Pharmaceutical Technology

JULY 29, 2022

Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. This treatment activates the OTC gene so that ammonia can be removed from the blood. This gene therapy will give the instructions to produce the enzyme arginine so that it can be hydrolysed to ornithine.

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RNA Gene Expression Gene Gene Therapy 245

Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. JMB: Rick Young’s publication shows that RNA binding TFs fine-tunes gene expression.

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Gene Expression Gene Genetic Disease RNA 59

Drug Discovery World

OCTOBER 6, 2023

Researchers uncover molecular pathways underlying depression Scientists have discovered the link between gene expression, DNA methylation, and brain structural changes in depression, revealing new potential therapeutic targets.

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Drugs Gene Editing Genome Genomics 52

Delveinsight

JULY 24, 2021

In addition to this, Angelman syndrome pipeline also involves gene therapy candidates such as GTX-101 which is a therapy of GeneTx Biotherapeutics. 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

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

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Gene Editing Gene Gene Therapy In-Vivo 144

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

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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Gene Therapy Gene Licensing Licensing 118

XTalks

MARCH 13, 2024

There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease. The Glaucoma Foundation has funded studies focusing on two specific areas within gene therapy.

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Gene Therapy Gene Life Science DNA 111

XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. BacTRL Gene Therapy Platform. Related: Red Meat Allergy Test Gets FDA Clearance. “We

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Clinical Trials Clinical Trials Vaccination Vaccine 75

Drug Discovery World

JULY 10, 2023

Artificial intelligence (AI), gene editing, synthetic biology and more, are beginning to prove their usefulness in drug design and development, taking therapies away from the one-size-fits-all approach, towards more definable targets that operate more effectively.

Gene Therapy 52

Gene Therapy Gene Scientist Gene Editing 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).

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Genetics Medicine Gene Therapy Marketing 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. Nature Communications, 2021.

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Drugs Gene Expression Big Data Gene 52

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.

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Manufacturing Gene Therapy Gene Gene Expression 52

Drug Discovery World

FEBRUARY 10, 2023

For instance, Swedish biotech Lokon Pharma worked with the Testa Center for nine weeks on a project to scale up its manufacturing process for its gene therapy candidate. Testa Center was able to help Lokon Pharma gain a better understanding of the manufacturing process for its oncolytic virus-based gene therapy.

Life Science 52

Life Science Drugs Gene Therapy Manufacturing 52

Roots Analysis

JANUARY 14, 2024

Usually, the desired gene, such as human insulin gene, when inserted into the plasmid of the host cell uses transcriptional and translational machinery of the host to express itself. It is worth mentioning that in vitro gene expression requires a suitable host for the production of a specific gene product.

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Contract Manufacturing Manufacturing Protein Bacteria 40

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.

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Gene Gene Therapy Gene Expression Regulation 40

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

The Pharma Data

NOVEMBER 8, 2020

DB-OTO, Decibel’s investigational gene therapy to restore hearing in children with congenital deafness due to a deficiency in the otoferlin gene, is in preclinical studies, and Decibel expects to initiate clinical testing in 2022. Thompson, M.D., a partner at OrbiMed, to its board of directors.

Gene Therapy 40

Gene Therapy Gene Gene Expression Genome 40

Drug Discovery World

FEBRUARY 29, 2024

They are called: Sitting down with… PharmEnable , Is single-cell gene expression the next trend in next generation sequencing? This is the latest episode of the free DDW narrated podcast, titled “What trends will speed up the journey from clinic to bedside?” which covers three written for Volume 23 – Issue 4, Fall 2022 of DDW.

Gene Expression 52

Gene Expression DNA Genome Genomics 52

XTalks

MAY 4, 2021

These modifications regulate gene expression without changing the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. It’s a great tool for controlling gene expression.”.

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DNA Gene Gene Silencing Genome 98

Drug Discovery World

OCTOBER 17, 2023

RA: With spatial biology being cross-disciplinary – in that it uses techniques in both gene expression analysis and immunofluorescence – is expertise in the field limited? Analysing spatial changes during disease progression or treatment response is key to fully understand pathologies. Let’s take CAR T cells as an example.

Gene Expression 52

Gene Expression Drugs Protein Gene 52

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.

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Marketing Gene Drugs Protein 52

XTalks

MAY 4, 2021

These modifications regulate gene expression without altering the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. It’s a great tool for controlling gene expression.”.

DNA 52

DNA Gene Gene Silencing Genome 52

Drug Discovery World

DECEMBER 15, 2022

Improve delivery: Clinical translation of gene therapy is dependent on the efficient and safe delivery of gene editing tools into cells, and this can occur either ex vivo or in vivo. These therapies could have even broader and more valuable impacts for the treatment of diseases caused by genetic abnormalities.

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Gene Editing Gene In-Vivo Genome 52

The Pharma Data

DECEMBER 27, 2020

STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 gene expression. RESTORE-1 is a Phase II trial of an intracerebral AAV-based gene therapy for Parkinson’s disease.

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Trials Antibody Gene Therapy Clinical Trials 52

Drug Discovery World

OCTOBER 17, 2022

For pharma and biotech companies – whether working on small or large molecule drugs, or cell and gene therapies – this means empowering scientists with better and faster data management approaches, best practices, and leading technologies so they can pinpoint new molecules and formulations and modify existing ones more easily and quickly.

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Marketing Scientist Gene Expression Drugs 52

Drug Discovery World

JANUARY 23, 2023

Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2. Single-cell RNA-sequencing of differentiating iPS cells reveals dynamic genetic effects on gene expression. 2020;13:dmm042317. doi:10.1242/dmm.042317. 13: Cuomo A, Seaton D, McCarthy D, et al.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. It also enables the downregulation of gene expression by directly knocking out the cytoplasmic mRNA transcripts.

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DNA Genome Genomics RNA 98

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 111

Life Science Vaccination Vaccine Gene 111

The Pharma Data

MAY 15, 2021

I was recently reminded of my journey out of the HCT nightmare as I watched a documentary about gene therapy. Gene therapy is a huge deal – some gene therapies are already achieving jaw-dropping results for people with previously untreatable illnesses. It’s fascinating stuff.

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Bacteria Gene Gene Therapy Gene Expression 52