Gene Silencing, Genetics, In-Vivo and Protein

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

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

In-Vivo Gene Editing Trials Drugs

The use of base editing in stem-cell based therapies

Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector.

Gene Editing

Gene Editing Gene Engineer DNA

Clinical Research Informer

Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

The use of base editing in stem-cell based therapies

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