Genetic Therapies Show Early Promise in Treating Obesity
BioSpace
JUNE 16, 2024
The plethora of genes involved in obesity presents an intriguing opportunity for both gene silencing and ex vivo gene therapy approaches.
Gene Silencing Gene Therapy Genetics 
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Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data
XTalks
OCTOBER 26, 2021
After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).
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Frontotemporal dementia: the state of treatment development
Pharmaceutical Technology
FEBRUARY 20, 2023
Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.
Novartis keeps a close eye on gene therapy with Vedere Bio buy
pharmaphorum
OCTOBER 29, 2020
Already a major player in gene therapy, Novartis has swooped on US startup Vedere Bio in a $280 million deal that builds its position in inherited eye diseases that can lead to blindness. Novartis’ big move in the gene therapy market came when it bought AveXis for $8.7 IRDs affect around 2 million people worldwide.
Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem
Pharma Marketing Network
DECEMBER 21, 2020
Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.
Three-in-one approach boosts the silencing power of CRISPR
Scienmag
JULY 2, 2021
A newly developed CRISPR-Cas9-based tool carries out efficient and long-term gene silencing by epigenetic editing Credit: BioDesign Research Originally discovered as a bacterial mode of defense against invading viruses, the remarkable ability of CRISPR-Cas9 to modify specific locations of DNA has made it a researcher favorite among gene editing tools. (..)
UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD
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). Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data.
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