Clinical Trials, Gene Expression and Gene Therapy

Clinical Trials

Gene Expression

Gene Therapy

Gene Therapy and Pharmacokinetics

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

Gene Therapy

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Astellas licenses companion drug for Pompe gene therapy

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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Bridging Innovation & Patient Care: The Growing Role of AI

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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

JULY 29, 2022

CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression.

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Bridging Innovation & Patient Care: The Growing Role of AI

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US FDA clears Transcenta’s IND for Rett syndrome treatment

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.

Gene Therapy

Gene Therapy Gene Gene Expression Regulation

Symvivo’s Oral COVID-19 Vaccine Enters Clinical Trials

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.

Clinical Trials

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Gene editing: beyond the hype

pharmaphorum

JANUARY 29, 2021

This growth reflects the continued and increasing interest in gene therapy – and with stock prices rising for editing companies across the board, Macrae says there has never been a more interesting time to be in genomic medicine. Zinc fingers are the most common control gene in the body,” Macrae explains. “We Macrae explains. “So

Gene Editing

Gene Editing Gene Gene Therapy In-Vivo

Angelman syndrome Pipeline: Unmet needs call for a robust pipeline

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.

Gene Therapy

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World Glaucoma Week 2024: New Advances in Glaucoma Care

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

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Zynteglo halt re-ignites viral vector safety concerns; analysts

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

Gene Therapy Gene Gene Sequencing Gene Expression

Gene Switch: A Novel Platform for Switching Genes On and Off

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

Gene Gene Therapy Gene Expression Regulation

Artificial intelligence could be new blueprint for precision drug discovery

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. Nature Communications, 2021.

Drugs

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Disruptive Technologies and Mature Regulatory Environment Vital for Cell Therapy Maturation

The Pharma Data

OCTOBER 15, 2020

“For cell therapy products to mature into ‘real’ products that deliver on the promises of 10 years ago, they must be scalable – which drives affordability – and they must solve their purity issues.”. For example, “The era of transplant medicine is unfolding before us,” Culley said. Source link.

Manufacturing

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Clinical Catch-Up: December 21-25 | BioSpace

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

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Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder

Delveinsight

JANUARY 18, 2021

However, DelveInsight’s Angelman syndrome market analysis report estimates that several approaches focused primarily on the Gene Therapies, Topoisomerase Inhibitors, Cannabidiol, Protein Phosphatase 2A inhibitor, among others are under development to find a cure for the Angelman syndrome.

Marketing

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2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Therapeutics.

Life Science

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Clinical Research Informer

Gene Therapy and Pharmacokinetics

Astellas licenses companion drug for Pompe gene therapy

Webinars

Trending Sources

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Webinars

US FDA clears Transcenta’s IND for Rett syndrome treatment

Symvivo’s Oral COVID-19 Vaccine Enters Clinical Trials

Gene editing: beyond the hype

Angelman syndrome Pipeline: Unmet needs call for a robust pipeline

World Glaucoma Week 2024: New Advances in Glaucoma Care

Zynteglo halt re-ignites viral vector safety concerns; analysts

Gene Switch: A Novel Platform for Switching Genes On and Off

Artificial intelligence could be new blueprint for precision drug discovery

Disruptive Technologies and Mature Regulatory Environment Vital for Cell Therapy Maturation

Clinical Catch-Up: December 21-25 | BioSpace

Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder

2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences

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