A vital building block in the treatment of disease
RhodaPhos® Phos Reagent has become an established ingredient in the manufacture of RNA (ribonucleic acid)-based therapeutics, such as antisense oligonucleotides (ASO) and small interfering RNA (siRNA). These innovative drug classes are used to help treat a wide range of conditions, ranging from rare genetic disorders, like Huntingdon’s and Crohn's, to more common diseases, like cardiovascular and metabolic illnesses.
ASO and siRNA therapies work by acting at the level of RNA, rather than proteins or cells. Many diseases are caused by an error in a patient’s DNA code. When DNA is transcribed into mRNA (messenger RNA), this error can be passed along, potentially resulting in a protein with an incorrect structure. RNA-based therapies offer a different approach. They can bind with the faulty mRNA and block or modify the translation of the disease-causing protein.
This mechanism has enabled the establishment of a new generation of oligonucleotide therapeutics — and RhodaPhos® Phos Reagent plays a crucial role in making them possible.
The role of RhodaPhos® Phos Reagent
ASO and siRNA drug development is promising for patients suffering from many different diseases. But how is Syensqo involved?
Our RhodaPhos® Phos Reagent is a high-purity, phosphorus-based pharmaceutical intermediate used to manufacture phosphoramidite monomers, which are the essential building blocks for synthesizing oligonucleotides. These monomers form the RNA strands required for ASO and siRNA therapeutics.
“We can think of RNA as being like a ladder whose rungs are made up of nucleosides,” explains Tina Zuo, Global Marketing Manager for Phosphorus Specialties at Syensqo. “The rungs are attached to the ladder with a phosphorus molecule, creating a sugar-phosphate backbone. RhodaPhos® Phos Reagent is part of the chemistry used to manufacture this backbone. It enables scientists to develop oligonucleotides that target specific genetic instructions within the body.”
One example of this is Inclisiran, an siRNA therapy used for treating the widespread cardiovascular condition dyslipidemia. As the first commercialized siRNA oligonucleotide drug targeting a chronic disease, Inclisiran reflects the expanding impact of RNA-based medicines, beyond their original application in rare genetic disorders.
It can help to think of the people who manufacture ASO and siRNA drugs as people who build skyscrapers. At Syensqo, we’re providing the steel for the skyscrapers. It’s a vital part of the process — and just as exciting as creating the drugs themselves!
Pure chemistry
While Syensqo doesn’t manufacture the oligonucleotides or the final drugs, it’s our chemistry that allows these complex molecules to come together at industrial scale.
“It can help to think of the people who manufacture ASO and siRNA drugs as people who build skyscrapers. At Syensqo, we’re providing the steel for the skyscrapers. It’s a vital part of the process — and just as exciting as creating the drugs themselves!” says Tina.
The key advantage that RhodaPhos® Phos Reagent offers is its exceptional purity. “In a typical ASO or siRNA molecule, the ladder can be from 20 to 100 rungs, and each of these rungs needs a phosphorus atom from our product,” explains Tina. “A small impurity in the phosphorus chemistry could get repeated many times and create errors in the final oligonucleotide, so we work to ensure that doesn’t happen.”
And because RhodaPhos® Phos Reagent is available in industrial quantities, it supports the large-scale production needed to ensure more oligonucleotide drugs continue to reach the market. This helps to produce drugs designed to treat genetic diseases and rare disorders, while also developing emerging treatments for cancers, metabolic diseases and cardiovascular conditions.
