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.

Gene Silencing 52

Gene Silencing RNA Protein Gene 52

Pharmaceutical Technology

FEBRUARY 20, 2023

The therapy is specifically aimed at treating FTD patients who have mutations in their granulin gene, which encodes for the protein progranulin (PGRN). This protein promotes lysosomal function. It was previously expected to enter clinical development last year, but no announcements were made.

Development 246

Development Gene Therapy Protein Gene 246

Drug Discovery World

OCTOBER 4, 2022

As it stands, there are currently 19 gene therapies approved for clinical use, alongside 18 RNA therapies and 59 non-genetically modified cell therapies, according to the Q2 report by the American Society of Cell & Gene Therapy. Knockout’ mice are bred in a laboratory with specific genes silenced or ‘knocked out’.

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52

The Pharma Data

NOVEMBER 24, 2020

The safety and efficacy of Oxlumo are also being evaluated in the ongoing ILLUMINATE-C Phase 3 clinical trial in patients of all ages with advanced PH1, including patients on dialysis. Visit OXLUMO.com for more information, including full Prescribing Information. Until today, there were no approved pharmaceutical therapies for PH1.

FDA Approval 52

FDA Approval Production RNA Medicine 52