Pharmaceutical Technology

JANUARY 24, 2023

Neurogene stated that the FDA IND clearance allows it to commence a Phase II/II trial of NGN-401 in female paediatric Rett syndrome patients. The multi-centre, open-label, single-arm clinical trial has been designed to evaluate the efficacy, tolerability, and safety of a single dose of NGN-401 delivered using a one-time ICV procedure.

Gene Therapy 208

Gene Therapy Gene Gene Expression Regulation 208

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.

Gene Expression 111

Gene Expression Drugs FDA Approval Gene 111

XTalks

FEBRUARY 21, 2025

In a Phase II trial involving patients with hyperphagia, ARD-101 was well tolerated and achieved an approximate eight-point reduction in hunger scores. Encouraged by these results, Aardvark has initiated a Phase III clinical trial, known as the HERO (Hunger Elimination or Reduction Objective) trial, with topline data expected in early 2026.

Genetics 59

Genetics Hormones Gene Expression Gene 59

Scienmag

AUGUST 4, 2020

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

Gene Expression 82

Gene Expression Gene Scientist Research 82

XTalks

DECEMBER 30, 2024

A subgroup analysis revealed even greater improvement in patients with high interferon-1 (IFN-1) gene expression, a known indicator of disease severity, with a -2.78 The cenerimod 4 mg group showed a significant improvement of -4.04 in mSLEDAI-2K compared to placebo. reduction compared to placebo.

Immune Response 52

Immune Response Gene Expression Drugs Trials 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 Register for this free webinar to learn what the FDA’s new draft guidance means for diverse and inclusive trials. Clinical Trials of Vtama. Live and On-Demand: Tuesday, July 12, 2022, at 11am EDT (4pm BST/UK). Dermavant’s Future Targets.

FDA Approval 98

FDA Approval Clinical Trials Clinical Trials Trials 98

pharmaphorum

JANUARY 10, 2023

. “This agreement provides an opportunity to deliver potentially transformative gene therapy treatments to a specific population of LOPD adult patients who might otherwise be ineligible for clinical trials or treatment with Astellas’ investigational product” commented Naoki Okamura, Astellas’ chief strategy officer.

Gene Therapy 119

Gene Therapy Gene Licensing Licensing 119

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 (..)

Gene Expression 52

Gene Expression Scientist Gene Clinical Trials 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.

Gene Therapy 96

Gene Therapy Gene Gene Expression RNA 96

XTalks

JUNE 17, 2022

It blocks the activity of JAK1 and JAK2 to interfere with the JAK-STAT signaling pathway to dampen the production of inflammatory cytokines through the modulation of gene expression. Olumiant is a Janus kinase (JAK) inhibitor that was first discovered by Incyte and then licensed to Eli Lilly.

FDA Approval 97

FDA Approval Gene Expression Cosmetics Trials 97

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.

Development 130

Development Bioequivalency Gene Expression Drugs 130

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.

Gene Therapy 111

Gene Therapy Gene Life Science DNA 111

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.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

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.

Drugs 52

Drugs Gene Expression Big Data Gene 52

XTalks

JUNE 18, 2024

XTALKS CLINICAL EDGE: Issue 2 — Elixirgen Therapeutics’ Interview Xtalks Clinical Edge is a magazine for clinical research professionals and all who want to be informed about the latest trends and happenings in clinical trials. This metaphor captures the essence of their therapeutic approach.

Protein 52

Protein RNA Genetics Gene Expression 52

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. InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells.

Trials 59

Trials Gene Silencing RNA Gene Expression 59

XTalks

MARCH 7, 2024

Experts discuss the therapeutic landscape, treatment gaps, regulatory considerations and clinical trial strategies. This on-demand webinar explores the perinatal period and related psychiatric conditions, focusing on perinatal mood disorders. Through this, researchers can pinpoint breast cancer cell types and their phenotypic states.

Genetics 115

Genetics Hormones Genomics Genome 115

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.

Gene Expression 52

Gene Expression Packaging Packaging Medicine 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. .

