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. Zinc fingers. The exact mechanism depends on the disease in question.

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Gene Editing Gene Gene Therapy In-Vivo 145

Pharmaceutical Technology

JANUARY 24, 2023

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

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Gene Therapy Gene Gene Expression Regulation 208

BioTech 365

DECEMBER 8, 2020

Rocket Pharmaceuticals Announces Positive Gene Expression, Clinical Biomarker and Preliminary Functional Data from Phase 1 Trial of RP-A501 for the Treatment of Danon Disease Rocket Pharmaceuticals Announces Positive Gene Expression, Clinical Biomarker and Preliminary Functional Data from Phase 1 Trial … Continue reading →

Gene Expression 40

Gene Expression Gene Trials 40

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

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.

Gene Therapy 59

Gene Therapy Gene Trials Gene Expression 59

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

MARCH 26, 2025

It works by increasing SCN1A gene expression, which is crucial for stabilizing brain signaling. In its Phase I/II trial, patients saw big reductions in seizure frequency. The 60-week trial will enroll 150 children and adolescents and measure seizure control, cognition and behavior.

Life Science 66

Life Science Gene Expression Trials Drugs 66

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

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. BacTRL Gene Therapy Platform. COVID-19 Clinical Trials.

Clinical Trials 75

Clinical Trials Clinical Trials Vaccination Vaccine 75

XTalks

DECEMBER 30, 2024

XTALKS WEBINAR: How to Overcome Trial Planning and Patient Recruitment Challenges Live and On-Demand: Friday, January 24, 2025, at 11am EST (4pm GMT/UK) Register for this free webinar to obtain insights to improve trial planning, stakeholder engagement and patient recruitment outcomes. in mSLEDAI-2K compared to placebo.

Immune Response 52

Immune Response Gene Expression Drugs Trials 52

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.

Antibody 75

Antibody Gene Expression Gene Trials 75

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

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

Delveinsight

JULY 24, 2021

Ovid Therapeutics is running Phase III clinical trials for its drug, Gaboxadol ( OV101 ). In addition to this, Angelman syndrome pipeline also involves gene therapy candidates such as GTX-101 which is a therapy of GeneTx Biotherapeutics.

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Gene Therapy Gene Gene Expression RNA 96

Roots Analysis

AUGUST 31, 2023

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

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.

Trials 59

Trials Gene Silencing RNA Gene Expression 59

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.

Regulation 52

Regulation Gene Gene Expression Packaging 52

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

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

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%

Trials 94

Trials DNA Genomics Genome 94

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

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. . million in Europe.

Gene Therapy 52

Gene Therapy Gene Gene Sequencing Gene Expression 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.

Development 130

Development Bioequivalency Gene Expression Drugs 130

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

Delveinsight

JANUARY 18, 2021

Angelman syndrome is a result of the missing or dysfunctional gene known as UBE3A. The gene is responsible for the normal and healthy development of an individual physically as well intellectually. Thus, restoring the presence of this particular gene can help to restore several of the functions of the brain.

Marketing 52

Marketing Gene Drugs Protein 52

XTalks

JUNE 18, 2024

The company is currently testing its lead candidate, EXG-34217 — an autologous cell therapy in Phase I/II trials for treating telomere biology disorders (TBDs), conditions known to cause bone marrow failure with treatments mainly limited to complicated bone marrow transplants. This metaphor captures the essence of their therapeutic approach.

Protein 52

Protein RNA Genetics Gene Expression 52

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

The Pharma Data

MARCH 3, 2022

This finding stemmed from more than two decades of her basic research into a gene in pancreatic islets called TXNIP. 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.

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. Non-COVID-19-Related.

Trials 52

Trials Antibody Gene Therapy Gene 52

Pharma Marketing Network

DECEMBER 21, 2020

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. greater safety, transient and reversible effect on gene expression).

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

The Pharma Data

OCTOBER 15, 2020

Immuno-oncology and CAR T cells energized the field of regenerative medicine, but for cell and gene to deliver on their promises, new, disruptive technologies and new modes of operation are needed. So far, 25 people have been treated in a Phase I/II trial. Other cell and gene companies are advancing solutions, too.

Manufacturing 52

Manufacturing Gene Therapy Gene Gene Expression 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.

Gene Expression 52

Gene Expression Packaging Packaging Medicine 52

XTalks

MARCH 22, 2021

The antibody accrued promising data in a randomized, placebo-controlled Phase II study in frontline advanced gastric or gastroesophageal junction (GEJ) cancer, and is now being prepared for Phase III trials. The tool analyzes the differential expression of over 90 genes to distinguish between 50 tumors types and subtypes.

Life Science 110

Life Science Development Marketing Gene Expression 110

The Pharma Data

NOVEMBER 9, 2020

Trial Also Met the Primary Endpoint in Patients With Low Levels of Eosinophils. In the subgroup of patients with baseline eosinophil counts less than 300 cells per microliter, the trial met the primary endpoint with tezepelumab demonstrating a statistically significant and clinically meaningful reduction in AAER.

Trials 52

Trials Production Sales Manufacturing 52

The Pharma Data

JANUARY 17, 2021

The trial is comprised of three dose escalation cohorts ranging from 30 ?g g with five patients in each group, and a total of 15 patients will be enrolled in the trial. ” The Company expects to report initial clinical data from the trial in 2021. . 1 and COX-2 gene expression. 1 and COX-2.

Gene Expression 52

Gene Expression Trials Development Production 52

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) 8) There were no clinically meaningful differences in safety results between the tezepelumab and placebo groups in the NAVIGATOR trial.(8)

Allergies 52

Allergies Trials In-Vivo Medicine 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 +.

Development 40

Development Drugs Trials FDA Approval 40