XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

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pharmaphorum

JANUARY 6, 2021

The regulator said it wanted more long-term safety and efficacy data from the REGENERATE study before making a final decision on the drug, which had been expected to be the first entrant into a likely multi-billion dollar market niche. The post High hopes as Inventiva takes NASH contender into phase 3 appeared first on.

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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.

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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. The Swiss pharma’s bet looks to have paid off as inclisiran is likely to be approved in Europe in the coming weeks after backing from CHMP regulators last month.

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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. AstraZeneca’s non-alcoholic steatohepatitis (or ‘NASH’) study is a prime example of the value of ASOs 2.

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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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Roots Analysis

OCTOBER 22, 2023

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. RNA interference (RNAi) Therapeutics : Fundamentally, RNAi is a natural process of post-transcriptional gene silencing, involving short strands of nucleic acids.

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