Much has been discussed over the last few years about the burgeoning world of digital biology. So many tools are emerging that are beginning to make their mark on the way we discover and develop new medicines. At Innovation Endeavors, we think some of the greatest achievements and impact of these tools will be in the life sciences.
This post, however, is not about what AI can do. Rather it is about drawing remarkable parallels between computation and biology, software and programmable therapeutics, and to announce our latest investment, Exsilio Therapeutics.
We’ve previously contemplated this analogy between biology and software. Biology computes. It can be much more expressive than an LLM, trained by its evolutionary history and able to operate diverse “functions” across many “instances.” And more and more, biology is becoming programmable, meaning we can model and design structures, encode those in the language of DNA, and produce entire constructs, all in the span of hours.
Moderna (and BioNTech and others) showcased this power on the world stage — going from a sequenced virus to a therapeutic candidate in weeks and then to an approved vaccine in record time. The speed and scale of impact was truly one of the most amazing achievements in the history of new medicine development. This was largely enabled by the emergence of modular components that are the hallmark of genetic medicines, in particular, in their case, an mRNA molecule (for which Katalin Karikó and Drew Weissman shared the 2023 Nobel Prize in Physiology or Medicine for foundational contributions to this class of medicines) and a lipid nanoparticle delivery vehicle.
For a long-time coming, academics and industry scientists have highlighted the potential of programmable therapeutics. Robert Plenge has good pieces (here, here) on this idea going back almost a decade, and Peter Marks has been drumming up measured excitement on the possibility for expedited development paths for medicines with previously validated components.
Over the last two decades, the field of genomic medicines has continued to accelerate and expand. The excitement stems from the ability to deliver functional cures for extremely challenging and debilitating diseases. LUXTURNA was the first directly administered gene therapy approved in the US in 2017; and last year brought the approval of the first treatment utilizing CRISPR gene editing technology. With these experiences in hand and many more clinical and commercial stage products, the FDA recently released draft guidance on platform technologies, intended to enable the development, manufacturing, and regulatory efficiencies we hope to enable with programmable medicines.
With that context, we’re excited to announce our investment today in a team and company building a platform to develop programmable genetic elements, Exsilio Therapeutics. They take inspiration from naturally occurring, programmable elements to develop constructs with the ability to insert whole genes. The approach aims to solve many of the current challenges of gene editing approaches — all-RNA payloads that can deliver gene-sized constructs into precise safe harbor sites in disease-relevant cells with a repeatable and titratable approach. This stands to bring forth a new pillar in genomic medicines.
The team bridges in silico and wet lab-based experimentation and is led by Chairman and Interim CEO, Tal Zaks, MD, PhD. As Tal says well, “mRNA-based medicines allow for a software-like approach to creating new medicines.” From his experience as the former Chief Medical Officer at Moderna, he has seen this firsthand. We completely agree and are excited to join our partners Novartis Venture Fund, Delos Capital, OrbiMed, Insight Partners, JP Morgan Life Sciences, CRISPR Therapeutics, Invus, Arc Ventures, and Deep Insight.
