Drug Discovery World

MARCH 6, 2024

The development of effective new disease-modifying treatments in neurodegenerative and neuropsychiatric disorders has been hindered by their inherent genetic complexity, environmental influences, and clinical variability. This is comparatively easier to accomplish in these diseases because they are relatively monogenic.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

XTalks

DECEMBER 13, 2023

3) Fulgent Genetics Compound annual growth rate: 260 percent Fulgent Genetics, headquartered in California, is a technology-driven company with a strong presence in clinical diagnostics and therapeutic development. The company achieved core revenues of $67 million, reflecting a remarkable 48 perecent year-over-year growth.

Genetics 111

Genetics Vaccination Vaccine DNA 111

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). In the UK, Libmeldy will be administered by a specialist service through the Centre for Genomic Medicine at Saint Mary’s hospital in Manchester.

Gene Therapy 98

Gene Therapy Drugs Gene Gene Silencing 98

XTalks

APRIL 15, 2021

New advances in heart failure genomics are helping to address this challenge. Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. Watch this on-demand webinar to hear from these experts.

Development 98

Development Drugs Genome Genomics 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. Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences.

DNA 52

DNA Gene Gene Silencing Genome 52

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. 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

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95