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 billion in cash.
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 licensed inclisiran to The Medicines Company, which was bought last year by Novartis for $9.7
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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 through epigenetic editing without altering the genetic sequence itself.
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.
Drug Discovery World
AUGUST 1, 2023
They are a promising approach for the treatment of genetic drivers of disease.” AstraZeneca’s non-alcoholic steatohepatitis (or ‘NASH’) study is a prime example of the value of ASOs 2. Though some genetic mutations are harmful, others can be protective.
Roots Analysis
OCTOBER 22, 2023
RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. These therapeutics are broadly classified into two categories, namely coding RNAs and non-coding RNAs.
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