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. Their results are published in the Proceedings of the National Academy of Sciences.

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Scienmag

SEPTEMBER 29, 2020

By changing gene expression first, some immunotherapies may work better, models show Credit: (courtesy of Hackensack Meridian Health) September 29, 2020 – Nutley, NJ – A one-two punch of changing gene expression, then deploying immune checkpoint inhibitors, shows promise in battling one of the most treatment-resistant types of cancer in (..)

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Gene Expression Scientist Gene Clinical Trials 52

XTalks

NOVEMBER 6, 2020

Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinical trials this week. The first healthy volunteer was dosed with the vaccine in Australia as part of the bacTRL-Spike COVID-19 Phase I clinical trial. COVID-19 Clinical Trials.

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Clinical Trials Clinical Trials Vaccination Vaccine 75

pharmaphorum

FEBRUARY 9, 2021

million ($22 million) in second-round financing as it prepares to start clinical trials of its lead drug candidate INT-1B3 for solid tumours. The start of dosing in the phase 1/1b trial of the drug has been delayed by the COVID-19 pandemic, but recruitment of the target 80 participants with advanced solid tumours is reported to be ongoing.

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pharmaphorum

OCTOBER 12, 2021

The research has focused on Alzheimer’s associated with an apolipoprotein E4 (APOE4) gene variant known to be a major risk factor for late-onset forms of the disease. All told, five drugs emerged with a gene expression signature that the researchers believed might help neutralise the disease, but the strongest candidate was bumetanide.

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Gene Expression Drugs FDA Approval Gene 111

Drug Discovery World

AUGUST 22, 2023

The company has already completed three trials in patients, including a positive open label trial in metastatic breast cancer using a prototype capsule formulation. The new tablet formulation of SFX-01 will now replace the prototype capsule for future regulatory submissions and clinical trials.

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Gene Expression In-Vitro Gene Trials 52

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 53

Scienmag

AUGUST 4, 2020

Histone deacetylases (HDACs) are enzymes that help modulate gene expression by removing […].

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Gene Expression Gene Scientist Research 82

Drug Discovery World

MARCH 21, 2023

In July 2022, DDW reported that molecular genetics company Mainz Biomed had enrolled its first patient in an international clinical study assessing the potential combination of gene expression (mRNA) biomarkers with a diagnostic test for colorectal cancer.

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Drugs Gene Expression RNA Clinical Trials 52

Drug Discovery World

MAY 16, 2023

FDA Orphan Drug Designation provides SiSaf with incentives such as tax credits for clinical trials, exemption from user fees, and expanded marketplace exclusivity. There are currently no approved treatments for Osteopetrosis ADO2 and no other treatments currently in clinical trials.

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Gene Expression RNA Clinical Trials Clinical Trials 52

Drug Discovery World

OCTOBER 10, 2023

Read more: Preclinical study proves antidepressant effects of drug Psychedelic-based drug options Later the same month, Cybin announced it had completed dosing in Cohort 5 of a Phase II trial evaluating CYB003, an investigational proprietary deuterated analog of psilocybin for the potential treatment of major depressive disorder (MDD).

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Drugs Gene Expression Gene Immune Response 59

Camargo

JULY 27, 2021

Additionally, gene editing allows us either to remove or to modify harmful genes. While most gene therapy clinical studies are ongoing, a number of products are in advanced clinical development, and several are approved by FDA. Gene Expression Considerations.

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Gene Therapy Gene Gene Editing Genetics 238

XTalks

JUNE 17, 2022

Olumiant’s FDA approval for AA was based on trial data that showed patients who had at least 50 percent scalp hair loss for more than six months experienced at least 80 percent scalp hair coverage by week 36 of treatment. The Olumiant approval is therefore a big win as it helps fulfill a significant unmet need in the area.

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FDA Approval Gene Expression Cosmetics Trials 97

Drug Discovery World

MARCH 3, 2023

Epigenetics discovery could lead to new class of cancer drugs 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.

