XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. The cells are then injected back into the patient.

Gene Therapy 98

Gene Therapy Drugs Gene Gene Silencing 98

Drug Discovery World

OCTOBER 12, 2023

ELRIG’s Drug Discovery 2023 will be taking place at ACC Liverpool, UK from 18-19 October 2023, focusing on the importance of a diverse life science ecosystem. Dr Michelle Fraser is Head of Cell & Gene Therapy at global health science solutions company, Revvity.

Drugs 59

Drugs Life Science Genome Genomics 59

XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Harnessing the Cellular Engineering Potential of CRISPR.

Gene Editing 98

Gene Editing DNA Gene 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

Drug Discovery World

MARCH 6, 2024

To find new ways of diagnosing and treating complex diseases we first must understand the mechanisms underpinning their key pathological drivers, how these relate to different patient subgroups, and which drugs might be useful in ameliorating their effects – this is the basis of precision neuroscience.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

XTalks

DECEMBER 13, 2023

The company’s clinical diagnostic division provides a wide array of services, including molecular diagnostic testing, comprehensive genetic testing and high-quality anatomic pathology laboratory services. The company boasts an expansive portfolio of assets that grant rights to future potential royalty and milestone payments.

Genetics 111

Genetics Vaccination Vaccine DNA 111

Drug Discovery World

APRIL 30, 2024

Rare diseases are largely treated using gene therapies which employ modified viruses to bring the therapy to the disease cells. We believe it can now do the same for rare diseases.” “We We have shown that mRNA holds an unprecedented therapeutic potential for incurable genetic diseases, in particular liver conditions.

RNA 52

RNA Protein Vaccination Vaccine 52