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Enhancing gene therapy with Circio

Drug Target Review

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?

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Unlocking the potential of mRNA for the future treatment of rare diseases 

Drug Discovery World

As we turn our focus to new potential applications and disease areas for the platform, scientists and companies must consider the potential life-saving impact on rare, inherited diseases, says Archana Gupta , PhD, staff scientist in genetic sciences at Thermo Fisher Scientific.

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Five Promising Treatment Areas in Early-Phase Drug Development in 2024

Alta Sciences

Gene Therapy Gene therapy is currently considered for diseases, often rare, that have no other cures. It is a growing field where new technology, and an increasing understanding of the science, are bringing advancements in drug development. Read The Altascientist , “Nonclinical Studies in Cell and Gene Therapy”. 3.

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Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset

The Pharma Data

As the number of patients treated with gene therapy around the world continues to grow, our goal is that more patients, and even new SMA patient populations, will be able to experience the transformative impact of this treatment,” said Sitra Tauscher-Wisniewski, MD, Vice President Clinical Development & Analytics, Novartis Gene Therapies.

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What to expect from PEGS Europe 2023: Day 3

Drug Discovery World

Jens Fischer, PhD, Program Manager, Large Molecule Research Therapeutic Modalities, Roche Pharma Research and Early Development (pRED), on: ‘ Bispecific and next-generation antibodies for non-oncology indications such as ophthalmologic and neurologic diseases’.