pharmaphorum

MAY 11, 2022

Novo Nordisk’s chief scientific officer Marcus Schindler called it “a new and innovative way of collaborating”, with the current plan that it will generate between three and five research programmes within the first three years. The move follows Novo Nordisk’s $3.3

Gene Silencing 126

Gene Silencing Medicine RNA Licensing 126

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 105

In-Vivo Gene Editing Trials Drugs 105

Scienmag

JUNE 7, 2021

Divita Mathur, Research Assistant Professor, is studying cytosolic access and instability of DNA nanoparticles. A number of candidate therapies such as CRISPR-Cas9 and gene silencing require the efficient delivery of functional nucleic acids to the cell cytosol and nucleus.

DNA 51

DNA Gene Silencing Gene Research 51

pharmaphorum

JULY 28, 2022

Alnylam Pharma has made a name for itself, developing gene-silencing therapies for rare disorders, but its latest discovery could take it into a much larger category – metabolic and cardiovascular disease. Transgenic mice in which the gene has been deleted have improved control of blood glucose and insulin sensitivity.

Biobanking 52

Biobanking Gene Gene Silencing Drugs 52

pharmaphorum

JULY 13, 2021

Novo Nordisk’s head of research and early development, Marcus Schindler, said the acquisition of PRX004 reinforces the company’s “dedication to advancing new disease-modifying therapies for the benefit of people with cardiovascular diseases which are the world’s leading cause of death.”

Gene Silencing 98

Gene Silencing Sales Gene Editing Clinical Trials 98

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 52

DNA Gene Gene Silencing Genomics 52

pharmaphorum

FEBRUARY 9, 2021

InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells.

Trials 59

Trials Gene Silencing RNA Gene Expression 59

pharmaphorum

DECEMBER 23, 2020

It has been a year dominated by the pandemic and many life sciences research projects were put on hold as big pharma turned its attention to vaccines and therapies. But there was some considerable progress in other fields of medicine even though research efforts were diverted away, reports Richard Staines. Rare disease progress.

Marketing 96

Marketing Gene Gene Silencing Drugs 96

The Pharma Data

JANUARY 26, 2021

The company’s lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. is a biopharmaceutical company developing transformational therapeutics capitalizing on groundbreaking scientific and medical discoveries from leading research and academic institutions. Scopus BioPharma Inc.

Gene Therapy 52

Gene Therapy Gene Silencing Gene Containment 52

Delveinsight

AUGUST 6, 2020

Hereditary ATTR amyloidosis , also known as Familial ATTR, is an autosomal dominant disease and a result of mutations in the TTR gene, thus it is passed down from one generation to the next one. Gene-silencing Therapies. However, recent research and clinical studies in the domain have widened the horizon for the hATTR treatment.

Protein 52

Protein Gene Silencing Marketing Gene 52

Roots Analysis

OCTOBER 22, 2023

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. In the last few years, researchers have become interested in using in vitro transcribed (IVT) mRNA as a drug delivery agent. RNA therapeutics offer several advantages over small molecules.

RNA 40

RNA Protein Genetics Gene Expression 40

The Pharma Data

NOVEMBER 24, 2020

About RNAi RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Until today, there were no approved pharmaceutical therapies for PH1.

FDA Approval 52

FDA Approval Production RNA Medicine 52