pharmaphorum

JUNE 28, 2021

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105

XTalks

MAY 4, 2021

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Genes can be switched on with a complementary tool called CRISPRon that has also been described in the paper. .

DNA 91

DNA Gene Gene Silencing Genomics 91

XTalks

MAY 4, 2021

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Genes can be switched on with a complementary tool called CRISPRon that has also been described in the paper. .

DNA 52

DNA Gene Gene Silencing Genomics 52

Pharma Marketing Network

DECEMBER 21, 2020

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e.,

Genetics 52

Genetics Medicine Gene Therapy Marketing 52