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.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105

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.

Biobanking 52

Biobanking Gene Gene Silencing Drugs 52

The Pharma Data

JANUARY 26, 2021

This drug candidate is highly distinctive, encompassing both gene therapy and immunotherapy by synthetically linking siRNA to an oligonucleotide TLR9 agonist, creating the potential for targeted gene silencing with simultaneous TLR stimulation and immune activation in the tumor microenvironment. Scopus BioPharma Inc.

Gene Therapy 52

Gene Therapy Gene Silencing Gene Containment 52

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. The research was published earlier this month in the journal Cell.

DNA 98

DNA Gene Gene Silencing Genome 98

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

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

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

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

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. The research was published earlier this month in the journal Cell. pyogenes dCas9.

DNA 52

DNA Gene Gene Silencing Genome 52

Delveinsight

AUGUST 6, 2020

Gene-silencing Therapies. Silencing or modifying gene TTR aims to reduce the effect of both variant and wild-type TTR gene, thus reducing hepatic production by targeting its mRNA. The past years have witnessed a shift from off-label and symptomatic therapies to approved TTR stabilizers and gene-silencing therapies.

Protein 52

Protein Gene Silencing Marketing Gene 52

Roots Analysis

OCTOBER 22, 2023

In the last few years, researchers have become interested in using in vitro transcribed (IVT) mRNA as a drug delivery agent. RNA interference (RNAi) Therapeutics : Fundamentally, RNAi is a natural process of post-transcriptional gene silencing, involving short strands of nucleic acids. Ronit holds a B.

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