Clinical Development Protein Expression RNA 
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?
The Pharma Data
DECEMBER 8, 2020
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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Alta Sciences
APRIL 17, 2024
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.
The Pharma Data
SEPTEMBER 22, 2020
Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. This guidance provides clarity on the path to registration for AVXS-101 IT.
Conversations in Drug Development Trends
MAY 30, 2024
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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