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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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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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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DNA Gene Expression Protein Gene 70

Pharmaceutical Technology

JANUARY 24, 2023

The US Food and Drug Administration (FDA) has granted clearance for Neurogene’s investigational new drug (IND) application for NGN-401 to treat Rett syndrome. We believe the preclinical profile for NGN-401 is highly compelling, with the strongest results generated to date across multiple animal models.

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XTalks

FEBRUARY 21, 2025

This capital will accelerate the companys clinical programs and support its pipeline of investigational drugs aimed at treatments for metabolic diseases like Prader-Willi syndrome (PWS). Aardvark Therapeutics, Inc. has priced its initial public offering (IPO) at $16.00 per share, offering 5,888,000 shares and targeting approximately $94.2

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XTalks

DECEMBER 30, 2024

has announced promising Phase IIb results for cenerimod, an investigational drug targeting immune system dysfunction in moderate-to-severe systemic lupus erythematosus (SLE). These results further support the ongoing Phase III Oral S1P1 Receptor ModUlation (OPUS) program, which aims to evaluate the drugs broader potential in treating SLE.

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XTalks

MARCH 26, 2025

Nearly 40% live with drug-resistant forms of the condition, meaning current treatments do not fully control their seizures. Unlike older drugs that broadly suppress brain activity, Xcopri targets persistent sodium currents and enhances gamma-aminobutyric acid (GABA) activity the brains main calming neurotransmitter to help restore balance.

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Life Science Gene Expression Trials Drugs 66

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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Pharmaceutical Technology

JUNE 13, 2023

The aberrant protein complex is essential for regulating leukaemia-promoting gene expression. The drug is currently being tested for expanded access in acute lymphocytic leukaemia, in addition to a planned study investigating the drug in combination with venetoclax in AML. years (22–86 years).

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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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Gene Expression Protein Gene Medicine 53

pharmaphorum

AUGUST 2, 2021

The agreement gives the company rights to spherical nucleic acid (SNA) drugs – nanoparticles with nucleic acids bound to their surfaces – which can be used to get oligonucleotides into cells to modify gene expression efficiently, and without the toxicity caused by some other delivery mechanisms.

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Gene Expression Gene Licensing Licensing 105

Pharmaceutical Technology

AUGUST 11, 2022

Alnylam has reported that it aims to develop a therapy that targets the INHBE gene and reduces the overall risk of T2D and coronary heart disease for at-risk patients. Targeting gene expression that results in increased abdominal fat and leads to obesity is a key factor for the reduction of T2D and wider metabolic syndrome.

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Camargo

JULY 8, 2021

At Camargo, we have worked with hundreds of investigators who have found new targets for established drugs. It is exciting to find new uses of drugs with known safety because these drugs can reach new groups of patients in a relatively short timeframe.

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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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Gene Therapy Gene Gene Expression RNA 96

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

Drugs 52

Drugs Gene Expression Big Data Gene 52

XTalks

JUNE 17, 2022

Eli Lilly’s Olumiant (baricitinib) has won US Food and Drug Administration (FDA) approval as the first systemic treatment for severe alopecia areata (AA), an autoimmune disorder that leads to patchy baldness. The drug was evaluated in 1,200 adult patients with severe AA.

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FDA Approval Gene Expression Cosmetics Trials 97

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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pharmaphorum

JANUARY 13, 2022

AstraZeneca has expanded its efforts to develop new therapies targeting transcription factors – proteins that regulate processes in the cell that have become a hot topic for drug discovery in cancer and other diseases. It’s not the big pharma’s first foray into the transcription factor area. Image by Patrizia08 from Pixabay .

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Protein Gene Expression Gene Regulation 59

pharmaphorum

OCTOBER 19, 2022

The dark genome – a loose term that covers non-coding regions of the genome that are capable of regulating the expression of genes, previously rather inaccurately referred to as junk DNA – is increasingly being explored by biopharma companies for new drug targets.

Genome 59

Genome Genomics Gene Gene Expression 59

XTalks

MAY 31, 2022

The spotlight for plaque psoriasis treatment is now on Vtama (tapinarof) cream (one percent), Dermavant’s discovery that has become the first steroid-free topical medication in its class approved by the US Food and Drug Administration (FDA). The Chemical Composition of Vtama. Clinical Trials of Vtama.

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XTalks

MAY 12, 2021

In this episode, Ayesha discusses a new tool that uses CRISPR to modulate gene expression without editing DNA sequences. Dubbed “CRISPRoff,” the technology targets the epigenome to silence genes involved in diseases, with applications in cancer, AML and other conditions with a heritable component.

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Delveinsight

JANUARY 18, 2021

For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation. Thus, restoring the presence of this particular gene can help to restore several of the functions of the brain.

