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.
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.
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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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
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. (..)
Pharmaceutical Technology
JANUARY 31, 2023
Gene therapy company uniQure has entered into a global licensing agreement with Apic Bio for APB-102 to treat patients with amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1). APB-102 is designed to be a one-time, intrathecally administered gene therapy for ALS patients.
XTalks
FEBRUARY 14, 2022
Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. It is an autosomal recessive disorder, which means that if both parents are carriers of the faulty gene that causes the disease, there is a one in four chance that their child will develop the disease.
Drug Discovery World
OCTOBER 4, 2022
This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. billion, compared to $19.9
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