Drug Discovery World
APRIL 17, 2024
JMB: Regulatory RNAs (or regRNAS) are molecules that directly control the expression of nearby protein-coding genes. CAMP4 is exploiting these regulatory interactions to specifically control the expression of genes tied to disease. What diseases are you currently targeting? What are the challenges of drug discovery in this area?
STAT News
SEPTEMBER 16, 2022
Enjoy, and see you soon … Intellia Therapeutics reported that the first six patients to receive its CRISPR-based treatment for a genetic swelling disorder have safely had small, corrective changes made to dysfunctional DNA inside their liver cells , STAT writes. Continue to STAT+ to read the full story…
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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.
Pharma Mirror
APRIL 21, 2024
With the rapid development of biotechnology and molecular medicine, the introduction of mRNA as a vaccine or therapeutic agent enables the production of almost any desired functional protein/peptide within the human body.
pharmaphorum
SEPTEMBER 6, 2021
PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down phenylalanine, an amino acid that is commonly found in many foods. So far, subjects in Phearless have received doses of either 2e13 vg/kg or 6e13 vg/kg of BMN 307, although the protocol does include a third, higher-dose group.
The Pharma Data
OCTOBER 18, 2020
(NASDAQ:CDXC) today highlighted a new study published in The European Molecular Biology Organization Journal looking at the effect of nicotinamide riboside (NR) on maintaining telomeres, the protective regions at the end of DNA strands. Telomeres are “caps” at the end of chromosomes that protect DNA from getting worn away as cells replicate.
XTalks
DECEMBER 13, 2023
The CRISPR system is dubbed to be molecular “scissors” that can cut out parts of genes that are abnormal, deactivating or replacing them with new strands of normal DNA. This has tremendous potential for curing diseases that are caused by faulty/mutated genes. It also affects Hispanic Americans, but at a lower prevalence.
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