Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program ...

Rett syndrome is a rare progressive neurodevelopmental ... the loss of independence as patients are unable to perform daily activities or communicate needs. They usually require 24/7 care and ...

The $9.2 million increase was driven by Good Manufacturing Practices batch activities for the intended commercial ... gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a ...

The $9.2 million increase was driven by Good Manufacturing Practices batch activities for the intended ... therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment ...

In a small study, patients with the syndrome were more likely to experience reactivation of Epstein-Barr virus and high levels of a coronavirus protein. By Apoorva Mandavilli The Covid-19 vaccines ...