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Drugs Gene Expression Genome Genomics 52

XTalks

MAY 31, 2022

XTALKS WEBINAR: All Means All: The Road to Inclusivity in Clinical Trials. Register for this free webinar to learn what the FDA’s new draft guidance means for diverse and inclusive trials. The featured speakers will discuss its shortcomings and what the industry still needs to address to make clinical trials more inclusive for all.

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FDA Approval Clinical Trials Clinical Trials Trials 98

pharmaphorum

JUNE 27, 2022

The safety of Astellas’ gene therapy portfolio has been thrust into the spotlight once again, after the FDA placed a clinical hold on a trial of its Pompe disease candidate AT845. The delivery vector – an AAV8 capsid – is designed to allow gene expression directly in target tissues like skeletal muscle.

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Gene Therapy Gene Gene Expression Trials 52

The Pharma Data

JULY 12, 2021

The AI approach helps model a disease using a map of successive changes in gene expression at the onset and during the progression of the disease. The underlying algorithms ensure that the identified gene expression patterns are ‘invariant’ regardless of different disease cohorts.

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Drugs Gene Expression Big Data Gene 52

Delveinsight

JULY 24, 2021

Ovid Therapeutics is running Phase III clinical trials for its drug, Gaboxadol ( OV101 ). Another therapy by the same company named GTX-102 is also an antisense therapy; however, it silences gene expression by binding to the RNA molecule that regulates its expression.

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Gene Therapy Gene Expression Gene RNA 96

Drug Discovery World

AUGUST 16, 2023

.” The findings suggest it might be possible to predict who will benefit from carboplatin and docetaxel using a range of different biomarkers and suggest further investigation of these biomarkers in clinical trials is warranted.

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Genetics Gene Hormones RNA 52

pharmaphorum

OCTOBER 27, 2022

Discussion was centred around ESMO 2022, where Dr Ray-Coquard presented collated data from a landmark five-year follow-up of the AstraZeneca/MSD PAOLA-1 phase 3 trial, the results from which suggested a clinically meaningful improvement in overall survival rates of certain patients with ovarian cancer. Furthermore, 65.5%

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Drug Discovery World

APRIL 16, 2023

The clinical benefit was observed regardless of the tumour mutational burden (TMB) status, according to results from the Phase IIb KEYNOTE-942 clinical trial presented at the AACR Annual Meeting 2023, held April 14-19. According to the results of the primary trial analysis, after 18 months, the RFS was 78.6%

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Vaccination Vaccine Immune Response Gene Expression 52

Drug Discovery World

JULY 10, 2023

The algorithms work by inputting the gene expression profiles of cancers cells to predict how each individual cancer will respond to chemotherapeutic drugs. For instance, this year saw Janssen discontinue a Phase III HIV clinical trial after the vaccine was shown to be non- effective in preventing HIV transmission.

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Gene Therapy Gene Scientist Gene Editing 52

pharmaphorum

NOVEMBER 22, 2022

Ut comes just as FogPharma is preparing to start clinical trials of alpha-helical polypeptide candidate FOG-001. The Series D comes a little over 18 months after the six-year-old biotech’s third-round financing netted it around $107 million, taking its total raised to date north of $360 million.

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Gene Expression Packaging Packaging Clinical Trials 52

pharmaphorum

FEBRUARY 22, 2021

Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. .

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Gene Therapy Gene Gene Sequencing Gene Expression 52

XTalks

MARCH 13, 2024

XTALKS WEBINAR: Addressing Ophthalmology Clinical Trial Design and Effective Data Monitoring Committee Strategies On-Demand: Thursday, February 8, 2024 Register for this free webinar gain insights into statistical data strategies for ophthalmology clinical trials and learn best practices for establishing and reporting ophthalmic data.

