Pharmaceutical Technology

JULY 29, 2022

Last week, CAMP4 Therapeutics announced the close of a $100 million Series B round , which will be used to advance their regulatory RNA (regRNA)-focused programs. However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea.

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RNA Gene Expression Gene Gene Therapy 245

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. DS: You describe your therapeutics as RNA Amplifiers. How do they work?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

Camargo

JULY 27, 2021

It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy. Additionally, gene editing allows us either to remove or to modify harmful genes. Formulation Considerations.

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Gene Therapy Gene Gene Editing Genetics 238

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. mRNA is a molecule made up of a single strand of ribonucleic acid (RNA). We believe it can now do the same for rare diseases.” “We

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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 through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”.

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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

The Pharma Data

DECEMBER 27, 2020

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 gene expression.

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Trials Antibody Gene Therapy Clinical Trials 52