria from being read.

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XTalks

AUGUST 5, 2020

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. The barcode sequence is followed by a unique molecular identifier, which enables accurate digital counting of gene transcripts.

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Scienmag

MARCH 17, 2021

An international team of scientists from the University of Turku, Finland and PennState University, USA have solved a long-standing mystery of how living organisms distinguish RNA and DNA building blocks during gene expression paving the way for the design of new antiviral drugs. All cellular […].

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Scienmag

SEPTEMBER 28, 2020

Brazilian research group shows that valproic acid (VPA), used to treat epilepsy since the 1960s, modulates gene expression in tumor gene models and acts on DNA conformation and the histones in chromatin Results of recent studies involving valproic acid, used for decades as an anti-convulsant drug, show that it can interact with the conformation of (..)

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Medical Xpress

NOVEMBER 22, 2022

A form of blood cancer known as mantle cell lymphoma is critically dependent on a protein that coordinates gene expression, such that blocking its activity with an experimental drug dramatically slows the growth of this lymphoma in preclinical tests, according to a study from Weill Cornell Medicine researchers.

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Medical Xpress

MARCH 8, 2023

A pilot trial by investigators from Brigham and Women's Hospital, a founding member of the Mass General Brigham health care system, tested the nasal administration of the drug Foralumab, an anti-CD3 monoclonal antibody.

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Drug Discovery World

APRIL 17, 2024

Pooled CRISPR screens offer the ability to knock out or perturb many genes in a single experiment from several hundred to tens of thousands of targets in parallel. Genome-wide screens are becoming the standard for drug discovery to power unbiased identification of drug targets across gene regulatory networks.

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Drug Discovery World

APRIL 3, 2024

Pooled CRISPR screens offer the ability to knock out or perturb many genes in a single experiment from several hundred to tens of thousands of targets in parallel. Genome-wide screens are becoming the standard for drug discovery to power unbiased identification of drug targets across gene regulatory networks.

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pharmaphorum

NOVEMBER 12, 2021

Researchers in the US have developed an artificial intelligence-based tool that is able to predict COVID-19 symptoms and suggest which FDA-approved drugs might be used to treat patients. It could also be made more powerful by incorporating personal genetic information and gene expression profiles in tissues such as the lungs, they suggest.

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Drug Discovery World

AUGUST 1, 2023

Synthetic biology is increasingly important to drug discovery and development. Evonetix will use the funding to brings its semiconductor chips to commercial scale and extend gene assembly capabilities for its binary assembly technology to deliver accurate, gene-length DNA in a benchtop instrument.

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pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. He added: “We have seen no significant gene misregulation attributable to the insertion event.

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Drug Discovery World

MARCH 21, 2023

Diana Spencer explores the different ways the drug discovery sector is tackling colorectal cancer. So, what are the recent developments and how is the drug discovery sector tackling this common cancer? Improved diagnostics Diagnostics and screening are essential to the early treatment of colorectal cancer.

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Drug Discovery World

FEBRUARY 27, 2024

BS: Genetic tuning, also called epigenetic editing, actively manipulates the epigenetic machinery to influence gene transcription. Orchestrating the naturally occurring conductors of gene expression can shift the functional state of cells and alter gene expression.

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Drug Discovery World

NOVEMBER 29, 2022

The technology can contribute to the development and production of vaccines and drugs for severe diseases much faster and at lower cost. . The AI designs synthetic DNA, where it is easy to modify its regulatory information in the desired direction of gene expression. . The next step is to use human cells.

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Drug Discovery World

MARCH 3, 2023

As well as providing extensive coverage of the SLAS2023 meeting in San Diego and Rare Disease Day , DDW has also reported on a number of developments in cancer drug discovery this week. The post This week in drug discovery (27 February – 3 March) appeared first on Drug Discovery World (DDW).

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Drug Discovery World

OCTOBER 4, 2023

Scientists have discovered the link between gene expression, DNA methylation, and brain structural changes in depression, revealing new potential therapeutic targets. Parallel findings in humans underscored the involvement of interferon-related genes in major depressive disorder.

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Drug Discovery World

FEBRUARY 13, 2023

Dr Stephen Barat, Senior Vice President and Therapeutics Division Head at HTG Molecular Diagnostics tells DDW how transcriptomics is driving drug discovery and precision medicines. RNA transcript expression levels can provide insight into underlying biology – eg. DDW: Could you explain the process of transcriptomics? SB: Certainly.

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Drug Discovery World

JULY 27, 2023

Currently prescribed drugs, such as anticonvulsants and antidepressants, can have severe adverse effects, are not well-tolerated, take a longer time to work, and only help a subset of patients. appeared first on Drug Discovery World (DDW). Opioids come with the risk of physical dependence and addiction.

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Drug Discovery World

DECEMBER 12, 2022

DDW highlights the most interesting global news and comments within the drug discovery and development sector this month. . Everyone enjoys a good news story and the drug discovery and development sector is no exception. The post The Dose: DDW’s drug discovery highlights appeared first on Drug Discovery World (DDW).

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Drug Discovery World

SEPTEMBER 27, 2023

Genetic variations inherited from our parents shape our response to drugs, disease susceptibility, physiology and metabolism. Epigenetics: the software of gene regulation Gene activity is the driving force behind our biological processes, and disruptions in normal gene activity can trigger diseases.

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The Pharma Data

JULY 12, 2021

online issue of Nature Communications, researchers at University of California San Diego School of Medicine describe a new approach that uses machine learning to hunt for disease targets and then predicts whether a drug is likely to receive FDA approval. the success rates in drug discovery?are

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Drug Discovery World

AUGUST 22, 2023

In addition to the biomarkers analysed in the study, changes in gene expression were measured by mRNA sequencing on participants’ blood, for placebo and SFX-01 treated subjects that received 600mg once daily. It delivers the drug at highly relevant blood levels within the timeframes expected of an enteric coated formulation.”

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Delveinsight

JULY 24, 2021

Ovid Therapeutics is running Phase III clinical trials for its drug, Gaboxadol ( OV101 ). The drug is a small molecule, which selectively activates GABAA receptors located on the surface of nerve cells and away from the synapse. OV101 is considered as the first compound that targets disrupted tonic inhibition.

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Drug Discovery World

AUGUST 16, 2023

A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy. “Our findings paint a complex picture – with many, dynamic factors driving how tumours respond to treatment.”

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The Pharma Data

FEBRUARY 24, 2022

UNC researchers now show that these two factors can directly associate with one another, modulating cancer-cell-specific programs of gene expression. ” They found that EZH2 possesses two different binding patterns on chromatin in acute leukemia cells, eliciting two distinct gene-regulatory programs (Figure 1). .”

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Drug Discovery World

FEBRUARY 14, 2024

A class of molecules playing a crucial role in the regulation of gene expression and other cellular processes can restore normal skin structure rather than producing a scar, a University of Manchester-led study suggests. The post Small RNAs promote wound-healing without scarring appeared first on Drug Discovery World (DDW).

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