Drugs, 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 post Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit appeared first on.

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 Dicerna has been a hot tip for takeover for a couple of years, as the list of approved drugs that work via RNAi has lengthened. billion in cash.

Gene Silencing

Gene Silencing RNA Protein Gene

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

The trends driving ELRIG Drug Discovery 2022

Drug Discovery World

OCTOBER 4, 2022

DDW Editor Reece Armstrong explores some of the trends that will be featured in sessions throughout ELRIG Drug Discovery 2022 and the news behind them. In the UK, effort has been made to scale-up the country’s cell and gene therapy manufacturing capabilities. Screening acts as the bedrock of many drug discovery activities.

Gene Therapy

Gene Therapy Drugs Gene Manufacturing

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

pharmaphorum

JULY 13, 2021

Diabetes giant Novo Nordisk has opened a new front in its rare disease R&D, thanks to an acquisition agreement with Prothena focusing on drugs for ATTR amyloidosis. In ATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body. billion by 2025.

Gene Silencing

Gene Silencing Sales Gene Editing Clinical Trials

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

pharmaphorum

OCTOBER 29, 2020

Earlier this year, the group also signed a licensing deal with Dyno Therapeutics for its AAV capsid-based gene delivery technology, also focused on ocular disease which remains one of Novartis’ core drug development targets despite the spin-off of the Alcon eyecare division in 2019.

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). Gene-silencing Therapies.

Gene Silencing

Gene Silencing Protein Marketing Gene

Frontotemporal dementia: the state of treatment development

Pharmaceutical Technology

FEBRUARY 20, 2023

The therapy is specifically aimed at treating FTD patients who have mutations in their granulin gene, which encodes for the protein progranulin (PGRN). This protein promotes lysosomal function. In addition to gene therapy, gene silencing, which can be done using ASOs, is also an interesting approach, says Barmada.

Development

Development Gene Therapy Protein Gene

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

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

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. In three more patients treated at 0.1mg/kg levels fell by 52%.

In-Vivo

In-Vivo Gene Editing Trials Drugs

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

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

The Pharma Data

NOVEMBER 24, 2020

Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. Injection site reactions (ISRs) were the most common drug-related adverse reaction.

FDA Approval

FDA Approval Production RNA Medicine

The use of base editing in stem-cell based therapies

Drug Discovery World

JANUARY 23, 2023

This precision editing approach allows for a host of potential therapeutic applications, including gene silencing, either via splice site disruption or the addition of premature stop codons, as well as gene correction in single nucleotide variant diseases. 2021;4:58–68. doi:10.1089/crispr.2020.0035. 2020.0035.

Gene Editing

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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

NICE backs Alnylam’s Amvuttra for rare form of amyloidosis

The trends driving ELRIG Drug Discovery 2022

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

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

Unfolding The Folds Of Transthyretin

Frontotemporal dementia: the state of treatment development

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

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

RNA Therapeutics: A Novel Approach to Treating Diseases

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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