The year 2023 has brought another great recognition to the field of RNA therapies, as evidenced by Nobel Prize in Physiology. RNA therapies, which involve targeting or the use of ribonucleic acid (RNA) molecules to treat diseases, represent a promising evolving frontier in medicine.
Over the past few decades researchers have made significant progress in developing RNA-based treatments for a wide range of conditions, from rare genetic disorders to cancer and viral infections. Several clinical breakthroughs have already been achieved in the field of RNA therapies, such as mRNA vaccines and RNA-interference. Yet only a handful of products achieved commercial success, with more than $1b in sales.
What to expect
In this article, we will look at how the main types of RNA therapies work, their application in the clinic, challenges researchers and companies face and new developments that can open doors to new breakthroughs.
The RNA landscape and why to invest
RNA therapies pipeline evolution
- RNA therapy pipeline has more than doubled over the last five years largely because of the mRNA-based vaccines, however other segments also saw significant growth
- RNAi has a proven application potential in rare diseases, mRNA in anti-infectives. Clinical success was demonstrated in prevalent diseases. New developments allow targeting cancer and other areas with high unmet medical need
- RNA therapies demonstrated both clinical and commercial proof-of-concept
- Potentially >$1b products in late-stage pipeline
Industry challenges and new developments. Why now?
RNA therapies offer exciting new avenues for the treatment of a wide range of diseases. However, there are several technical challenges that limit the pace of achievements, primarily within the tissue-specific nucleic acid delivery, stability and durability of protein expression.
In this article we provide an overview and discuss how the new developments in the field help to overcome these bottlenecks.
Upcoming near-term catalysts and milestones
Some of the catalysts and milestones that could mark the next round of evolution and further accelerate RNA therapies success are as follows:
- mRNA to demonstrate further clinical benefit in oncology and rare diseases as well as vaccines beyond COVID
- RNAi therapies to reach a blockbuster status in prevalent conditions
- ASOs to confirm clinical benefit in rare diseases
RNA therapy pipeline has more than doubled over the last five years
RNA therapies demonstrated both clinical and commercial proof-of-concept
Recent technological advances allow to achieve tissue specificity and enhanced durability
Despite the major breakthroughs, cumulative valuation of the publicly traded RNA therapy companies is returning to pre-pandemic levels (see chart in publication)
Where we can help
Investment, Commercial & Corporate strategy
Target search
One of the most frequent mistakes in the deal origination is a reactive approach, where the “buy-side” engages in transactions without having a clear high-resolution map of the field. Only the proactive approach, where the field is segmented and carefully mapped, allows maximizing the deal outcomes.
Asset / company due diligence
In-depth assessment of product potential and company valuation may help reduce the risk of asset mispricing when it comes to in/out-licensing or M&A. This is applicable for both buy- and sell-side / licensee and licensor.
Maximizing deal outcomes
Companies preparing for a new financing round, including crossover or an IPO, need to be particularly careful about their valuation as well as the targeted raise size. An environment assessment, investor sentiment analysis and clear company strategy going forward are some of the key prerequisites of success.
Portfolio strategy
It’s not uncommon for new entrants in the space to select “low hanging fruit” indications that may be less risky from both, scientific and the clinical perspective. However, this leads to increasing competition in certain indications and the “first-to-market” vs ”best in class” problem.
Operations
Manufacturing and Supply Chain
RNA manufacturing as well as next-generation vehicle development and synthesis require new capabilities. Robust supply of high-quality raw materials may be challenging to secure given the high demand on the market and limited capacities. It’s critical to be able to identify the right solutions or capabilities, know how to build them internally or acquire and integrate such capabilities and capacities to future-proof the organization.
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