Gene Expression, Gene Therapy and RNA - Clinical Research Informer

Gene Expression

Gene Therapy

RNA

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

Gene Therapy Gene Gene Editing Genetics

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

Pharmaceutical Technology

JULY 29, 2022

Last week, CAMP4 Therapeutics announced the close of a $100 million Series B round , which will be used to advance their regulatory RNA (regRNA)-focused programs. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression.

RNA

RNA Gene Expression Gene Gene Therapy

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Gene expression delivery tool ‘slides’ instructions into cells

Drug Discovery World

NOVEMBER 1, 2022

If further studies verify the results, this could be a new method for targeting specific cell types for a variety of disorders that could be treated with gene therapies. Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

Gene Expression

Gene Expression Gene Packaging Packaging

Turning science into business: Amplifying mRNA by targeting regRNAs

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. DS: You describe your therapeutics as RNA Amplifiers. How do they work?

Gene Expression

Gene Expression Gene Genetic Disease RNA

Angelman syndrome Pipeline: Unmet needs call for a robust pipeline

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.

Gene Therapy

Gene Therapy Gene Expression Gene RNA

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

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

Genetics Medicine Gene Therapy Marketing

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

Roots Analysis

AUGUST 31, 2023

Mechanism of Gene Switch During the transcription process, the promoter region, which is located near the upstream end of each gene, binds to transcription factor, which is a specific type of protein. An additional level of genetic control is provided by gene switches that are located upstream of the promoter region.

Gene

Gene Gene Therapy Gene Expression Regulation

The future outlook for mRNA therapies

Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. mRNA is a molecule made up of a single strand of ribonucleic acid (RNA).

RNA

RNA Protein Vaccination Vaccine

Podcast: What trends will speed up the journey from clinic to bedside?

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

Gene Expression DNA Genome Genomics

What spatial biology can tell us about disease and drug discovery

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? The first step to reach this goal will be to generate protein and RNA data from the same sample. Let’s take CAR T cells as an example.

Gene Expression

Gene Expression Drugs Protein Gene

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

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.”.

DNA

DNA Gene Gene Silencing Genome

CRISPR breakthroughs: New solutions for common diseases

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. CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg. parvovirus B19, Flaviviridae, Ebola, and Coronaviridae), DNA viruses (e.g.,

DNA

DNA Genome Genomics RNA

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

Marketing Gene Drugs Protein

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

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

DNA Gene Gene Silencing Genome

Delivering on the promise of gene editing

Drug Discovery World

DECEMBER 15, 2022

Prime editing, while also employing a single strand nick, expands this scope to all 12 possible changes using a more complex prime editing guide RNA (pegRNA). 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.

Gene Editing

Gene Editing Gene In-Vivo Genome

The use of base editing in stem-cell based therapies

Drug Discovery World

JANUARY 23, 2023

Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2. Base editing uses a guide RNA in partnership with a nickase version of Cas9, mutated to cut only a single strand of DNA, along with a deaminase enzyme to enable alteration of a single nucleotide 23,24.

Gene Editing

Gene Editing Gene Engineer DNA

Clinical Catch-Up: December 21-25 | BioSpace

The Pharma Data

DECEMBER 27, 2020

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. 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.

Trials

Trials Antibody Gene Therapy Clinical Trials

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

Life Science Vaccination Vaccine Gene

Gene Therapy and Pharmacokinetics

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

Trending Sources

Gene expression delivery tool ‘slides’ instructions into cells

Turning science into business: Amplifying mRNA by targeting regRNAs

Angelman syndrome Pipeline: Unmet needs call for a robust pipeline

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

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

The future outlook for mRNA therapies

Podcast: What trends will speed up the journey from clinic to bedside?

What spatial biology can tell us about disease and drug discovery

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

CRISPR breakthroughs: New solutions for common diseases

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

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

Delivering on the promise of gene editing

The use of base editing in stem-cell based therapies

Clinical Catch-Up: December 21-25 | BioSpace

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

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