New Wave of RNAi Startups Targets Wider Disease Horizons
A new generation of startups is looking into sophisticated delivery technology to take RNAi beyond its primary organ-specific application in the liver and into the more complicated diseases of organs like the kidneys, lungs, and CNS with strong investor support and biotech pioneers breathing new life into RNA interference (RNAi) technology.
Gene silencing therapies are stepping into the new age this Nobel Prize-winning technique, wherein the disease-causing genes alter to silence them, has advanced so much with improving delivery methods. Though the big companies like Alnylam Pharmaceuticals pioneered RNAi treatments, this new wave of biotechnology startups brings it to the edge.
RNAi Landscape: From Niche to New Horizons
Since 2018, six RNAi drugs have received approval for market use. Most of these are focused on rare genetic diseases and conditions like high cholesterol. However, most of them target the liver because RNAi tends to accumulate there. New delivery strategies will overcome this limitation. The latest advances are pushing RNAi therapy into areas long considered inaccessible for RNAi, such as the kidneys, brain, lungs, and muscles.
Small companies like Switch Therapeutics and Aro Biotherapeutics will be at the forefront of building delivery systems specific to each of these tissues, ushering in hopes of treating CNS disorders, genetic lung conditions, and muscle diseases.
Alnylam’s Heritage to New Startups
The RNAi industry started off as a venture based on the pioneering work of innovators like Alnylam, Arrowhead, and Dicerna. Much had to be developed to get RNAi from something which has the potential but is so toxic and therefore unsafe as a therapeutic option. For Alnylam, partnership and concentrated research efforts on hepatopathologies, using lipid nanoparticles as well as sugar-based drug delivery for increased drug exposure and reduced toxicity, was the way to success. Despite these setbacks, this tenacity has resulted in significant successes: Onpattro was the first approval in 2018, and had provided a firmer foundation for RNAi, a very promising therapeutic area. This foundation gives newer players the confidence to progress beyond liver disease and develop new innovations and delivery strategies that Alnylam and others might find difficult to prioritize in their current scaling phase.
New Strategies: Multi-target and Ultraprecise Delivery
Novel strategies are employed by companies in this industry to increase the therapeutic delivery of RNAi. For example, Switch Therapeutics attaches “sensors” on RNAi drugs so that it activates just in specific cell types, providing a new dimension of targeting disease. Aro Biotherapeutics is working on an entirely new “Centyrin” protein platform to help deliver RNAi therapies into muscle tissue. All the different startup companies are playing with various approaches to overcome delivery barriers-be it by conjugation to a ligand or forming compact RNA-to ensure that the efficacy of the drug reaches the desired location inside the patient’s body.
Investment and a New Generation of Genetic Therapies
A wave of investment is also inflating RNAi’s resurgence, as City Therapeutics and Judo Bio are raising more than $100 million combined. Judo Bio’s effort to develop an RNAi platform focused on the kidneys, using ligands with specific molecular properties designed to facilitate target-rich selective delivery. This interest reflects broader confidence in the future of RNAi, especially as RNAi technology is now considered less risky thanks to the scientific groundwork laid by Alnylam and other early movers. The infusion of capital and interest in RNAi aligns with the growth of the genetic medicine field, where other technologies such as CRISPR have sparked excitement at the promise of durable effects and an ability to treat complex genetic disorders in various ways.
A Transformatory Moment for RNAi?
These new start-ups open new doors for RNAi which is now becoming so much closer to reality will now push past the rare disorders barrier, and conquer even those diseases that were considered insurmountable. Companies are applying “bits of knowledge” that cropped up over the last twenty years and no one thought could be possible. As Jeff Goater, lead investor in Judo Bio and from The Column Group, puts it, “aha moments” define these new breakthroughs and how every innovation opens new doors to genetic medicine.
The Next Frontier of RNAi
The future of RNAi will be one of accessibility, precision, and broad scope for therapy. These emerging companies, with helmsmen such as John Maraganore and others, are poised to take RNAi far beyond the borders conventionally defined.
While Alnylam’s work laid the foundation, the commitment of these young companies to improving delivery and targeting strategies will probably push RNAi to a whole new level of gene therapy. At this moment, maybe RNAi, once confined to the liver, is made pliable enough for small, ambitious players to ride the transition wave into revolutionary treatments across multiple fields.
Know more about this article here.
