pharmaphorum

JUNE 1, 2022

The US biotech has just reported phase 2 trial results with the small, interfering RNA (siRNA) gene-silencing drug showing that it an cause a 90% or greater reduction in Lp(a) levels – a risk factor for cardiovascular disease – that was sustained over 48 weeks of follow-up.

Gene Silencing 59

Gene Silencing Drugs Medicine RNA 59

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

FDA Approval 52

FDA Approval Gene Silencing Drugs RNA 52

pharmaphorum

OCTOBER 29, 2020

Incubated by Atlas Ventures, Cambridge, Massachusetts Vedere has been flying under the radar since it was founded in 2019 to develop adeno-associated virus (AAV) based gene therapies for ocular diseases, but didn’t escape the beady eye of Novartis. Novartis’ big move in the gene therapy market came when it bought AveXis for $8.7

Gene Therapy 40

Gene Therapy Gene Gene Silencing Protein 40