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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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Biopharma Money on the Move: December 2 – 8

The Pharma Data

Now poised to advance a robust therapeutics pipeline to clinical development, Nuance will use the funds for ongoing R&D of existing products and business development of potential new assets. Laying down a new track for RNA processing, Remix launched with $81 million in financing. Sigilon Therapeutics .

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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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Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Conversations in Drug Development Trends

Differences in regulatory sentiments and industry sponsors’ subsequent clinical development strategies historically restricted access based on geography. The targeted ASOs aim to downregulate the expression of gene mutations that are associated with gain-of-function toxicity that leads to motor neuron loss in some ALS cases.

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

Alta Sciences

Oligonucleotide-Based Techniques Most oligonucleotide therapies act through antisense mechanisms and are directed against various RNA species. 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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What to expect from PEGS Europe 2023: Day 3

Drug Discovery World

Nageatte Ibrahim, MD, Vice President, Oncology & Global Clinical Development, MSD, on: ‘The new age of immunotherapy: From checkpoint inhibitors to vaccines’. Jana Langhoff, Tecan, on: ‘Opportunities and challenges of automating high throughput protein purification’.