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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

The half-life of proteins expressed through the mRNA technology is also much longer than those of direct protein delivery, which suggests that it could be more effective as a treatment strategy for rare diseases. Therefore, the risk for off-target insertional mutagenesis is greatly reduced.

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Reflecting on PEGS Europe 2023 

Drug Discovery World

This presentation covered three primary drivers for a protein expression model: speed, cost and sustainability. “It Field also covered the capabilities and strategies that enable acceleration of Fc-fusion, Fab fragment and bispecific antibodies through pre-clinical development.

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

Alta Sciences

Several viral vectors are being used currently, in addition to non-viral vectors, such as oligonucleotides, naked DNA, and lipoplexes and polyplexes. They work by binding to specific sequences of nucleotides present within the mRNA structure and can induce mechanisms that either decrease, restore, or modify protein expression.

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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

Thereafter, there will be two luncheon presentations: Joseph Rucker, PhD, Vice President, Research & Development, Integral Molecular, on: ‘A humanised chicken antibody platform that delivers diverse and developable therapeutic candidates’.