Epoch 30: Metabolic RNA Silencing

A Older Modality Matures into the Prime Time

Hello Avatar! Welcome back for another week of biotech analysis. Today is Sunday, which means this is our Building Biotech newsletter that is focused on discussing biopharma strategy topics. Recently there has been much excitement over the role of oligonucleotide-based therapeutics in the treatment of metabolic diseases. Leveraging the latest advancements in RNA chemistry and innovative delivery platforms, these therapies offer the promise of highly specific, effective, and safer alternatives to conventional treatments. As research continues to evolve, it is anticipated that the scope of oligonucleotide therapies will broaden, encompassing a wider array of metabolic conditions and thereby significantly enhancing patient outcomes.  Today we will deep dive into this modality, how it has evolved, and who are the biggest players in the space.

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Introduction

Recently there has been increased focus on oligonucleotide therapeutics. Companies like Alynlam, Arrowood, and Ionis have been leading the way in terms of generating compelling clinical data.  Many of these programs appear to be on their way to approval and offer tremendous advantages over current standards of care.  

In terms of therapeutic areas of interest there is nothing hotter than cardiovascular at the moment.  This is particularly driven by advances in metabolic disorders - mainly obesity.  In this space, we have noted the rise of RNA silencing as a therapeutic strategy. Today, we will take you through why this is an area to keep an eye on and which companies/programs are leading the way.

Oligonucleotide-Based Therapeutics

Oligonucleotide-based therapeutic approaches, including RNA interference (RNAi) and antisense oligonucleotides (ASOs), are being extensively explored for treating metabolic diseases. These therapies manipulate gene expression by binding to target RNA, using natural cellular processes to inhibit gene expression, thereby suppressing disease-causing genes or mutations.  As you will see in the coming sections of this essay, the high level take-away is that for therapeutic targets requiring silencing or regulation of expression, oligonucleotide based approaches can be quite powerful.