Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What diseases are you currently targeting? Can you give some examples?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

Pharmaceutical Technology

JULY 29, 2022

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. Typically, nitrogen in the blood is removed in the form of urea.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

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

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

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