Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease. was swooped up by Real Chemistry in January.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

XTalks

DECEMBER 22, 2021

The RNA Revolution: From mRNA Vaccines to RNA Editing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. RNA technology is not new nor has its potential been surprising. RNA in the Making. So why did this perceived RNA ‘revolution’ take so long?

Life Science 98

Life Science RNA Vaccination Vaccine 98

pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. Severely-affected patients need regular blood transfusions to maintain their haemoglobin levels. .

Genetic Disease 85

Genetic Disease Protein RNA Doctors 85

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. It’s a great tool for controlling gene expression.”.

DNA 98

DNA Gene Gene Silencing Genome 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 Genome 52

XTalks

DECEMBER 13, 2023

Related: Top 30 Pharma Companies in 2023: Statistics and Trends 1) Moderna Compound annual growth rate: 415 percent Moderna, headquartered in Massachusetts, is a prominent biotechnology company specializing in RNA therapeutics, particularly mRNA vaccines. Alpine Immune Sciences has recently announced an augmentation in its R&D investment.

Genetics 111

Genetics Vaccination Vaccine DNA 111

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. Its job is to carry coding information that is essential to the translation and processing of functional proteins.

RNA 52

RNA Protein Vaccination Vaccine 52

pharmaphorum

JANUARY 10, 2022

Previously, messenger RNA (mRNA) therapies were a niche part of the global R&D pipeline, now a wide section of the public is at least familiar with the name of this type of therapy. The company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and genetic diseases.

RNA 99

RNA Vaccination Vaccine Pharma Companies 99

Drug Discovery World

FEBRUARY 28, 2023

Interestingly, at least 80% of all rare diseases originate from monogenic mutations, meaning that they arise from mutations in a single gene 4. Therefore, the risk for off-target insertional mutagenesis is greatly reduced. In addition, it may be easier to target the therapeutic molecule to the correct cell or tissue.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 52

The Pharma Data

DECEMBER 27, 2020

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. The drug is a topical ointment applied to the lower lid to address the build-up and shedding of proteins at the opening of the Meibomian gland. The drug is an autologous IL13Ralpha2-CAR-T cell product.

Trials 52

Trials Antibody Gene Therapy Clinical Trials 52

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

FDA Approval 52

FDA Approval Production RNA Medicine 52

Drug Discovery World

FEBRUARY 22, 2024

Indeed, we have shown that the causative mutations of some leukodystrophies affect the subunits of an important cellular enzyme, RNA polymerase III, preventing its normal assembly – it turned out that Riluzole can counteract this assembly defect,” says Maxime Pinard, the researcher responsible for the project in Coulombe’s lab. ‘’For

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52