Marketing 52

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pharmaphorum

NOVEMBER 22, 2022

Cambridge, Massachusetts-based FogPharma has closed another impressive fundraising round, adding $178 million to the pot as it ushers its polypeptide-based drug candidates towards the clinic. Ut comes just as FogPharma is preparing to start clinical trials of alpha-helical polypeptide candidate FOG-001.

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Gene Expression Packaging Packaging Medicine 52

XTalks

MARCH 13, 2024

Therapeutic Developments Related to Glaucoma The introduction of new classes of glaucoma medications, such as prostaglandin analogs, nitric oxide-donating drugs and Rho kinase inhibitors, has added to the pharmacological options available for managing IOP.

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Gene Therapy Gene Life Science DNA 111

pharmaphorum

MARCH 10, 2021

Known as Zynteglo in Europe where it is approved to treat the rare blood disorder beta-thalassemia, the gene therapy has been given the all-clear by bluebird’s scientists after reviewing the case of AML that emerged in a trial of the drug in sickle cell disease. Shares in bluebird ticked up following the announcement.

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Gene Therapy Gene Trials Gene Expression 59

The Pharma Data

MARCH 3, 2022

Use of the drug verapamil to treat Type 1 diabetes continues to show benefits lasting at least two years, researchers report in the journal Nature Communications. The suggestion that verapamil might serve as a potential Type 1 diabetes drug was the serendipitous discovery of study leader Anath Shalev, M.D.,

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Insulin Gene Expression Gene Protein 52

Roots Analysis

AUGUST 12, 2024

Hybridization Tests – These tests utilize complementary DNA or RNA probes to detect and quantify specific genetic targets, enabling the identification of pathogens, genetic variations, and gene expression patterns. Such test can be used for gene expression profiling, genotyping and detecting chromosomal abnormalities.

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Genetics Gene Expression RNA DNA 40

The Pharma Data

NOVEMBER 8, 2020

The program, developed in collaboration with Regeneron Pharmaceuticals, uses a proprietary, cell-selective promoter to precisely control gene expression in cochlear hair cells.

Gene Therapy 40

Gene Therapy Gene Gene Expression Genome 40

The Pharma Data

JANUARY 17, 2021

a biopharmaceutical company engaged in the discovery and development of RNAi therapeutics against cancer and fibrotic diseases, today announced dose administration for the first patient in a Phase 2a clinical study of the company’s lead drug candidate, STP705, for the treatment of cutaneous basal cell carcinoma. GAITHERSBURG, Md.

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Gene Expression Trials Development Production 52

Roots Analysis

NOVEMBER 3, 2023

NAMPT inhibitors are a class of drugs that target the enzyme nicotinamide phosphoribosyltransferase (NAMPT). NAD + is involved in a wide range of cellular processes, including energy metabolism, DNA repair, and gene expression. Several trials focused on evaluation of such drugs were discontinued in the past.

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The Pharma Data

JANUARY 19, 2021

Fulcrum’s proprietary product engine identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression.

Gene Expression 40

Gene Expression Containment Genetics Clinical Development 40

Camargo

JULY 27, 2021

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.

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Gene Therapy Gene Gene Editing Genetics 238

The Pharma Data

OCTOBER 19, 2020

In addition, the live CTCs harvested can be cultured, which offers the potential for testing response to drugs outside the patient. This is important because cancer develops and changes over time and there is a clear medical need for up-to-date information on the status.

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The Pharma Data

NOVEMBER 9, 2020

Food and Drug Administration Breakthrough Therapy Designation granted to tezepelumab in Sept. Food and Drug Administration, and no conclusions can or should be drawn regarding the safety or effectiveness of the product candidates. Food and Drug Administration for the products. Such product candidates are not approved by the U.S.

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Scienmag

DECEMBER 1, 2021

BOSTON – An important player in the healthy development of female embryos turns out also to play a key role in regulating the behavior of chromosome loops and gene expression in both sexes, according to a new study by researchers at Massachusetts General Hospital (MGH).

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Gene Expression Gene RNA Research 61

pharmaphorum

JANUARY 10, 2023

Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene therapy for inherited neuromuscular disorder Pompe disease. The post Astellas licenses companion drug for Pompe gene therapy appeared first on.

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Gene Therapy Gene Licensing Licensing 119

pharmaphorum

JANUARY 29, 2021

Since that discovery, a flurry of gene-editing focused biopharma companies have launched – including Intellia Therapeutics, CRISPR Therapeutics, Caribou Biosciences and Mammoth Biosciences – and the first drug therapies based on the technology are now in human testing for diseases like cancer. billion in funding.

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