Pharmaceutical Technology

JANUARY 31, 2023

Under the terms of the deal, uniQure will obtain the worldwide rights to develop and market the clinical stage gene therapy, APB-102. uniQure stated that the license of APB-102 further strengthens its gene therapies pipeline developed for the treatment of neurological disorders as well as miRNA-based gene silencing programmes.

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pharmaphorum

NOVEMBER 18, 2021

Danish drugmaker Novo Nordisk must like what it has seen in its two-year-old alliance with gene-silencing specialist Dicerna Pharma – it has just agreed to acquire the biotech for $3.3 Once in the blood, the GalXC molecules travel to the liver, where they enter hepatocyte cells and can switch protein production on or off.

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Gene Silencing RNA Protein Gene 52

pharmaphorum

JUNE 8, 2022

The UK medicines regulator has awarded Alnylam’s RNAi-based therapy zilebesiran for hypertension an ‘innovation passport ‘, a designation designed to speed up NHS access to promising new medicines.

Gene Silencing 52

Gene Silencing Drugs Medicine Regulation 52

pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. Alnylam is hoping that revenue will stream from its new products and payments from Novartis, which owns the rights to cholesterol lowering drug inclisiran.

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pharmaphorum

MARCH 4, 2021

But with Novartis failing to convince European regulators about the merits of Xiidra last year, and Allergan’s ageing Restasis potentially facing generic competition, there’s a lot to play for and two biotechs are aiming to disrupt the market. Novartis paid Takeda $3.4

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Gene Silencing FDA Approval Trials Marketing 98

Drug Discovery World

AUGUST 1, 2023

As per a definition provided by AstraZeneca: “Once inside, ASOs bind with high specificity to target mRNA or pre-mRNA, inducing its degradation – effectively silencing it – to prevent its translation into a detrimental protein product.

RNA 52

RNA Drugs Gene Silencing Vaccination 52

Roots Analysis

OCTOBER 22, 2023

ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein. RNA interference (RNAi) Therapeutics : Fundamentally, RNAi is a natural process of post-transcriptional gene silencing, involving short strands of nucleic acids.

RNA 40

RNA Protein Genetics Gene Expression 40