Gene Silencing, Genetics and Protein - Clinical Research Informer

Study resolves long-running controversy over critical step in gene silencing

Scienmag

JANUARY 4, 2021

BOSTON – A long-running debate over how an important gene-silencing protein identifies its targets has been resolved by researchers at Massachusetts General Hospital (MGH). The findings could yield important implications for development of drugs to treat cancer and other diseases.

Gene Silencing

Gene Silencing Gene Protein Development

Novo Nordisk snaps up RNAi partner Dicerna in $3.3bn takeover deal

pharmaphorum

NOVEMBER 18, 2021

Danish drugmaker Novo Nordisk must like what it has seen in its two-year-old alliance with gene-silencing specialist Dicerna Pharma – it has just agreed to acquire the biotech for $3.3 Once in the blood, the GalXC molecules travel to the liver, where they enter hepatocyte cells and can switch protein production on or off.

Gene Silencing

Gene Silencing RNA Protein Gene

Novartis keeps a close eye on gene therapy with Vedere Bio buy

pharmaphorum

OCTOBER 29, 2020

Shortly after, Novartis teamed up with Sangamo in a deal focused on the adjacent gene therapy discipline of gene silencing, targeting neurodevelopmental disorders like autism. It also gets two preclinical-stage development projects. IRDs affect around 2 million people worldwide.

Gene Therapy

Gene Therapy Gene Gene Silencing Protein

Unfolding The Folds Of Transthyretin

Delveinsight

AUGUST 6, 2020

Transthyretin (TTR) is a protein that the liver produces to facilitate the transfer of thyroid hormone and vitamin A in the blood. However, due to specific reasons, the TTR protein begins to misfold and aggregate as fibrils, a process known as amyloidosis of Transthyretin (ATTR). hATTR Market: Present Treatment Options.

Gene Silencing

Gene Silencing Protein Marketing Gene

Frontotemporal dementia: the state of treatment development

Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.

Development

Development Gene Therapy Protein Gene

The trends driving ELRIG Drug Discovery 2022

Drug Discovery World

OCTOBER 4, 2022

The pandemic might have affected the number of clinical trials taking place for cell and gene therapies, with activity decreasing by around 15% compared to 2020, but major therapies such as Novartis’ gene therapy drug for spinal muscular atrophy (SMA) Zolgensma still made it to market. .

Gene Therapy

Gene Therapy Drugs Gene Manufacturing

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

pharmaphorum

JUNE 28, 2021

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo

In-Vivo Gene Editing Trials Drugs

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.

DNA

DNA Gene Gene Silencing Genome

World RNA Day: What impact RNA it had on drug discovery?

Drug Discovery World

AUGUST 1, 2023

1 August was first chosen as World RNA Day in 2018 as a play on AUG (adenine, uracil and guanine), a triple sequence of RNA (called a codon) that initiates protein synthesis by the cell. Since then, it has been observed to publicise the importance of this molecule in the generation of proteins in the body.

RNA

RNA Drugs Gene Silencing Vaccination

RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

OCTOBER 22, 2023

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. These therapeutics are broadly classified into two categories, namely coding RNAs and non-coding RNAs.

RNA

RNA Protein Genetics Gene Expression

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

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA

DNA Gene Gene Silencing Genome

REGENERON AND ALNYLAM REPORT POSITIVE INTERIM PHASE 1

The Pharma Data

APRIL 27, 2023

(Nasdaq: ALNY ) announced today positive interim results from the ongoing single ascending dose part of the Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy (CAA). Chief Medical Officer of Alnylam. “We

Gene Silencing

Gene Silencing Protein Gene Engineer

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. About Primary Hyperoxaluria Type 1 (PH1) PH1 is an ultra-rare genetic disease that affects an estimated one to three individuals per million in the United States and Europe. Oxlumo should be administered by a healthcare professional.

FDA Approval

FDA Approval Production RNA Medicine

The use of base editing in stem-cell based therapies

Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector. Single-cell RNA-sequencing of differentiating iPS cells reveals dynamic genetic effects on gene expression.

Gene Editing

Gene Editing Gene Engineer DNA

Clinical Research Informer

Study resolves long-running controversy over critical step in gene silencing

Novo Nordisk snaps up RNAi partner Dicerna in $3.3bn takeover deal

Trending Sources

Novartis keeps a close eye on gene therapy with Vedere Bio buy

Unfolding The Folds Of Transthyretin

Frontotemporal dementia: the state of treatment development

The trends driving ELRIG Drug Discovery 2022

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

World RNA Day: What impact RNA it had on drug discovery?

RNA Therapeutics: A Novel Approach to Treating Diseases

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

REGENERON AND ALNYLAM REPORT POSITIVE INTERIM PHASE 1

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

The use of base editing in stem-cell based therapies

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