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Gene Therapy Gene Life Science DNA 111

Drug Discovery World

AUGUST 1, 2023

Towards the end of last year, Kite, a Gilead company, entered into an exclusive license agreement with synthetic biology company Refuge to access its gene expression platform to develop potential treatments for blood cancers.

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Drugs Gene Expression Engineer Gene 52

Camargo

JULY 8, 2021

The UCLA researchers measured monoamine oxidase A (MAOA) gene expression in tumors from cancer patients and found that high MAOA expression was associated with shorter survival times and turned to a mouse model to investigate these observations with promising results.

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Development Bioequivalency Gene Expression Drugs 130

The Pharma Data

JULY 11, 2021

Eli Lilly and Company (NYSE: LLY) announced new Phase 2 data showing that gene expression changes induced by mirikizumab in patients with ulcerative colitis (UC) over a 12-week induction treatment were maintained for up to one year. For methodology, see the “About the Studies” section below.

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Regulation Gene Gene Expression Packaging 52

Roots Analysis

NOVEMBER 3, 2023

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. NAMPT inhibitors work by blocking the activity of NAMPT, which in turn reduces the production of NAD +.

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Development Drugs Trials FDA Approval 40

The Pharma Data

MARCH 3, 2022

In 2014, Shalev’s UAB research lab reported that verapamil completely reversed diabetes in animal models, and she announced plans to test the effects of the drug in a human clinical trial. The United States Food and Drug Administration approved verapamil for the treatment of high blood pressure in 1981.

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Insulin Gene Expression Gene Protein 52

Drug Discovery World

OCTOBER 3, 2022

The researchers performed flow-cytometry EMT characterisation of CTCs as compared to EMT gene expression in tumour tissue and investigated expression correlation with respect to clinical outcomes. The expression of EpCAM and N-cadherin were utilised to characterise CTC status as epithelial or EMT.

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Gene Expression Genome Genomics Clinical Trials 52

Drug Discovery World

DECEMBER 12, 2022

In Nature 3 , we learnt about the CRISPR cancer trial success leading the way to personalised treatments. According to the publication, “A small clinical trial has shown that researchers can use CRISPR gene editing to alter immune cells so that they will recognise mutated proteins specific to a person’s tumours.

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Drugs Gene Editing Genome Genomics 52

Drug Discovery World

FEBRUARY 13, 2023

DDW: Can RNA profiling be used past the point of early development to monitor how drugs perform within clinical trials? Diseases ranging from cancer to viral immune response, from cardiovascular to reproductive health, and from metabolic to nervous systems all have the potential to benefit from in-depth transcriptomics. SB: Certainly.

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RNA Drugs Gene Expression Gene 52

Drug Discovery World

MARCH 26, 2024

Mutations in genes encoding urea cycle enzymes result in insufficient levels of these important proteins. CMP-CPS-001 is an antisense oligonucleotide (ASO) designed to amplify CPS1 mRNA by harnessing fundamental cellular gene expression control mechanisms.

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Trials Gene Expression Protein Gene 52

The Pharma Data

OCTOBER 15, 2020

Manual manipulation affects gene expression,” he pointed out, so minimizing that, as well as the vast quantities of plastics typically required, results in a more controlled process and a more consistent product. So far, 25 people have been treated in a Phase I/II trial.

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Manufacturing Gene Therapy Gene Gene Expression 52

pharmaphorum

JANUARY 29, 2021

ZFPs can also be engineered to make ZFP transcription-factors, or ZFP-TFs, which are proteins that can be used to regulate genomes by selectively increasing or decreasing gene expression. Zinc fingers are the most common control gene in the body,” Macrae explains. “We The exact mechanism depends on the disease in question.

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pharmaphorum

JUNE 3, 2021

Further evidence of its potential efficacy as a drug candidate was also established based on diminished hypothalamic inflammatory gene expression. Demonstrating a robust safety profile can be considered a major milestone because previous melanocortin-based drug candidates had significant cardiovascular side-effects in clinical trials.

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