BIOTECH
Pioneering Nucleoside Building Blocks for Next-Level Therapeutic Impact
ASOs: Precision RNA Therapeutics that Target the “Undruggable”
Oligonucleotide therapeutics are groundbreaking in their ability to directly target and modulate genes linked to disease. At their forefront are antisense oligonucleotides (ASOs), short, single-stranded RNA molecules designed to bind messenger RNA and interrupt the production of pathogenic proteins, halting disease progression at its genetic roots.
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This innovative approach has the potential to revolutionize the treatment of undruggable or rare diseases where conventional small molecules or biologics have fallen short, offering renewed hope to patients.
Overcoming Barriers to Delivery and Safety
Despite their promise, ASO therapies face major obstacles, particularly in delivering treatments to difficult-to-access tissues such as the muscle, lung, and central nervous system. A key example is the blood-brain barrier, which presents unique complexities in targeting the CNS and its many tissue types. Ensuring safety and reducing off-target effects are also paramount for the clinical viability of ASOs.
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However, the limited chemical diversity in ASOs, due to the outdated and insufficient nucleoside building blocks currently available, has stifled their ability to fully realize their position as a third pillar of therapeutics alongside small molecules and biologics. Expanding this chemical space is essential to unlocking the transformative potential of ASOs, and more broadly, of all oligonucleotide-based therapeutics.
Fundamentally changing how we build RNA medicines
Cloudburst’s world-class expertise in nucleoside chemistry has culminated in RAvIN, a platform that replaces complex and low-yield approaches with sustainable processes that simplify synthesis and increase yields by tens of folds.
By creating novel and precisely tailored nucleoside monomers efficiently and at scale, RAvIN unlocks new chemical space and provides access to med-chem-ready cores for rapid diversification to enable the next generation of precision therapeutics.
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We are rigorously testing our medicinal chemistry libraries in ASO sequences to evaluate their clinical potential against rare and challenging conditions including genetic, oncological, pulmonary, cardiac, and neurodegenerative diseases. Our mission is to unlock a new generation of targeted RNA therapies that transform lives.