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.

Gene Therapy 59

Gene Therapy Gene Trials Gene Expression 59

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., 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.

Genetics 52

Genetics Medicine Gene Therapy Marketing 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 (..)

Gene Expression 52

Gene Expression Scientist Gene Clinical Trials 52

XTalks

MAY 31, 2022

The precise cause of psoriasis is unknown, but risk factors include genetics and a family history of the condition, heavy alcohol consumption, stress, smoking and dry or cold weather. XTALKS WEBINAR: All Means All: The Road to Inclusivity in Clinical Trials. Clinical Trials of Vtama. Dermavant’s Future Targets.

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FDA Approval Clinical Trials Clinical Trials Trials 98

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

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.

Protein 52

Protein RNA Genetics 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.

Gene Therapy 111

Gene Therapy Gene Life Science DNA 111

The Pharma Data

JULY 12, 2021

“This is mostly because drugs that work perfectly in preclinical inbred models, such as laboratory mice, that are genetically or otherwise identical to each other, don’t translate to patients in the clinic, where each individual and their disease is unique. HUMANOID Center of Research Excellence?

Drugs 52

Drugs Gene Expression Big Data Gene 52

Roots Analysis

AUGUST 31, 2023

It is worth noting that the transcription factor is responsible for recruiting RNA polymerase to bind to the gene and produce messenger RNA, which is then translated into the protein. An additional level of genetic control is provided by gene switches that are located upstream of the promoter region.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

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

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

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

XTalks

MARCH 22, 2021

Using a simple, standard blood sample, the assay can detect actionable genetic mutations in cancers, including cancer-driving alterations. Both of Biotheranostics’ diagnostics are PCR-based gene expression tests.

Life Science 110

Life Science Development Marketing Gene Expression 110

Delveinsight

JANUARY 18, 2021

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. The company was running Phase III trials of the therapy for AS; however, the therapy flunked in the trial failing to meet its primary endpoint. As a consequence, Ovid halted everything related to OV101 and Angelman syndrome.

Marketing 52

Marketing Gene Drugs Protein 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

The Pharma Data

DECEMBER 14, 2020

Combining DetermaRx with our products for genetic testing and MRD detection (currently under R&D), we can provide a comprehensive testing strategy for oncologists to ultimately benefit Chinese early-stage NSCLC patients by improving their survival and quality of life.

Licensing 52

Licensing Licensing Marketing Gene Expression 52

XTalks

DECEMBER 24, 2020

However, for virologists like Lori Frappier, PhD, professor in the department of molecular genetics at the University of Toronto, the outbreak of SARS-CoV-2 was less of a surprise and more of an inevitability. CRISPR Gene Editing Inventors Win Nobel Prize. But nevertheless, 2021 into 2022 will be the time.”.

Life Science 111

Life Science Vaccine Vaccination Gene 111

The Pharma Data

MAY 13, 2021

based weight loss intervention in adults with obesity: A randomized clinical trial. Reduced Reward-driven Eating Accounts for the Impact of a Mindfulness-Based Diet and Exercise Intervention on Weight Loss: Data from the SHINE Randomized Controlled Trial. Neural and Genetic Correlates of the Social Sharing of Happiness.

Insulin 52

Insulin Production Hormones Medicine 52