Drug Target Review

MAY 22, 2024

What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context. DNA vectors in mouse models?

Therapies 59

Therapies RNA Protein Expression DNA 59

Drug Discovery World

FEBRUARY 28, 2023

With only 10% of these conditions indicated for an approved therapy, there is a sizable opportunity for scientists and companies to develop treatments for patients with no other options to turn to. At this stage, qPCR can be used to check for IVT efficiency, residual DNA contamination and adventitious agents such as microbes.

Disease 130

Disease Treatment Vaccine DNA 130

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

Drug Development 52

Drug Development Treatment FDA Approval Drugs 52

The Pharma Data

MARCH 20, 2023

2,3 These data are among a Zolgensma data set being presented during the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, which also include, in part, real-world evidence data from the RESTORE registry. Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5

Licensing 40

Licensing Licensing Clinical Trials Therapies 40