Development, Gene Silencing and Protein - Clinical Research Informer

Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit

pharmaphorum

JANUARY 6, 2021

Two years after starting to work together, Novo Nordisk and Dicerna have selected the first candidate from a joint project to find new, gene-silencing drugs for liver-related cardiometabolic diseases. The nomination of a first candidate in the collaboration that will advance into development triggers a $2.5

Gene Silencing

Gene Silencing Gene RNA Contract Manufacturing

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

Scienmag

JANUARY 4, 2021

The findings could yield important implications for development of drugs to treat cancer and other diseases. 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).

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

Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

pharmaphorum

JULY 13, 2021

The bulk of Novo Nordisk’s revenues come from its diabetes products, although it also active in the development and sale of drugs for growth disorders, haemophilia and sickle cell disease. In ATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body.

Gene Silencing

Gene Silencing Sales Gene Editing Clinical Trials

NICE backs Alnylam’s Amvuttra for rare form of amyloidosis

pharmaphorum

JANUARY 19, 2023

Alnylam’s gene-silencing drug Amvuttra has been recommended as a treatment for hereditary transthyretin-related (ATTR) amyloidosis by NICE, paving the way for the drug to be made available on the NHS in England and Wales.

Gene Silencing

Gene Silencing RNA Production Protein

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

pharmaphorum

OCTOBER 29, 2020

Already a major player in gene therapy, Novartis has swooped on US startup Vedere Bio in a $280 million deal that builds its position in inherited eye diseases that can lead to blindness. It also gets two preclinical-stage development projects.

Gene Therapy

Gene Therapy Gene Gene Silencing Protein

Frontotemporal dementia: the state of treatment development

Pharmaceutical Technology

FEBRUARY 20, 2023

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. This is also one of the major challenges that slow the development of effective treatments for FTD, he adds. This protein promotes lysosomal function.

Development

Development Gene Therapy Protein Gene

The trends driving ELRIG Drug Discovery 2022

Drug Discovery World

OCTOBER 4, 2022

As it stands, there are currently 19 gene therapies approved for clinical use, alongside 18 RNA therapies and 59 non-genetically modified cell therapies, according to the Q2 report by the American Society of Cell & Gene Therapy. million by Horizon Europe which will go towards its goal of developing bioprocessing technologies. .

Gene Therapy

Gene Therapy Drugs Gene Manufacturing

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

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

RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

OCTOBER 22, 2023

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein.

RNA

RNA Protein Genetics Gene Expression

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). Yancopoulos, M.D.,

Gene Silencing

Gene Silencing Protein Gene Engineer

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. Epigenome Editing with CRISPR.

DNA

DNA Gene Gene Silencing Genome

The use of base editing in stem-cell based therapies

Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. In addition, iPSC lines, despite their pluripotency, have skewed suitability towards certain cell types 12,13.

Gene Editing

Gene Editing Gene Engineer DNA

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

The Pharma Data

NOVEMBER 24, 2020

Together, the ILLUMINATE studies comprise a comprehensive clinical development program intended to demonstrate the safety and efficacy of Oxlumo across the full spectrum of patients diagnosed with PH1. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development.

FDA Approval

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Clinical Research Informer

Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit

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

Trending Sources

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

Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

NICE backs Alnylam’s Amvuttra for rare form of amyloidosis

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

Frontotemporal dementia: the state of treatment development

The trends driving ELRIG Drug Discovery 2022

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

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

RNA Therapeutics: A Novel Approach to Treating Diseases

REGENERON AND ALNYLAM REPORT POSITIVE INTERIM PHASE 1

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

The use of base editing in stem-cell based therapies

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

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