Gene Therapy 52

Gene Therapy Gene Gene Sequencing Gene Expression 52

Roots Analysis

AUGUST 31, 2023

The two types of gene regulation have been discussed below: Positive Gene Regulation: Activator is a protein molecule that helps to initiate a positive gene regulation. When activator binds to the operon, it either speeds up or permits gene expression.

Gene 40

Gene Gene Therapy Gene Expression Regulation 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.

Insulin 52

Insulin Gene Expression Gene Protein 52

The Pharma Data

DECEMBER 27, 2020

Even with the holidays among us, there were a number of clinical trial announcements. Vir Biotechnology and GlaxoSmithKline dosed the first patient in a new sub-trial of a Phase III study of monoclonal antibody VIR-7831 for hospitalized adults with COVID-19. Here’s a look. COVID-19-Related.

Trials 52

Trials Antibody Gene Therapy Gene 52

The Pharma Data

JANUARY 17, 2021

” The Company expects to report initial clinical data from the trial in 2021. Additional information about this clinical trial is available at clinicaltrials.gov using the identifier: NCT04669808. 1 and COX-2 gene expression.

Gene Expression 52

Gene Expression Trials Development Production 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. The advantage is that the cells exist in a very homogenous space and are 99% pure.”.

Manufacturing 52

Manufacturing Gene Therapy Gene Gene Expression 52

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. Although, it can be relieving that other players turned to different approaches for finding a cure for Angelman syndrome.

Marketing 52

Marketing Gene Drugs Protein 52

The Pharma Data

DECEMBER 14, 2020

Oncocyte has also launched DetermaIO , a gene expression test that identifies patients more likely to respond to checkpoint inhibitor immunotherapies, for research and pharma clinical trial use.

Licensing 52

Licensing Licensing Marketing Gene Expression 52

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.

Gene Expression 40

Gene Expression Research Drugs Medicine 40

Roots Analysis

NOVEMBER 3, 2023

NAD + is involved in a wide range of cellular processes, including energy metabolism, DNA repair, and gene expression. Several factors have contributed to the failure of NAMPT inhibitors in clinical trials, including poor pharmacokinetic properties, limited efficacy, and toxicity issues.

Development 40

Development Drugs Trials FDA Approval 40

The Pharma Data

JULY 8, 2021

8) There were no clinically meaningful differences in safety results between the tezepelumab and placebo groups in the NAVIGATOR trial.(8) About the NAVIGATOR and the PATHFINDER Clinical Trial Program. Building on the Phase 2b PATHWAY trial, the Phase 3 PATHFINDER program included two trials, NAVIGATOR8,18 and SOURCE.19,20

Allergies 52

Allergies Trials In-Vivo Medicine 52

The Pharma Data

NOVEMBER 9, 2020

1,2 Expression of TSLP is increased in the airways of patients with asthma and has been correlated with disease severity. NAVIGATOR and the PATHFINDER clinical trial program Building on the Phase 2b PATHWAY trial, the Phase 3 PATHFINDER program included two trials, NAVIGATOR and SOURCE. EMJ Allergy Immunol.

Trials 52

Trials Production Sales Manufacturing 52

XTalks

DECEMBER 24, 2020

As of November, the WHO has discouraged the use of the drug as the agency’s review of data from four different clinical trials yielded no worthy effect on mortality, need for mechanical ventilation, time to clinical improvement or any other important patient outcomes. CRISPR Therapeutics.

Life Science 111

Life Science Vaccination Vaccine Gene 111

The Pharma Data

AUGUST 3, 2021

HDACs catalyze the removal of acetyl groups from acetylated lysine residues in histones, resulting in the modulation of gene expression. Reactivation of hepatitis B virus infection was reported in 1% of patients in clinical trials. HDACs also deacetylate non-histone proteins, such as transcription factors.

In-Vitro 52

In-Vitro Nurses Gene Expression Medicine 52

The Pharma Data

MAY 13, 2021

based weight loss intervention in adults with obesity: A randomized clinical trial. Opiate-like effects of sugar on gene expression in reward areas of the rat brain. Journal of Obesity. 2011;2011:651936. DOI: 10.1155/2011/651936. . Daubenmier J, Moran PJ, Kristeller J, et al. Effects of a mindfulness?based De Araujo IE.

Insulin 52

Insulin Production Hormones Medicine 52