DNA Gene Genetic Disease Protein 
Camargo
JULY 27, 2021
How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.
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 are the advantages compared to other gene modulating approaches?
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XTalks
DECEMBER 13, 2023
The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.
pharmaphorum
SEPTEMBER 6, 2021
BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal.
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…
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.
Drug Discovery World
APRIL 30, 2024
Its job is to carry coding information that is essential to the translation and processing of functional proteins. This is essential to its use as a therapeutic agent and gives the technology a vast versatility making it suitable to treating a wide range of diseases – especially those that have high protein expression.
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